AVAHO

A humanized mouse model suggests that head and neck cancer growth may stem from chronic stress. The study found that animals had immunophenotypic changes and a greater propensity towards tumor growth and metastasis.

It is not uncommon for low-income patients with head and neck cancer to present with more aggressive disease at diagnosis. Other studies have shown this may be caused by the lack of access to health care services or poor quality care. but the difference remains even after adjusting for these factors, according to researchers writing in Head and Neck.

Led by Heather A. Himburg, PhD, associate professor of radiation oncology with the Medical College of Wisconsin, Milwaukee, researchers conducted a study of head and neck cancer models in which tumor cells were implanted into a mouse with a humanized immune system.

Their theory was that psychosocial stress may contribute to the growth of head and neck tumors. The stress of poverty, social deprivation and social isolation can lead to the up-regulation of proinflammatory markers in circulating blood leukocytes, and this has been tied to worse outcomes in hematologic malignancies and breast cancer. Many such studies examined social adversity and found an association with greater tumor growth rates and treatment resistance.

Other researchers have used mouse models to study the phenomenon, but the results have been inconclusive. For example, some research linked the beta-adrenergic pathway to head and neck cancer, but clinical trials of beta-blockers showed no benefit, and even potential harm, for patients with head and neck cancers. Those results imply that this pathway does not drive tumor growth and metastasis in the presence of chronic stress.

Previous research used immunocompromised or nonhumanized mice. However, neither type of model reproduces the human tumor microenvironment, which may contribute to ensuing clinical failures. In the new study, researchers describe results from a preclinical model created using a human head and neck cancer xenograft in a mouse with a humanized immune system.
 

How the study was conducted

The animals were randomly assigned to normal housing of two or three animals from the same litter to a cage, or social isolation from littermates. There were five male and five female animals in each arm, and the animals were housed in their separate conditions for 4 weeks before tumor implantation.

The isolated animals experienced increased growth and metastasis of the xenografts, compared with controls. The results are consistent with findings in immunodeficient or syngeneic mice, but the humanized nature of the new model could lead to better translation of findings into clinical studies. “The humanized model system in this study demonstrated the presence of both human myeloid and lymphoid lineages as well as expression of at least 40 human cytokines. These data indicate that our model is likely to well-represent the human condition and better predict human clinical responses as compared to both immunodeficient and syngeneic models,” the authors wrote.

The researchers also found that chronic stress may act through an immunoregulatory effect, since there was greater human immune infiltrate into the tumors of stressed animals. Increased presence of regulatory components like myeloid-derived suppressor cells or regulatory T cells, or eroded function of tumor-infiltrating lymphocytes, might explain this finding. The researchers also identified a proinflammatory change in peripheral blood monocular cells in the stressed group. When they analyzed samples from patients who were low income earners of less than $45,000 in annual household income, they found a similar pattern. “This suggests that chronic socioeconomic stress may induce a similar proinflammatory immune state as our chronic stress model system,” the authors wrote.

Tumors were also different between the two groups of mice. Tumors in stressed animals had a higher percentage of cancer stem cells, which is associated with more aggressive tumors and worse disease-free survival. The researchers suggested that up-regulated levels of the chemokine SDF-1 seen in the stressed animals may be driving the higher proportion of stem cells through its effects on the CXCR4 receptor, which is expressed by stem cells in various organs and may cause migration, proliferation, and cell survival.

The study was funded by an endowment from Advancing a Healthier Wisconsin and a grant from the National Center for Advancing Translational Sciences. The authors reported no conflicts of interest.

A humanized mouse model suggests that head and neck cancer growth may stem from chronic stress. The study found that animals had immunophenotypic changes and a greater propensity towards tumor growth and metastasis.

It is not uncommon for low-income patients with head and neck cancer to present with more aggressive disease at diagnosis. Other studies have shown this may be caused by the lack of access to health care services or poor quality care. but the difference remains even after adjusting for these factors, according to researchers writing in Head and Neck.

Led by Heather A. Himburg, PhD, associate professor of radiation oncology with the Medical College of Wisconsin, Milwaukee, researchers conducted a study of head and neck cancer models in which tumor cells were implanted into a mouse with a humanized immune system.

Their theory was that psychosocial stress may contribute to the growth of head and neck tumors. The stress of poverty, social deprivation and social isolation can lead to the up-regulation of proinflammatory markers in circulating blood leukocytes, and this has been tied to worse outcomes in hematologic malignancies and breast cancer. Many such studies examined social adversity and found an association with greater tumor growth rates and treatment resistance.

Other researchers have used mouse models to study the phenomenon, but the results have been inconclusive. For example, some research linked the beta-adrenergic pathway to head and neck cancer, but clinical trials of beta-blockers showed no benefit, and even potential harm, for patients with head and neck cancers. Those results imply that this pathway does not drive tumor growth and metastasis in the presence of chronic stress.

Previous research used immunocompromised or nonhumanized mice. However, neither type of model reproduces the human tumor microenvironment, which may contribute to ensuing clinical failures. In the new study, researchers describe results from a preclinical model created using a human head and neck cancer xenograft in a mouse with a humanized immune system.
 

How the study was conducted

The animals were randomly assigned to normal housing of two or three animals from the same litter to a cage, or social isolation from littermates. There were five male and five female animals in each arm, and the animals were housed in their separate conditions for 4 weeks before tumor implantation.

The isolated animals experienced increased growth and metastasis of the xenografts, compared with controls. The results are consistent with findings in immunodeficient or syngeneic mice, but the humanized nature of the new model could lead to better translation of findings into clinical studies. “The humanized model system in this study demonstrated the presence of both human myeloid and lymphoid lineages as well as expression of at least 40 human cytokines. These data indicate that our model is likely to well-represent the human condition and better predict human clinical responses as compared to both immunodeficient and syngeneic models,” the authors wrote.

The researchers also found that chronic stress may act through an immunoregulatory effect, since there was greater human immune infiltrate into the tumors of stressed animals. Increased presence of regulatory components like myeloid-derived suppressor cells or regulatory T cells, or eroded function of tumor-infiltrating lymphocytes, might explain this finding. The researchers also identified a proinflammatory change in peripheral blood monocular cells in the stressed group. When they analyzed samples from patients who were low income earners of less than $45,000 in annual household income, they found a similar pattern. “This suggests that chronic socioeconomic stress may induce a similar proinflammatory immune state as our chronic stress model system,” the authors wrote.

Tumors were also different between the two groups of mice. Tumors in stressed animals had a higher percentage of cancer stem cells, which is associated with more aggressive tumors and worse disease-free survival. The researchers suggested that up-regulated levels of the chemokine SDF-1 seen in the stressed animals may be driving the higher proportion of stem cells through its effects on the CXCR4 receptor, which is expressed by stem cells in various organs and may cause migration, proliferation, and cell survival.

The study was funded by an endowment from Advancing a Healthier Wisconsin and a grant from the National Center for Advancing Translational Sciences. The authors reported no conflicts of interest.

A humanized mouse model suggests that head and neck cancer growth may stem from chronic stress. The study found that animals had immunophenotypic changes and a greater propensity towards tumor growth and metastasis.

It is not uncommon for low-income patients with head and neck cancer to present with more aggressive disease at diagnosis. Other studies have shown this may be caused by the lack of access to health care services or poor quality care. but the difference remains even after adjusting for these factors, according to researchers writing in Head and Neck.

Led by Heather A. Himburg, PhD, associate professor of radiation oncology with the Medical College of Wisconsin, Milwaukee, researchers conducted a study of head and neck cancer models in which tumor cells were implanted into a mouse with a humanized immune system.

Their theory was that psychosocial stress may contribute to the growth of head and neck tumors. The stress of poverty, social deprivation and social isolation can lead to the up-regulation of proinflammatory markers in circulating blood leukocytes, and this has been tied to worse outcomes in hematologic malignancies and breast cancer. Many such studies examined social adversity and found an association with greater tumor growth rates and treatment resistance.

Other researchers have used mouse models to study the phenomenon, but the results have been inconclusive. For example, some research linked the beta-adrenergic pathway to head and neck cancer, but clinical trials of beta-blockers showed no benefit, and even potential harm, for patients with head and neck cancers. Those results imply that this pathway does not drive tumor growth and metastasis in the presence of chronic stress.

Previous research used immunocompromised or nonhumanized mice. However, neither type of model reproduces the human tumor microenvironment, which may contribute to ensuing clinical failures. In the new study, researchers describe results from a preclinical model created using a human head and neck cancer xenograft in a mouse with a humanized immune system.
 

How the study was conducted

The animals were randomly assigned to normal housing of two or three animals from the same litter to a cage, or social isolation from littermates. There were five male and five female animals in each arm, and the animals were housed in their separate conditions for 4 weeks before tumor implantation.

The isolated animals experienced increased growth and metastasis of the xenografts, compared with controls. The results are consistent with findings in immunodeficient or syngeneic mice, but the humanized nature of the new model could lead to better translation of findings into clinical studies. “The humanized model system in this study demonstrated the presence of both human myeloid and lymphoid lineages as well as expression of at least 40 human cytokines. These data indicate that our model is likely to well-represent the human condition and better predict human clinical responses as compared to both immunodeficient and syngeneic models,” the authors wrote.

The researchers also found that chronic stress may act through an immunoregulatory effect, since there was greater human immune infiltrate into the tumors of stressed animals. Increased presence of regulatory components like myeloid-derived suppressor cells or regulatory T cells, or eroded function of tumor-infiltrating lymphocytes, might explain this finding. The researchers also identified a proinflammatory change in peripheral blood monocular cells in the stressed group. When they analyzed samples from patients who were low income earners of less than $45,000 in annual household income, they found a similar pattern. “This suggests that chronic socioeconomic stress may induce a similar proinflammatory immune state as our chronic stress model system,” the authors wrote.

Tumors were also different between the two groups of mice. Tumors in stressed animals had a higher percentage of cancer stem cells, which is associated with more aggressive tumors and worse disease-free survival. The researchers suggested that up-regulated levels of the chemokine SDF-1 seen in the stressed animals may be driving the higher proportion of stem cells through its effects on the CXCR4 receptor, which is expressed by stem cells in various organs and may cause migration, proliferation, and cell survival.

The study was funded by an endowment from Advancing a Healthier Wisconsin and a grant from the National Center for Advancing Translational Sciences. The authors reported no conflicts of interest.

Encouraging improvements in survival have been reported by surgeons who used liver transplants from live donors as a treatment for patients with colorectal cancer (CRC) and unresectable liver metastases. These patients usually have a poor prognosis, and for many, palliative chemotherapy is the standard of care.

“For the first time, we have been able to demonstrate [outside of Norway] that liver transplantation for patients with unresectable liver metastases is feasible with good outcomes,” lead author Gonzalo Sapisochin, MD, PhD, an assistant professor of surgery at the University of Toronto, said in an interview.

“Furthermore, this is the first time we are able to prove that living donation may be a good strategy in this setting,” Dr. Sapisochin said of the series of 10 cases that they published in JAMA Surgery.

The series showed “excellent perioperative outcomes for both donors and recipients,” noted the authors of an accompanying commentary. They said the team “should be commended for adding liver-donor live transplantation to the armamentarium of surgical options for patients with CRC liver metastases.”

However, they express concern about the relatively short follow-up of 1.5 years and the “very high” recurrence rate of 30%.

Commenting in an interview, lead editorialist Shimul Shah, MD, an associate professor of surgery and the chief of solid organ transplantation at the University of Cincinnati, said: “I agree that overall survival is an important measure to look at, but it’s hard to look at overall survival with [1.5] years of follow-up.”

Other key areas of concern are the need for more standardized practices and for more data on how liver transplantation compares with patients who just continue to receive chemotherapy.

“I certainly think that there’s a role for liver transplantation in these patients, and I am a big fan of this,” Dr. Shah emphasized, noting that four patients at his own center have recently received liver transplants, including three from deceased donors.

“However, I just think that as a community, we need to be cautious and not get too excited too early,” he said. “We need to keep studying it and take it one step at a time.”

Moving from deceased to living donors

Nearly 70% of patients with CRC develop liver metastases, and when these are unresectable, the prognosis is poor, with 5-year survival rates of less than 10%.

The option of liver transplantation was first reported in 2015 by a group in Norway. Their study included 21 patients with CRC and unresectable liver tumors. They reported a striking improvement in overall survival at 5 years (56% vs. 9% among patients who started first-line chemotherapy).

But with shortages of donor livers, this approach has not caught on. Deceased-donor liver allografts are in short supply in most countries, and recent allocation changes have further shifted available organs away from patients with liver tumors.

An alternative is to use living donors. In a recent study, Dr. Sapisochin and colleagues showed viability and a survival advantage, compared with deceased-donor liver transplantation.

Building on that work, they established treatment protocols at three centers – the University of Rochester (N.Y.) Medical Center, the Cleveland Clinic, , and the University Health Network in Toronto.

Of 91 evaluated patients who were prospectively enrolled with liver-confined, unresectable CRC liver metastases, 10 met all inclusion criteria and received living-donor liver transplants between December 2017 and May 2021. The median age of the patients was 45 years; six were men, and four were women.

These patients all had primary tumors greater than stage T2 (six T3 and four T4b). Lymphovascular invasion was present in two patients, and perineural invasion was present in one patient.

The median time from diagnosis of the liver metastases to liver transplant was 1.7 years (range, 1.1-7.8 years).

At a median follow-up of 1.5 years (range, 0.4-2.9 years), recurrences occurred in three patients, with a rate of recurrence-free survival, using Kaplan-Meier estimates, of 62% and a rate of overall survival of 100%.

Rates of morbidity associated with transplantation were no higher than those observed in established standards for the donors or recipients, the authors noted.

Among transplant recipients, three patients had no Clavien-Dindo complications; three had grade II, and four had grade III complications. Among donors, five had no complications, four had grade I, and one had grade III complications.

All 10 donors were discharged from the hospital 4-7 days after surgery and recovered fully.

All three patients who experienced recurrences were treated with palliative chemotherapy. One died of disease after 3 months of treatment. As of the time of publication of the study, the other two had survived for 2 or more years following their live donor liver transplant.
 

Patient selection key

The authors are now investigating tumor subtypes, responses in CRC liver metastases, and other factors, with the aim of developing a novel screening method to identify appropriate candidates more quickly.

In the meantime, they emphasized that indicators of disease biology, such as the Oslo Score, the Clinical Risk Score, and sustained clinical response to systemic therapy, “remain the key filters through which to select patients who have sufficient opportunity for long-term cancer control, which is necessary to justify the risk to a living donor.”

Dr. Sapisochin reported receiving grants from Roche and Bayer and personal fees from Integra, Roche, AstraZeneca, and Novartis outside the submitted work. Dr. Shah disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Encouraging improvements in survival have been reported by surgeons who used liver transplants from live donors as a treatment for patients with colorectal cancer (CRC) and unresectable liver metastases. These patients usually have a poor prognosis, and for many, palliative chemotherapy is the standard of care.

“For the first time, we have been able to demonstrate [outside of Norway] that liver transplantation for patients with unresectable liver metastases is feasible with good outcomes,” lead author Gonzalo Sapisochin, MD, PhD, an assistant professor of surgery at the University of Toronto, said in an interview.

“Furthermore, this is the first time we are able to prove that living donation may be a good strategy in this setting,” Dr. Sapisochin said of the series of 10 cases that they published in JAMA Surgery.

The series showed “excellent perioperative outcomes for both donors and recipients,” noted the authors of an accompanying commentary. They said the team “should be commended for adding liver-donor live transplantation to the armamentarium of surgical options for patients with CRC liver metastases.”

However, they express concern about the relatively short follow-up of 1.5 years and the “very high” recurrence rate of 30%.

Commenting in an interview, lead editorialist Shimul Shah, MD, an associate professor of surgery and the chief of solid organ transplantation at the University of Cincinnati, said: “I agree that overall survival is an important measure to look at, but it’s hard to look at overall survival with [1.5] years of follow-up.”

Other key areas of concern are the need for more standardized practices and for more data on how liver transplantation compares with patients who just continue to receive chemotherapy.

“I certainly think that there’s a role for liver transplantation in these patients, and I am a big fan of this,” Dr. Shah emphasized, noting that four patients at his own center have recently received liver transplants, including three from deceased donors.

“However, I just think that as a community, we need to be cautious and not get too excited too early,” he said. “We need to keep studying it and take it one step at a time.”

Moving from deceased to living donors

Nearly 70% of patients with CRC develop liver metastases, and when these are unresectable, the prognosis is poor, with 5-year survival rates of less than 10%.

The option of liver transplantation was first reported in 2015 by a group in Norway. Their study included 21 patients with CRC and unresectable liver tumors. They reported a striking improvement in overall survival at 5 years (56% vs. 9% among patients who started first-line chemotherapy).

But with shortages of donor livers, this approach has not caught on. Deceased-donor liver allografts are in short supply in most countries, and recent allocation changes have further shifted available organs away from patients with liver tumors.

An alternative is to use living donors. In a recent study, Dr. Sapisochin and colleagues showed viability and a survival advantage, compared with deceased-donor liver transplantation.

Building on that work, they established treatment protocols at three centers – the University of Rochester (N.Y.) Medical Center, the Cleveland Clinic, , and the University Health Network in Toronto.

Of 91 evaluated patients who were prospectively enrolled with liver-confined, unresectable CRC liver metastases, 10 met all inclusion criteria and received living-donor liver transplants between December 2017 and May 2021. The median age of the patients was 45 years; six were men, and four were women.

These patients all had primary tumors greater than stage T2 (six T3 and four T4b). Lymphovascular invasion was present in two patients, and perineural invasion was present in one patient.

The median time from diagnosis of the liver metastases to liver transplant was 1.7 years (range, 1.1-7.8 years).

At a median follow-up of 1.5 years (range, 0.4-2.9 years), recurrences occurred in three patients, with a rate of recurrence-free survival, using Kaplan-Meier estimates, of 62% and a rate of overall survival of 100%.

Rates of morbidity associated with transplantation were no higher than those observed in established standards for the donors or recipients, the authors noted.

Among transplant recipients, three patients had no Clavien-Dindo complications; three had grade II, and four had grade III complications. Among donors, five had no complications, four had grade I, and one had grade III complications.

All 10 donors were discharged from the hospital 4-7 days after surgery and recovered fully.

All three patients who experienced recurrences were treated with palliative chemotherapy. One died of disease after 3 months of treatment. As of the time of publication of the study, the other two had survived for 2 or more years following their live donor liver transplant.
 

Patient selection key

The authors are now investigating tumor subtypes, responses in CRC liver metastases, and other factors, with the aim of developing a novel screening method to identify appropriate candidates more quickly.

In the meantime, they emphasized that indicators of disease biology, such as the Oslo Score, the Clinical Risk Score, and sustained clinical response to systemic therapy, “remain the key filters through which to select patients who have sufficient opportunity for long-term cancer control, which is necessary to justify the risk to a living donor.”

