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Patient Navigators for Serious Illnesses Can Now Bill Under New Medicare Codes
In a move that acknowledges the gauntlet the US health system poses for people facing serious and fatal illnesses, Medicare will pay for a new class of workers to help patients manage treatments for conditions like cancer and heart failure.
The 2024 Medicare physician fee schedule includes new billing codes, including G0023, to pay for 60 minutes a month of care coordination by certified or trained auxiliary personnel working under the direction of a clinician.
A diagnosis of cancer or another serious illness takes a toll beyond the physical effects of the disease. Patients often scramble to make adjustments in family and work schedules to manage treatment, said Samyukta Mullangi, MD, MBA, medical director of oncology at Thyme Care, a Nashville, Tennessee–based firm that provides navigation and coordination services to oncology practices and insurers.
“It just really does create a bit of a pressure cooker for patients,” Dr. Mullangi told this news organization.
Medicare has for many years paid for medical professionals to help patients cope with the complexities of disease, such as chronic care management (CCM) provided by physicians, nurses, and physician assistants.
The new principal illness navigation (PIN) payments are intended to pay for work that to date typically has been done by people without medical degrees, including those involved in peer support networks and community health programs. The US Centers for Medicare and Medicaid Services(CMS) expects these navigators will undergo training and work under the supervision of clinicians.
The new navigators may coordinate care transitions between medical settings, follow up with patients after emergency department (ED) visits, or communicate with skilled nursing facilities regarding the psychosocial needs and functional deficits of a patient, among other functions.
CMS expects the new navigators may:
- Conduct assessments to understand a patient’s life story, strengths, needs, goals, preferences, and desired outcomes, including understanding cultural and linguistic factors.
- Provide support to accomplish the clinician’s treatment plan.
- Coordinate the receipt of needed services from healthcare facilities, home- and community-based service providers, and caregivers.
Peers as Navigators
The new navigators can be former patients who have undergone similar treatments for serious diseases, CMS said. This approach sets the new program apart from other care management services Medicare already covers, program officials wrote in the 2024 physician fee schedule.
“For some conditions, patients are best able to engage with the healthcare system and access care if they have assistance from a single, dedicated individual who has ‘lived experience,’ ” according to the rule.
The agency has taken a broad initial approach in defining what kinds of illnesses a patient may have to qualify for services. Patients must have a serious condition that is expected to last at least 3 months, such as cancer, heart failure, or substance use disorder.
But those without a definitive diagnosis may also qualify to receive navigator services.
In the rule, CMS cited a case in which a CT scan identified a suspicious mass in a patient’s colon. A clinician might decide this person would benefit from navigation services due to the potential risks for an undiagnosed illness.
“Regardless of the definitive diagnosis of the mass, presence of a colonic mass for that patient may be a serious high-risk condition that could, for example, cause obstruction and lead the patient to present to the emergency department, as well as be potentially indicative of an underlying life-threatening illness such as colon cancer,” CMS wrote in the rule.
Navigators often start their work when cancer patients are screened and guide them through initial diagnosis, potential surgery, radiation, or chemotherapy, said Sharon Gentry, MSN, RN, a former nurse navigator who is now the editor in chief of the Journal of the Academy of Oncology Nurse & Patient Navigators.
The navigators are meant to be a trusted and continual presence for patients, who otherwise might be left to start anew in finding help at each phase of care.
The navigators “see the whole picture. They see the whole journey the patient takes, from pre-diagnosis all the way through diagnosis care out through survival,” Ms. Gentry said.
Gaining a special Medicare payment for these kinds of services will elevate this work, she said.
Many newer drugs can target specific mechanisms and proteins of cancer. Often, oncology treatment involves testing to find out if mutations are allowing the cancer cells to evade a patient’s immune system.
Checking these biomarkers takes time, however. Patients sometimes become frustrated because they are anxious to begin treatment. Patients may receive inaccurate information from friends or family who went through treatment previously. Navigators can provide knowledge on the current state of care for a patient’s disease, helping them better manage anxieties.
“You have to explain to them that things have changed since the guy you drink coffee with was diagnosed with cancer, and there may be a drug that could target that,” Ms. Gentry said.
Potential Challenges
Initial uptake of the new PIN codes may be slow going, however, as clinicians and health systems may already use well-established codes. These include CCM and principal care management services, which may pay higher rates, Mullangi said.
“There might be sensitivity around not wanting to cannibalize existing programs with a new program,” Dr. Mullangi said.
In addition, many patients will have a copay for the services of principal illness navigators, Dr. Mullangi said.
While many patients have additional insurance that would cover the service, not all do. People with traditional Medicare coverage can sometimes pay 20% of the cost of some medical services.
“I think that may give patients pause, particularly if they’re already feeling the financial burden of a cancer treatment journey,” Dr. Mullangi said.
Pay rates for PIN services involve calculations of regional price differences, which are posted publicly by CMS, and potential added fees for services provided by hospital-affiliated organizations.
Consider payments for code G0023, covering 60 minutes of principal navigation services provided in a single month.
A set reimbursement for patients cared for in independent medical practices exists, with variation for local costs. Medicare’s non-facility price for G0023 would be $102.41 in some parts of Silicon Valley in California, including San Jose. In Arkansas, where costs are lower, reimbursement would be $73.14 for this same service.
Patients who get services covered by code G0023 in independent medical practices would have monthly copays of about $15-$20, depending on where they live.
The tab for patients tends to be higher for these same services if delivered through a medical practice owned by a hospital, as this would trigger the addition of facility fees to the payments made to cover the services. Facility fees are difficult for the public to ascertain before getting a treatment or service.
Dr. Mullangi and Ms. Gentry reported no relevant financial disclosures outside of their employers.
A version of this article first appeared on Medscape.com.
In a move that acknowledges the gauntlet the US health system poses for people facing serious and fatal illnesses, Medicare will pay for a new class of workers to help patients manage treatments for conditions like cancer and heart failure.
The 2024 Medicare physician fee schedule includes new billing codes, including G0023, to pay for 60 minutes a month of care coordination by certified or trained auxiliary personnel working under the direction of a clinician.
A diagnosis of cancer or another serious illness takes a toll beyond the physical effects of the disease. Patients often scramble to make adjustments in family and work schedules to manage treatment, said Samyukta Mullangi, MD, MBA, medical director of oncology at Thyme Care, a Nashville, Tennessee–based firm that provides navigation and coordination services to oncology practices and insurers.
“It just really does create a bit of a pressure cooker for patients,” Dr. Mullangi told this news organization.
Medicare has for many years paid for medical professionals to help patients cope with the complexities of disease, such as chronic care management (CCM) provided by physicians, nurses, and physician assistants.
The new principal illness navigation (PIN) payments are intended to pay for work that to date typically has been done by people without medical degrees, including those involved in peer support networks and community health programs. The US Centers for Medicare and Medicaid Services(CMS) expects these navigators will undergo training and work under the supervision of clinicians.
The new navigators may coordinate care transitions between medical settings, follow up with patients after emergency department (ED) visits, or communicate with skilled nursing facilities regarding the psychosocial needs and functional deficits of a patient, among other functions.
CMS expects the new navigators may:
- Conduct assessments to understand a patient’s life story, strengths, needs, goals, preferences, and desired outcomes, including understanding cultural and linguistic factors.
- Provide support to accomplish the clinician’s treatment plan.
- Coordinate the receipt of needed services from healthcare facilities, home- and community-based service providers, and caregivers.
Peers as Navigators
The new navigators can be former patients who have undergone similar treatments for serious diseases, CMS said. This approach sets the new program apart from other care management services Medicare already covers, program officials wrote in the 2024 physician fee schedule.
“For some conditions, patients are best able to engage with the healthcare system and access care if they have assistance from a single, dedicated individual who has ‘lived experience,’ ” according to the rule.
The agency has taken a broad initial approach in defining what kinds of illnesses a patient may have to qualify for services. Patients must have a serious condition that is expected to last at least 3 months, such as cancer, heart failure, or substance use disorder.
But those without a definitive diagnosis may also qualify to receive navigator services.
In the rule, CMS cited a case in which a CT scan identified a suspicious mass in a patient’s colon. A clinician might decide this person would benefit from navigation services due to the potential risks for an undiagnosed illness.
“Regardless of the definitive diagnosis of the mass, presence of a colonic mass for that patient may be a serious high-risk condition that could, for example, cause obstruction and lead the patient to present to the emergency department, as well as be potentially indicative of an underlying life-threatening illness such as colon cancer,” CMS wrote in the rule.
Navigators often start their work when cancer patients are screened and guide them through initial diagnosis, potential surgery, radiation, or chemotherapy, said Sharon Gentry, MSN, RN, a former nurse navigator who is now the editor in chief of the Journal of the Academy of Oncology Nurse & Patient Navigators.
The navigators are meant to be a trusted and continual presence for patients, who otherwise might be left to start anew in finding help at each phase of care.
The navigators “see the whole picture. They see the whole journey the patient takes, from pre-diagnosis all the way through diagnosis care out through survival,” Ms. Gentry said.
Gaining a special Medicare payment for these kinds of services will elevate this work, she said.
Many newer drugs can target specific mechanisms and proteins of cancer. Often, oncology treatment involves testing to find out if mutations are allowing the cancer cells to evade a patient’s immune system.
Checking these biomarkers takes time, however. Patients sometimes become frustrated because they are anxious to begin treatment. Patients may receive inaccurate information from friends or family who went through treatment previously. Navigators can provide knowledge on the current state of care for a patient’s disease, helping them better manage anxieties.
“You have to explain to them that things have changed since the guy you drink coffee with was diagnosed with cancer, and there may be a drug that could target that,” Ms. Gentry said.
Potential Challenges
Initial uptake of the new PIN codes may be slow going, however, as clinicians and health systems may already use well-established codes. These include CCM and principal care management services, which may pay higher rates, Mullangi said.
“There might be sensitivity around not wanting to cannibalize existing programs with a new program,” Dr. Mullangi said.
In addition, many patients will have a copay for the services of principal illness navigators, Dr. Mullangi said.
While many patients have additional insurance that would cover the service, not all do. People with traditional Medicare coverage can sometimes pay 20% of the cost of some medical services.
“I think that may give patients pause, particularly if they’re already feeling the financial burden of a cancer treatment journey,” Dr. Mullangi said.
Pay rates for PIN services involve calculations of regional price differences, which are posted publicly by CMS, and potential added fees for services provided by hospital-affiliated organizations.
Consider payments for code G0023, covering 60 minutes of principal navigation services provided in a single month.
A set reimbursement for patients cared for in independent medical practices exists, with variation for local costs. Medicare’s non-facility price for G0023 would be $102.41 in some parts of Silicon Valley in California, including San Jose. In Arkansas, where costs are lower, reimbursement would be $73.14 for this same service.
Patients who get services covered by code G0023 in independent medical practices would have monthly copays of about $15-$20, depending on where they live.
The tab for patients tends to be higher for these same services if delivered through a medical practice owned by a hospital, as this would trigger the addition of facility fees to the payments made to cover the services. Facility fees are difficult for the public to ascertain before getting a treatment or service.