Dr. Sapisochin reported receiving grants from Roche and Bayer and personal fees from Integra, Roche, AstraZeneca, and Novartis outside the submitted work. Dr. Shah disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Encouraging improvements in survival have been reported by surgeons who used liver transplants from live donors as a treatment for patients with colorectal cancer (CRC) and unresectable liver metastases. These patients usually have a poor prognosis, and for many, palliative chemotherapy is the standard of care.

“For the first time, we have been able to demonstrate [outside of Norway] that liver transplantation for patients with unresectable liver metastases is feasible with good outcomes,” lead author Gonzalo Sapisochin, MD, PhD, an assistant professor of surgery at the University of Toronto, said in an interview.

“Furthermore, this is the first time we are able to prove that living donation may be a good strategy in this setting,” Dr. Sapisochin said of the series of 10 cases that they published in JAMA Surgery.

The series showed “excellent perioperative outcomes for both donors and recipients,” noted the authors of an accompanying commentary. They said the team “should be commended for adding liver-donor live transplantation to the armamentarium of surgical options for patients with CRC liver metastases.”

However, they express concern about the relatively short follow-up of 1.5 years and the “very high” recurrence rate of 30%.

Commenting in an interview, lead editorialist Shimul Shah, MD, an associate professor of surgery and the chief of solid organ transplantation at the University of Cincinnati, said: “I agree that overall survival is an important measure to look at, but it’s hard to look at overall survival with [1.5] years of follow-up.”

Other key areas of concern are the need for more standardized practices and for more data on how liver transplantation compares with patients who just continue to receive chemotherapy.

“I certainly think that there’s a role for liver transplantation in these patients, and I am a big fan of this,” Dr. Shah emphasized, noting that four patients at his own center have recently received liver transplants, including three from deceased donors.

“However, I just think that as a community, we need to be cautious and not get too excited too early,” he said. “We need to keep studying it and take it one step at a time.”

Moving from deceased to living donors

Nearly 70% of patients with CRC develop liver metastases, and when these are unresectable, the prognosis is poor, with 5-year survival rates of less than 10%.

The option of liver transplantation was first reported in 2015 by a group in Norway. Their study included 21 patients with CRC and unresectable liver tumors. They reported a striking improvement in overall survival at 5 years (56% vs. 9% among patients who started first-line chemotherapy).

But with shortages of donor livers, this approach has not caught on. Deceased-donor liver allografts are in short supply in most countries, and recent allocation changes have further shifted available organs away from patients with liver tumors.

An alternative is to use living donors. In a recent study, Dr. Sapisochin and colleagues showed viability and a survival advantage, compared with deceased-donor liver transplantation.

Building on that work, they established treatment protocols at three centers – the University of Rochester (N.Y.) Medical Center, the Cleveland Clinic, , and the University Health Network in Toronto.

Of 91 evaluated patients who were prospectively enrolled with liver-confined, unresectable CRC liver metastases, 10 met all inclusion criteria and received living-donor liver transplants between December 2017 and May 2021. The median age of the patients was 45 years; six were men, and four were women.

These patients all had primary tumors greater than stage T2 (six T3 and four T4b). Lymphovascular invasion was present in two patients, and perineural invasion was present in one patient.

The median time from diagnosis of the liver metastases to liver transplant was 1.7 years (range, 1.1-7.8 years).

At a median follow-up of 1.5 years (range, 0.4-2.9 years), recurrences occurred in three patients, with a rate of recurrence-free survival, using Kaplan-Meier estimates, of 62% and a rate of overall survival of 100%.

Rates of morbidity associated with transplantation were no higher than those observed in established standards for the donors or recipients, the authors noted.

Among transplant recipients, three patients had no Clavien-Dindo complications; three had grade II, and four had grade III complications. Among donors, five had no complications, four had grade I, and one had grade III complications.

All 10 donors were discharged from the hospital 4-7 days after surgery and recovered fully.

All three patients who experienced recurrences were treated with palliative chemotherapy. One died of disease after 3 months of treatment. As of the time of publication of the study, the other two had survived for 2 or more years following their live donor liver transplant.
 

Patient selection key

The authors are now investigating tumor subtypes, responses in CRC liver metastases, and other factors, with the aim of developing a novel screening method to identify appropriate candidates more quickly.

In the meantime, they emphasized that indicators of disease biology, such as the Oslo Score, the Clinical Risk Score, and sustained clinical response to systemic therapy, “remain the key filters through which to select patients who have sufficient opportunity for long-term cancer control, which is necessary to justify the risk to a living donor.”

Dr. Sapisochin reported receiving grants from Roche and Bayer and personal fees from Integra, Roche, AstraZeneca, and Novartis outside the submitted work. Dr. Shah disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

The preclinical phase of chronic lymphocytic leukemia (CLL) may be exist longer than previously thought, even in adverse-prognostic cases, as suggested by a sequencing analysis of blood samples obtained up to 22 years prior to CLL diagnosis.

Previous analyses showed that monoclonal B-cell lymphocytosis (MBL), a CLL precursor state, has been detected up to 6 years before CLL diagnosis, the investigators explained, noting that “[a]nother prognostically relevant immunogenetic feature of CLL concerns the stereotype of the B-cell receptor immunoglobulins (BcR IG).”

“Indeed, distinct stereotyped subsets can be defined by the expression of shared sequence motifs and are associated with particular presentation and outcomes,” P. Martijn Kolijn, PhD, a researcher in the department of immunology at Erasmus Medical Center, Rotterdam, the Netherlands, and colleagues wrote in a brief report published online in Blood. In an effort to “gain insight into the composition of the BcR IG repertoire during the early stages of CLL,” the investigators utilized next-generation sequencing to analyze 124 blood samples taken from healthy individuals up to 22 years before they received a diagnosis of CLL or small lymphocytic leukemia (SLL). An additional 118 matched control samples were also analyzed.

Study subjects were participants in the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort.

“First, unsurprisingly, we observed a significant difference in the frequency of the dominant clonotype in CLL patients versus controls with a median frequency of 54.9%, compared to only 0.38% in controls,” they wrote.

Among 28 patients whose lymphocyte counts were measured at baseline, 10 showed evidence of lymphocytosis up to 8 years before CLL diagnosis.

This suggests undiagnosed instances of high-count MBL (cases with a cell count above 0.5x 109 cells/L, which can progress to CLL) or asymptomatic CLL, they explained.

“In contrast, next-generation sequencing results showed detectable skewing of the IGH gene repertoire in 21/28 patients up to 15 years before CLL diagnosis, often in the absence of elevated lymphocyte counts,” they wrote. “Remarkably, some patients with CLL requiring treatment and clinical transformation to an aggressive B-cell lymphoma displayed considerable skewing in the IGH gene repertoire even 16 years before CLL diagnosis.”

Patients with a prediagnostic IGHV-unmutated dominant clonotype had significantly shorter overall survival after CLL diagnosis than did those with an IGHV-mutated clonotype, they noted.

“Furthermore, at early timepoints (>10 years before diagnosis), patients with a high dominant clonotype frequency were more likely to be IGHV mutated, whereas closer to diagnosis this tendency was lost, indicating that the prediagnostic phase may be even longer than 16 years for [mutated] CLL patients,” they added.

The investigators also found that:

• Twenty-five patients carried stereotyped BcR IG up to 17 years prior to CLL diagnosis, and of these, 10 clonotypes were assigned to minor subsets and 15 to major CLL subsets. Among the latter, 14 of the 15 belonged to high-risk subsets, and most of those showed a trend for faster disease evolution.
• High frequency of the dominant clonotype was evident in samples obtained less than 6 years before diagnosis, whereas high-risk stereotyped clonotypes found longer before diagnosis (as early as 16 years) tended to have a lower dominant clonotype frequency (<20% of IGH gene repertoire)
• The stereotyped BcR IG matched the clonotype at diagnosis for both patients with diagnostic material.
• No stereotyped subsets were identified among the dominant clonotypes of the healthy controls.

“To our knowledge, the dynamics of the emergence of biclonality in an MBL patient and subsequent progression to CLL have never been captured in such a convincing manner,” they noted.

The findings “extend current knowledge on the evolution of the IGH repertoire prior to CLL diagnosis, highlighting that even high-risk CLL subtypes may display a prolonged indolent preclinical stage,” they added, speculating that “somatic genetic aberrations, (auto)stimulation, epigenetic and/or microenvironmental influences are required for the transformation into overt CLL.”

The investigators also noted that since the observed skewing in the IGH gene repertoire often occurs prior to B-cell lymphocytosis, they consider the findings “a novel extension to the characterization of MBL.”

“Further studies may prove invaluable in the clinical distinction between ‘progressing’ MBL versus ‘stable’ MBL. Notwithstanding the above, we emphasize that early detection is only warranted if it provides clear benefits to patient care,” they concluded.

In a related commentary, Gerald Marti, MD, PhD, of the National Heart, Lung, and Blood Institute, emphasized that the findings “represent the earliest detection of a clonotypic precursor cell for CLL.” .

They also raise new questions and point to new directions for research, Dr. Marti noted.

“Where do we go from here? CLL has a long evolutionary history in which early branching may start as an oligoclonal process (antigen stimulation) and include driver mutations,” he wrote. “A long-term analysis of the B-cell repertoire in familial CLL might shed light on this process. Further clarification of the mechanisms of age-related immune senescence is also of interest.”

The study authors and Dr. Marti reported having no competing financial interests.

The preclinical phase of chronic lymphocytic leukemia (CLL) may be exist longer than previously thought, even in adverse-prognostic cases, as suggested by a sequencing analysis of blood samples obtained up to 22 years prior to CLL diagnosis.

Previous analyses showed that monoclonal B-cell lymphocytosis (MBL), a CLL precursor state, has been detected up to 6 years before CLL diagnosis, the investigators explained, noting that “[a]nother prognostically relevant immunogenetic feature of CLL concerns the stereotype of the B-cell receptor immunoglobulins (BcR IG).”

“Indeed, distinct stereotyped subsets can be defined by the expression of shared sequence motifs and are associated with particular presentation and outcomes,” P. Martijn Kolijn, PhD, a researcher in the department of immunology at Erasmus Medical Center, Rotterdam, the Netherlands, and colleagues wrote in a brief report published online in Blood. In an effort to “gain insight into the composition of the BcR IG repertoire during the early stages of CLL,” the investigators utilized next-generation sequencing to analyze 124 blood samples taken from healthy individuals up to 22 years before they received a diagnosis of CLL or small lymphocytic leukemia (SLL). An additional 118 matched control samples were also analyzed.

Study subjects were participants in the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort.

“First, unsurprisingly, we observed a significant difference in the frequency of the dominant clonotype in CLL patients versus controls with a median frequency of 54.9%, compared to only 0.38% in controls,” they wrote.

Among 28 patients whose lymphocyte counts were measured at baseline, 10 showed evidence of lymphocytosis up to 8 years before CLL diagnosis.

This suggests undiagnosed instances of high-count MBL (cases with a cell count above 0.5x 109 cells/L, which can progress to CLL) or asymptomatic CLL, they explained.

“In contrast, next-generation sequencing results showed detectable skewing of the IGH gene repertoire in 21/28 patients up to 15 years before CLL diagnosis, often in the absence of elevated lymphocyte counts,” they wrote. “Remarkably, some patients with CLL requiring treatment and clinical transformation to an aggressive B-cell lymphoma displayed considerable skewing in the IGH gene repertoire even 16 years before CLL diagnosis.”

Patients with a prediagnostic IGHV-unmutated dominant clonotype had significantly shorter overall survival after CLL diagnosis than did those with an IGHV-mutated clonotype, they noted.

“Furthermore, at early timepoints (>10 years before diagnosis), patients with a high dominant clonotype frequency were more likely to be IGHV mutated, whereas closer to diagnosis this tendency was lost, indicating that the prediagnostic phase may be even longer than 16 years for [mutated] CLL patients,” they added.

The investigators also found that:

• Twenty-five patients carried stereotyped BcR IG up to 17 years prior to CLL diagnosis, and of these, 10 clonotypes were assigned to minor subsets and 15 to major CLL subsets. Among the latter, 14 of the 15 belonged to high-risk subsets, and most of those showed a trend for faster disease evolution.
• High frequency of the dominant clonotype was evident in samples obtained less than 6 years before diagnosis, whereas high-risk stereotyped clonotypes found longer before diagnosis (as early as 16 years) tended to have a lower dominant clonotype frequency (<20% of IGH gene repertoire)
• The stereotyped BcR IG matched the clonotype at diagnosis for both patients with diagnostic material.
• No stereotyped subsets were identified among the dominant clonotypes of the healthy controls.

“To our knowledge, the dynamics of the emergence of biclonality in an MBL patient and subsequent progression to CLL have never been captured in such a convincing manner,” they noted.

The findings “extend current knowledge on the evolution of the IGH repertoire prior to CLL diagnosis, highlighting that even high-risk CLL subtypes may display a prolonged indolent preclinical stage,” they added, speculating that “somatic genetic aberrations, (auto)stimulation, epigenetic and/or microenvironmental influences are required for the transformation into overt CLL.”

The investigators also noted that since the observed skewing in the IGH gene repertoire often occurs prior to B-cell lymphocytosis, they consider the findings “a novel extension to the characterization of MBL.”

“Further studies may prove invaluable in the clinical distinction between ‘progressing’ MBL versus ‘stable’ MBL. Notwithstanding the above, we emphasize that early detection is only warranted if it provides clear benefits to patient care,” they concluded.

In a related commentary, Gerald Marti, MD, PhD, of the National Heart, Lung, and Blood Institute, emphasized that the findings “represent the earliest detection of a clonotypic precursor cell for CLL.” .

They also raise new questions and point to new directions for research, Dr. Marti noted.

“Where do we go from here? CLL has a long evolutionary history in which early branching may start as an oligoclonal process (antigen stimulation) and include driver mutations,” he wrote. “A long-term analysis of the B-cell repertoire in familial CLL might shed light on this process. Further clarification of the mechanisms of age-related immune senescence is also of interest.”

The study authors and Dr. Marti reported having no competing financial interests.

The preclinical phase of chronic lymphocytic leukemia (CLL) may be exist longer than previously thought, even in adverse-prognostic cases, as suggested by a sequencing analysis of blood samples obtained up to 22 years prior to CLL diagnosis.

Previous analyses showed that monoclonal B-cell lymphocytosis (MBL), a CLL precursor state, has been detected up to 6 years before CLL diagnosis, the investigators explained, noting that “[a]nother prognostically relevant immunogenetic feature of CLL concerns the stereotype of the B-cell receptor immunoglobulins (BcR IG).”

“Indeed, distinct stereotyped subsets can be defined by the expression of shared sequence motifs and are associated with particular presentation and outcomes,” P. Martijn Kolijn, PhD, a researcher in the department of immunology at Erasmus Medical Center, Rotterdam, the Netherlands, and colleagues wrote in a brief report published online in Blood. In an effort to “gain insight into the composition of the BcR IG repertoire during the early stages of CLL,” the investigators utilized next-generation sequencing to analyze 124 blood samples taken from healthy individuals up to 22 years before they received a diagnosis of CLL or small lymphocytic leukemia (SLL). An additional 118 matched control samples were also analyzed.

Study subjects were participants in the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort.

“First, unsurprisingly, we observed a significant difference in the frequency of the dominant clonotype in CLL patients versus controls with a median frequency of 54.9%, compared to only 0.38% in controls,” they wrote.

Among 28 patients whose lymphocyte counts were measured at baseline, 10 showed evidence of lymphocytosis up to 8 years before CLL diagnosis.

This suggests undiagnosed instances of high-count MBL (cases with a cell count above 0.5x 109 cells/L, which can progress to CLL) or asymptomatic CLL, they explained.

“In contrast, next-generation sequencing results showed detectable skewing of the IGH gene repertoire in 21/28 patients up to 15 years before CLL diagnosis, often in the absence of elevated lymphocyte counts,” they wrote. “Remarkably, some patients with CLL requiring treatment and clinical transformation to an aggressive B-cell lymphoma displayed considerable skewing in the IGH gene repertoire even 16 years before CLL diagnosis.”

Patients with a prediagnostic IGHV-unmutated dominant clonotype had significantly shorter overall survival after CLL diagnosis than did those with an IGHV-mutated clonotype, they noted.

“Furthermore, at early timepoints (>10 years before diagnosis), patients with a high dominant clonotype frequency were more likely to be IGHV mutated, whereas closer to diagnosis this tendency was lost, indicating that the prediagnostic phase may be even longer than 16 years for [mutated] CLL patients,” they added.

The investigators also found that:

• Twenty-five patients carried stereotyped BcR IG up to 17 years prior to CLL diagnosis, and of these, 10 clonotypes were assigned to minor subsets and 15 to major CLL subsets. Among the latter, 14 of the 15 belonged to high-risk subsets, and most of those showed a trend for faster disease evolution.
• High frequency of the dominant clonotype was evident in samples obtained less than 6 years before diagnosis, whereas high-risk stereotyped clonotypes found longer before diagnosis (as early as 16 years) tended to have a lower dominant clonotype frequency (<20% of IGH gene repertoire)
• The stereotyped BcR IG matched the clonotype at diagnosis for both patients with diagnostic material.
• No stereotyped subsets were identified among the dominant clonotypes of the healthy controls.

“To our knowledge, the dynamics of the emergence of biclonality in an MBL patient and subsequent progression to CLL have never been captured in such a convincing manner,” they noted.

The findings “extend current knowledge on the evolution of the IGH repertoire prior to CLL diagnosis, highlighting that even high-risk CLL subtypes may display a prolonged indolent preclinical stage,” they added, speculating that “somatic genetic aberrations, (auto)stimulation, epigenetic and/or microenvironmental influences are required for the transformation into overt CLL.”

The investigators also noted that since the observed skewing in the IGH gene repertoire often occurs prior to B-cell lymphocytosis, they consider the findings “a novel extension to the characterization of MBL.”

“Further studies may prove invaluable in the clinical distinction between ‘progressing’ MBL versus ‘stable’ MBL. Notwithstanding the above, we emphasize that early detection is only warranted if it provides clear benefits to patient care,” they concluded.

In a related commentary, Gerald Marti, MD, PhD, of the National Heart, Lung, and Blood Institute, emphasized that the findings “represent the earliest detection of a clonotypic precursor cell for CLL.” .

They also raise new questions and point to new directions for research, Dr. Marti noted.

“Where do we go from here? CLL has a long evolutionary history in which early branching may start as an oligoclonal process (antigen stimulation) and include driver mutations,” he wrote. “A long-term analysis of the B-cell repertoire in familial CLL might shed light on this process. Further clarification of the mechanisms of age-related immune senescence is also of interest.”

The study authors and Dr. Marti reported having no competing financial interests.

Adjuvant pembrolizumab significantly improves disease-free survival (DFS) compared to placebo in patients with early-stage non–small cell lung cancer (NSCLC) who have undergone complete resection, according to findings from the phase 3 PEARLS/KEYNOTE-091 (PEARLS) study.

Patients in the pembrolizumab arm demonstrated median DFS nearly 12 months longer than those in the placebo arm (53.6 vs. 42.0 months). Investigators observed a DFS benefit for patients with any programmed death-ligand 1 (PD-L1) expression.