Dr. Mullangi and Ms. Gentry reported no relevant financial disclosures outside of their employers.
A version of this article first appeared on Medscape.com.
In a move that acknowledges the gauntlet the US health system poses for people facing serious and fatal illnesses, Medicare will pay for a new class of workers to help patients manage treatments for conditions like cancer and heart failure.
The 2024 Medicare physician fee schedule includes new billing codes, including G0023, to pay for 60 minutes a month of care coordination by certified or trained auxiliary personnel working under the direction of a clinician.
A diagnosis of cancer or another serious illness takes a toll beyond the physical effects of the disease. Patients often scramble to make adjustments in family and work schedules to manage treatment, said Samyukta Mullangi, MD, MBA, medical director of oncology at Thyme Care, a Nashville, Tennessee–based firm that provides navigation and coordination services to oncology practices and insurers.
“It just really does create a bit of a pressure cooker for patients,” Dr. Mullangi told this news organization.
Medicare has for many years paid for medical professionals to help patients cope with the complexities of disease, such as chronic care management (CCM) provided by physicians, nurses, and physician assistants.
The new principal illness navigation (PIN) payments are intended to pay for work that to date typically has been done by people without medical degrees, including those involved in peer support networks and community health programs. The US Centers for Medicare and Medicaid Services(CMS) expects these navigators will undergo training and work under the supervision of clinicians.
The new navigators may coordinate care transitions between medical settings, follow up with patients after emergency department (ED) visits, or communicate with skilled nursing facilities regarding the psychosocial needs and functional deficits of a patient, among other functions.
CMS expects the new navigators may:
- Conduct assessments to understand a patient’s life story, strengths, needs, goals, preferences, and desired outcomes, including understanding cultural and linguistic factors.
- Provide support to accomplish the clinician’s treatment plan.
- Coordinate the receipt of needed services from healthcare facilities, home- and community-based service providers, and caregivers.
Peers as Navigators
The new navigators can be former patients who have undergone similar treatments for serious diseases, CMS said. This approach sets the new program apart from other care management services Medicare already covers, program officials wrote in the 2024 physician fee schedule.
“For some conditions, patients are best able to engage with the healthcare system and access care if they have assistance from a single, dedicated individual who has ‘lived experience,’ ” according to the rule.
The agency has taken a broad initial approach in defining what kinds of illnesses a patient may have to qualify for services. Patients must have a serious condition that is expected to last at least 3 months, such as cancer, heart failure, or substance use disorder.
But those without a definitive diagnosis may also qualify to receive navigator services.
In the rule, CMS cited a case in which a CT scan identified a suspicious mass in a patient’s colon. A clinician might decide this person would benefit from navigation services due to the potential risks for an undiagnosed illness.
“Regardless of the definitive diagnosis of the mass, presence of a colonic mass for that patient may be a serious high-risk condition that could, for example, cause obstruction and lead the patient to present to the emergency department, as well as be potentially indicative of an underlying life-threatening illness such as colon cancer,” CMS wrote in the rule.
Navigators often start their work when cancer patients are screened and guide them through initial diagnosis, potential surgery, radiation, or chemotherapy, said Sharon Gentry, MSN, RN, a former nurse navigator who is now the editor in chief of the Journal of the Academy of Oncology Nurse & Patient Navigators.
The navigators are meant to be a trusted and continual presence for patients, who otherwise might be left to start anew in finding help at each phase of care.
The navigators “see the whole picture. They see the whole journey the patient takes, from pre-diagnosis all the way through diagnosis care out through survival,” Ms. Gentry said.
Gaining a special Medicare payment for these kinds of services will elevate this work, she said.
Many newer drugs can target specific mechanisms and proteins of cancer. Often, oncology treatment involves testing to find out if mutations are allowing the cancer cells to evade a patient’s immune system.
Checking these biomarkers takes time, however. Patients sometimes become frustrated because they are anxious to begin treatment. Patients may receive inaccurate information from friends or family who went through treatment previously. Navigators can provide knowledge on the current state of care for a patient’s disease, helping them better manage anxieties.
“You have to explain to them that things have changed since the guy you drink coffee with was diagnosed with cancer, and there may be a drug that could target that,” Ms. Gentry said.
Potential Challenges
Initial uptake of the new PIN codes may be slow going, however, as clinicians and health systems may already use well-established codes. These include CCM and principal care management services, which may pay higher rates, Mullangi said.
“There might be sensitivity around not wanting to cannibalize existing programs with a new program,” Dr. Mullangi said.
In addition, many patients will have a copay for the services of principal illness navigators, Dr. Mullangi said.
While many patients have additional insurance that would cover the service, not all do. People with traditional Medicare coverage can sometimes pay 20% of the cost of some medical services.
“I think that may give patients pause, particularly if they’re already feeling the financial burden of a cancer treatment journey,” Dr. Mullangi said.
Pay rates for PIN services involve calculations of regional price differences, which are posted publicly by CMS, and potential added fees for services provided by hospital-affiliated organizations.
Consider payments for code G0023, covering 60 minutes of principal navigation services provided in a single month.
A set reimbursement for patients cared for in independent medical practices exists, with variation for local costs. Medicare’s non-facility price for G0023 would be $102.41 in some parts of Silicon Valley in California, including San Jose. In Arkansas, where costs are lower, reimbursement would be $73.14 for this same service.
Patients who get services covered by code G0023 in independent medical practices would have monthly copays of about $15-$20, depending on where they live.
The tab for patients tends to be higher for these same services if delivered through a medical practice owned by a hospital, as this would trigger the addition of facility fees to the payments made to cover the services. Facility fees are difficult for the public to ascertain before getting a treatment or service.
Dr. Mullangi and Ms. Gentry reported no relevant financial disclosures outside of their employers.
A version of this article first appeared on Medscape.com.
Building an AI Army of Digital Twins to Fight Cancer
A patient has cancer. It’s decision time.
Clinician and patient alike face, really, the ultimate challenge when making those decisions. They have to consider the patient’s individual circumstances, available treatment options, potential side effects, relevant clinical data such as the patient’s genetic profile and cancer specifics, and more.
“That’s a lot of information to hold,” said Uzma Asghar, PhD, MRCP, a British consultant medical oncologist at The Royal Marsden Hospital and a chief scientific officer at Concr LTD.
What if there were a way to test — quickly and accurately — all the potential paths forward?
That’s the goal of digital twins.
“What the [digital twin] model can do for the clinician is to hold all that information and process it really quickly, within a couple of minutes,” Asghar noted.
A digital twin is more than just a computer model or simulation because it copies a real-world person and relies on real-world data. Some digital twin programs also integrate new information as it becomes available. This technology holds promise for personalized medicine, drug discovery, developing screening strategies, and better understanding diseases.
How to Deliver a Twin
To create a digital twin, experts develop a computer model with data to hone its expertise in an area of medicine, such as cancer types and treatments. Then “you train the model on information it’s seen, and then introduce a patient and patient’s information,” said Asghar.
Asghar is currently working with colleagues to develop digital twins that could eventually help solve the aforementioned cancer scenario — a doctor and patient decide the best course of cancer treatment. But their applications are manifold, particularly in clinical research.
Digital twins often include a machine learning component, which would fall under the umbrella term of AI, said Asghar, but it’s not like ChatGPT or other generative AI modules many people are now familiar with.
“The difference here is the model is not there to replace the clinician or to replace clinical trials,” Asghar noted. Instead, digital twins help make decisions faster in a way that can be more affordable.
Digital Twins to Predict Cancer Outcomes
Asghar is currently involved in UK clinical trials enrolling patients with cancer to test the accuracy of digital twin programs.
At this point, these studies do not yet use digital twins to guide the course of treatment, which is something they hope to do eventually. For now, they are still at the validation phase — the digital twin program makes predictions about the treatments and then the researchers later evaluate how accurate the predictions turned out to be based on real information from the enrolled patients.
Their current model gives predictions for RECIST (response evaluation criteria in solid tumor), treatment response, and survival. In addition to collecting data from ongoing clinical trials, they’ve used retrospective data, such as from the Cancer Tumor Atlas, to test the model.
“We’ve clinically validated it now in over 9000 patients,” said Asghar, who noted that they are constantly testing it on new patients. Their data include 30 chemotherapies and 23 cancer types, but they are focusing on four: Triple-negative breast cancer, cancer of unknown primary, pancreatic cancer, and colorectal cancer.
“The reason for choosing those four cancer types is that they are aggressive, their response to chemotherapy isn’t as great, and the outcome for those patient populations, there’s significant room for improvement,” Asghar explained.
Currently, Asghar said, the model is around 80%-90% correct in predicting what the actual clinical outcomes turn out to be.
The final stage of their work, before it becomes widely available to clinicians, will be to integrate it into a clinical trial in which some clinicians use the model to make decisions about treatment vs some who don’t use the model. By studying patient outcomes in both groups, they will be able to determine the value of the digital twin program they created.
What Else Can a Twin Do? A Lot
While a model that helps clinicians make decisions about cancer treatments may be among the first digital twin programs that become widely available, there are many other kinds of digital twins in the works.
For example, a digital twin could be used as a benchmark for a patient to determine how their cancer might have progressed without treatment. Say a patient’s tumor grew during treatment, it might seem like the treatment failed, but a digital twin might show that if left untreated, the tumor would have grown five times as fast, said Paul Macklin, PhD, professor in the Department of Intelligent Systems Engineering at Indiana University Bloomington.
Alternatively, if the virtual patient’s tumor is around the same size as the real patient’s tumor, “that means that treatment has lost its efficacy. It’s time to do something new,” said Macklin. And a digital twin could help with not only choosing a therapy but also choosing a dosing schedule, he noted.
The models can also be updated as new treatments come out, which could help clinicians virtually explore how they might affect a patient before having that patient switch treatments.
Digital twins could also assist in decision-making based on a patient’s priorities and real-life circumstances. “Maybe your priority is not necessarily to shrink this [tumor] at all costs ... maybe your priority is some mix of that and also quality of life,” Macklin said, referring to potential side effects. Or if someone lives 3 hours from the nearest cancer center, a digital twin could help determine whether less frequent treatments could still be effective.
And while much of the activity around digital twins in biomedical research has been focused on cancer, Asghar said the technology has the potential to be applied to other diseases as well. A digital twin for cardiovascular disease could help doctors choose the best treatment. It could also integrate new information from a smartwatch or glucose monitor to make better predictions and help doctors adjust the treatment plan.
Faster, More Effective Research With Twins
Because digital twin programs can quickly analyze large datasets, they can also make real-world studies more effective and efficient.
Though digital twins would not fully replace real clinical trials, they could help run through preliminary scenarios before starting a full clinical trial, which would “save everybody some money, time and pain and risk,” said Macklin.
It’s also possible to use digital twins to design better screening strategies for early cancer detection and monitoring, said Ioannis Zervantonakis, PhD, a bioengineering professor at the University of Pittsburgh.
Zervantonakis is tapping digital twin technology for research that homes in on understanding tumors. In this case, the digital twin is a virtual representation of a real tumor, complete with its complex network of cells and the surrounding tissue.
Zervantonakis’ lab is using the technology to study cell-cell interactions in the tumor microenvironment, with a focus on human epidermal growth factor receptor 2–targeted therapy resistance in breast cancer. The digital twin they developed will simulate tumor growth, predict drug response, analyze cellular interactions, and optimize treatment strategies.
The Long Push Forward
One big hurdle to making digital twins more widely available is that regulation for the technology is still in progress.
“We’re developing the technology, and what’s also happening is the regulatory framework is being developed in parallel. So we’re almost developing things blindly on the basis that we think this is what the regulators would want,” explained Asghar.
“It’s really important that these technologies are regulated properly, just like drugs, and that’s what we’re pushing and advocating for,” said Asghar, noting that people need to know that like drugs, a digital twin has strengths and limitations.
And while a digital twin can be a cost-saving approach in the long run, it does require funding to get a program built, and finding funds can be difficult because not everyone knows about the technology. More funding means more trials.
With more data, Asghar is hopeful that within a few years, a digital twin model could be available for clinicians to use to help inform treatment decisions. This could lead to more effective treatments and, ultimately, better patient outcomes.
A version of this article appeared on Medscape.com.
A patient has cancer. It’s decision time.
Clinician and patient alike face, really, the ultimate challenge when making those decisions. They have to consider the patient’s individual circumstances, available treatment options, potential side effects, relevant clinical data such as the patient’s genetic profile and cancer specifics, and more.
“That’s a lot of information to hold,” said Uzma Asghar, PhD, MRCP, a British consultant medical oncologist at The Royal Marsden Hospital and a chief scientific officer at Concr LTD.
What if there were a way to test — quickly and accurately — all the potential paths forward?
That’s the goal of digital twins.
“What the [digital twin] model can do for the clinician is to hold all that information and process it really quickly, within a couple of minutes,” Asghar noted.
A digital twin is more than just a computer model or simulation because it copies a real-world person and relies on real-world data. Some digital twin programs also integrate new information as it becomes available. This technology holds promise for personalized medicine, drug discovery, developing screening strategies, and better understanding diseases.
How to Deliver a Twin
To create a digital twin, experts develop a computer model with data to hone its expertise in an area of medicine, such as cancer types and treatments. Then “you train the model on information it’s seen, and then introduce a patient and patient’s information,” said Asghar.
Asghar is currently working with colleagues to develop digital twins that could eventually help solve the aforementioned cancer scenario — a doctor and patient decide the best course of cancer treatment. But their applications are manifold, particularly in clinical research.
Digital twins often include a machine learning component, which would fall under the umbrella term of AI, said Asghar, but it’s not like ChatGPT or other generative AI modules many people are now familiar with.
“The difference here is the model is not there to replace the clinician or to replace clinical trials,” Asghar noted. Instead, digital twins help make decisions faster in a way that can be more affordable.
Digital Twins to Predict Cancer Outcomes
Asghar is currently involved in UK clinical trials enrolling patients with cancer to test the accuracy of digital twin programs.
At this point, these studies do not yet use digital twins to guide the course of treatment, which is something they hope to do eventually. For now, they are still at the validation phase — the digital twin program makes predictions about the treatments and then the researchers later evaluate how accurate the predictions turned out to be based on real information from the enrolled patients.
Their current model gives predictions for RECIST (response evaluation criteria in solid tumor), treatment response, and survival. In addition to collecting data from ongoing clinical trials, they’ve used retrospective data, such as from the Cancer Tumor Atlas, to test the model.
“We’ve clinically validated it now in over 9000 patients,” said Asghar, who noted that they are constantly testing it on new patients. Their data include 30 chemotherapies and 23 cancer types, but they are focusing on four: Triple-negative breast cancer, cancer of unknown primary, pancreatic cancer, and colorectal cancer.
“The reason for choosing those four cancer types is that they are aggressive, their response to chemotherapy isn’t as great, and the outcome for those patient populations, there’s significant room for improvement,” Asghar explained.
Currently, Asghar said, the model is around 80%-90% correct in predicting what the actual clinical outcomes turn out to be.
The final stage of their work, before it becomes widely available to clinicians, will be to integrate it into a clinical trial in which some clinicians use the model to make decisions about treatment vs some who don’t use the model. By studying patient outcomes in both groups, they will be able to determine the value of the digital twin program they created.
What Else Can a Twin Do? A Lot
While a model that helps clinicians make decisions about cancer treatments may be among the first digital twin programs that become widely available, there are many other kinds of digital twins in the works.
For example, a digital twin could be used as a benchmark for a patient to determine how their cancer might have progressed without treatment. Say a patient’s tumor grew during treatment, it might seem like the treatment failed, but a digital twin might show that if left untreated, the tumor would have grown five times as fast, said Paul Macklin, PhD, professor in the Department of Intelligent Systems Engineering at Indiana University Bloomington.
Alternatively, if the virtual patient’s tumor is around the same size as the real patient’s tumor, “that means that treatment has lost its efficacy. It’s time to do something new,” said Macklin. And a digital twin could help with not only choosing a therapy but also choosing a dosing schedule, he noted.
The models can also be updated as new treatments come out, which could help clinicians virtually explore how they might affect a patient before having that patient switch treatments.
Digital twins could also assist in decision-making based on a patient’s priorities and real-life circumstances. “Maybe your priority is not necessarily to shrink this [tumor] at all costs ... maybe your priority is some mix of that and also quality of life,” Macklin said, referring to potential side effects. Or if someone lives 3 hours from the nearest cancer center, a digital twin could help determine whether less frequent treatments could still be effective.
And while much of the activity around digital twins in biomedical research has been focused on cancer, Asghar said the technology has the potential to be applied to other diseases as well. A digital twin for cardiovascular disease could help doctors choose the best treatment. It could also integrate new information from a smartwatch or glucose monitor to make better predictions and help doctors adjust the treatment plan.
Faster, More Effective Research With Twins
Because digital twin programs can quickly analyze large datasets, they can also make real-world studies more effective and efficient.
Though digital twins would not fully replace real clinical trials, they could help run through preliminary scenarios before starting a full clinical trial, which would “save everybody some money, time and pain and risk,” said Macklin.
It’s also possible to use digital twins to design better screening strategies for early cancer detection and monitoring, said Ioannis Zervantonakis, PhD, a bioengineering professor at the University of Pittsburgh.
Zervantonakis is tapping digital twin technology for research that homes in on understanding tumors. In this case, the digital twin is a virtual representation of a real tumor, complete with its complex network of cells and the surrounding tissue.
Zervantonakis’ lab is using the technology to study cell-cell interactions in the tumor microenvironment, with a focus on human epidermal growth factor receptor 2–targeted therapy resistance in breast cancer. The digital twin they developed will simulate tumor growth, predict drug response, analyze cellular interactions, and optimize treatment strategies.
The Long Push Forward
One big hurdle to making digital twins more widely available is that regulation for the technology is still in progress.
“We’re developing the technology, and what’s also happening is the regulatory framework is being developed in parallel. So we’re almost developing things blindly on the basis that we think this is what the regulators would want,” explained Asghar.
“It’s really important that these technologies are regulated properly, just like drugs, and that’s what we’re pushing and advocating for,” said Asghar, noting that people need to know that like drugs, a digital twin has strengths and limitations.
And while a digital twin can be a cost-saving approach in the long run, it does require funding to get a program built, and finding funds can be difficult because not everyone knows about the technology. More funding means more trials.
With more data, Asghar is hopeful that within a few years, a digital twin model could be available for clinicians to use to help inform treatment decisions. This could lead to more effective treatments and, ultimately, better patient outcomes.
A version of this article appeared on Medscape.com.
A patient has cancer. It’s decision time.
Clinician and patient alike face, really, the ultimate challenge when making those decisions. They have to consider the patient’s individual circumstances, available treatment options, potential side effects, relevant clinical data such as the patient’s genetic profile and cancer specifics, and more.
“That’s a lot of information to hold,” said Uzma Asghar, PhD, MRCP, a British consultant medical oncologist at The Royal Marsden Hospital and a chief scientific officer at Concr LTD.
What if there were a way to test — quickly and accurately — all the potential paths forward?
That’s the goal of digital twins.
“What the [digital twin] model can do for the clinician is to hold all that information and process it really quickly, within a couple of minutes,” Asghar noted.
A digital twin is more than just a computer model or simulation because it copies a real-world person and relies on real-world data. Some digital twin programs also integrate new information as it becomes available. This technology holds promise for personalized medicine, drug discovery, developing screening strategies, and better understanding diseases.
How to Deliver a Twin
To create a digital twin, experts develop a computer model with data to hone its expertise in an area of medicine, such as cancer types and treatments. Then “you train the model on information it’s seen, and then introduce a patient and patient’s information,” said Asghar.
Asghar is currently working with colleagues to develop digital twins that could eventually help solve the aforementioned cancer scenario — a doctor and patient decide the best course of cancer treatment. But their applications are manifold, particularly in clinical research.
Digital twins often include a machine learning component, which would fall under the umbrella term of AI, said Asghar, but it’s not like ChatGPT or other generative AI modules many people are now familiar with.
“The difference here is the model is not there to replace the clinician or to replace clinical trials,” Asghar noted. Instead, digital twins help make decisions faster in a way that can be more affordable.
Digital Twins to Predict Cancer Outcomes
Asghar is currently involved in UK clinical trials enrolling patients with cancer to test the accuracy of digital twin programs.
At this point, these studies do not yet use digital twins to guide the course of treatment, which is something they hope to do eventually. For now, they are still at the validation phase — the digital twin program makes predictions about the treatments and then the researchers later evaluate how accurate the predictions turned out to be based on real information from the enrolled patients.
Their current model gives predictions for RECIST (response evaluation criteria in solid tumor), treatment response, and survival. In addition to collecting data from ongoing clinical trials, they’ve used retrospective data, such as from the Cancer Tumor Atlas, to test the model.
“We’ve clinically validated it now in over 9000 patients,” said Asghar, who noted that they are constantly testing it on new patients. Their data include 30 chemotherapies and 23 cancer types, but they are focusing on four: Triple-negative breast cancer, cancer of unknown primary, pancreatic cancer, and colorectal cancer.
“The reason for choosing those four cancer types is that they are aggressive, their response to chemotherapy isn’t as great, and the outcome for those patient populations, there’s significant room for improvement,” Asghar explained.
Currently, Asghar said, the model is around 80%-90% correct in predicting what the actual clinical outcomes turn out to be.
The final stage of their work, before it becomes widely available to clinicians, will be to integrate it into a clinical trial in which some clinicians use the model to make decisions about treatment vs some who don’t use the model. By studying patient outcomes in both groups, they will be able to determine the value of the digital twin program they created.
What Else Can a Twin Do? A Lot
While a model that helps clinicians make decisions about cancer treatments may be among the first digital twin programs that become widely available, there are many other kinds of digital twins in the works.