“We believe that pembrolizumab has the potential to become a new adjuvant treatment option for patient with [stage IB to IIIA] non–small cell lung cancer following complete resection and adjuvant chemotherapy when recommended,” concluded first author Luis Paz-Ares, MD, chair of the clinical research unit at Hospital Universitario 12 de Octubre, CNIO & Universidad Complutense, Madrid. “Pembrolizumab provided a benefit regardless of pathological stage and PD-L1 progression subgroup.”

The findings were presented by Dr. Paz-Ares at the European Society for Medical Oncology (ESMO) March virtual plenary session and published March 17 in Annals of Oncology.

Pembrolizumab is the standard treatment for patients with advanced NSCLC, but its efficacy in early-stage disease remains unclear. To determine whether patients with early-stage disease benefit from pembrolizumab, Dr. Paz-Ares and colleagues randomized 1,177 adults with stage IB, II, or IIIA NSCLC to 200 mg of pembrolizumab (n = 590) or placebo (n = 587) every 3 weeks.

All patients had Eastern Cooperative Oncology Group performance status of 0-1, and any level of PD-L1 expression. Of the study participants, 168 in the pembrolizumab arm and 165 in the placebo arm had PD-L1 expression and a tumor proportion score (TPS) of at least 50%.

Overall, patients receiving pembrolizumab had a DFS of 53.6 months compared to 42.0 months in the placebo arm (hazard ratio [HR], 0.76; P = .0014). The DFS benefit was generally consistent across patients with PD-L1 TPS <1%, 1%-49%, and ≥50%. In the subset of patients with PD-L1 TPS ≥50%, a slightly higher percentage of patients in the pembrolizumab group demonstrated DFS at 18 months (71.7% vs. 70.2%), but the difference did not reach statistical significance (HR, 0.82; P = .14).

Overall survival (OS) at 18 months was 91.7% in the treatment arm and 91.3% in the placebo arm (HR, 0.87; P = .17), but the data were immature.

“The disease-free survival benefit was observed across most prespecified subgroups,” Dr. Paz-Ares said.

No new safety concerns were raised. Grade 3 or greater adverse events occurred in 34.1% of patients in the treatment arm and 25.8% in the placebo arm. Adverse events led to discontinuation in 19.8% of patients receiving pembrolizumab and 5.9% of patients in the placebo group.

Invited discussant Martin Reck, MD, said these findings represent forward progress. “We do see many patients with distant relapse, which indicates that we have to improve our control of the systemic relapse,” said Dr. Reck, head of the department of thoracic oncology and the clinical trial department at the Lungen Clinic Grosshansdorf, Germany.

Prior data provide a rationale for using immune checkpoint inhibition in early-stage NSCLC, and both the PEARLS study and the IMpower010 trial evaluating atezolizumab in a similar setting have demonstrated relevant improvements in DFS.

“I think we are entering the times of perioperative immunotherapies. We are seeing the first signals of efficacy for adjuvant immunotherapy in two large, randomized trials,” Dr. Reck said.

Based on the PEARLS trial results, Dr. Reck said that PD-L1 appears to have predictive and prognostic value but noted that “several other clinical trials say PD-L1 expression is a poor prognostic marker” for sensitivity to immune checkpoint inhibitor. Given this potential inconsistency, Dr. Reck called for further follow-up in this patient population and for studies in larger groups of patients to further delineate the role of PD-L1 as well as EGFR mutations and adjuvant chemotherapy in patients with early NSCLC.

The PEARLS study was funded by Merck Sharp & Dohme Corp. Dr. Paz-Ares and Dr. Reck disclosed numerous relationships with pharmaceutical companies.

Adjuvant pembrolizumab significantly improves disease-free survival (DFS) compared to placebo in patients with early-stage non–small cell lung cancer (NSCLC) who have undergone complete resection, according to findings from the phase 3 PEARLS/KEYNOTE-091 (PEARLS) study.

Patients in the pembrolizumab arm demonstrated median DFS nearly 12 months longer than those in the placebo arm (53.6 vs. 42.0 months). Investigators observed a DFS benefit for patients with any programmed death-ligand 1 (PD-L1) expression.

“We believe that pembrolizumab has the potential to become a new adjuvant treatment option for patient with [stage IB to IIIA] non–small cell lung cancer following complete resection and adjuvant chemotherapy when recommended,” concluded first author Luis Paz-Ares, MD, chair of the clinical research unit at Hospital Universitario 12 de Octubre, CNIO & Universidad Complutense, Madrid. “Pembrolizumab provided a benefit regardless of pathological stage and PD-L1 progression subgroup.”

The findings were presented by Dr. Paz-Ares at the European Society for Medical Oncology (ESMO) March virtual plenary session and published March 17 in Annals of Oncology.

Pembrolizumab is the standard treatment for patients with advanced NSCLC, but its efficacy in early-stage disease remains unclear. To determine whether patients with early-stage disease benefit from pembrolizumab, Dr. Paz-Ares and colleagues randomized 1,177 adults with stage IB, II, or IIIA NSCLC to 200 mg of pembrolizumab (n = 590) or placebo (n = 587) every 3 weeks.

All patients had Eastern Cooperative Oncology Group performance status of 0-1, and any level of PD-L1 expression. Of the study participants, 168 in the pembrolizumab arm and 165 in the placebo arm had PD-L1 expression and a tumor proportion score (TPS) of at least 50%.

Overall, patients receiving pembrolizumab had a DFS of 53.6 months compared to 42.0 months in the placebo arm (hazard ratio [HR], 0.76; P = .0014). The DFS benefit was generally consistent across patients with PD-L1 TPS <1%, 1%-49%, and ≥50%. In the subset of patients with PD-L1 TPS ≥50%, a slightly higher percentage of patients in the pembrolizumab group demonstrated DFS at 18 months (71.7% vs. 70.2%), but the difference did not reach statistical significance (HR, 0.82; P = .14).

Overall survival (OS) at 18 months was 91.7% in the treatment arm and 91.3% in the placebo arm (HR, 0.87; P = .17), but the data were immature.

“The disease-free survival benefit was observed across most prespecified subgroups,” Dr. Paz-Ares said.

No new safety concerns were raised. Grade 3 or greater adverse events occurred in 34.1% of patients in the treatment arm and 25.8% in the placebo arm. Adverse events led to discontinuation in 19.8% of patients receiving pembrolizumab and 5.9% of patients in the placebo group.

Invited discussant Martin Reck, MD, said these findings represent forward progress. “We do see many patients with distant relapse, which indicates that we have to improve our control of the systemic relapse,” said Dr. Reck, head of the department of thoracic oncology and the clinical trial department at the Lungen Clinic Grosshansdorf, Germany.

Prior data provide a rationale for using immune checkpoint inhibition in early-stage NSCLC, and both the PEARLS study and the IMpower010 trial evaluating atezolizumab in a similar setting have demonstrated relevant improvements in DFS.

“I think we are entering the times of perioperative immunotherapies. We are seeing the first signals of efficacy for adjuvant immunotherapy in two large, randomized trials,” Dr. Reck said.

Based on the PEARLS trial results, Dr. Reck said that PD-L1 appears to have predictive and prognostic value but noted that “several other clinical trials say PD-L1 expression is a poor prognostic marker” for sensitivity to immune checkpoint inhibitor. Given this potential inconsistency, Dr. Reck called for further follow-up in this patient population and for studies in larger groups of patients to further delineate the role of PD-L1 as well as EGFR mutations and adjuvant chemotherapy in patients with early NSCLC.

The PEARLS study was funded by Merck Sharp & Dohme Corp. Dr. Paz-Ares and Dr. Reck disclosed numerous relationships with pharmaceutical companies.

Adjuvant pembrolizumab significantly improves disease-free survival (DFS) compared to placebo in patients with early-stage non–small cell lung cancer (NSCLC) who have undergone complete resection, according to findings from the phase 3 PEARLS/KEYNOTE-091 (PEARLS) study.

Patients in the pembrolizumab arm demonstrated median DFS nearly 12 months longer than those in the placebo arm (53.6 vs. 42.0 months). Investigators observed a DFS benefit for patients with any programmed death-ligand 1 (PD-L1) expression.

“We believe that pembrolizumab has the potential to become a new adjuvant treatment option for patient with [stage IB to IIIA] non–small cell lung cancer following complete resection and adjuvant chemotherapy when recommended,” concluded first author Luis Paz-Ares, MD, chair of the clinical research unit at Hospital Universitario 12 de Octubre, CNIO & Universidad Complutense, Madrid. “Pembrolizumab provided a benefit regardless of pathological stage and PD-L1 progression subgroup.”

The findings were presented by Dr. Paz-Ares at the European Society for Medical Oncology (ESMO) March virtual plenary session and published March 17 in Annals of Oncology.

Pembrolizumab is the standard treatment for patients with advanced NSCLC, but its efficacy in early-stage disease remains unclear. To determine whether patients with early-stage disease benefit from pembrolizumab, Dr. Paz-Ares and colleagues randomized 1,177 adults with stage IB, II, or IIIA NSCLC to 200 mg of pembrolizumab (n = 590) or placebo (n = 587) every 3 weeks.

All patients had Eastern Cooperative Oncology Group performance status of 0-1, and any level of PD-L1 expression. Of the study participants, 168 in the pembrolizumab arm and 165 in the placebo arm had PD-L1 expression and a tumor proportion score (TPS) of at least 50%.

Overall, patients receiving pembrolizumab had a DFS of 53.6 months compared to 42.0 months in the placebo arm (hazard ratio [HR], 0.76; P = .0014). The DFS benefit was generally consistent across patients with PD-L1 TPS <1%, 1%-49%, and ≥50%. In the subset of patients with PD-L1 TPS ≥50%, a slightly higher percentage of patients in the pembrolizumab group demonstrated DFS at 18 months (71.7% vs. 70.2%), but the difference did not reach statistical significance (HR, 0.82; P = .14).

Overall survival (OS) at 18 months was 91.7% in the treatment arm and 91.3% in the placebo arm (HR, 0.87; P = .17), but the data were immature.

“The disease-free survival benefit was observed across most prespecified subgroups,” Dr. Paz-Ares said.

No new safety concerns were raised. Grade 3 or greater adverse events occurred in 34.1% of patients in the treatment arm and 25.8% in the placebo arm. Adverse events led to discontinuation in 19.8% of patients receiving pembrolizumab and 5.9% of patients in the placebo group.

Invited discussant Martin Reck, MD, said these findings represent forward progress. “We do see many patients with distant relapse, which indicates that we have to improve our control of the systemic relapse,” said Dr. Reck, head of the department of thoracic oncology and the clinical trial department at the Lungen Clinic Grosshansdorf, Germany.

Prior data provide a rationale for using immune checkpoint inhibition in early-stage NSCLC, and both the PEARLS study and the IMpower010 trial evaluating atezolizumab in a similar setting have demonstrated relevant improvements in DFS.

“I think we are entering the times of perioperative immunotherapies. We are seeing the first signals of efficacy for adjuvant immunotherapy in two large, randomized trials,” Dr. Reck said.

Based on the PEARLS trial results, Dr. Reck said that PD-L1 appears to have predictive and prognostic value but noted that “several other clinical trials say PD-L1 expression is a poor prognostic marker” for sensitivity to immune checkpoint inhibitor. Given this potential inconsistency, Dr. Reck called for further follow-up in this patient population and for studies in larger groups of patients to further delineate the role of PD-L1 as well as EGFR mutations and adjuvant chemotherapy in patients with early NSCLC.

The PEARLS study was funded by Merck Sharp & Dohme Corp. Dr. Paz-Ares and Dr. Reck disclosed numerous relationships with pharmaceutical companies.

Even by cautious calculations, the worldwide incidence of cutaneous melanoma is high and predicted to rise sharply over the next 2 decades, cancer epidemiologists warn.

An estimated 325,000 people worldwide received a new diagnosis of cutaneous melanoma in 2020, and if present trends continue, the incidence of new cases is predicted to increase by about 50% in 2040, with melanoma deaths expected to rise by almost 70%, Melina Arnold, PhD, from the Cancer Surveillance Branch of the International Agency for Research on Cancer in Lyon, France, and colleagues reported.

“Melanoma is the most lethal form of skin cancer; this epidemiological assessment found a heavy public health and economic burden, and our projections suggest that it will remain so in the coming decades,” they wrote in a study published online in JAMA Dermatology.

In an accompanying editorial, Mavis Obeng-Kusi, MPharm and Ivo Abraham, PhD from the Center for Health Outcomes and PharmacoEconomic Research at the University of Arizona, Tucson, commented that the findings are “sobering,” but may substantially underestimate the gravity of the problem in low- and middle-income countries (LMIC).

“The study by Arnold et al. brings to the fore a public health concern that requires global attention and initiates conversations particularly related to LMIC settings, where the incidence and mortality of melanoma is thought to be minimal and for which preventive measures may be insufficient,” they wrote.

Down Under nations lead

Dr. Arnold and colleagues looked at data on age-standardized melanoma incidence and mortality rates per 100,000 person-years (PY) by country, each of 20 world regions as defined by the United Nations, and according to the UN’s four-tier Human Development Index, which stratifies countries into low-, medium-, high-, and very high–income categories.

As noted previously, the researchers estimated that there were 325,000 new melanoma cases worldwide in 2020 (174,000 cases in males and 151,000 in females). There were 57,000 estimated melanoma deaths the same year (32,000 in males and 25,000 in females.

The highest incidence rates were seen in Australia and New Zealand, at 42 per 100,000 PY among males and 31 per 100,000 PY in females, followed by Western Europe with 19 per 100,000 PY in both males and females, North America with 18 and 14 cases per 100,000 PY in males and females respectively, and Northern Europe, with 17 per 100,000 PY in males, and 18 per 100,000 PY in females.

In contrast, in most African and Asian countries melanoma was rare, with rates commonly less than 1 per 100,000 PY, the investigators noted.

The melanoma mortality rate was highest in New Zealand, at 5 per 100,000 PY. Mortality rates worldwide varied less widely than incidence rates. In most other regions of the world, mortality rates were “much lower,” ranging between 0.2-1.0 per 100,000 PY, they wrote.

The authors estimated that, if 2020 rates remain stable, the global burden from melanoma in 2040 will increase to approximately 510,000 new cases and 96,000 deaths.

Public health efforts needed

In their editorial, Ms. Obeng-Kusi and Dr. Abraham pointed out that the study was hampered by the limited availability of cancer data from LMICs, leading the authors to estimate incidence and mortality rates based on proxy data, such as statistical modeling or averaged rates from neighboring countries.

They emphasized the need for going beyond the statistics: “Specific to cutaneous melanoma data, what is most important globally, knowing the exact numbers of cases and deaths or understanding the order of magnitude of the present and future epidemiology? No doubt the latter. Melanoma can be treated more easily if caught at earlier stages.”

Projections such as those provided by Dr. Arnold and colleagues could help to raise awareness of the importance of decreasing exposure to UV radiation, which accounts for three-fourths of all incident melanomas, the editorialists said.

The study was funded in part by a grant to coauthor Anna E. Cust, PhD, MPH. Dr. Cust reported receiving a fellowship from the Australian National Health and Medical Research Council outside the submitted work. Dr. Arnold had no conflicts of interested to disclose. Dr. Abraham reported financial relationships with various entities. Ms. Obeng-Kusi had no disclosures.

Even by cautious calculations, the worldwide incidence of cutaneous melanoma is high and predicted to rise sharply over the next 2 decades, cancer epidemiologists warn.

An estimated 325,000 people worldwide received a new diagnosis of cutaneous melanoma in 2020, and if present trends continue, the incidence of new cases is predicted to increase by about 50% in 2040, with melanoma deaths expected to rise by almost 70%, Melina Arnold, PhD, from the Cancer Surveillance Branch of the International Agency for Research on Cancer in Lyon, France, and colleagues reported.

“Melanoma is the most lethal form of skin cancer; this epidemiological assessment found a heavy public health and economic burden, and our projections suggest that it will remain so in the coming decades,” they wrote in a study published online in JAMA Dermatology.

In an accompanying editorial, Mavis Obeng-Kusi, MPharm and Ivo Abraham, PhD from the Center for Health Outcomes and PharmacoEconomic Research at the University of Arizona, Tucson, commented that the findings are “sobering,” but may substantially underestimate the gravity of the problem in low- and middle-income countries (LMIC).

“The study by Arnold et al. brings to the fore a public health concern that requires global attention and initiates conversations particularly related to LMIC settings, where the incidence and mortality of melanoma is thought to be minimal and for which preventive measures may be insufficient,” they wrote.

Down Under nations lead

Dr. Arnold and colleagues looked at data on age-standardized melanoma incidence and mortality rates per 100,000 person-years (PY) by country, each of 20 world regions as defined by the United Nations, and according to the UN’s four-tier Human Development Index, which stratifies countries into low-, medium-, high-, and very high–income categories.

As noted previously, the researchers estimated that there were 325,000 new melanoma cases worldwide in 2020 (174,000 cases in males and 151,000 in females). There were 57,000 estimated melanoma deaths the same year (32,000 in males and 25,000 in females.

The highest incidence rates were seen in Australia and New Zealand, at 42 per 100,000 PY among males and 31 per 100,000 PY in females, followed by Western Europe with 19 per 100,000 PY in both males and females, North America with 18 and 14 cases per 100,000 PY in males and females respectively, and Northern Europe, with 17 per 100,000 PY in males, and 18 per 100,000 PY in females.

In contrast, in most African and Asian countries melanoma was rare, with rates commonly less than 1 per 100,000 PY, the investigators noted.

The melanoma mortality rate was highest in New Zealand, at 5 per 100,000 PY. Mortality rates worldwide varied less widely than incidence rates. In most other regions of the world, mortality rates were “much lower,” ranging between 0.2-1.0 per 100,000 PY, they wrote.

The authors estimated that, if 2020 rates remain stable, the global burden from melanoma in 2040 will increase to approximately 510,000 new cases and 96,000 deaths.

Public health efforts needed

In their editorial, Ms. Obeng-Kusi and Dr. Abraham pointed out that the study was hampered by the limited availability of cancer data from LMICs, leading the authors to estimate incidence and mortality rates based on proxy data, such as statistical modeling or averaged rates from neighboring countries.

They emphasized the need for going beyond the statistics: “Specific to cutaneous melanoma data, what is most important globally, knowing the exact numbers of cases and deaths or understanding the order of magnitude of the present and future epidemiology? No doubt the latter. Melanoma can be treated more easily if caught at earlier stages.”

Projections such as those provided by Dr. Arnold and colleagues could help to raise awareness of the importance of decreasing exposure to UV radiation, which accounts for three-fourths of all incident melanomas, the editorialists said.

The study was funded in part by a grant to coauthor Anna E. Cust, PhD, MPH. Dr. Cust reported receiving a fellowship from the Australian National Health and Medical Research Council outside the submitted work. Dr. Arnold had no conflicts of interested to disclose. Dr. Abraham reported financial relationships with various entities. Ms. Obeng-Kusi had no disclosures.

Even by cautious calculations, the worldwide incidence of cutaneous melanoma is high and predicted to rise sharply over the next 2 decades, cancer epidemiologists warn.

An estimated 325,000 people worldwide received a new diagnosis of cutaneous melanoma in 2020, and if present trends continue, the incidence of new cases is predicted to increase by about 50% in 2040, with melanoma deaths expected to rise by almost 70%, Melina Arnold, PhD, from the Cancer Surveillance Branch of the International Agency for Research on Cancer in Lyon, France, and colleagues reported.

“Melanoma is the most lethal form of skin cancer; this epidemiological assessment found a heavy public health and economic burden, and our projections suggest that it will remain so in the coming decades,” they wrote in a study published online in JAMA Dermatology.

In an accompanying editorial, Mavis Obeng-Kusi, MPharm and Ivo Abraham, PhD from the Center for Health Outcomes and PharmacoEconomic Research at the University of Arizona, Tucson, commented that the findings are “sobering,” but may substantially underestimate the gravity of the problem in low- and middle-income countries (LMIC).

“The study by Arnold et al. brings to the fore a public health concern that requires global attention and initiates conversations particularly related to LMIC settings, where the incidence and mortality of melanoma is thought to be minimal and for which preventive measures may be insufficient,” they wrote.

Down Under nations lead

Dr. Arnold and colleagues looked at data on age-standardized melanoma incidence and mortality rates per 100,000 person-years (PY) by country, each of 20 world regions as defined by the United Nations, and according to the UN’s four-tier Human Development Index, which stratifies countries into low-, medium-, high-, and very high–income categories.

As noted previously, the researchers estimated that there were 325,000 new melanoma cases worldwide in 2020 (174,000 cases in males and 151,000 in females). There were 57,000 estimated melanoma deaths the same year (32,000 in males and 25,000 in females.

The highest incidence rates were seen in Australia and New Zealand, at 42 per 100,000 PY among males and 31 per 100,000 PY in females, followed by Western Europe with 19 per 100,000 PY in both males and females, North America with 18 and 14 cases per 100,000 PY in males and females respectively, and Northern Europe, with 17 per 100,000 PY in males, and 18 per 100,000 PY in females.

In contrast, in most African and Asian countries melanoma was rare, with rates commonly less than 1 per 100,000 PY, the investigators noted.

The melanoma mortality rate was highest in New Zealand, at 5 per 100,000 PY. Mortality rates worldwide varied less widely than incidence rates. In most other regions of the world, mortality rates were “much lower,” ranging between 0.2-1.0 per 100,000 PY, they wrote.

The authors estimated that, if 2020 rates remain stable, the global burden from melanoma in 2040 will increase to approximately 510,000 new cases and 96,000 deaths.

Public health efforts needed

In their editorial, Ms. Obeng-Kusi and Dr. Abraham pointed out that the study was hampered by the limited availability of cancer data from LMICs, leading the authors to estimate incidence and mortality rates based on proxy data, such as statistical modeling or averaged rates from neighboring countries.

They emphasized the need for going beyond the statistics: “Specific to cutaneous melanoma data, what is most important globally, knowing the exact numbers of cases and deaths or understanding the order of magnitude of the present and future epidemiology? No doubt the latter. Melanoma can be treated more easily if caught at earlier stages.”

Projections such as those provided by Dr. Arnold and colleagues could help to raise awareness of the importance of decreasing exposure to UV radiation, which accounts for three-fourths of all incident melanomas, the editorialists said.

The study was funded in part by a grant to coauthor Anna E. Cust, PhD, MPH. Dr. Cust reported receiving a fellowship from the Australian National Health and Medical Research Council outside the submitted work. Dr. Arnold had no conflicts of interested to disclose. Dr. Abraham reported financial relationships with various entities. Ms. Obeng-Kusi had no disclosures.

The 5 days that she spent at her mother’s bedside were eye-opening for an oncologist used to being on the other side of the clinician–patient relationship.

“As a physician, I thought I had a unique perspective of things that were done well – and things that were not,” commented Pamela Kunz, MD.

Dr. Kunz, who was named the 2021 Woman Oncologist of the Year, is director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and of the Yale Cancer Center, New Haven, Conn.

But she was propelled into quite a different role when her mother was admitted to the hospital.

Her mom, who has trouble hearing, was easily confused by jargon and by “all of the people coming in and out with no introductions,” she explained.

“She needed someone to translate what was going on because she didn’t feel well,” she added.

Seeing inpatient care through her mother’s eyes was enlightening, and at times it was “shocking to be on the other side.”

Physicians get used to “checking boxes, getting through the day,” she said. “It’s easy to forget the human side.”

“Seeing a loved one sick, [struggling] through this – I just wished I had seen things done differently,” added Dr. Kunz.

The experience prompted Dr. Kunz to share several “communication pearls” via Twitter. Her thread has since garnered thousands of “likes” and scores of comments and retweets.

She began the Twitter thread explaining what prompted her comments:

“I spent many hours last week observing the practice of medicine while sitting at my mom’s hospital bedside and was reminded of some important communication pearls. Some musings ...”

“1. Introduce yourself by full name, role, and team and have ID badges visible. It can get very confusing for [patients] and family members with the number of people in and out of rooms. E.g. ‘My name is Dr. X. I’m the intern on the primary internal medicine team.’

2.  End your patient visit with a summary of the plan for the day.

3. Avoid medical jargon & speak slowly, clearly, and logically. Remember you are a teacher for your [patients] and their family.

4. Masks make it harder to hear, especially for [patients] with hearing loss (and they no longer have the aid of lip reading).

5. Many older [patients] get confused in the hospital. Repetition is a good thing.

6. Speak to a family member at least once per day to relay the plan.

7. Try to avoid last minute or surprise discharges – they make [patients] and family members anxious. Talk about discharge planning from day 1 and what milestones must occur prior to a safe discharge. ‘In order for you to leave the hospital, X, Y, X must happen.’

8. Talk with your [patients] about something other than what brought them to the hospital (a tip I once learned from a wise mentor).

9.  When possible, sit at eye level with your patient (I love these stools from @YNHH).

10. Take time to listen.”

Dr. Kunz closed with her golden rule: “Lastly, treat your patients how you would want your own family member treated.”

Twitter user @BrunaPellini replied: “I love this, especially ‘Treat your patients how you would want your own family member treated.’ My mom and grandma always said that to me since I was a med student, and this is definitely one of my core values.”

Other clinicians shared similar experiences, and some added to Dr. Kunz’s list.

“Agree entirely, love the list – and while none of us can always practice perfectly, my experiences with my own mother’s illness taught me an enormous amount about communication,” @hoperugo responded.

Twitter user @mariejacork added: “Everyone in health care please read ... if you are lucky enough to not have had a loved one unwell in hospital, these may get forgotten. Having sat with my dad for a few days before he died a few years ago, I felt a lot of these, and it changed my practice forever.”

@bjcohenmd provided additional advice: “And use the dry erase board that should be in every room. Never start a medication without explaining it. Many docs will see the patient and then go to the computer, decide to order a med, but never go back to explain it.”

Patients also shared experiences and offered suggestions.

“As a chronic pain patient I’d add – we know it’s frustrating you can’t cure us but PLEASE do not SIGH if we say something didn’t work or [tell] us to be more positive. Just say ‘I know this is very hard, I’m here to listen.’ We don’t expect a cure, we do expect to be believed,” said @ppenguinsmt. “It makes me feel like I’m causing distress to you if I say the pain has been unrelenting. I leave feeling worse. ...You may have heard 10 [people] in pain before me but this is MY only [appointment].”

Twitter user @KatieCahoots added: “These are perfect. I wish doctors would do this not only in the hospital but in the doctor’s office, as well. I would add one caveat: When you try not to use medical jargon, don’t dumb it down as though I don’t know anything about science or haven’t done any of my own research.”

Dr. Kunz said she was taken aback but pleased by the response to her Tweet.

“It’s an example of the human side of medicine, so it resonates with physicians and with patients,” she commented. Seeing through her mom’s eyes how care was provided made her realize that medical training should include more emphasis on communication, including “real-time feedback to interns, residents, fellows, and students.”

Yes, it takes time, and “we don’t all have a lot of extra time,” she acknowledged.

“But some of these elements don’t take that much more time to do. They can help build trust and can, in the long run, actually save time if patients understand and family members feel engaged and like they are participants,” she said. “I think a little time investment will go a long way.”

In her case, she very much appreciated the one trainee who tried to call her and update her about her mother’s care each afternoon. “I really valued that,” she said.

A version of this article first appeared on Medscape.com.

The 5 days that she spent at her mother’s bedside were eye-opening for an oncologist used to being on the other side of the clinician–patient relationship.

“As a physician, I thought I had a unique perspective of things that were done well – and things that were not,” commented Pamela Kunz, MD.

Dr. Kunz, who was named the 2021 Woman Oncologist of the Year, is director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and of the Yale Cancer Center, New Haven, Conn.

But she was propelled into quite a different role when her mother was admitted to the hospital.

Her mom, who has trouble hearing, was easily confused by jargon and by “all of the people coming in and out with no introductions,” she explained.

“She needed someone to translate what was going on because she didn’t feel well,” she added.

Seeing inpatient care through her mother’s eyes was enlightening, and at times it was “shocking to be on the other side.”

Physicians get used to “checking boxes, getting through the day,” she said. “It’s easy to forget the human side.”

“Seeing a loved one sick, [struggling] through this – I just wished I had seen things done differently,” added Dr. Kunz.

The experience prompted Dr. Kunz to share several “communication pearls” via Twitter. Her thread has since garnered thousands of “likes” and scores of comments and retweets.

She began the Twitter thread explaining what prompted her comments:

“I spent many hours last week observing the practice of medicine while sitting at my mom’s hospital bedside and was reminded of some important communication pearls. Some musings ...”

“1. Introduce yourself by full name, role, and team and have ID badges visible. It can get very confusing for [patients] and family members with the number of people in and out of rooms. E.g. ‘My name is Dr. X. I’m the intern on the primary internal medicine team.’

2.  End your patient visit with a summary of the plan for the day.

3. Avoid medical jargon & speak slowly, clearly, and logically. Remember you are a teacher for your [patients] and their family.

4. Masks make it harder to hear, especially for [patients] with hearing loss (and they no longer have the aid of lip reading).

5. Many older [patients] get confused in the hospital. Repetition is a good thing.

6. Speak to a family member at least once per day to relay the plan.

7. Try to avoid last minute or surprise discharges – they make [patients] and family members anxious. Talk about discharge planning from day 1 and what milestones must occur prior to a safe discharge. ‘In order for you to leave the hospital, X, Y, X must happen.’

8. Talk with your [patients] about something other than what brought them to the hospital (a tip I once learned from a wise mentor).

9.  When possible, sit at eye level with your patient (I love these stools from @YNHH).

10. Take time to listen.”

Dr. Kunz closed with her golden rule: “Lastly, treat your patients how you would want your own family member treated.”

Twitter user @BrunaPellini replied: “I love this, especially ‘Treat your patients how you would want your own family member treated.’ My mom and grandma always said that to me since I was a med student, and this is definitely one of my core values.”

Other clinicians shared similar experiences, and some added to Dr. Kunz’s list.

“Agree entirely, love the list – and while none of us can always practice perfectly, my experiences with my own mother’s illness taught me an enormous amount about communication,” @hoperugo responded.

Twitter user @mariejacork added: “Everyone in health care please read ... if you are lucky enough to not have had a loved one unwell in hospital, these may get forgotten. Having sat with my dad for a few days before he died a few years ago, I felt a lot of these, and it changed my practice forever.”

@bjcohenmd provided additional advice: “And use the dry erase board that should be in every room. Never start a medication without explaining it. Many docs will see the patient and then go to the computer, decide to order a med, but never go back to explain it.”

Patients also shared experiences and offered suggestions.

“As a chronic pain patient I’d add – we know it’s frustrating you can’t cure us but PLEASE do not SIGH if we say something didn’t work or [tell] us to be more positive. Just say ‘I know this is very hard, I’m here to listen.’ We don’t expect a cure, we do expect to be believed,” said @ppenguinsmt. “It makes me feel like I’m causing distress to you if I say the pain has been unrelenting. I leave feeling worse. ...You may have heard 10 [people] in pain before me but this is MY only [appointment].”

Twitter user @KatieCahoots added: “These are perfect. I wish doctors would do this not only in the hospital but in the doctor’s office, as well. I would add one caveat: When you try not to use medical jargon, don’t dumb it down as though I don’t know anything about science or haven’t done any of my own research.”

Dr. Kunz said she was taken aback but pleased by the response to her Tweet.

“It’s an example of the human side of medicine, so it resonates with physicians and with patients,” she commented. Seeing through her mom’s eyes how care was provided made her realize that medical training should include more emphasis on communication, including “real-time feedback to interns, residents, fellows, and students.”

Yes, it takes time, and “we don’t all have a lot of extra time,” she acknowledged.

“But some of these elements don’t take that much more time to do. They can help build trust and can, in the long run, actually save time if patients understand and family members feel engaged and like they are participants,” she said. “I think a little time investment will go a long way.”

In her case, she very much appreciated the one trainee who tried to call her and update her about her mother’s care each afternoon. “I really valued that,” she said.

A version of this article first appeared on Medscape.com.

The 5 days that she spent at her mother’s bedside were eye-opening for an oncologist used to being on the other side of the clinician–patient relationship.

“As a physician, I thought I had a unique perspective of things that were done well – and things that were not,” commented Pamela Kunz, MD.

Dr. Kunz, who was named the 2021 Woman Oncologist of the Year, is director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and of the Yale Cancer Center, New Haven, Conn.

But she was propelled into quite a different role when her mother was admitted to the hospital.

Her mom, who has trouble hearing, was easily confused by jargon and by “all of the people coming in and out with no introductions,” she explained.

“She needed someone to translate what was going on because she didn’t feel well,” she added.

Seeing inpatient care through her mother’s eyes was enlightening, and at times it was “shocking to be on the other side.”

Physicians get used to “checking boxes, getting through the day,” she said. “It’s easy to forget the human side.”

“Seeing a loved one sick, [struggling] through this – I just wished I had seen things done differently,” added Dr. Kunz.

The experience prompted Dr. Kunz to share several “communication pearls” via Twitter. Her thread has since garnered thousands of “likes” and scores of comments and retweets.

She began the Twitter thread explaining what prompted her comments:

“I spent many hours last week observing the practice of medicine while sitting at my mom’s hospital bedside and was reminded of some important communication pearls. Some musings ...”

“1. Introduce yourself by full name, role, and team and have ID badges visible. It can get very confusing for [patients] and family members with the number of people in and out of rooms. E.g. ‘My name is Dr. X. I’m the intern on the primary internal medicine team.’

2.  End your patient visit with a summary of the plan for the day.

3. Avoid medical jargon & speak slowly, clearly, and logically. Remember you are a teacher for your [patients] and their family.

4. Masks make it harder to hear, especially for [patients] with hearing loss (and they no longer have the aid of lip reading).

5. Many older [patients] get confused in the hospital. Repetition is a good thing.

6. Speak to a family member at least once per day to relay the plan.

7. Try to avoid last minute or surprise discharges – they make [patients] and family members anxious. Talk about discharge planning from day 1 and what milestones must occur prior to a safe discharge. ‘In order for you to leave the hospital, X, Y, X must happen.’

8. Talk with your [patients] about something other than what brought them to the hospital (a tip I once learned from a wise mentor).

9.  When possible, sit at eye level with your patient (I love these stools from @YNHH).

10. Take time to listen.”

Dr. Kunz closed with her golden rule: “Lastly, treat your patients how you would want your own family member treated.”

Twitter user @BrunaPellini replied: “I love this, especially ‘Treat your patients how you would want your own family member treated.’ My mom and grandma always said that to me since I was a med student, and this is definitely one of my core values.”

Other clinicians shared similar experiences, and some added to Dr. Kunz’s list.

“Agree entirely, love the list – and while none of us can always practice perfectly, my experiences with my own mother’s illness taught me an enormous amount about communication,” @hoperugo responded.

Twitter user @mariejacork added: “Everyone in health care please read ... if you are lucky enough to not have had a loved one unwell in hospital, these may get forgotten. Having sat with my dad for a few days before he died a few years ago, I felt a lot of these, and it changed my practice forever.”

@bjcohenmd provided additional advice: “And use the dry erase board that should be in every room. Never start a medication without explaining it. Many docs will see the patient and then go to the computer, decide to order a med, but never go back to explain it.”

Patients also shared experiences and offered suggestions.

“As a chronic pain patient I’d add – we know it’s frustrating you can’t cure us but PLEASE do not SIGH if we say something didn’t work or [tell] us to be more positive. Just say ‘I know this is very hard, I’m here to listen.’ We don’t expect a cure, we do expect to be believed,” said @ppenguinsmt. “It makes me feel like I’m causing distress to you if I say the pain has been unrelenting. I leave feeling worse. ...You may have heard 10 [people] in pain before me but this is MY only [appointment].”

Twitter user @KatieCahoots added: “These are perfect. I wish doctors would do this not only in the hospital but in the doctor’s office, as well. I would add one caveat: When you try not to use medical jargon, don’t dumb it down as though I don’t know anything about science or haven’t done any of my own research.”

Dr. Kunz said she was taken aback but pleased by the response to her Tweet.

“It’s an example of the human side of medicine, so it resonates with physicians and with patients,” she commented. Seeing through her mom’s eyes how care was provided made her realize that medical training should include more emphasis on communication, including “real-time feedback to interns, residents, fellows, and students.”

Yes, it takes time, and “we don’t all have a lot of extra time,” she acknowledged.

“But some of these elements don’t take that much more time to do. They can help build trust and can, in the long run, actually save time if patients understand and family members feel engaged and like they are participants,” she said. “I think a little time investment will go a long way.”

In her case, she very much appreciated the one trainee who tried to call her and update her about her mother’s care each afternoon. “I really valued that,” she said.

A version of this article first appeared on Medscape.com.

Just about every patient-reported outcome favors adding immunotherapy to standard chemotherapy for patients with metastatic non-small cell lung cancer, according to an analysis presented at the annual European Lung Cancer Congress (ELCC) on March 30.

“Overall, it is very clear that chemotherapy plus immunotherapy prolongs the time to symptom deterioration and actually improves symptoms” in this patient population, said study discussant Luis Paz-Ares, MD, PhD, chair of medical oncology at the Hospital Universitario 12 de Octubre, Madrid, who was not involved in the research.

Last September, investigators reported efficacy outcomes from the phase 3 POSEIDON trial, which randomized 1,013 patients with EGFR/ALK wild-type mNSCLC to one of three first-line options: chemotherapy alone, chemotherapy plus the checkpoint inhibitor durvalumab, or chemotherapy plus two check-point inhibitors, durvalumab and tremelimumab. The analysis showed improved progression-free survival in both immunotherapy arms as well as a significant 2.3-month overall survival advantage with dual immunotherapy and a nonsignificant 1.6-month advantage with single agent durvalumab.