For example, a digital twin could be used as a benchmark for a patient to determine how their cancer might have progressed without treatment. Say a patient’s tumor grew during treatment, it might seem like the treatment failed, but a digital twin might show that if left untreated, the tumor would have grown five times as fast, said Paul Macklin, PhD, professor in the Department of Intelligent Systems Engineering at Indiana University Bloomington.
Alternatively, if the virtual patient’s tumor is around the same size as the real patient’s tumor, “that means that treatment has lost its efficacy. It’s time to do something new,” said Macklin. And a digital twin could help with not only choosing a therapy but also choosing a dosing schedule, he noted.
The models can also be updated as new treatments come out, which could help clinicians virtually explore how they might affect a patient before having that patient switch treatments.
Digital twins could also assist in decision-making based on a patient’s priorities and real-life circumstances. “Maybe your priority is not necessarily to shrink this [tumor] at all costs ... maybe your priority is some mix of that and also quality of life,” Macklin said, referring to potential side effects. Or if someone lives 3 hours from the nearest cancer center, a digital twin could help determine whether less frequent treatments could still be effective.
And while much of the activity around digital twins in biomedical research has been focused on cancer, Asghar said the technology has the potential to be applied to other diseases as well. A digital twin for cardiovascular disease could help doctors choose the best treatment. It could also integrate new information from a smartwatch or glucose monitor to make better predictions and help doctors adjust the treatment plan.
Faster, More Effective Research With Twins
Because digital twin programs can quickly analyze large datasets, they can also make real-world studies more effective and efficient.
Though digital twins would not fully replace real clinical trials, they could help run through preliminary scenarios before starting a full clinical trial, which would “save everybody some money, time and pain and risk,” said Macklin.
It’s also possible to use digital twins to design better screening strategies for early cancer detection and monitoring, said Ioannis Zervantonakis, PhD, a bioengineering professor at the University of Pittsburgh.
Zervantonakis is tapping digital twin technology for research that homes in on understanding tumors. In this case, the digital twin is a virtual representation of a real tumor, complete with its complex network of cells and the surrounding tissue.
Zervantonakis’ lab is using the technology to study cell-cell interactions in the tumor microenvironment, with a focus on human epidermal growth factor receptor 2–targeted therapy resistance in breast cancer. The digital twin they developed will simulate tumor growth, predict drug response, analyze cellular interactions, and optimize treatment strategies.
The Long Push Forward
One big hurdle to making digital twins more widely available is that regulation for the technology is still in progress.
“We’re developing the technology, and what’s also happening is the regulatory framework is being developed in parallel. So we’re almost developing things blindly on the basis that we think this is what the regulators would want,” explained Asghar.
“It’s really important that these technologies are regulated properly, just like drugs, and that’s what we’re pushing and advocating for,” said Asghar, noting that people need to know that like drugs, a digital twin has strengths and limitations.
And while a digital twin can be a cost-saving approach in the long run, it does require funding to get a program built, and finding funds can be difficult because not everyone knows about the technology. More funding means more trials.
With more data, Asghar is hopeful that within a few years, a digital twin model could be available for clinicians to use to help inform treatment decisions. This could lead to more effective treatments and, ultimately, better patient outcomes.
A version of this article appeared on Medscape.com.
Smokeless Tobacco, Areca Nut Chewing Behind 1 in 3 Oral Cancers: IARC Report
“Smokeless tobacco and areca nut products are available to consumers in many different forms across the world, but consuming smokeless tobacco and areca nut is linked to multiple diseases, including oral cancer,” Harriet Rumgay, PhD, a scientist in the Cancer Surveillance Branch at IARC and first author of the study in Lancet Oncology, said in a news release.
Worldwide, about 300 million people use smokeless tobacco and 600 million people use areca (also called betel) nut, one of the most popular psychoactive substances in the world after nicotine, alcohol, and caffeine. Smokeless tobacco products are consumed without burning and can be chewed, sucked, inhaled, applied locally, or ingested. Areca nut is the seed of the areca palm and can be consumed in various forms.
“Our estimates highlight the burden these products pose on health care and the importance of prevention strategies to reduce consumption of smokeless tobacco and areca nut,” Rumgay said.
According to the new report, in 2022, an estimated 120,200 of the 389,800 (30.8%) global cases of oral cancer were attributable to these products.
More than three quarters (77%) of attributable cases were among men and about one quarter (23%) among women.
The vast majority (96%) of all oral cancer cases caused by smokeless tobacco and areca nut use occurred in low- and middle-income countries.
Regions with the highest burden of oral cancers from these products were Southcentral Asia — with 105,500 of 120,200 cases (nearly 88%), including 83,400 in India, 9700 in Bangladesh, 8900 in Pakistan, and 1300 in Sri Lanka — followed by Southeastern Asia with a total of 3900 cases (1600 in Myanmar, 990 in Indonesia, and 785 in Thailand) and East Asia with 3300 cases (3200 in China).
Limitations and Action Points
The authors noted a limitation of the analysis is not accounting for the potential synergistic effects of combined use of smokeless tobacco or areca nut products with other risk factors for oral cancer, such as smoking tobacco or drinking alcohol.
The researchers explained that combined consumption of smokeless tobacco or areca nut, smoked tobacco, and alcohol has a “multiplicative effect” on oral cancer risk, with reported odds ratios increasing from 2.7 for smokeless tobacco only, 7.0 for smoked tobacco only, and 1.6 for alcohol only to 16.2 for all three exposures (vs no use).
However, the proportion of people who chewed tobacco and also smoked in countries with high smokeless tobacco or areca nut use was small. In India, for example, 6% of men and 0.5% of women in 2016-2017 were dual users of both smoked and smokeless tobacco, compared with 23% of men and 12% of women who only used smokeless tobacco.
Overall, curbing or preventing smokeless tobacco and areca nut use could help avoid many instances of oral cancer.
Despite “encouraging trends” in control of tobacco smoking in many regions of the world over the past two decades, progress in reducing the prevalence of smokeless tobacco consumption has stalled in many countries that are major consumers, the authors said.
Compounding the problem, areca nut does not fall within the WHO framework of tobacco control and there are very few areca nut control policies worldwide.
Smokeless tobacco control must be “prioritized” and a framework on areca nut control should be developed with guidelines to incorporate areca nut prevention into cancer control programs, the authors concluded.
Funding for the study was provided by the French National Cancer Institute. The authors had no relevant disclosures.
A version of this article first appeared on Medscape.com.
“Smokeless tobacco and areca nut products are available to consumers in many different forms across the world, but consuming smokeless tobacco and areca nut is linked to multiple diseases, including oral cancer,” Harriet Rumgay, PhD, a scientist in the Cancer Surveillance Branch at IARC and first author of the study in Lancet Oncology, said in a news release.
Worldwide, about 300 million people use smokeless tobacco and 600 million people use areca (also called betel) nut, one of the most popular psychoactive substances in the world after nicotine, alcohol, and caffeine. Smokeless tobacco products are consumed without burning and can be chewed, sucked, inhaled, applied locally, or ingested. Areca nut is the seed of the areca palm and can be consumed in various forms.
“Our estimates highlight the burden these products pose on health care and the importance of prevention strategies to reduce consumption of smokeless tobacco and areca nut,” Rumgay said.
According to the new report, in 2022, an estimated 120,200 of the 389,800 (30.8%) global cases of oral cancer were attributable to these products.
More than three quarters (77%) of attributable cases were among men and about one quarter (23%) among women.
The vast majority (96%) of all oral cancer cases caused by smokeless tobacco and areca nut use occurred in low- and middle-income countries.
Regions with the highest burden of oral cancers from these products were Southcentral Asia — with 105,500 of 120,200 cases (nearly 88%), including 83,400 in India, 9700 in Bangladesh, 8900 in Pakistan, and 1300 in Sri Lanka — followed by Southeastern Asia with a total of 3900 cases (1600 in Myanmar, 990 in Indonesia, and 785 in Thailand) and East Asia with 3300 cases (3200 in China).
Limitations and Action Points
The authors noted a limitation of the analysis is not accounting for the potential synergistic effects of combined use of smokeless tobacco or areca nut products with other risk factors for oral cancer, such as smoking tobacco or drinking alcohol.
The researchers explained that combined consumption of smokeless tobacco or areca nut, smoked tobacco, and alcohol has a “multiplicative effect” on oral cancer risk, with reported odds ratios increasing from 2.7 for smokeless tobacco only, 7.0 for smoked tobacco only, and 1.6 for alcohol only to 16.2 for all three exposures (vs no use).
However, the proportion of people who chewed tobacco and also smoked in countries with high smokeless tobacco or areca nut use was small. In India, for example, 6% of men and 0.5% of women in 2016-2017 were dual users of both smoked and smokeless tobacco, compared with 23% of men and 12% of women who only used smokeless tobacco.
Overall, curbing or preventing smokeless tobacco and areca nut use could help avoid many instances of oral cancer.
Despite “encouraging trends” in control of tobacco smoking in many regions of the world over the past two decades, progress in reducing the prevalence of smokeless tobacco consumption has stalled in many countries that are major consumers, the authors said.
Compounding the problem, areca nut does not fall within the WHO framework of tobacco control and there are very few areca nut control policies worldwide.
Smokeless tobacco control must be “prioritized” and a framework on areca nut control should be developed with guidelines to incorporate areca nut prevention into cancer control programs, the authors concluded.
Funding for the study was provided by the French National Cancer Institute. The authors had no relevant disclosures.
A version of this article first appeared on Medscape.com.
“Smokeless tobacco and areca nut products are available to consumers in many different forms across the world, but consuming smokeless tobacco and areca nut is linked to multiple diseases, including oral cancer,” Harriet Rumgay, PhD, a scientist in the Cancer Surveillance Branch at IARC and first author of the study in Lancet Oncology, said in a news release.
Worldwide, about 300 million people use smokeless tobacco and 600 million people use areca (also called betel) nut, one of the most popular psychoactive substances in the world after nicotine, alcohol, and caffeine. Smokeless tobacco products are consumed without burning and can be chewed, sucked, inhaled, applied locally, or ingested. Areca nut is the seed of the areca palm and can be consumed in various forms.
“Our estimates highlight the burden these products pose on health care and the importance of prevention strategies to reduce consumption of smokeless tobacco and areca nut,” Rumgay said.
According to the new report, in 2022, an estimated 120,200 of the 389,800 (30.8%) global cases of oral cancer were attributable to these products.
More than three quarters (77%) of attributable cases were among men and about one quarter (23%) among women.
The vast majority (96%) of all oral cancer cases caused by smokeless tobacco and areca nut use occurred in low- and middle-income countries.
Regions with the highest burden of oral cancers from these products were Southcentral Asia — with 105,500 of 120,200 cases (nearly 88%), including 83,400 in India, 9700 in Bangladesh, 8900 in Pakistan, and 1300 in Sri Lanka — followed by Southeastern Asia with a total of 3900 cases (1600 in Myanmar, 990 in Indonesia, and 785 in Thailand) and East Asia with 3300 cases (3200 in China).
Limitations and Action Points
The authors noted a limitation of the analysis is not accounting for the potential synergistic effects of combined use of smokeless tobacco or areca nut products with other risk factors for oral cancer, such as smoking tobacco or drinking alcohol.