At the ELCC meeting, study presenter and lead investigator Edward Garon, MD, reported the latest data on the trial’s secondary endpoints: patient-reported outcomes. Global health status, functioning, and symptom scores were assessed using two questionnaires, the EORTC QLQ-C30 and EORTC QLQ-LC13.

Overall, Dr. Garon and colleagues reported a longer time to deterioration in all three areas – global health status, functioning, and symptoms – for patients who received immunotherapy versus chemotherapy alone, with similar results in both immunotherapy arms.

Time to deterioration in global health status, for instance, was a median of about 8 months on both immunotherapy regimens versus 5.6 months with chemotherapy alone. The positive findings held for many patient-reported treatment side effects, including dyspnea, hemoptysis, nausea/vomiting, and insomnia, but the benefits of adding immunotherapy weren’t always statistically significant.

Adding one or both checkpoint inhibitors to chemotherapy “improved efficacy while delaying deterioration in symptoms, functioning, and [health-related quality of life] versus chemotherapy alone in patients with mNSCLC,” concluded Dr. Garon, a thoracic medical oncologist at the University of California, Los Angeles. Plus, he added, “the pattern was observed across nearly all prespecified symptoms and domains of interest.”

According to study discussant Dr. Paz-Ares, “the data seem to be very consistent with all the trials asking similar questions.” The important thing here is figuring out the ideal candidates for dual inhibitor therapy, he said.

With positive efficacy and patient-reported outcomes for single and dual immunotherapy in this trial, it’s a “relatively straightforward” decision to add immunotherapy to chemotherapy for patients with mNSCLC, Massimo Di Maio, a medical oncologist at the University of Turin, Italy, said in an editorial on the ELCC’s news site.

However, that’s not always the case for every cancer type, which makes patient-reported outcomes “crucial” for determining the right treatment for each patient. Some might opt for a modest survival benefit regardless of the side effects, while others might favor a less toxic approach, even it means not living quite as long, he said.

The problem, he stressed, is that trials often release efficacy data well before patient-reported outcomes, which makes weighing the benefits and risks of a treat-ment option more difficult. The delay between efficacy and patient-reported outcome data was about 6 months in the POSEIDON trial.

“Timing is key when it comes to using [patient reported outcomes] for decision-making in oncology,” Dr. Di Maio said. “In fact, to enable a full assessment of a treatment, results should be published concurrently with the efficacy and safety data. Unfortunately, this is generally not the case.”

POSEIDON was funded by AstraZeneca, which markets durvalumab and is developing tremelimumab. Dr. Garon reported grants from the company. Dr. Paz-Ares reported honoraria and institutional research grants from AstraZeneca. Dr. Di Maio is a consultant for AstraZeneca and reported receiving honoraria and personal fees from the company.

Just about every patient-reported outcome favors adding immunotherapy to standard chemotherapy for patients with metastatic non-small cell lung cancer, according to an analysis presented at the annual European Lung Cancer Congress (ELCC) on March 30.

“Overall, it is very clear that chemotherapy plus immunotherapy prolongs the time to symptom deterioration and actually improves symptoms” in this patient population, said study discussant Luis Paz-Ares, MD, PhD, chair of medical oncology at the Hospital Universitario 12 de Octubre, Madrid, who was not involved in the research.

Last September, investigators reported efficacy outcomes from the phase 3 POSEIDON trial, which randomized 1,013 patients with EGFR/ALK wild-type mNSCLC to one of three first-line options: chemotherapy alone, chemotherapy plus the checkpoint inhibitor durvalumab, or chemotherapy plus two check-point inhibitors, durvalumab and tremelimumab. The analysis showed improved progression-free survival in both immunotherapy arms as well as a significant 2.3-month overall survival advantage with dual immunotherapy and a nonsignificant 1.6-month advantage with single agent durvalumab.

At the ELCC meeting, study presenter and lead investigator Edward Garon, MD, reported the latest data on the trial’s secondary endpoints: patient-reported outcomes. Global health status, functioning, and symptom scores were assessed using two questionnaires, the EORTC QLQ-C30 and EORTC QLQ-LC13.

Overall, Dr. Garon and colleagues reported a longer time to deterioration in all three areas – global health status, functioning, and symptoms – for patients who received immunotherapy versus chemotherapy alone, with similar results in both immunotherapy arms.

Time to deterioration in global health status, for instance, was a median of about 8 months on both immunotherapy regimens versus 5.6 months with chemotherapy alone. The positive findings held for many patient-reported treatment side effects, including dyspnea, hemoptysis, nausea/vomiting, and insomnia, but the benefits of adding immunotherapy weren’t always statistically significant.

Adding one or both checkpoint inhibitors to chemotherapy “improved efficacy while delaying deterioration in symptoms, functioning, and [health-related quality of life] versus chemotherapy alone in patients with mNSCLC,” concluded Dr. Garon, a thoracic medical oncologist at the University of California, Los Angeles. Plus, he added, “the pattern was observed across nearly all prespecified symptoms and domains of interest.”

According to study discussant Dr. Paz-Ares, “the data seem to be very consistent with all the trials asking similar questions.” The important thing here is figuring out the ideal candidates for dual inhibitor therapy, he said.

With positive efficacy and patient-reported outcomes for single and dual immunotherapy in this trial, it’s a “relatively straightforward” decision to add immunotherapy to chemotherapy for patients with mNSCLC, Massimo Di Maio, a medical oncologist at the University of Turin, Italy, said in an editorial on the ELCC’s news site.

However, that’s not always the case for every cancer type, which makes patient-reported outcomes “crucial” for determining the right treatment for each patient. Some might opt for a modest survival benefit regardless of the side effects, while others might favor a less toxic approach, even it means not living quite as long, he said.

The problem, he stressed, is that trials often release efficacy data well before patient-reported outcomes, which makes weighing the benefits and risks of a treat-ment option more difficult. The delay between efficacy and patient-reported outcome data was about 6 months in the POSEIDON trial.

“Timing is key when it comes to using [patient reported outcomes] for decision-making in oncology,” Dr. Di Maio said. “In fact, to enable a full assessment of a treatment, results should be published concurrently with the efficacy and safety data. Unfortunately, this is generally not the case.”

POSEIDON was funded by AstraZeneca, which markets durvalumab and is developing tremelimumab. Dr. Garon reported grants from the company. Dr. Paz-Ares reported honoraria and institutional research grants from AstraZeneca. Dr. Di Maio is a consultant for AstraZeneca and reported receiving honoraria and personal fees from the company.

Just about every patient-reported outcome favors adding immunotherapy to standard chemotherapy for patients with metastatic non-small cell lung cancer, according to an analysis presented at the annual European Lung Cancer Congress (ELCC) on March 30.

“Overall, it is very clear that chemotherapy plus immunotherapy prolongs the time to symptom deterioration and actually improves symptoms” in this patient population, said study discussant Luis Paz-Ares, MD, PhD, chair of medical oncology at the Hospital Universitario 12 de Octubre, Madrid, who was not involved in the research.

Last September, investigators reported efficacy outcomes from the phase 3 POSEIDON trial, which randomized 1,013 patients with EGFR/ALK wild-type mNSCLC to one of three first-line options: chemotherapy alone, chemotherapy plus the checkpoint inhibitor durvalumab, or chemotherapy plus two check-point inhibitors, durvalumab and tremelimumab. The analysis showed improved progression-free survival in both immunotherapy arms as well as a significant 2.3-month overall survival advantage with dual immunotherapy and a nonsignificant 1.6-month advantage with single agent durvalumab.

At the ELCC meeting, study presenter and lead investigator Edward Garon, MD, reported the latest data on the trial’s secondary endpoints: patient-reported outcomes. Global health status, functioning, and symptom scores were assessed using two questionnaires, the EORTC QLQ-C30 and EORTC QLQ-LC13.

Overall, Dr. Garon and colleagues reported a longer time to deterioration in all three areas – global health status, functioning, and symptoms – for patients who received immunotherapy versus chemotherapy alone, with similar results in both immunotherapy arms.

Time to deterioration in global health status, for instance, was a median of about 8 months on both immunotherapy regimens versus 5.6 months with chemotherapy alone. The positive findings held for many patient-reported treatment side effects, including dyspnea, hemoptysis, nausea/vomiting, and insomnia, but the benefits of adding immunotherapy weren’t always statistically significant.

Adding one or both checkpoint inhibitors to chemotherapy “improved efficacy while delaying deterioration in symptoms, functioning, and [health-related quality of life] versus chemotherapy alone in patients with mNSCLC,” concluded Dr. Garon, a thoracic medical oncologist at the University of California, Los Angeles. Plus, he added, “the pattern was observed across nearly all prespecified symptoms and domains of interest.”

According to study discussant Dr. Paz-Ares, “the data seem to be very consistent with all the trials asking similar questions.” The important thing here is figuring out the ideal candidates for dual inhibitor therapy, he said.

With positive efficacy and patient-reported outcomes for single and dual immunotherapy in this trial, it’s a “relatively straightforward” decision to add immunotherapy to chemotherapy for patients with mNSCLC, Massimo Di Maio, a medical oncologist at the University of Turin, Italy, said in an editorial on the ELCC’s news site.

However, that’s not always the case for every cancer type, which makes patient-reported outcomes “crucial” for determining the right treatment for each patient. Some might opt for a modest survival benefit regardless of the side effects, while others might favor a less toxic approach, even it means not living quite as long, he said.

The problem, he stressed, is that trials often release efficacy data well before patient-reported outcomes, which makes weighing the benefits and risks of a treat-ment option more difficult. The delay between efficacy and patient-reported outcome data was about 6 months in the POSEIDON trial.

“Timing is key when it comes to using [patient reported outcomes] for decision-making in oncology,” Dr. Di Maio said. “In fact, to enable a full assessment of a treatment, results should be published concurrently with the efficacy and safety data. Unfortunately, this is generally not the case.”

POSEIDON was funded by AstraZeneca, which markets durvalumab and is developing tremelimumab. Dr. Garon reported grants from the company. Dr. Paz-Ares reported honoraria and institutional research grants from AstraZeneca. Dr. Di Maio is a consultant for AstraZeneca and reported receiving honoraria and personal fees from the company.

In patients undergoing transcatheter aortic valve replacement (TAVR), the incidence of leaflet thrombosis was numerically lower in those treated with the anticoagulant edoxaban for 6 months after the procedure than in those who received dual antiplatelet therapy, although the difference was not statistically significant, in the ADAPT-TAVR study.

There was no difference in new cerebral thromboembolism or neurologic/neurocognitive function between the two groups in the study.

Also, there was no significant relation between subclinical leaflet thrombosis and increased risk for cerebral thromboembolism and neurologic dysfunction.

The ADAPT-TAVR trial was presented April 4 at the American College of Cardiology (ACC) 2022 Scientific Session by Duk-Woo Park, MD, Asan Medical Center, Seoul, South Korea. It was simultaneously published online in Circulation.

A version of this article first appeared on Medscape.com.

In patients undergoing transcatheter aortic valve replacement (TAVR), the incidence of leaflet thrombosis was numerically lower in those treated with the anticoagulant edoxaban for 6 months after the procedure than in those who received dual antiplatelet therapy, although the difference was not statistically significant, in the ADAPT-TAVR study.

There was no difference in new cerebral thromboembolism or neurologic/neurocognitive function between the two groups in the study.

Also, there was no significant relation between subclinical leaflet thrombosis and increased risk for cerebral thromboembolism and neurologic dysfunction.

The ADAPT-TAVR trial was presented April 4 at the American College of Cardiology (ACC) 2022 Scientific Session by Duk-Woo Park, MD, Asan Medical Center, Seoul, South Korea. It was simultaneously published online in Circulation.

A version of this article first appeared on Medscape.com.

In patients undergoing transcatheter aortic valve replacement (TAVR), the incidence of leaflet thrombosis was numerically lower in those treated with the anticoagulant edoxaban for 6 months after the procedure than in those who received dual antiplatelet therapy, although the difference was not statistically significant, in the ADAPT-TAVR study.

There was no difference in new cerebral thromboembolism or neurologic/neurocognitive function between the two groups in the study.

Also, there was no significant relation between subclinical leaflet thrombosis and increased risk for cerebral thromboembolism and neurologic dysfunction.

The ADAPT-TAVR trial was presented April 4 at the American College of Cardiology (ACC) 2022 Scientific Session by Duk-Woo Park, MD, Asan Medical Center, Seoul, South Korea. It was simultaneously published online in Circulation.

A version of this article first appeared on Medscape.com.

This transcript of a video roundtable, which is available on Medscape.com, has been edited for clarity.

Hope S. Rugo, MD: Hello. I’m Hope Rugo, a breast medical oncologist from the University of California, San Francisco. I’m joined here by three of my friends and colleagues to discuss the toxicity of new agents in the treatment of breast cancer. Fatima, do you want to start by introducing yourself?

Fatima F. Cardoso, MD: Sure. Hello, everyone. I’m Fatima Cardoso, a breast medical oncologist in Lisbon, Portugal.

Dr. Rugo: Sheila.

Sheila Pettiford: Hi, I’m Sheila Pettiford. I am a metastatic [breast cancer] patient and have been for almost 8 years in April. I used to live in Philadelphia, Pennsylvania, but moved to Delaware in the last couple of years during the pandemic. I’m happy to be here.

Dr. Rugo: Julia.

Julia Maués: Hi, everyone. I also am a person living with metastatic breast cancer. I was diagnosed in 2013, so it’s going to be 9 years, also in April.
 

Effective monitoring and management of side effects: A team effort

Dr. Rugo: We have an amazing group and an international representation, which is also really nice because we get different perspectives. What we’re going to talk about is important to providers and patients across the board. With the host of new agents for the treatment of breast cancer – most of which have really moved us forward in terms of having effective treatment options – we’ve also been faced with a lot of new toxicities or side effects that we haven’t seen before or that we might not have expected from the specific agent.

Those toxicities across the board include side effects that are quite familiar to us, like low blood counts, but we may not advise people well enough about other side effects such as mouth sores, inflammation of the lungs, immune toxicities, and skin toxicities.

Fatima, do you want to start and talk about how we can think about these toxicities and address them?

Dr. Cardoso: Sure. Thank you. From the health care provider point of view, what I would highlight is to educate. Educate before we start the treatment. It’s very important to inform the patient but in a balanced way, so we don’t overexaggerate certain types of side effects or underestimate certain types of side effects.

It’s very important because an informed patient will be attentive to the types of side effects that can happen. Also, teach the patient when it is a [cause for] alarm or something for which they might need to contact their health care team and when that’s not the case. I think this is one crucial topic.

The other one is to monitor. Find ways how to best communicate between the patient and the health care team but in a way that you can monitor, so you can act very early on. Most of these new side effects, if you act early on, will not become severe. It is very important to know about them and to act early on.

I believe there is something important that we don’t think about all the time, and that is prophylaxis. Do not be shy about using prophylactic measures, be it for the mouth sores, nausea and vomiting, diarrhea, and other things that really impact the quality of life of patients. Those, to start, are my three major points of attention for health care professionals.

Dr. Rugo: I think that’s so incredibly important – the comments that you’ve made – and also that prevention and prophylaxis are so important. You don’t want to have a patient have diarrhea in the middle of the night and not have any antipropulsive agents at home. Just as a very straightforward example, it’s really important.

Also, the ability to know what you should be looking for and how you can manage it [is important]. There are many examples of times when, even with some education, providers may not have communicated well to the patient. Then the patient is surprised and unhappy with the situation and unable to manage it.
 

The importance of education

Sheila, your comments on this from the patient perspective are so important. How important is the education piece, and how do you manage the fear of side effects vs actually managing the side effects that might be caused by the treatment you’re taking?

Ms. Pettiford: Thank you for that question. I really think it’s a dance. It’s a dance between the patient and the health care team. Yes, education is absolutely important. However, the health care professionals have to establish a relationship of trust with the patient. My own circumstances were that – and I was very fortunate in that my oncologist, who I chose just by looking and not by a recommendation – I did find an oncologist who listened to me.

When it came time for me to deal with a new medication, the education she provided me was sufficient because of the fact that there was a lot of listening that had gone on prior to the new medicine being given to me. I trusted what I was hearing, and it felt like there was a balanced situation that came about from what I was being told. I could look it up, too.

There still is that part of the patient who will be participating in the process, as well. They can still look up things, and that’s one of the downfalls of the information age we are in. It is a dance. I just want to go back to that. There’s a dance between the patient and the health care providers.

Dr. Rugo: Julia, from the patient’s side, how do you balance the benefits you might get from a treatment versus the side effects and how best to manage them?

Ms. Maués: I think it’s interesting that when we talk with our doctors, and especially when we read about a certain treatment, the attention is focused on the very severe and unlikely side effects that a drug has. We don’t talk as much about the side effects that are most likely to happen and will affect us but may not be life threatening.

Especially for those of us with metastatic cancer who are going to ideally be on a drug for a very long time, we’re then faced with low-grade nausea for the rest of our lives. That’s not okay either, right? I think it’s important to talk about all of the levels of toxicities and everything that can be done to avoid this.
 

Communication is key

Ms. Pettiford: I just want to add something that Dr. Cardoso said about monitoring that is absolutely important. We’re in a day and time when it’s very difficult to get someone on the telephone, but we do have digital charts and other ways that monitoring can take place. I was at a large teaching university, and I had to go monthly for my treatments. Every month, there were questions that were asked about my life and my condition. I could always get in contact with somebody through the digital chart.

Dr. Rugo: That’s an incredibly important comment, Sheila, about communication and how patients can feel like they have someone to go to in real time who can help manage things. Fatima, I’m interested in your comment on that.

Also, just to go to the next step, which is that when we see data reported on clinical trials and how the agent we’ve added or substituted is better than the standard, the toxicity tables are side effects that occur in at least 10% or more patients and sometimes even 20%. Then they’re graded, where often the division is grade 3 or greater. That may not actually reflect much about what the individual patient experience is. How do we interpret these data? Communication and interpretation?

Dr. Cardoso: Absolutely. I always call attention that perhaps, since we focus so much on grade 3 and 4 [side effects], that is the reason why we don’t see in the usual reporting differences quality of life between treatments. Quality of life is affected also significantly by grade 2 side effects. Or, like Julia was mentioning, even grade 1, if they are persistent, will eventually affect your quality of life.

Sometimes, like I was saying, don’t underestimate – it’s a little bit like that. We focus on explaining, “Look, this new immunotherapy can give you all these different side effects.” But then we forget to say: “Oh, by the way, it may also give you some nausea.” Actually, the nausea will affect the patient’s quality of life. I think that’s why it is so important to balance the way we provide the information.

I would like also to take on what Sheila said that sometimes too much information is not very helpful. That’s why sometimes we have to go stepwise. The first time you’re about to start the treatment, advise [the patient] on the most frequent side effects. Later on, you have time to say: “Okay, by the way, this can also give a rare side effect. This is what you should look for. If you have it, please contact your health care team.”

I think the most difficult part, at least from my experience, is for patients to understand what is really a sign of a severe side effect and what is normal for that type of treatment. Some of the new ways of communicating, like using some patient-reported outcome (PRO) apps, actually help the patient by saying, “This that you are feeling is normal. It can wait for your next appointment. This that you are feeling, it’s better if you try to reach your health care team right away. Or, this is an urgent thing and go to the emergency room near you.”

For this kind of triage, there are now new apps that can help. I think this is the most difficult part because when you are a patient, you don’t know if what you are feeling is actually a sign of something very severe or if it’s normal for the type of treatment you are receiving.

Dr. Rugo: I think that’s so important, and these new PRO apps may help with this. Of course, nothing substitutes for talking in the end if you’re confused or it doesn’t fit into whatever’s in that paradigm. I think it’s important.
 

Best practices in focusing on the individual patient

Julia, what do you think the best way of educating the patient is when you’re going to start a new treatment? You might be newly diagnosed with cancer or you might have had cancer for a number of years. You’re going to start a new treatment. What’s the best way to know what to look for and how to manage it?

Ms. Maués: I think the key here is that everyone’s different, so have that conversation, the doctor and the patient, about what the best way [of education] is for that specific person. Do they want a flyer listing all of the side effects? Do they want a link to a video they can watch and understand? Do they want someone to come in and give an extra explanation about things? Everyone learns so differently, and I think it’s really hard to assume there’s one way that all patients will understand.

I think the PRO apps are great, and also another benefit is that you keep track of your side effects. Sometimes we don’t even remember well. When did you have nausea? Was it in the morning? Was it in the evening? Is it every day? If you track it with these apps, then you will have the data stored there in the form to answer those questions.

Dr. Cardoso: There was recently a publication – I found it quite interesting – from Lesley Fallowfield’s group saying that the majority of patients would better absorb the information if it is not just text, but if it somehow has a video component, an image, or an infographic that would help them memorize a little bit more information.

Dr. Rugo: There’s been a move toward trying to make videos because the amount of education that’s needed on the providers’ side from our nurses and advanced practice providers may be overwhelming, so things might get missed. The idea of having videos to get everybody on the same page is very popular right now for this reason, and Lesley’s work is really groundbreaking.

Sheila, what do you think is the best way to communicate information?

Ms. Pettiford: Well, I definitely think it’s important for the doctors to recognize, as Julia said, that everyone is different, and all their patients are different. They could come with the same exact subtype of whatever cancer they have – in this situation, breast cancer – and still have so many different reactions. It’s so important for everybody on the health care team to listen to what the patient says because the patient is the one who is living with the illness and knows their body, hopefully.

It’s just one of those things. It’s not a one-size-fits-all situation. You give the standards, but I think it’s important to offer various ways of communicating to a patient because some people are visual. Some people want an overwhelming amount of information so they can sort through it. Then, you have some people who just want the bullet points. Again, it is important not to try to do it as a one-size-fits-all type thing.

Dr. Rugo: Yes, that’s such a good point. I’m always struck by the fact that some patients are totally on top of it and listen to it all, and then other people, we just can’t get them to even call in regarding their side effects. In some ways, it’s frightening for people to call in with issues. Maybe they’re afraid they won’t get the treatment, or that it is related to their cancer progressing, too. Trying to meet people on their own level is a real challenge and an important one.

We talked about education for providers. Fatima, how should we be best educating for these new drugs and new side effects? So many different manifestations can occur, and as we talked about, they might be quite uncommon. We just want people to keep their ears up for any kind of unusual toxicity we see. We all know that the presentation of efficacy data is not adequate for education.

Dr. Cardoso: When we present a new treatment, we focus usually on efficacy, right? Then we say a few things about safety, particularly if there is a new or a severe side effect, but we don’t go through details on how to best manage this in clinical practice.

Anecdotally, I remember that I contacted you because I was going to start using a new treatment and you had some experience. I asked, “What about nausea and vomiting? What do you do for prophylaxis?” I couldn’t find it anywhere in the manuscripts or the presentations. I think we need to focus a little bit more on practical tips. If you are about to start this new treatment, what you should think about and not just the very severe and rare side effects?

Of course, as health care professionals, we need to keep this in our minds. For example, with immunotherapy, side effects can often occur even after stopping the treatment. For other types of new treatments, we need to gain knowledge about endocrinology, for example, which is something that oncologists wouldn’t have to deal with that often in the past. Now, new skills are needed.

It’s also what makes our profession so exciting. There’s always something new to learn, and I like to look at it from that perspective. It’s not boring at all. We are always learning new things.

Dr. Rugo: Indeed. Certainly, you and I have worked together on trying to encourage our pharmaceutical colleagues to publish these papers alongside their urgency of distributing the efficacy data and publishing the papers on efficacy, and also to do a nitty-gritty review of safety and talk about management strategies. I’m really pleased that there seems to be a little more focus on that earlier now in the drug process – although still not early enough – but it’s getting there. That’s a good thing.

Ms. Pettiford: Julia, you mentioned earlier how important it is for the individual patient’s quality of life to understand how these side effects can affect them. It really is one of those things in which we have to make personal decisions. What might be good for one person in terms of what happens with side effects, and their ability to function might not work with someone else.

If you are a person who’s dealing with metastatic disease who has children, a household, a dog, and a cat to take care of, what I can handle being that I’m a single person is not what they can handle. That’s all a part of the education piece. That’s all part of the teamwork. That’s all part of the communication process. It all comes into play.

Dr. Rugo: That’s such an incredibly important point. As we’re wrapping up, it would be great if everybody had some points to make that pulled together some of our conversation. Julia, do you want to start?

Ms. Maués: Yes, I was going to add specifically about the topic you were just discussing, with all that an oncologist’s team has to know and all the different areas of our health being affected by these new treatments. One tip for patients and their teams is that the other providers around the patients may not be as informed about the disease and the treatments they are on. Sometimes we patients end up getting information that isn’t up to date with the latest drugs and things like that.

When we do talk with someone about our issues, make sure they are informed about the new drugs. For example, we often have skin issues. There are dermatologists that work with cancer patients often, and they’re very informed about the side effects that come with these drugs. There are others who never see these sorts of issues and may assume it’s something completely different.

I usually just go to doctors that my oncologist’s team collaborates with and gets referrals from because they send their patients to these doctors often. These are doctors that see cancer patients. We’re a very unique group.

Dr. Rugo: That’s a really good point. I have the same thing. We all have a little stable of people we refer to for various issues that we can reach on speed dial.
 

The importance of diversity in clinical trials to obtain the most useful outcomes

Fatima, there’s recently been, appropriately so, more of a push to try and evaluate side effects by racial and ethnic subgroups. I think we’re still pretty crummy at it, but we are making some progress. How important is that to you when you think about patients and managing them?

Dr. Cardoso: I think this is quite important. One area of research that is underused, really, is all the new genomics and sequencing technologies to understand why people react differently to the same treatment. Why is it that for some people, either for ethnic or other reasons, you have a different metabolism or something else that justifies a very high rate of side effects from a certain treatment, whereas in other regions of the world this doesn’t happen?

Not to go into these new drugs, but when using a very old drug like a taxane, I found a difference in reaction between the Portuguese patients and the Belgian patients, the two countries where I’ve worked. I even found that the cause might be genetic because the Portuguese living in Belgium reacted differently than the Belgians themselves.

Maybe there is something in the genetics that justifies the type of side effects that you have. I make a plea also for us to dedicate research to understanding why certain side effects are related to race and others are related to maybe some other types of genetic alterations that will lead to an increased side effect.

Dr. Rugo: Sheila, comments?

Ms. Pettiford: That is just excellent. It’s excellent to even consider it because it is so obvious. To me, it’s an obvious situation because there are things that are underneath the skin that we don’t understand. We have to take that into consideration when we are dealing with all these wonderful – I call them miracle – drugs that have come about in the last 20 years.

There still is much more to be done, and I try to participate in any type of organization that’s encouraging diversity in clinical trials because you need to have people of all different ethnicities in order for us to get to these answers. It’s fascinating that you found this out, doctor.
 

The patient-centered dosing initiative

Ms. Maués: I have the pleasure of being a member of a patient-led initiative called the Patient-Centered Dosing Initiative (PCDI). We are highlighting the discussion around dosages of drugs, especially in the metastatic setting. Metastatic breast cancer is what we’re focusing on, although it could apply to any type of cancer. We are advised by a number of wonderful, world-renowned physicians, Dr. Rugo being one of them. Anne Loeser, the leader of our group, has spoken at ASCO about this topic of dosage. What we’re seeing is that the dosage determination for oncology drugs is still done in the same way it used to be done decades ago and mostly with the curative intent of early-stage disease — metastatic cancer patients back then really didn’t live long at all. What we’re seeing right now is people with metastatic breast cancer that are able to, in some cases, live a long life managing their disease.

Patients are put on doses that are too high for them to be able to manage the side effects, and then they end up having to go off the drug, which means they have lost one of the tools in their toolbox. So, what we like to say about dosing is that, for metastatic cancer patients, it’s a marathon and not a sprint. If we throw all the poison at the patient from the very beginning, they won’t be able to take this for a very long time. And in the metastatic setting, the goal is to stay on each therapy for as long as possible. If we burn one of the cards early on, you have to move on to the next one. This is finite because at some point, there are not enough drugs that can help a particular patient. The PCDI is really getting a lot of visibility with the FDA and experts. People are talking more about dosages, and the FDA is now providing guidance for pharmaceutical companies to study different dosages in the clinical trials from the very beginning. This initiative is almost 3 years old, and we have made a tremendous impact since then.

Dr. Rugo: I think this is an incredibly important area moving forward, and thankfully, there’s so much interest now in not only promoting diversity, enrollment in trials, and education to promote diversity but also in looking at differences in efficacy and side effects.

I’ll just thank everybody for your contributions and amazing perspectives in this incredibly important area. As we move forward with better agents, we need to also make sure we’re understanding what the side effects are, managing them, and hearing the voices of our patients. Thanks very much.

Ms. Pettiford: Thank you so much.

Dr. Cardoso: Thank you.

Ms. Maués: Thank you.
 

Editor’s note: Our panelists would like to highlight these points:

• The patient and the health care team must build trust with each other. 
• African Americans have historical reasons for not trusting the health care industry. Much outreach is still needed. 
• Inform and educate before the start of treatment and during the treatment.
• Be balanced and do not underestimate common side effects or overestimate rare ones. Adapt the amount and the detail of the information to the wishes of the individual patient. Offer various methods of delivery (e.g., videos, pamphlets, fact sheets).
• Patients will research their condition and treatments online. Instead of trying to stop this, help them find the best sources.
• Patients will connect with others in the patient community and learn from each other’s experiences. Keep in mind that everyone is different, and decisions should always be made together with the medical team.
• Monitor patients regularly, especially during the first few treatment cycles.
• Use different forms of communication between the patient and health care providers (e.g., apps, digital charts, oncology nurses/nurse navigators, responsive oncologists, different forms of telemedicine), but don’t forget to speak directly with the patient.
• The use of new PRO apps can be very useful to help patients differentiate between urgent and nonurgent signs and symptoms.
• As much as possible, use preventive/prophylactic measures, namely for nausea, vomiting, diarrhea/constipation, and mucositis.
• Be aware of late side effects, especially with immunotherapy.
• Don’t forget that grade 1-2 side effects can substantially impact quality of life, particularly if they are persistent.
• Consider quality-of-life issues for each patient. What is acceptable for one patient may not be for another.
• Learn how to manage new and specific side effects (e.g., endocrine, skin related, pneumonitis).
• Keep an open dialogue about treatment and side effects. Things can change, and there are different ways to address issues such as medications for side effects and dosing changes.
• Listen to your patient and respond in a timely fashion.
• Ethnicity and genetics should be studied as a factor for individual side effects. Standard industry dosages of a new anticancer medication might not be as effective in one ethnic group as another due to the lack of diversity in clinical trials.
• Medications with hard-to-manage or dangerous side effects may be counterproductive regardless of effectiveness.
• Cancer treatment varies vastly depending on region and type of treatment facility. There are many unmet needs in rural areas because of lack of oncology personnel, finances, transportation, etc.

Dr. Rugo is a professor in the department of medicine, University of California San Francisco Comprehensive Cancer Center; director, Breast Oncology and Clinical Trials Education, Cancer Infusion Services, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco. Dr. Cardoso is director, breast unit, Champalimaud Clinical Centre, Lisbon. Financial disclosures for both Dr. Rugo and Dr. Cardoso are available on Medscape.com, where this article first appeared. Julia Maués is a patient in Washington. She has disclosed no relevant financial relationships. Sheila Pettiford is a patient in Middletown, Del. She has disclosed no relevant financial relationships.

This transcript of a video roundtable, which is available on Medscape.com, has been edited for clarity.

Hope S. Rugo, MD: Hello. I’m Hope Rugo, a breast medical oncologist from the University of California, San Francisco. I’m joined here by three of my friends and colleagues to discuss the toxicity of new agents in the treatment of breast cancer. Fatima, do you want to start by introducing yourself?

Fatima F. Cardoso, MD: Sure. Hello, everyone. I’m Fatima Cardoso, a breast medical oncologist in Lisbon, Portugal.

Dr. Rugo: Sheila.

Sheila Pettiford: Hi, I’m Sheila Pettiford. I am a metastatic [breast cancer] patient and have been for almost 8 years in April. I used to live in Philadelphia, Pennsylvania, but moved to Delaware in the last couple of years during the pandemic. I’m happy to be here.

Dr. Rugo: Julia.

Julia Maués: Hi, everyone. I also am a person living with metastatic breast cancer. I was diagnosed in 2013, so it’s going to be 9 years, also in April.
 

Effective monitoring and management of side effects: A team effort

Dr. Rugo: We have an amazing group and an international representation, which is also really nice because we get different perspectives. What we’re going to talk about is important to providers and patients across the board. With the host of new agents for the treatment of breast cancer – most of which have really moved us forward in terms of having effective treatment options – we’ve also been faced with a lot of new toxicities or side effects that we haven’t seen before or that we might not have expected from the specific agent.

Those toxicities across the board include side effects that are quite familiar to us, like low blood counts, but we may not advise people well enough about other side effects such as mouth sores, inflammation of the lungs, immune toxicities, and skin toxicities.

Fatima, do you want to start and talk about how we can think about these toxicities and address them?

Dr. Cardoso: Sure. Thank you. From the health care provider point of view, what I would highlight is to educate. Educate before we start the treatment. It’s very important to inform the patient but in a balanced way, so we don’t overexaggerate certain types of side effects or underestimate certain types of side effects.

It’s very important because an informed patient will be attentive to the types of side effects that can happen. Also, teach the patient when it is a [cause for] alarm or something for which they might need to contact their health care team and when that’s not the case. I think this is one crucial topic.

The other one is to monitor. Find ways how to best communicate between the patient and the health care team but in a way that you can monitor, so you can act very early on. Most of these new side effects, if you act early on, will not become severe. It is very important to know about them and to act early on.

I believe there is something important that we don’t think about all the time, and that is prophylaxis. Do not be shy about using prophylactic measures, be it for the mouth sores, nausea and vomiting, diarrhea, and other things that really impact the quality of life of patients. Those, to start, are my three major points of attention for health care professionals.

Dr. Rugo: I think that’s so incredibly important – the comments that you’ve made – and also that prevention and prophylaxis are so important. You don’t want to have a patient have diarrhea in the middle of the night and not have any antipropulsive agents at home. Just as a very straightforward example, it’s really important.

Also, the ability to know what you should be looking for and how you can manage it [is important]. There are many examples of times when, even with some education, providers may not have communicated well to the patient. Then the patient is surprised and unhappy with the situation and unable to manage it.
 

The importance of education

Sheila, your comments on this from the patient perspective are so important. How important is the education piece, and how do you manage the fear of side effects vs actually managing the side effects that might be caused by the treatment you’re taking?

Ms. Pettiford: Thank you for that question. I really think it’s a dance. It’s a dance between the patient and the health care team. Yes, education is absolutely important. However, the health care professionals have to establish a relationship of trust with the patient. My own circumstances were that – and I was very fortunate in that my oncologist, who I chose just by looking and not by a recommendation – I did find an oncologist who listened to me.

When it came time for me to deal with a new medication, the education she provided me was sufficient because of the fact that there was a lot of listening that had gone on prior to the new medicine being given to me. I trusted what I was hearing, and it felt like there was a balanced situation that came about from what I was being told. I could look it up, too.

There still is that part of the patient who will be participating in the process, as well. They can still look up things, and that’s one of the downfalls of the information age we are in. It is a dance. I just want to go back to that. There’s a dance between the patient and the health care providers.

Dr. Rugo: Julia, from the patient’s side, how do you balance the benefits you might get from a treatment versus the side effects and how best to manage them?

Ms. Maués: I think it’s interesting that when we talk with our doctors, and especially when we read about a certain treatment, the attention is focused on the very severe and unlikely side effects that a drug has. We don’t talk as much about the side effects that are most likely to happen and will affect us but may not be life threatening.

Especially for those of us with metastatic cancer who are going to ideally be on a drug for a very long time, we’re then faced with low-grade nausea for the rest of our lives. That’s not okay either, right? I think it’s important to talk about all of the levels of toxicities and everything that can be done to avoid this.
 

Communication is key

Ms. Pettiford: I just want to add something that Dr. Cardoso said about monitoring that is absolutely important. We’re in a day and time when it’s very difficult to get someone on the telephone, but we do have digital charts and other ways that monitoring can take place. I was at a large teaching university, and I had to go monthly for my treatments. Every month, there were questions that were asked about my life and my condition. I could always get in contact with somebody through the digital chart.

Dr. Rugo: That’s an incredibly important comment, Sheila, about communication and how patients can feel like they have someone to go to in real time who can help manage things. Fatima, I’m interested in your comment on that.

Also, just to go to the next step, which is that when we see data reported on clinical trials and how the agent we’ve added or substituted is better than the standard, the toxicity tables are side effects that occur in at least 10% or more patients and sometimes even 20%. Then they’re graded, where often the division is grade 3 or greater. That may not actually reflect much about what the individual patient experience is. How do we interpret these data? Communication and interpretation?

Dr. Cardoso: Absolutely. I always call attention that perhaps, since we focus so much on grade 3 and 4 [side effects], that is the reason why we don’t see in the usual reporting differences quality of life between treatments. Quality of life is affected also significantly by grade 2 side effects. Or, like Julia was mentioning, even grade 1, if they are persistent, will eventually affect your quality of life.

Sometimes, like I was saying, don’t underestimate – it’s a little bit like that. We focus on explaining, “Look, this new immunotherapy can give you all these different side effects.” But then we forget to say: “Oh, by the way, it may also give you some nausea.” Actually, the nausea will affect the patient’s quality of life. I think that’s why it is so important to balance the way we provide the information.

I would like also to take on what Sheila said that sometimes too much information is not very helpful. That’s why sometimes we have to go stepwise. The first time you’re about to start the treatment, advise [the patient] on the most frequent side effects. Later on, you have time to say: “Okay, by the way, this can also give a rare side effect. This is what you should look for. If you have it, please contact your health care team.”