The researchers explained that combined consumption of smokeless tobacco or areca nut, smoked tobacco, and alcohol has a “multiplicative effect” on oral cancer risk, with reported odds ratios increasing from 2.7 for smokeless tobacco only, 7.0 for smoked tobacco only, and 1.6 for alcohol only to 16.2 for all three exposures (vs no use).
However, the proportion of people who chewed tobacco and also smoked in countries with high smokeless tobacco or areca nut use was small. In India, for example, 6% of men and 0.5% of women in 2016-2017 were dual users of both smoked and smokeless tobacco, compared with 23% of men and 12% of women who only used smokeless tobacco.
Overall, curbing or preventing smokeless tobacco and areca nut use could help avoid many instances of oral cancer.
Despite “encouraging trends” in control of tobacco smoking in many regions of the world over the past two decades, progress in reducing the prevalence of smokeless tobacco consumption has stalled in many countries that are major consumers, the authors said.
Compounding the problem, areca nut does not fall within the WHO framework of tobacco control and there are very few areca nut control policies worldwide.
Smokeless tobacco control must be “prioritized” and a framework on areca nut control should be developed with guidelines to incorporate areca nut prevention into cancer control programs, the authors concluded.
Funding for the study was provided by the French National Cancer Institute. The authors had no relevant disclosures.
A version of this article first appeared on Medscape.com.
FROM THE LANCET ONCOLOGY
Does Radiation Timing Affect QOL After Prostate Surgery?
TOPLINE:
METHODOLOGY:
- Delaying radiotherapy after prostatectomy can help avoid overtreatment and mitigate genitourinary and erectile toxic effects. However, few studies have compared long-term patient-reported health-related quality-of-life outcomes on the basis of the timing of postprostatectomy radiotherapy.
- Researchers evaluated 1203 men (median age, 60.5 years; 92% were White and 6.8% were Black) with localized prostate cancer who underwent radical prostatectomy from the PROST-QA (2003-2006) and RP2 Consortium (2010-2013). Among these patients, 1082 underwent surgery only, 57 received early radiotherapy (within 12 months of surgery), and 64 underwent late radiotherapy (12 months or more after surgery).
- Patients who received early radiotherapy were more likely to receive androgen deprivation therapy than those who underwent late radiotherapy (40.4% vs 12.5%; P < .001).
- Primary outcome was health-related quality of life measured using the Expanded Prostate Cancer Index Composite at baseline, 2, 6, and 12 months, and annually after that. Health-related quality-of-life measures included sexual function, urinary incontinence, urinary irritation and/or obstruction, and bowel or rectal function.
- The median follow-up duration was 85.6 months.
TAKEAWAY:
- Postprostatectomy radiotherapy was associated with a significantly greater decline in health-related quality of life across all domains, including sexual function and urinary incontinence.
- Patients who received early radiation initially experienced worse urinary incontinence and sexual health, compared with patients in the late group, but the early group also had higher-risk disease and were more likely to receive concurrent androgen deprivation therapy.
- In the long term, the early radiotherapy group experienced more pronounced recovery of sexual function, urinary irritation, and urinary incontinence than the late radiotherapy group.
- Ultimately, patients in the early radiotherapy group had similar, potentially better, long-term health-related quality-of-life domain scores than those in the late group over the long term. For instance, the likelihood of being pad free increased for patients treated early with radiation, while it decreased for those treated late. In patients who received early radiation, the rate of freedom from pad use increased from 39% before radiation to 67% at the sixth follow-up visit after radiation, while it decreased from 73% to 48% in those who received late radiation.
IN PRACTICE:
“Long-term patient-reported sexual, incontinence, and urinary irritative outcomes did not significantly differ between early vs late postprostatectomy [radiotherapy],” the authors said. In fact, “men receiving early [radiation] experienced greater recovery of these toxicity domains and achieved similar, and possibly better, domain scores as those receiving late [radiation] at long-term follow-up.” Overall, “these results may help guide treatment counseling and support consideration of early [radiotherapy] after prostatectomy for men at particularly high risk of recurrence and metastasis.”
SOURCE:
The study, led by Sagar A. Patel, MD, MSc, Emory University in Atlanta, was published online in JAMA Network Open.
LIMITATIONS:
The early and late postprostatectomy radiotherapy groups were relatively small and underpowered to detect statistically significant differences between groups. The study has a nonrandomized design, which may introduce unaccounted for imbalances among the different groups. The study did not directly compare health-related quality of life between patients receiving adjuvant vs salvage radiotherapy.
DISCLOSURES:
This study received funding from National Institutes of Health grants and the Paul Calabresi Career Development Award for Clinical Oncology. Several authors reported receiving personal fees, grants, and having other ties with various sources. Additional disclosures are noted in the original article.
A version of this article appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
- Delaying radiotherapy after prostatectomy can help avoid overtreatment and mitigate genitourinary and erectile toxic effects. However, few studies have compared long-term patient-reported health-related quality-of-life outcomes on the basis of the timing of postprostatectomy radiotherapy.
- Researchers evaluated 1203 men (median age, 60.5 years; 92% were White and 6.8% were Black) with localized prostate cancer who underwent radical prostatectomy from the PROST-QA (2003-2006) and RP2 Consortium (2010-2013). Among these patients, 1082 underwent surgery only, 57 received early radiotherapy (within 12 months of surgery), and 64 underwent late radiotherapy (12 months or more after surgery).
- Patients who received early radiotherapy were more likely to receive androgen deprivation therapy than those who underwent late radiotherapy (40.4% vs 12.5%; P < .001).
- Primary outcome was health-related quality of life measured using the Expanded Prostate Cancer Index Composite at baseline, 2, 6, and 12 months, and annually after that. Health-related quality-of-life measures included sexual function, urinary incontinence, urinary irritation and/or obstruction, and bowel or rectal function.
- The median follow-up duration was 85.6 months.
TAKEAWAY:
- Postprostatectomy radiotherapy was associated with a significantly greater decline in health-related quality of life across all domains, including sexual function and urinary incontinence.
- Patients who received early radiation initially experienced worse urinary incontinence and sexual health, compared with patients in the late group, but the early group also had higher-risk disease and were more likely to receive concurrent androgen deprivation therapy.
- In the long term, the early radiotherapy group experienced more pronounced recovery of sexual function, urinary irritation, and urinary incontinence than the late radiotherapy group.
- Ultimately, patients in the early radiotherapy group had similar, potentially better, long-term health-related quality-of-life domain scores than those in the late group over the long term. For instance, the likelihood of being pad free increased for patients treated early with radiation, while it decreased for those treated late. In patients who received early radiation, the rate of freedom from pad use increased from 39% before radiation to 67% at the sixth follow-up visit after radiation, while it decreased from 73% to 48% in those who received late radiation.
IN PRACTICE:
“Long-term patient-reported sexual, incontinence, and urinary irritative outcomes did not significantly differ between early vs late postprostatectomy [radiotherapy],” the authors said. In fact, “men receiving early [radiation] experienced greater recovery of these toxicity domains and achieved similar, and possibly better, domain scores as those receiving late [radiation] at long-term follow-up.” Overall, “these results may help guide treatment counseling and support consideration of early [radiotherapy] after prostatectomy for men at particularly high risk of recurrence and metastasis.”
SOURCE:
The study, led by Sagar A. Patel, MD, MSc, Emory University in Atlanta, was published online in JAMA Network Open.
LIMITATIONS:
The early and late postprostatectomy radiotherapy groups were relatively small and underpowered to detect statistically significant differences between groups. The study has a nonrandomized design, which may introduce unaccounted for imbalances among the different groups. The study did not directly compare health-related quality of life between patients receiving adjuvant vs salvage radiotherapy.
DISCLOSURES:
This study received funding from National Institutes of Health grants and the Paul Calabresi Career Development Award for Clinical Oncology. Several authors reported receiving personal fees, grants, and having other ties with various sources. Additional disclosures are noted in the original article.
A version of this article appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
- Delaying radiotherapy after prostatectomy can help avoid overtreatment and mitigate genitourinary and erectile toxic effects. However, few studies have compared long-term patient-reported health-related quality-of-life outcomes on the basis of the timing of postprostatectomy radiotherapy.
- Researchers evaluated 1203 men (median age, 60.5 years; 92% were White and 6.8% were Black) with localized prostate cancer who underwent radical prostatectomy from the PROST-QA (2003-2006) and RP2 Consortium (2010-2013). Among these patients, 1082 underwent surgery only, 57 received early radiotherapy (within 12 months of surgery), and 64 underwent late radiotherapy (12 months or more after surgery).
- Patients who received early radiotherapy were more likely to receive androgen deprivation therapy than those who underwent late radiotherapy (40.4% vs 12.5%; P < .001).
- Primary outcome was health-related quality of life measured using the Expanded Prostate Cancer Index Composite at baseline, 2, 6, and 12 months, and annually after that. Health-related quality-of-life measures included sexual function, urinary incontinence, urinary irritation and/or obstruction, and bowel or rectal function.
- The median follow-up duration was 85.6 months.
TAKEAWAY:
- Postprostatectomy radiotherapy was associated with a significantly greater decline in health-related quality of life across all domains, including sexual function and urinary incontinence.
- Patients who received early radiation initially experienced worse urinary incontinence and sexual health, compared with patients in the late group, but the early group also had higher-risk disease and were more likely to receive concurrent androgen deprivation therapy.
- In the long term, the early radiotherapy group experienced more pronounced recovery of sexual function, urinary irritation, and urinary incontinence than the late radiotherapy group.
- Ultimately, patients in the early radiotherapy group had similar, potentially better, long-term health-related quality-of-life domain scores than those in the late group over the long term. For instance, the likelihood of being pad free increased for patients treated early with radiation, while it decreased for those treated late. In patients who received early radiation, the rate of freedom from pad use increased from 39% before radiation to 67% at the sixth follow-up visit after radiation, while it decreased from 73% to 48% in those who received late radiation.
IN PRACTICE:
“Long-term patient-reported sexual, incontinence, and urinary irritative outcomes did not significantly differ between early vs late postprostatectomy [radiotherapy],” the authors said. In fact, “men receiving early [radiation] experienced greater recovery of these toxicity domains and achieved similar, and possibly better, domain scores as those receiving late [radiation] at long-term follow-up.” Overall, “these results may help guide treatment counseling and support consideration of early [radiotherapy] after prostatectomy for men at particularly high risk of recurrence and metastasis.”
SOURCE:
The study, led by Sagar A. Patel, MD, MSc, Emory University in Atlanta, was published online in JAMA Network Open.
LIMITATIONS:
The early and late postprostatectomy radiotherapy groups were relatively small and underpowered to detect statistically significant differences between groups. The study has a nonrandomized design, which may introduce unaccounted for imbalances among the different groups. The study did not directly compare health-related quality of life between patients receiving adjuvant vs salvage radiotherapy.