I think the most difficult part, at least from my experience, is for patients to understand what is really a sign of a severe side effect and what is normal for that type of treatment. Some of the new ways of communicating, like using some patient-reported outcome (PRO) apps, actually help the patient by saying, “This that you are feeling is normal. It can wait for your next appointment. This that you are feeling, it’s better if you try to reach your health care team right away. Or, this is an urgent thing and go to the emergency room near you.”

For this kind of triage, there are now new apps that can help. I think this is the most difficult part because when you are a patient, you don’t know if what you are feeling is actually a sign of something very severe or if it’s normal for the type of treatment you are receiving.

Dr. Rugo: I think that’s so important, and these new PRO apps may help with this. Of course, nothing substitutes for talking in the end if you’re confused or it doesn’t fit into whatever’s in that paradigm. I think it’s important.
 

Best practices in focusing on the individual patient

Julia, what do you think the best way of educating the patient is when you’re going to start a new treatment? You might be newly diagnosed with cancer or you might have had cancer for a number of years. You’re going to start a new treatment. What’s the best way to know what to look for and how to manage it?

Ms. Maués: I think the key here is that everyone’s different, so have that conversation, the doctor and the patient, about what the best way [of education] is for that specific person. Do they want a flyer listing all of the side effects? Do they want a link to a video they can watch and understand? Do they want someone to come in and give an extra explanation about things? Everyone learns so differently, and I think it’s really hard to assume there’s one way that all patients will understand.

I think the PRO apps are great, and also another benefit is that you keep track of your side effects. Sometimes we don’t even remember well. When did you have nausea? Was it in the morning? Was it in the evening? Is it every day? If you track it with these apps, then you will have the data stored there in the form to answer those questions.

Dr. Cardoso: There was recently a publication – I found it quite interesting – from Lesley Fallowfield’s group saying that the majority of patients would better absorb the information if it is not just text, but if it somehow has a video component, an image, or an infographic that would help them memorize a little bit more information.

Dr. Rugo: There’s been a move toward trying to make videos because the amount of education that’s needed on the providers’ side from our nurses and advanced practice providers may be overwhelming, so things might get missed. The idea of having videos to get everybody on the same page is very popular right now for this reason, and Lesley’s work is really groundbreaking.

Sheila, what do you think is the best way to communicate information?

Ms. Pettiford: Well, I definitely think it’s important for the doctors to recognize, as Julia said, that everyone is different, and all their patients are different. They could come with the same exact subtype of whatever cancer they have – in this situation, breast cancer – and still have so many different reactions. It’s so important for everybody on the health care team to listen to what the patient says because the patient is the one who is living with the illness and knows their body, hopefully.

It’s just one of those things. It’s not a one-size-fits-all situation. You give the standards, but I think it’s important to offer various ways of communicating to a patient because some people are visual. Some people want an overwhelming amount of information so they can sort through it. Then, you have some people who just want the bullet points. Again, it is important not to try to do it as a one-size-fits-all type thing.

Dr. Rugo: Yes, that’s such a good point. I’m always struck by the fact that some patients are totally on top of it and listen to it all, and then other people, we just can’t get them to even call in regarding their side effects. In some ways, it’s frightening for people to call in with issues. Maybe they’re afraid they won’t get the treatment, or that it is related to their cancer progressing, too. Trying to meet people on their own level is a real challenge and an important one.

We talked about education for providers. Fatima, how should we be best educating for these new drugs and new side effects? So many different manifestations can occur, and as we talked about, they might be quite uncommon. We just want people to keep their ears up for any kind of unusual toxicity we see. We all know that the presentation of efficacy data is not adequate for education.

Dr. Cardoso: When we present a new treatment, we focus usually on efficacy, right? Then we say a few things about safety, particularly if there is a new or a severe side effect, but we don’t go through details on how to best manage this in clinical practice.

Anecdotally, I remember that I contacted you because I was going to start using a new treatment and you had some experience. I asked, “What about nausea and vomiting? What do you do for prophylaxis?” I couldn’t find it anywhere in the manuscripts or the presentations. I think we need to focus a little bit more on practical tips. If you are about to start this new treatment, what you should think about and not just the very severe and rare side effects?

Of course, as health care professionals, we need to keep this in our minds. For example, with immunotherapy, side effects can often occur even after stopping the treatment. For other types of new treatments, we need to gain knowledge about endocrinology, for example, which is something that oncologists wouldn’t have to deal with that often in the past. Now, new skills are needed.

It’s also what makes our profession so exciting. There’s always something new to learn, and I like to look at it from that perspective. It’s not boring at all. We are always learning new things.

Dr. Rugo: Indeed. Certainly, you and I have worked together on trying to encourage our pharmaceutical colleagues to publish these papers alongside their urgency of distributing the efficacy data and publishing the papers on efficacy, and also to do a nitty-gritty review of safety and talk about management strategies. I’m really pleased that there seems to be a little more focus on that earlier now in the drug process – although still not early enough – but it’s getting there. That’s a good thing.

Ms. Pettiford: Julia, you mentioned earlier how important it is for the individual patient’s quality of life to understand how these side effects can affect them. It really is one of those things in which we have to make personal decisions. What might be good for one person in terms of what happens with side effects, and their ability to function might not work with someone else.

If you are a person who’s dealing with metastatic disease who has children, a household, a dog, and a cat to take care of, what I can handle being that I’m a single person is not what they can handle. That’s all a part of the education piece. That’s all part of the teamwork. That’s all part of the communication process. It all comes into play.

Dr. Rugo: That’s such an incredibly important point. As we’re wrapping up, it would be great if everybody had some points to make that pulled together some of our conversation. Julia, do you want to start?

Ms. Maués: Yes, I was going to add specifically about the topic you were just discussing, with all that an oncologist’s team has to know and all the different areas of our health being affected by these new treatments. One tip for patients and their teams is that the other providers around the patients may not be as informed about the disease and the treatments they are on. Sometimes we patients end up getting information that isn’t up to date with the latest drugs and things like that.

When we do talk with someone about our issues, make sure they are informed about the new drugs. For example, we often have skin issues. There are dermatologists that work with cancer patients often, and they’re very informed about the side effects that come with these drugs. There are others who never see these sorts of issues and may assume it’s something completely different.

I usually just go to doctors that my oncologist’s team collaborates with and gets referrals from because they send their patients to these doctors often. These are doctors that see cancer patients. We’re a very unique group.

Dr. Rugo: That’s a really good point. I have the same thing. We all have a little stable of people we refer to for various issues that we can reach on speed dial.
 

The importance of diversity in clinical trials to obtain the most useful outcomes

Fatima, there’s recently been, appropriately so, more of a push to try and evaluate side effects by racial and ethnic subgroups. I think we’re still pretty crummy at it, but we are making some progress. How important is that to you when you think about patients and managing them?

Dr. Cardoso: I think this is quite important. One area of research that is underused, really, is all the new genomics and sequencing technologies to understand why people react differently to the same treatment. Why is it that for some people, either for ethnic or other reasons, you have a different metabolism or something else that justifies a very high rate of side effects from a certain treatment, whereas in other regions of the world this doesn’t happen?

Not to go into these new drugs, but when using a very old drug like a taxane, I found a difference in reaction between the Portuguese patients and the Belgian patients, the two countries where I’ve worked. I even found that the cause might be genetic because the Portuguese living in Belgium reacted differently than the Belgians themselves.

Maybe there is something in the genetics that justifies the type of side effects that you have. I make a plea also for us to dedicate research to understanding why certain side effects are related to race and others are related to maybe some other types of genetic alterations that will lead to an increased side effect.

Dr. Rugo: Sheila, comments?

Ms. Pettiford: That is just excellent. It’s excellent to even consider it because it is so obvious. To me, it’s an obvious situation because there are things that are underneath the skin that we don’t understand. We have to take that into consideration when we are dealing with all these wonderful – I call them miracle – drugs that have come about in the last 20 years.

There still is much more to be done, and I try to participate in any type of organization that’s encouraging diversity in clinical trials because you need to have people of all different ethnicities in order for us to get to these answers. It’s fascinating that you found this out, doctor.
 

The patient-centered dosing initiative

Ms. Maués: I have the pleasure of being a member of a patient-led initiative called the Patient-Centered Dosing Initiative (PCDI). We are highlighting the discussion around dosages of drugs, especially in the metastatic setting. Metastatic breast cancer is what we’re focusing on, although it could apply to any type of cancer. We are advised by a number of wonderful, world-renowned physicians, Dr. Rugo being one of them. Anne Loeser, the leader of our group, has spoken at ASCO about this topic of dosage. What we’re seeing is that the dosage determination for oncology drugs is still done in the same way it used to be done decades ago and mostly with the curative intent of early-stage disease — metastatic cancer patients back then really didn’t live long at all. What we’re seeing right now is people with metastatic breast cancer that are able to, in some cases, live a long life managing their disease.

Patients are put on doses that are too high for them to be able to manage the side effects, and then they end up having to go off the drug, which means they have lost one of the tools in their toolbox. So, what we like to say about dosing is that, for metastatic cancer patients, it’s a marathon and not a sprint. If we throw all the poison at the patient from the very beginning, they won’t be able to take this for a very long time. And in the metastatic setting, the goal is to stay on each therapy for as long as possible. If we burn one of the cards early on, you have to move on to the next one. This is finite because at some point, there are not enough drugs that can help a particular patient. The PCDI is really getting a lot of visibility with the FDA and experts. People are talking more about dosages, and the FDA is now providing guidance for pharmaceutical companies to study different dosages in the clinical trials from the very beginning. This initiative is almost 3 years old, and we have made a tremendous impact since then.

Dr. Rugo: I think this is an incredibly important area moving forward, and thankfully, there’s so much interest now in not only promoting diversity, enrollment in trials, and education to promote diversity but also in looking at differences in efficacy and side effects.

I’ll just thank everybody for your contributions and amazing perspectives in this incredibly important area. As we move forward with better agents, we need to also make sure we’re understanding what the side effects are, managing them, and hearing the voices of our patients. Thanks very much.

Ms. Pettiford: Thank you so much.

Dr. Cardoso: Thank you.

Ms. Maués: Thank you.
 

Editor’s note: Our panelists would like to highlight these points:

• The patient and the health care team must build trust with each other. 
• African Americans have historical reasons for not trusting the health care industry. Much outreach is still needed. 
• Inform and educate before the start of treatment and during the treatment.
• Be balanced and do not underestimate common side effects or overestimate rare ones. Adapt the amount and the detail of the information to the wishes of the individual patient. Offer various methods of delivery (e.g., videos, pamphlets, fact sheets).
• Patients will research their condition and treatments online. Instead of trying to stop this, help them find the best sources.
• Patients will connect with others in the patient community and learn from each other’s experiences. Keep in mind that everyone is different, and decisions should always be made together with the medical team.
• Monitor patients regularly, especially during the first few treatment cycles.
• Use different forms of communication between the patient and health care providers (e.g., apps, digital charts, oncology nurses/nurse navigators, responsive oncologists, different forms of telemedicine), but don’t forget to speak directly with the patient.
• The use of new PRO apps can be very useful to help patients differentiate between urgent and nonurgent signs and symptoms.
• As much as possible, use preventive/prophylactic measures, namely for nausea, vomiting, diarrhea/constipation, and mucositis.
• Be aware of late side effects, especially with immunotherapy.
• Don’t forget that grade 1-2 side effects can substantially impact quality of life, particularly if they are persistent.
• Consider quality-of-life issues for each patient. What is acceptable for one patient may not be for another.
• Learn how to manage new and specific side effects (e.g., endocrine, skin related, pneumonitis).
• Keep an open dialogue about treatment and side effects. Things can change, and there are different ways to address issues such as medications for side effects and dosing changes.
• Listen to your patient and respond in a timely fashion.
• Ethnicity and genetics should be studied as a factor for individual side effects. Standard industry dosages of a new anticancer medication might not be as effective in one ethnic group as another due to the lack of diversity in clinical trials.
• Medications with hard-to-manage or dangerous side effects may be counterproductive regardless of effectiveness.
• Cancer treatment varies vastly depending on region and type of treatment facility. There are many unmet needs in rural areas because of lack of oncology personnel, finances, transportation, etc.

Dr. Rugo is a professor in the department of medicine, University of California San Francisco Comprehensive Cancer Center; director, Breast Oncology and Clinical Trials Education, Cancer Infusion Services, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco. Dr. Cardoso is director, breast unit, Champalimaud Clinical Centre, Lisbon. Financial disclosures for both Dr. Rugo and Dr. Cardoso are available on Medscape.com, where this article first appeared. Julia Maués is a patient in Washington. She has disclosed no relevant financial relationships. Sheila Pettiford is a patient in Middletown, Del. She has disclosed no relevant financial relationships.

This transcript of a video roundtable, which is available on Medscape.com, has been edited for clarity.

Hope S. Rugo, MD: Hello. I’m Hope Rugo, a breast medical oncologist from the University of California, San Francisco. I’m joined here by three of my friends and colleagues to discuss the toxicity of new agents in the treatment of breast cancer. Fatima, do you want to start by introducing yourself?

Fatima F. Cardoso, MD: Sure. Hello, everyone. I’m Fatima Cardoso, a breast medical oncologist in Lisbon, Portugal.

Dr. Rugo: Sheila.

Sheila Pettiford: Hi, I’m Sheila Pettiford. I am a metastatic [breast cancer] patient and have been for almost 8 years in April. I used to live in Philadelphia, Pennsylvania, but moved to Delaware in the last couple of years during the pandemic. I’m happy to be here.

Dr. Rugo: Julia.

Julia Maués: Hi, everyone. I also am a person living with metastatic breast cancer. I was diagnosed in 2013, so it’s going to be 9 years, also in April.
 

Effective monitoring and management of side effects: A team effort

Dr. Rugo: We have an amazing group and an international representation, which is also really nice because we get different perspectives. What we’re going to talk about is important to providers and patients across the board. With the host of new agents for the treatment of breast cancer – most of which have really moved us forward in terms of having effective treatment options – we’ve also been faced with a lot of new toxicities or side effects that we haven’t seen before or that we might not have expected from the specific agent.

Those toxicities across the board include side effects that are quite familiar to us, like low blood counts, but we may not advise people well enough about other side effects such as mouth sores, inflammation of the lungs, immune toxicities, and skin toxicities.

Fatima, do you want to start and talk about how we can think about these toxicities and address them?

Dr. Cardoso: Sure. Thank you. From the health care provider point of view, what I would highlight is to educate. Educate before we start the treatment. It’s very important to inform the patient but in a balanced way, so we don’t overexaggerate certain types of side effects or underestimate certain types of side effects.

It’s very important because an informed patient will be attentive to the types of side effects that can happen. Also, teach the patient when it is a [cause for] alarm or something for which they might need to contact their health care team and when that’s not the case. I think this is one crucial topic.

The other one is to monitor. Find ways how to best communicate between the patient and the health care team but in a way that you can monitor, so you can act very early on. Most of these new side effects, if you act early on, will not become severe. It is very important to know about them and to act early on.

I believe there is something important that we don’t think about all the time, and that is prophylaxis. Do not be shy about using prophylactic measures, be it for the mouth sores, nausea and vomiting, diarrhea, and other things that really impact the quality of life of patients. Those, to start, are my three major points of attention for health care professionals.

Dr. Rugo: I think that’s so incredibly important – the comments that you’ve made – and also that prevention and prophylaxis are so important. You don’t want to have a patient have diarrhea in the middle of the night and not have any antipropulsive agents at home. Just as a very straightforward example, it’s really important.

Also, the ability to know what you should be looking for and how you can manage it [is important]. There are many examples of times when, even with some education, providers may not have communicated well to the patient. Then the patient is surprised and unhappy with the situation and unable to manage it.
 

The importance of education

Sheila, your comments on this from the patient perspective are so important. How important is the education piece, and how do you manage the fear of side effects vs actually managing the side effects that might be caused by the treatment you’re taking?

Ms. Pettiford: Thank you for that question. I really think it’s a dance. It’s a dance between the patient and the health care team. Yes, education is absolutely important. However, the health care professionals have to establish a relationship of trust with the patient. My own circumstances were that – and I was very fortunate in that my oncologist, who I chose just by looking and not by a recommendation – I did find an oncologist who listened to me.

When it came time for me to deal with a new medication, the education she provided me was sufficient because of the fact that there was a lot of listening that had gone on prior to the new medicine being given to me. I trusted what I was hearing, and it felt like there was a balanced situation that came about from what I was being told. I could look it up, too.

There still is that part of the patient who will be participating in the process, as well. They can still look up things, and that’s one of the downfalls of the information age we are in. It is a dance. I just want to go back to that. There’s a dance between the patient and the health care providers.

Dr. Rugo: Julia, from the patient’s side, how do you balance the benefits you might get from a treatment versus the side effects and how best to manage them?

Ms. Maués: I think it’s interesting that when we talk with our doctors, and especially when we read about a certain treatment, the attention is focused on the very severe and unlikely side effects that a drug has. We don’t talk as much about the side effects that are most likely to happen and will affect us but may not be life threatening.

Especially for those of us with metastatic cancer who are going to ideally be on a drug for a very long time, we’re then faced with low-grade nausea for the rest of our lives. That’s not okay either, right? I think it’s important to talk about all of the levels of toxicities and everything that can be done to avoid this.
 

Communication is key

Ms. Pettiford: I just want to add something that Dr. Cardoso said about monitoring that is absolutely important. We’re in a day and time when it’s very difficult to get someone on the telephone, but we do have digital charts and other ways that monitoring can take place. I was at a large teaching university, and I had to go monthly for my treatments. Every month, there were questions that were asked about my life and my condition. I could always get in contact with somebody through the digital chart.

Dr. Rugo: That’s an incredibly important comment, Sheila, about communication and how patients can feel like they have someone to go to in real time who can help manage things. Fatima, I’m interested in your comment on that.

Also, just to go to the next step, which is that when we see data reported on clinical trials and how the agent we’ve added or substituted is better than the standard, the toxicity tables are side effects that occur in at least 10% or more patients and sometimes even 20%. Then they’re graded, where often the division is grade 3 or greater. That may not actually reflect much about what the individual patient experience is. How do we interpret these data? Communication and interpretation?

Dr. Cardoso: Absolutely. I always call attention that perhaps, since we focus so much on grade 3 and 4 [side effects], that is the reason why we don’t see in the usual reporting differences quality of life between treatments. Quality of life is affected also significantly by grade 2 side effects. Or, like Julia was mentioning, even grade 1, if they are persistent, will eventually affect your quality of life.

Sometimes, like I was saying, don’t underestimate – it’s a little bit like that. We focus on explaining, “Look, this new immunotherapy can give you all these different side effects.” But then we forget to say: “Oh, by the way, it may also give you some nausea.” Actually, the nausea will affect the patient’s quality of life. I think that’s why it is so important to balance the way we provide the information.

I would like also to take on what Sheila said that sometimes too much information is not very helpful. That’s why sometimes we have to go stepwise. The first time you’re about to start the treatment, advise [the patient] on the most frequent side effects. Later on, you have time to say: “Okay, by the way, this can also give a rare side effect. This is what you should look for. If you have it, please contact your health care team.”

I think the most difficult part, at least from my experience, is for patients to understand what is really a sign of a severe side effect and what is normal for that type of treatment. Some of the new ways of communicating, like using some patient-reported outcome (PRO) apps, actually help the patient by saying, “This that you are feeling is normal. It can wait for your next appointment. This that you are feeling, it’s better if you try to reach your health care team right away. Or, this is an urgent thing and go to the emergency room near you.”

For this kind of triage, there are now new apps that can help. I think this is the most difficult part because when you are a patient, you don’t know if what you are feeling is actually a sign of something very severe or if it’s normal for the type of treatment you are receiving.

Dr. Rugo: I think that’s so important, and these new PRO apps may help with this. Of course, nothing substitutes for talking in the end if you’re confused or it doesn’t fit into whatever’s in that paradigm. I think it’s important.
 

Best practices in focusing on the individual patient

Julia, what do you think the best way of educating the patient is when you’re going to start a new treatment? You might be newly diagnosed with cancer or you might have had cancer for a number of years. You’re going to start a new treatment. What’s the best way to know what to look for and how to manage it?

Ms. Maués: I think the key here is that everyone’s different, so have that conversation, the doctor and the patient, about what the best way [of education] is for that specific person. Do they want a flyer listing all of the side effects? Do they want a link to a video they can watch and understand? Do they want someone to come in and give an extra explanation about things? Everyone learns so differently, and I think it’s really hard to assume there’s one way that all patients will understand.

I think the PRO apps are great, and also another benefit is that you keep track of your side effects. Sometimes we don’t even remember well. When did you have nausea? Was it in the morning? Was it in the evening? Is it every day? If you track it with these apps, then you will have the data stored there in the form to answer those questions.

Dr. Cardoso: There was recently a publication – I found it quite interesting – from Lesley Fallowfield’s group saying that the majority of patients would better absorb the information if it is not just text, but if it somehow has a video component, an image, or an infographic that would help them memorize a little bit more information.

Dr. Rugo: There’s been a move toward trying to make videos because the amount of education that’s needed on the providers’ side from our nurses and advanced practice providers may be overwhelming, so things might get missed. The idea of having videos to get everybody on the same page is very popular right now for this reason, and Lesley’s work is really groundbreaking.

Sheila, what do you think is the best way to communicate information?

Ms. Pettiford: Well, I definitely think it’s important for the doctors to recognize, as Julia said, that everyone is different, and all their patients are different. They could come with the same exact subtype of whatever cancer they have – in this situation, breast cancer – and still have so many different reactions. It’s so important for everybody on the health care team to listen to what the patient says because the patient is the one who is living with the illness and knows their body, hopefully.

It’s just one of those things. It’s not a one-size-fits-all situation. You give the standards, but I think it’s important to offer various ways of communicating to a patient because some people are visual. Some people want an overwhelming amount of information so they can sort through it. Then, you have some people who just want the bullet points. Again, it is important not to try to do it as a one-size-fits-all type thing.

Dr. Rugo: Yes, that’s such a good point. I’m always struck by the fact that some patients are totally on top of it and listen to it all, and then other people, we just can’t get them to even call in regarding their side effects. In some ways, it’s frightening for people to call in with issues. Maybe they’re afraid they won’t get the treatment, or that it is related to their cancer progressing, too. Trying to meet people on their own level is a real challenge and an important one.

We talked about education for providers. Fatima, how should we be best educating for these new drugs and new side effects? So many different manifestations can occur, and as we talked about, they might be quite uncommon. We just want people to keep their ears up for any kind of unusual toxicity we see. We all know that the presentation of efficacy data is not adequate for education.

Dr. Cardoso: When we present a new treatment, we focus usually on efficacy, right? Then we say a few things about safety, particularly if there is a new or a severe side effect, but we don’t go through details on how to best manage this in clinical practice.

Anecdotally, I remember that I contacted you because I was going to start using a new treatment and you had some experience. I asked, “What about nausea and vomiting? What do you do for prophylaxis?” I couldn’t find it anywhere in the manuscripts or the presentations. I think we need to focus a little bit more on practical tips. If you are about to start this new treatment, what you should think about and not just the very severe and rare side effects?

Of course, as health care professionals, we need to keep this in our minds. For example, with immunotherapy, side effects can often occur even after stopping the treatment. For other types of new treatments, we need to gain knowledge about endocrinology, for example, which is something that oncologists wouldn’t have to deal with that often in the past. Now, new skills are needed.

It’s also what makes our profession so exciting. There’s always something new to learn, and I like to look at it from that perspective. It’s not boring at all. We are always learning new things.

Dr. Rugo: Indeed. Certainly, you and I have worked together on trying to encourage our pharmaceutical colleagues to publish these papers alongside their urgency of distributing the efficacy data and publishing the papers on efficacy, and also to do a nitty-gritty review of safety and talk about management strategies. I’m really pleased that there seems to be a little more focus on that earlier now in the drug process – although still not early enough – but it’s getting there. That’s a good thing.

Ms. Pettiford: Julia, you mentioned earlier how important it is for the individual patient’s quality of life to understand how these side effects can affect them. It really is one of those things in which we have to make personal decisions. What might be good for one person in terms of what happens with side effects, and their ability to function might not work with someone else.

If you are a person who’s dealing with metastatic disease who has children, a household, a dog, and a cat to take care of, what I can handle being that I’m a single person is not what they can handle. That’s all a part of the education piece. That’s all part of the teamwork. That’s all part of the communication process. It all comes into play.

Dr. Rugo: That’s such an incredibly important point. As we’re wrapping up, it would be great if everybody had some points to make that pulled together some of our conversation. Julia, do you want to start?

Ms. Maués: Yes, I was going to add specifically about the topic you were just discussing, with all that an oncologist’s team has to know and all the different areas of our health being affected by these new treatments. One tip for patients and their teams is that the other providers around the patients may not be as informed about the disease and the treatments they are on. Sometimes we patients end up getting information that isn’t up to date with the latest drugs and things like that.

When we do talk with someone about our issues, make sure they are informed about the new drugs. For example, we often have skin issues. There are dermatologists that work with cancer patients often, and they’re very informed about the side effects that come with these drugs. There are others who never see these sorts of issues and may assume it’s something completely different.

I usually just go to doctors that my oncologist’s team collaborates with and gets referrals from because they send their patients to these doctors often. These are doctors that see cancer patients. We’re a very unique group.

Dr. Rugo: That’s a really good point. I have the same thing. We all have a little stable of people we refer to for various issues that we can reach on speed dial.
 

The importance of diversity in clinical trials to obtain the most useful outcomes

Fatima, there’s recently been, appropriately so, more of a push to try and evaluate side effects by racial and ethnic subgroups. I think we’re still pretty crummy at it, but we are making some progress. How important is that to you when you think about patients and managing them?

Dr. Cardoso: I think this is quite important. One area of research that is underused, really, is all the new genomics and sequencing technologies to understand why people react differently to the same treatment. Why is it that for some people, either for ethnic or other reasons, you have a different metabolism or something else that justifies a very high rate of side effects from a certain treatment, whereas in other regions of the world this doesn’t happen?

Not to go into these new drugs, but when using a very old drug like a taxane, I found a difference in reaction between the Portuguese patients and the Belgian patients, the two countries where I’ve worked. I even found that the cause might be genetic because the Portuguese living in Belgium reacted differently than the Belgians themselves.

Maybe there is something in the genetics that justifies the type of side effects that you have. I make a plea also for us to dedicate research to understanding why certain side effects are related to race and others are related to maybe some other types of genetic alterations that will lead to an increased side effect.

Dr. Rugo: Sheila, comments?

Ms. Pettiford: That is just excellent. It’s excellent to even consider it because it is so obvious. To me, it’s an obvious situation because there are things that are underneath the skin that we don’t understand. We have to take that into consideration when we are dealing with all these wonderful – I call them miracle – drugs that have come about in the last 20 years.

There still is much more to be done, and I try to participate in any type of organization that’s encouraging diversity in clinical trials because you need to have people of all different ethnicities in order for us to get to these answers. It’s fascinating that you found this out, doctor.
 

The patient-centered dosing initiative

Ms. Maués: I have the pleasure of being a member of a patient-led initiative called the Patient-Centered Dosing Initiative (PCDI). We are highlighting the discussion around dosages of drugs, especially in the metastatic setting. Metastatic breast cancer is what we’re focusing on, although it could apply to any type of cancer. We are advised by a number of wonderful, world-renowned physicians, Dr. Rugo being one of them. Anne Loeser, the leader of our group, has spoken at ASCO about this topic of dosage. What we’re seeing is that the dosage determination for oncology drugs is still done in the same way it used to be done decades ago and mostly with the curative intent of early-stage disease — metastatic cancer patients back then really didn’t live long at all. What we’re seeing right now is people with metastatic breast cancer that are able to, in some cases, live a long life managing their disease.

Patients are put on doses that are too high for them to be able to manage the side effects, and then they end up having to go off the drug, which means they have lost one of the tools in their toolbox. So, what we like to say about dosing is that, for metastatic cancer patients, it’s a marathon and not a sprint. If we throw all the poison at the patient from the very beginning, they won’t be able to take this for a very long time. And in the metastatic setting, the goal is to stay on each therapy for as long as possible. If we burn one of the cards early on, you have to move on to the next one. This is finite because at some point, there are not enough drugs that can help a particular patient. The PCDI is really getting a lot of visibility with the FDA and experts. People are talking more about dosages, and the FDA is now providing guidance for pharmaceutical companies to study different dosages in the clinical trials from the very beginning. This initiative is almost 3 years old, and we have made a tremendous impact since then.

Dr. Rugo: I think this is an incredibly important area moving forward, and thankfully, there’s so much interest now in not only promoting diversity, enrollment in trials, and education to promote diversity but also in looking at differences in efficacy and side effects.

I’ll just thank everybody for your contributions and amazing perspectives in this incredibly important area. As we move forward with better agents, we need to also make sure we’re understanding what the side effects are, managing them, and hearing the voices of our patients. Thanks very much.

Ms. Pettiford: Thank you so much.

Dr. Cardoso: Thank you.

Ms. Maués: Thank you.
 

Editor’s note: Our panelists would like to highlight these points:

• The patient and the health care team must build trust with each other. 
• African Americans have historical reasons for not trusting the health care industry. Much outreach is still needed. 
• Inform and educate before the start of treatment and during the treatment.
• Be balanced and do not underestimate common side effects or overestimate rare ones. Adapt the amount and the detail of the information to the wishes of the individual patient. Offer various methods of delivery (e.g., videos, pamphlets, fact sheets).
• Patients will research their condition and treatments online. Instead of trying to stop this, help them find the best sources.
• Patients will connect with others in the patient community and learn from each other’s experiences. Keep in mind that everyone is different, and decisions should always be made together with the medical team.
• Monitor patients regularly, especially during the first few treatment cycles.
• Use different forms of communication between the patient and health care providers (e.g., apps, digital charts, oncology nurses/nurse navigators, responsive oncologists, different forms of telemedicine), but don’t forget to speak directly with the patient.
• The use of new PRO apps can be very useful to help patients differentiate between urgent and nonurgent signs and symptoms.
• As much as possible, use preventive/prophylactic measures, namely for nausea, vomiting, diarrhea/constipation, and mucositis.
• Be aware of late side effects, especially with immunotherapy.
• Don’t forget that grade 1-2 side effects can substantially impact quality of life, particularly if they are persistent.
• Consider quality-of-life issues for each patient. What is acceptable for one patient may not be for another.
• Learn how to manage new and specific side effects (e.g., endocrine, skin related, pneumonitis).
• Keep an open dialogue about treatment and side effects. Things can change, and there are different ways to address issues such as medications for side effects and dosing changes.
• Listen to your patient and respond in a timely fashion.
• Ethnicity and genetics should be studied as a factor for individual side effects. Standard industry dosages of a new anticancer medication might not be as effective in one ethnic group as another due to the lack of diversity in clinical trials.
• Medications with hard-to-manage or dangerous side effects may be counterproductive regardless of effectiveness.
• Cancer treatment varies vastly depending on region and type of treatment facility. There are many unmet needs in rural areas because of lack of oncology personnel, finances, transportation, etc.

Dr. Rugo is a professor in the department of medicine, University of California San Francisco Comprehensive Cancer Center; director, Breast Oncology and Clinical Trials Education, Cancer Infusion Services, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco. Dr. Cardoso is director, breast unit, Champalimaud Clinical Centre, Lisbon. Financial disclosures for both Dr. Rugo and Dr. Cardoso are available on Medscape.com, where this article first appeared. Julia Maués is a patient in Washington. She has disclosed no relevant financial relationships. Sheila Pettiford is a patient in Middletown, Del. She has disclosed no relevant financial relationships.

The United States has never achieved a single high standard of medical care equity for all of its people, and the trend line does not appear favorable. The closest we have reached is basic Medicare (Parts A and B), military medicine, the Veterans Health Administration, and large nonprofit groups like Kaiser Permanente. It seems that the nature of we individualistic Americans is to always try to seek an advantage.

But even achieving equity in medical care would not ensure equity in health. The social determinants of health (income level, education, politics, government, geography, neighborhood, country of origin, language spoken, literacy, gender, and yes – race and ethnicity) have far more influence on health equity than does medical care.

Narratives can both reflect and influence culture. Considering the harmful effects of the current political divisiveness in the United States, the timing is ideal for our three leading medical and health education organizations – the American Medical Association, the Association of American Medical Colleges (AAMC), and the Centers for Disease Control and Prevention – to publish a definitive position paper called “Advancing Health Equity: A Guide to Language, Narrative and Concepts.”
 

What’s in a word?

According to William Shakespeare, “A rose by any other name would smell as sweet” (Romeo and Juliet). Maybe. But if the word used were “thorn” or “thistle,” it just would not be the same.

Words comprise language and wield enormous power with human beings. Wars are fought over geographic boundaries often defined by the language spoken by the people: think 2022, Russian-speaking Ukrainians. Think Winston Churchill’s massive 1,500-page “A History of the English-Speaking Peoples.” Think about the political power of French in Quebec, Canada.

Thus, it should be no surprise that words, acronyms, and abbreviations become rallying cries for political activists of all stripes: PC, January 6, Woke, 1619, BLM, Critical Race Theory, 1776, Remember Pearl Harbor, Remember the Alamo, the Civil War or the War Between the States, the War for Southern Independence, the War of Northern Aggression, the War of the Rebellion, or simply “The Lost Cause.” How about Realpolitik?

Is “medical language” the language of the people or of the profession? Physicians must understand each other, and physicians also must communicate clearly with patients using words that convey neutral meanings and don’t interfere with objective understanding. Medical editors prefer the brevity of one or a few words to clearly convey meaning.

A version of this article first appeared on Medscape.com.

The United States has never achieved a single high standard of medical care equity for all of its people, and the trend line does not appear favorable. The closest we have reached is basic Medicare (Parts A and B), military medicine, the Veterans Health Administration, and large nonprofit groups like Kaiser Permanente. It seems that the nature of we individualistic Americans is to always try to seek an advantage.

But even achieving equity in medical care would not ensure equity in health. The social determinants of health (income level, education, politics, government, geography, neighborhood, country of origin, language spoken, literacy, gender, and yes – race and ethnicity) have far more influence on health equity than does medical care.

Narratives can both reflect and influence culture. Considering the harmful effects of the current political divisiveness in the United States, the timing is ideal for our three leading medical and health education organizations – the American Medical Association, the Association of American Medical Colleges (AAMC), and the Centers for Disease Control and Prevention – to publish a definitive position paper called “Advancing Health Equity: A Guide to Language, Narrative and Concepts.”
 

What’s in a word?

According to William Shakespeare, “A rose by any other name would smell as sweet” (Romeo and Juliet). Maybe. But if the word used were “thorn” or “thistle,” it just would not be the same.

Words comprise language and wield enormous power with human beings. Wars are fought over geographic boundaries often defined by the language spoken by the people: think 2022, Russian-speaking Ukrainians. Think Winston Churchill’s massive 1,500-page “A History of the English-Speaking Peoples.” Think about the political power of French in Quebec, Canada.

Thus, it should be no surprise that words, acronyms, and abbreviations become rallying cries for political activists of all stripes: PC, January 6, Woke, 1619, BLM, Critical Race Theory, 1776, Remember Pearl Harbor, Remember the Alamo, the Civil War or the War Between the States, the War for Southern Independence, the War of Northern Aggression, the War of the Rebellion, or simply “The Lost Cause.” How about Realpolitik?

Is “medical language” the language of the people or of the profession? Physicians must understand each other, and physicians also must communicate clearly with patients using words that convey neutral meanings and don’t interfere with objective understanding. Medical editors prefer the brevity of one or a few words to clearly convey meaning.

A version of this article first appeared on Medscape.com.

The United States has never achieved a single high standard of medical care equity for all of its people, and the trend line does not appear favorable. The closest we have reached is basic Medicare (Parts A and B), military medicine, the Veterans Health Administration, and large nonprofit groups like Kaiser Permanente. It seems that the nature of we individualistic Americans is to always try to seek an advantage.

But even achieving equity in medical care would not ensure equity in health. The social determinants of health (income level, education, politics, government, geography, neighborhood, country of origin, language spoken, literacy, gender, and yes – race and ethnicity) have far more influence on health equity than does medical care.

Narratives can both reflect and influence culture. Considering the harmful effects of the current political divisiveness in the United States, the timing is ideal for our three leading medical and health education organizations – the American Medical Association, the Association of American Medical Colleges (AAMC), and the Centers for Disease Control and Prevention – to publish a definitive position paper called “Advancing Health Equity: A Guide to Language, Narrative and Concepts.”
 

What’s in a word?

According to William Shakespeare, “A rose by any other name would smell as sweet” (Romeo and Juliet). Maybe. But if the word used were “thorn” or “thistle,” it just would not be the same.

Words comprise language and wield enormous power with human beings. Wars are fought over geographic boundaries often defined by the language spoken by the people: think 2022, Russian-speaking Ukrainians. Think Winston Churchill’s massive 1,500-page “A History of the English-Speaking Peoples.” Think about the political power of French in Quebec, Canada.

Thus, it should be no surprise that words, acronyms, and abbreviations become rallying cries for political activists of all stripes: PC, January 6, Woke, 1619, BLM, Critical Race Theory, 1776, Remember Pearl Harbor, Remember the Alamo, the Civil War or the War Between the States, the War for Southern Independence, the War of Northern Aggression, the War of the Rebellion, or simply “The Lost Cause.” How about Realpolitik?

Is “medical language” the language of the people or of the profession? Physicians must understand each other, and physicians also must communicate clearly with patients using words that convey neutral meanings and don’t interfere with objective understanding. Medical editors prefer the brevity of one or a few words to clearly convey meaning.

A version of this article first appeared on Medscape.com.