DISCLOSURES:
This study received funding from National Institutes of Health grants and the Paul Calabresi Career Development Award for Clinical Oncology. Several authors reported receiving personal fees, grants, and having other ties with various sources. Additional disclosures are noted in the original article.
A version of this article appeared on Medscape.com.
What Matters Most for Young Patients With CRC: Survey Highlights Top Concerns
“We tend to think of cancer as a disease of older populations, but it’s impacting younger people who are in important developmental stages of their lives,” said Samantha Savitch, MD, in a podcast from the American College of Surgeons (ACS) Clinical Congress 2024, where she presented her research.
In fact, since 1994, cases of young-onset CRC have increased by more than 50%, according to the National Cancer Institute.
“Our goal with the study was to better understand what young adults with colorectal cancer really care about, so that we can ensure that we’re properly addressing their needs as part of like comprehensive cancer care,” Savitch, with the University of Michigan, Ann Arbor, Michigan, explained.
The researchers interviewed a sample of 35 patients who were diagnosed with CRC before the age of 50 years. The researchers asked patients open-ended questions about the influence their CRC diagnosis had on their lives, the daily challenges they experienced, as well as concerns about the future.
Patients expressed the greatest concern about four areas of health and well-being: Physical health, mental health, family planning, and career.
For physical health, patients worried about incontinence, loss of vitality, and expenses related to healthcare. On the mental health front, patients expressed concern about the uncertainty surrounding long-term survival and anxiety about the timing of their diagnosis. Family planning was a key issue as well, with patients highlighting uncertainties about fertility after chemotherapy. On the career front, patients also noted concerns surrounding job security, challenges pursuing advanced degrees, and a reliance on benefits from employment.
These concerns were not gender-specific. Career, physical health, financial security, mental health, fertility, and family planning were equally important to men and women.
Savitch provided a sample of quotes from interviewees that illustrated their specific concerns in each category.
A 47-year-old man reflected on his physical health now that his rectum is gone. “I no longer have that feeling of sensation like in my cheeks; basically, the cheeks and the anus area is all dead,” he said. A 48-year-old woman discussed the havoc chemotherapy wrecked on her teeth. “I don’t want to get emotional, I just went to the dentist yesterday, and I just get so frustrated ... All these things to pay. I should be happy to be alive,” she said. But “I have so much money in my mouth.”
On the mental health front, a 34-year-old woman described the fear she felt about a cancer recurrence following the birth of her daughter. After a CT scan, she had to experience 2 weeks of limbo, thinking, “I have cancer again.” She had begun a journal dedicated to her daughter in case she had a recurrence and died. “I always think that I am going to die. I think about death every day.”
Reflecting on her future fertility, a 22-year-old woman recalled the uncertainty surrounding whether chemotherapy would affect her ability to have children. “I would get really nervous,” she said, “so I was like, ‘I will do the injections. I just want to save a few of my eggs just in case.’ ” A 33-year-old man opted not to freeze his sperm because “I didn’t know if I was going to live or die, I didn’t know anything ... I barely had any money. So, like, do I risk putting this money up to freeze something when I don’t even know if I am going to be here or not?”
On the career front, a 48-year-old man highlighted how his cancer completely changed his family’s life.”I went from being a provider for my family, making enough money to take care of my family, where my wife was staying home, to now not being able to work and her having to pick up little side jobs and stuff just to try to help make ends meet.”
“These aspects of cancer care are rarely discussed, so it is important to acknowledge that patients care about fertility and family planning, their career aspirations, building assets — all things they must put on hold because of their cancer diagnosis,” Savitch said in a news release.
“This goes beyond just colorectal cancer,” Savitch added. “There are a lot of patients experiencing similar challenges, so we need more research to better understand these issues in patients with colorectal cancer as well as other cancers and, ultimately, to restructure our comprehensive cancer programs to make sure we are treating the patient and not just the disease.”
Support for the study was provided by the Rogel Cancer Center at the University of Michigan. Savitch had no relevant disclosures.
A version of this article first appeared on Medscape.com.
“We tend to think of cancer as a disease of older populations, but it’s impacting younger people who are in important developmental stages of their lives,” said Samantha Savitch, MD, in a podcast from the American College of Surgeons (ACS) Clinical Congress 2024, where she presented her research.
In fact, since 1994, cases of young-onset CRC have increased by more than 50%, according to the National Cancer Institute.
“Our goal with the study was to better understand what young adults with colorectal cancer really care about, so that we can ensure that we’re properly addressing their needs as part of like comprehensive cancer care,” Savitch, with the University of Michigan, Ann Arbor, Michigan, explained.
The researchers interviewed a sample of 35 patients who were diagnosed with CRC before the age of 50 years. The researchers asked patients open-ended questions about the influence their CRC diagnosis had on their lives, the daily challenges they experienced, as well as concerns about the future.
Patients expressed the greatest concern about four areas of health and well-being: Physical health, mental health, family planning, and career.
For physical health, patients worried about incontinence, loss of vitality, and expenses related to healthcare. On the mental health front, patients expressed concern about the uncertainty surrounding long-term survival and anxiety about the timing of their diagnosis. Family planning was a key issue as well, with patients highlighting uncertainties about fertility after chemotherapy. On the career front, patients also noted concerns surrounding job security, challenges pursuing advanced degrees, and a reliance on benefits from employment.
These concerns were not gender-specific. Career, physical health, financial security, mental health, fertility, and family planning were equally important to men and women.
Savitch provided a sample of quotes from interviewees that illustrated their specific concerns in each category.
A 47-year-old man reflected on his physical health now that his rectum is gone. “I no longer have that feeling of sensation like in my cheeks; basically, the cheeks and the anus area is all dead,” he said. A 48-year-old woman discussed the havoc chemotherapy wrecked on her teeth. “I don’t want to get emotional, I just went to the dentist yesterday, and I just get so frustrated ... All these things to pay. I should be happy to be alive,” she said. But “I have so much money in my mouth.”
On the mental health front, a 34-year-old woman described the fear she felt about a cancer recurrence following the birth of her daughter. After a CT scan, she had to experience 2 weeks of limbo, thinking, “I have cancer again.” She had begun a journal dedicated to her daughter in case she had a recurrence and died. “I always think that I am going to die. I think about death every day.”
Reflecting on her future fertility, a 22-year-old woman recalled the uncertainty surrounding whether chemotherapy would affect her ability to have children. “I would get really nervous,” she said, “so I was like, ‘I will do the injections. I just want to save a few of my eggs just in case.’ ” A 33-year-old man opted not to freeze his sperm because “I didn’t know if I was going to live or die, I didn’t know anything ... I barely had any money. So, like, do I risk putting this money up to freeze something when I don’t even know if I am going to be here or not?”
On the career front, a 48-year-old man highlighted how his cancer completely changed his family’s life.”I went from being a provider for my family, making enough money to take care of my family, where my wife was staying home, to now not being able to work and her having to pick up little side jobs and stuff just to try to help make ends meet.”
“These aspects of cancer care are rarely discussed, so it is important to acknowledge that patients care about fertility and family planning, their career aspirations, building assets — all things they must put on hold because of their cancer diagnosis,” Savitch said in a news release.
“This goes beyond just colorectal cancer,” Savitch added. “There are a lot of patients experiencing similar challenges, so we need more research to better understand these issues in patients with colorectal cancer as well as other cancers and, ultimately, to restructure our comprehensive cancer programs to make sure we are treating the patient and not just the disease.”
Support for the study was provided by the Rogel Cancer Center at the University of Michigan. Savitch had no relevant disclosures.
A version of this article first appeared on Medscape.com.
“We tend to think of cancer as a disease of older populations, but it’s impacting younger people who are in important developmental stages of their lives,” said Samantha Savitch, MD, in a podcast from the American College of Surgeons (ACS) Clinical Congress 2024, where she presented her research.
In fact, since 1994, cases of young-onset CRC have increased by more than 50%, according to the National Cancer Institute.
“Our goal with the study was to better understand what young adults with colorectal cancer really care about, so that we can ensure that we’re properly addressing their needs as part of like comprehensive cancer care,” Savitch, with the University of Michigan, Ann Arbor, Michigan, explained.
The researchers interviewed a sample of 35 patients who were diagnosed with CRC before the age of 50 years. The researchers asked patients open-ended questions about the influence their CRC diagnosis had on their lives, the daily challenges they experienced, as well as concerns about the future.
Patients expressed the greatest concern about four areas of health and well-being: Physical health, mental health, family planning, and career.
For physical health, patients worried about incontinence, loss of vitality, and expenses related to healthcare. On the mental health front, patients expressed concern about the uncertainty surrounding long-term survival and anxiety about the timing of their diagnosis. Family planning was a key issue as well, with patients highlighting uncertainties about fertility after chemotherapy. On the career front, patients also noted concerns surrounding job security, challenges pursuing advanced degrees, and a reliance on benefits from employment.
These concerns were not gender-specific. Career, physical health, financial security, mental health, fertility, and family planning were equally important to men and women.
Savitch provided a sample of quotes from interviewees that illustrated their specific concerns in each category.
A 47-year-old man reflected on his physical health now that his rectum is gone. “I no longer have that feeling of sensation like in my cheeks; basically, the cheeks and the anus area is all dead,” he said. A 48-year-old woman discussed the havoc chemotherapy wrecked on her teeth. “I don’t want to get emotional, I just went to the dentist yesterday, and I just get so frustrated ... All these things to pay. I should be happy to be alive,” she said. But “I have so much money in my mouth.”
On the mental health front, a 34-year-old woman described the fear she felt about a cancer recurrence following the birth of her daughter. After a CT scan, she had to experience 2 weeks of limbo, thinking, “I have cancer again.” She had begun a journal dedicated to her daughter in case she had a recurrence and died. “I always think that I am going to die. I think about death every day.”
Reflecting on her future fertility, a 22-year-old woman recalled the uncertainty surrounding whether chemotherapy would affect her ability to have children. “I would get really nervous,” she said, “so I was like, ‘I will do the injections. I just want to save a few of my eggs just in case.’ ” A 33-year-old man opted not to freeze his sperm because “I didn’t know if I was going to live or die, I didn’t know anything ... I barely had any money. So, like, do I risk putting this money up to freeze something when I don’t even know if I am going to be here or not?”
On the career front, a 48-year-old man highlighted how his cancer completely changed his family’s life.”I went from being a provider for my family, making enough money to take care of my family, where my wife was staying home, to now not being able to work and her having to pick up little side jobs and stuff just to try to help make ends meet.”
“These aspects of cancer care are rarely discussed, so it is important to acknowledge that patients care about fertility and family planning, their career aspirations, building assets — all things they must put on hold because of their cancer diagnosis,” Savitch said in a news release.
“This goes beyond just colorectal cancer,” Savitch added. “There are a lot of patients experiencing similar challenges, so we need more research to better understand these issues in patients with colorectal cancer as well as other cancers and, ultimately, to restructure our comprehensive cancer programs to make sure we are treating the patient and not just the disease.”
Support for the study was provided by the Rogel Cancer Center at the University of Michigan. Savitch had no relevant disclosures.
A version of this article first appeared on Medscape.com.
FROM ACS 2024
Many Patients With Cancer Visit EDs Before Diagnosis
Researchers examined Institute for Clinical Evaluative Sciences (ICES) data that had been gathered from January 1, 2014, to December 31, 2021. The study focused on patients aged 18 years or older with confirmed primary cancer diagnoses.
Factors associated with an increased likelihood of an ED visit ahead of diagnosis included having certain cancers, living in rural areas, and having less access to primary care, according to study author Keerat Grewal, MD, an emergency physician and clinician scientist at the Schwartz/Reisman Emergency Medicine Institute at Sinai Health in Toronto, Ontario, Canada, and coauthors.
“The ED is a distressing environment for patients to receive a possible cancer diagnosis,” the authors wrote. “Moreover, it is frequently ill equipped to provide ongoing continuity of care, which can lead patients down a poorly defined diagnostic pathway before receiving a confirmed diagnosis based on tissue and a subsequent treatment plan.”
The findings were published online on November 4 in CMAJ).
Neurologic Cancers Prominent
In an interview, Grewal said in an interview that the study reflects her desire as an emergency room physician to understand why so many patients with cancer get the initial reports about their disease from clinicians whom they often have just met for the first time.
Among patients with an ED visit before cancer diagnosis, 51.4% were admitted to hospital from the most recent visit.
Compared with patients with a family physician on whom they could rely for routine care, those who had no outpatient visits (odds ratio [OR], 2.09) or fewer than three outpatient visits (OR, 1.41) in the 6-30 months before cancer diagnosis were more likely to have an ED visit before their cancer diagnosis.
Other factors associated with increased odds of ED use before cancer diagnosis included rurality (OR, 1.15), residence in northern Ontario (northeast region: OR, 1.14 and northwest region: OR, 1.27 vs Toronto region), and living in the most marginalized areas (material resource deprivation: OR, 1.37 and housing stability: OR, 1.09 vs least marginalized area).
The researchers also found that patients with certain cancers were more likely to have sought care in the ED. They compared these cancers with breast cancer, which is often detected through screening.
“Patients with neurologic cancers had extremely high odds of ED use before cancer diagnosis,” the authors wrote. “This is likely because of the emergent nature of presentation, with acute neurologic symptoms such as weakness, confusion, or seizures, which require urgent assessment.” On the other hand, pancreatic, liver, or thoracic cancer can trigger nonspecific symptoms that may be ignored until they reach a crisis level that prompts an ED visit.
The limitations of the study included its inability to identify cancer-related ED visits and its narrow focus on patients in Ontario, according to the researchers. But the use of the ICES databases also allowed researchers access to a broader pool of data than are available in many other cases.
The findings in the new paper echo those of previous research, the authors noted. Research in the United Kingdom found that 24%-31% of cancer diagnoses involved the ED. In addition, a study of people enrolled in the US Medicare program, which serves patients aged 65 years or older, found that 23% were seen in the ED in the 30 days before diagnosis.
‘Unpacking the Data’
The current findings also are consistent with those of an International Cancer Benchmarking Partnership study that was published in 2022 in The Lancet Oncology, said Erika Nicholson, MHS, vice president of cancer systems and innovation at the Canadian Partnership Against Cancer. The latter study analyzed cancer registration and linked hospital admissions data from 14 jurisdictions in Australia, Canada, Denmark, New Zealand, Norway, and the United Kingdom.
“We see similar trends in terms of people visiting EDs and being diagnosed through EDs internationally,” Nicholson said. “We’re working with partners to put in place different strategies to address the challenges” that this phenomenon presents in terms of improving screening and follow-up care.
“Cancer is not one disease, but many diseases,” she said. “They present differently. We’re focused on really unpacking the data and understanding them.”
All this research highlights the need for more services and personnel to address cancer, including people who are trained to help patients cope after getting concerning news through emergency care, she said.
“That means having a system that fully supports you and helps you navigate through that diagnostic process,” Nicholson said. Addressing the added challenges for patients who don’t have secure housing is a special need, she added.
This study was supported by the Canadian Institutes of Health Research (CIHR). Grewal reported receiving grants from CIHR and the Canadian Association of Emergency Physicians. Nicholson reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
Researchers examined Institute for Clinical Evaluative Sciences (ICES) data that had been gathered from January 1, 2014, to December 31, 2021. The study focused on patients aged 18 years or older with confirmed primary cancer diagnoses.
Factors associated with an increased likelihood of an ED visit ahead of diagnosis included having certain cancers, living in rural areas, and having less access to primary care, according to study author Keerat Grewal, MD, an emergency physician and clinician scientist at the Schwartz/Reisman Emergency Medicine Institute at Sinai Health in Toronto, Ontario, Canada, and coauthors.
“The ED is a distressing environment for patients to receive a possible cancer diagnosis,” the authors wrote. “Moreover, it is frequently ill equipped to provide ongoing continuity of care, which can lead patients down a poorly defined diagnostic pathway before receiving a confirmed diagnosis based on tissue and a subsequent treatment plan.”
The findings were published online on November 4 in CMAJ).
Neurologic Cancers Prominent
In an interview, Grewal said in an interview that the study reflects her desire as an emergency room physician to understand why so many patients with cancer get the initial reports about their disease from clinicians whom they often have just met for the first time.
Among patients with an ED visit before cancer diagnosis, 51.4% were admitted to hospital from the most recent visit.
Compared with patients with a family physician on whom they could rely for routine care, those who had no outpatient visits (odds ratio [OR], 2.09) or fewer than three outpatient visits (OR, 1.41) in the 6-30 months before cancer diagnosis were more likely to have an ED visit before their cancer diagnosis.
Other factors associated with increased odds of ED use before cancer diagnosis included rurality (OR, 1.15), residence in northern Ontario (northeast region: OR, 1.14 and northwest region: OR, 1.27 vs Toronto region), and living in the most marginalized areas (material resource deprivation: OR, 1.37 and housing stability: OR, 1.09 vs least marginalized area).
The researchers also found that patients with certain cancers were more likely to have sought care in the ED. They compared these cancers with breast cancer, which is often detected through screening.
“Patients with neurologic cancers had extremely high odds of ED use before cancer diagnosis,” the authors wrote. “This is likely because of the emergent nature of presentation, with acute neurologic symptoms such as weakness, confusion, or seizures, which require urgent assessment.” On the other hand, pancreatic, liver, or thoracic cancer can trigger nonspecific symptoms that may be ignored until they reach a crisis level that prompts an ED visit.
The limitations of the study included its inability to identify cancer-related ED visits and its narrow focus on patients in Ontario, according to the researchers. But the use of the ICES databases also allowed researchers access to a broader pool of data than are available in many other cases.
The findings in the new paper echo those of previous research, the authors noted. Research in the United Kingdom found that 24%-31% of cancer diagnoses involved the ED. In addition, a study of people enrolled in the US Medicare program, which serves patients aged 65 years or older, found that 23% were seen in the ED in the 30 days before diagnosis.
‘Unpacking the Data’
The current findings also are consistent with those of an International Cancer Benchmarking Partnership study that was published in 2022 in The Lancet Oncology, said Erika Nicholson, MHS, vice president of cancer systems and innovation at the Canadian Partnership Against Cancer. The latter study analyzed cancer registration and linked hospital admissions data from 14 jurisdictions in Australia, Canada, Denmark, New Zealand, Norway, and the United Kingdom.
“We see similar trends in terms of people visiting EDs and being diagnosed through EDs internationally,” Nicholson said. “We’re working with partners to put in place different strategies to address the challenges” that this phenomenon presents in terms of improving screening and follow-up care.
“Cancer is not one disease, but many diseases,” she said. “They present differently. We’re focused on really unpacking the data and understanding them.”
All this research highlights the need for more services and personnel to address cancer, including people who are trained to help patients cope after getting concerning news through emergency care, she said.
“That means having a system that fully supports you and helps you navigate through that diagnostic process,” Nicholson said. Addressing the added challenges for patients who don’t have secure housing is a special need, she added.
This study was supported by the Canadian Institutes of Health Research (CIHR). Grewal reported receiving grants from CIHR and the Canadian Association of Emergency Physicians. Nicholson reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
Researchers examined Institute for Clinical Evaluative Sciences (ICES) data that had been gathered from January 1, 2014, to December 31, 2021. The study focused on patients aged 18 years or older with confirmed primary cancer diagnoses.
Factors associated with an increased likelihood of an ED visit ahead of diagnosis included having certain cancers, living in rural areas, and having less access to primary care, according to study author Keerat Grewal, MD, an emergency physician and clinician scientist at the Schwartz/Reisman Emergency Medicine Institute at Sinai Health in Toronto, Ontario, Canada, and coauthors.
“The ED is a distressing environment for patients to receive a possible cancer diagnosis,” the authors wrote. “Moreover, it is frequently ill equipped to provide ongoing continuity of care, which can lead patients down a poorly defined diagnostic pathway before receiving a confirmed diagnosis based on tissue and a subsequent treatment plan.”
The findings were published online on November 4 in CMAJ).
Neurologic Cancers Prominent
In an interview, Grewal said in an interview that the study reflects her desire as an emergency room physician to understand why so many patients with cancer get the initial reports about their disease from clinicians whom they often have just met for the first time.
Among patients with an ED visit before cancer diagnosis, 51.4% were admitted to hospital from the most recent visit.
Compared with patients with a family physician on whom they could rely for routine care, those who had no outpatient visits (odds ratio [OR], 2.09) or fewer than three outpatient visits (OR, 1.41) in the 6-30 months before cancer diagnosis were more likely to have an ED visit before their cancer diagnosis.
Other factors associated with increased odds of ED use before cancer diagnosis included rurality (OR, 1.15), residence in northern Ontario (northeast region: OR, 1.14 and northwest region: OR, 1.27 vs Toronto region), and living in the most marginalized areas (material resource deprivation: OR, 1.37 and housing stability: OR, 1.09 vs least marginalized area).
The researchers also found that patients with certain cancers were more likely to have sought care in the ED. They compared these cancers with breast cancer, which is often detected through screening.
“Patients with neurologic cancers had extremely high odds of ED use before cancer diagnosis,” the authors wrote. “This is likely because of the emergent nature of presentation, with acute neurologic symptoms such as weakness, confusion, or seizures, which require urgent assessment.” On the other hand, pancreatic, liver, or thoracic cancer can trigger nonspecific symptoms that may be ignored until they reach a crisis level that prompts an ED visit.
The limitations of the study included its inability to identify cancer-related ED visits and its narrow focus on patients in Ontario, according to the researchers. But the use of the ICES databases also allowed researchers access to a broader pool of data than are available in many other cases.
The findings in the new paper echo those of previous research, the authors noted. Research in the United Kingdom found that 24%-31% of cancer diagnoses involved the ED. In addition, a study of people enrolled in the US Medicare program, which serves patients aged 65 years or older, found that 23% were seen in the ED in the 30 days before diagnosis.
‘Unpacking the Data’
The current findings also are consistent with those of an International Cancer Benchmarking Partnership study that was published in 2022 in The Lancet Oncology, said Erika Nicholson, MHS, vice president of cancer systems and innovation at the Canadian Partnership Against Cancer. The latter study analyzed cancer registration and linked hospital admissions data from 14 jurisdictions in Australia, Canada, Denmark, New Zealand, Norway, and the United Kingdom.
“We see similar trends in terms of people visiting EDs and being diagnosed through EDs internationally,” Nicholson said. “We’re working with partners to put in place different strategies to address the challenges” that this phenomenon presents in terms of improving screening and follow-up care.
“Cancer is not one disease, but many diseases,” she said. “They present differently. We’re focused on really unpacking the data and understanding them.”
All this research highlights the need for more services and personnel to address cancer, including people who are trained to help patients cope after getting concerning news through emergency care, she said.
“That means having a system that fully supports you and helps you navigate through that diagnostic process,” Nicholson said. Addressing the added challenges for patients who don’t have secure housing is a special need, she added.
This study was supported by the Canadian Institutes of Health Research (CIHR). Grewal reported receiving grants from CIHR and the Canadian Association of Emergency Physicians. Nicholson reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
FROM CMAJ
Plasma Omega-6 and Omega-3 Fatty Acids Inversely Associated With Cancer
TOPLINE:
Higher plasma levels of omega-6 and omega-3 fatty acids are associated with a lower incidence of cancer. However, omega-3 fatty acids are linked to an increased risk for prostate cancer, specifically.
METHODOLOGY:
- Researchers looked for associations of plasma omega-3 and omega-6 polyunsaturated fatty acids (PUFAs) with the incidence of cancer overall and 19 site-specific cancers in the large population-based prospective UK Biobank cohort.
- They included 253,138 participants aged 37-73 years who were followed for an average of 12.9 years, with 29,838 diagnosed with cancer.
- Plasma levels of omega-3 and omega-6 fatty acids were measured using nuclear magnetic resonance and expressed as percentages of total fatty acids.
- Participants with cancer diagnoses at baseline, those who withdrew from the study, and those with missing data on plasma PUFAs were excluded.
- The study adjusted for multiple covariates, including age, sex, ethnicity, socioeconomic status, lifestyle behaviors, and family history of diseases.
TAKEAWAY:
- Higher plasma levels of omega-6 and omega-3 fatty acids were associated with a 2% and 1% reduction in overall cancer risk per SD increase, respectively (P = .001 and P = .03).
- Omega-6 fatty acids were inversely associated with 14 site-specific cancers, whereas omega-3 fatty acids were inversely associated with five site-specific cancers.
- Prostate cancer was positively associated with omega-3 fatty acids, with a 3% increased risk per SD increase (P = .049).
- A higher omega-6/omega-3 ratio was associated with an increased risk for overall cancer, and three site-specific cancers showed positive associations with the ratio. “Each standard deviation increase, corresponding to a 13.13 increase in the omega ratio, was associated with a 2% increase in the risk of rectum cancer,” for example, the authors wrote.
IN PRACTICE:
“Overall, our findings provide support for possible small net protective roles of omega-3 and omega-6 PUFAs in the development of new cancer incidence. Our study also suggests that the usage of circulating blood biomarkers captures different aspects of dietary intake, reduces measurement errors, and thus enhances statistical power. The differential effects of omega-6% and omega-3% in age and sex subgroups warrant future investigation,” wrote the authors of the study.
SOURCE:
The study was led by Yuchen Zhang of the University of Georgia in Athens, Georgia. It was published online in the International Journal of Cancer.
LIMITATIONS:
The study’s potential for selective bias persists due to the participant sample skewing heavily toward European ancestry and White ethnicity. The number of events was small for some specific cancer sites, which may have limited the statistical power. The study focused on total omega-3 and omega-6 PUFAs, with only two individual fatty acids measured. Future studies are needed to examine the roles of other individual PUFAs and specific genetic variants.
DISCLOSURES:
This study was supported by grants from the National Institute of General Medical Sciences of the National Institutes of Health. No relevant conflicts of interest were disclosed by the authors.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
TOPLINE:
Higher plasma levels of omega-6 and omega-3 fatty acids are associated with a lower incidence of cancer. However, omega-3 fatty acids are linked to an increased risk for prostate cancer, specifically.
METHODOLOGY:
- Researchers looked for associations of plasma omega-3 and omega-6 polyunsaturated fatty acids (PUFAs) with the incidence of cancer overall and 19 site-specific cancers in the large population-based prospective UK Biobank cohort.
- They included 253,138 participants aged 37-73 years who were followed for an average of 12.9 years, with 29,838 diagnosed with cancer.
- Plasma levels of omega-3 and omega-6 fatty acids were measured using nuclear magnetic resonance and expressed as percentages of total fatty acids.
- Participants with cancer diagnoses at baseline, those who withdrew from the study, and those with missing data on plasma PUFAs were excluded.
- The study adjusted for multiple covariates, including age, sex, ethnicity, socioeconomic status, lifestyle behaviors, and family history of diseases.
TAKEAWAY:
- Higher plasma levels of omega-6 and omega-3 fatty acids were associated with a 2% and 1% reduction in overall cancer risk per SD increase, respectively (P = .001 and P = .03).
- Omega-6 fatty acids were inversely associated with 14 site-specific cancers, whereas omega-3 fatty acids were inversely associated with five site-specific cancers.
- Prostate cancer was positively associated with omega-3 fatty acids, with a 3% increased risk per SD increase (P = .049).
- A higher omega-6/omega-3 ratio was associated with an increased risk for overall cancer, and three site-specific cancers showed positive associations with the ratio. “Each standard deviation increase, corresponding to a 13.13 increase in the omega ratio, was associated with a 2% increase in the risk of rectum cancer,” for example, the authors wrote.
IN PRACTICE:
“Overall, our findings provide support for possible small net protective roles of omega-3 and omega-6 PUFAs in the development of new cancer incidence. Our study also suggests that the usage of circulating blood biomarkers captures different aspects of dietary intake, reduces measurement errors, and thus enhances statistical power. The differential effects of omega-6% and omega-3% in age and sex subgroups warrant future investigation,” wrote the authors of the study.
SOURCE:
The study was led by Yuchen Zhang of the University of Georgia in Athens, Georgia. It was published online in the International Journal of Cancer.
LIMITATIONS:
The study’s potential for selective bias persists due to the participant sample skewing heavily toward European ancestry and White ethnicity. The number of events was small for some specific cancer sites, which may have limited the statistical power. The study focused on total omega-3 and omega-6 PUFAs, with only two individual fatty acids measured. Future studies are needed to examine the roles of other individual PUFAs and specific genetic variants.
DISCLOSURES:
This study was supported by grants from the National Institute of General Medical Sciences of the National Institutes of Health. No relevant conflicts of interest were disclosed by the authors.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
TOPLINE:
Higher plasma levels of omega-6 and omega-3 fatty acids are associated with a lower incidence of cancer. However, omega-3 fatty acids are linked to an increased risk for prostate cancer, specifically.
METHODOLOGY:
- Researchers looked for associations of plasma omega-3 and omega-6 polyunsaturated fatty acids (PUFAs) with the incidence of cancer overall and 19 site-specific cancers in the large population-based prospective UK Biobank cohort.
- They included 253,138 participants aged 37-73 years who were followed for an average of 12.9 years, with 29,838 diagnosed with cancer.
- Plasma levels of omega-3 and omega-6 fatty acids were measured using nuclear magnetic resonance and expressed as percentages of total fatty acids.
- Participants with cancer diagnoses at baseline, those who withdrew from the study, and those with missing data on plasma PUFAs were excluded.
- The study adjusted for multiple covariates, including age, sex, ethnicity, socioeconomic status, lifestyle behaviors, and family history of diseases.
TAKEAWAY:
- Higher plasma levels of omega-6 and omega-3 fatty acids were associated with a 2% and 1% reduction in overall cancer risk per SD increase, respectively (P = .001 and P = .03).
- Omega-6 fatty acids were inversely associated with 14 site-specific cancers, whereas omega-3 fatty acids were inversely associated with five site-specific cancers.
- Prostate cancer was positively associated with omega-3 fatty acids, with a 3% increased risk per SD increase (P = .049).
- A higher omega-6/omega-3 ratio was associated with an increased risk for overall cancer, and three site-specific cancers showed positive associations with the ratio. “Each standard deviation increase, corresponding to a 13.13 increase in the omega ratio, was associated with a 2% increase in the risk of rectum cancer,” for example, the authors wrote.
IN PRACTICE:
“Overall, our findings provide support for possible small net protective roles of omega-3 and omega-6 PUFAs in the development of new cancer incidence. Our study also suggests that the usage of circulating blood biomarkers captures different aspects of dietary intake, reduces measurement errors, and thus enhances statistical power. The differential effects of omega-6% and omega-3% in age and sex subgroups warrant future investigation,” wrote the authors of the study.
SOURCE:
The study was led by Yuchen Zhang of the University of Georgia in Athens, Georgia. It was published online in the International Journal of Cancer.
LIMITATIONS:
The study’s potential for selective bias persists due to the participant sample skewing heavily toward European ancestry and White ethnicity. The number of events was small for some specific cancer sites, which may have limited the statistical power. The study focused on total omega-3 and omega-6 PUFAs, with only two individual fatty acids measured. Future studies are needed to examine the roles of other individual PUFAs and specific genetic variants.
DISCLOSURES:
This study was supported by grants from the National Institute of General Medical Sciences of the National Institutes of Health. No relevant conflicts of interest were disclosed by the authors.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Prostate Cancer Treatment Associated With More Complications
TOPLINE:
Radiotherapy increases the risk for bladder cancer and radiation-specific complications, according to the new cohort study.
METHODOLOGY:
- Researchers conducted a cohort study to try to characterize long-term treatment-related adverse effects and complications in patients treated for prostate cancer, compared with a general population of older males.
- They used data from the Prostate Cancer Prevention Trial and the Selenium and Vitamin E Cancer Prevention Trial, linked with Medicare claims. A total of 29,196 participants were included in the study’s control group. Of 3946 patients diagnosed with prostate cancer, 655 were treated with prostatectomy, and 1056 were treated with radiotherapy.
- Participants were followed for a median of 10.2 years, with specific follow-up durations being 10.5 years and 8.5 years for the prostatectomy and radiotherapy groups, respectively.
- The study analyzed ten potential treatment-related complications using Medicare claims data, including urinary incontinence, erectile dysfunction, and secondary cancers.
- Multivariable Cox regression was used to adjust for age, race, and year of time-at-risk initiation, with stratification by study and intervention arm.
TAKEAWAY:
- At 12 years, there was a 7.23 increase in hazard risk for urinary or sexual complications for patients who had prostatectomy, compared with controls (P < .001).
- Radiotherapy-treated patients had a nearly three times greater hazard risk for bladder cancer and a 100-fold increased hazard risk for radiation-specific complications, such as radiation cystitis and radiation proctitis (P < .001).
- The incidence of any treatment-related complication per 1000 person-years was 124.26 for prostatectomy, 62.15 for radiotherapy, and 23.61 for untreated participants.
- The authors stated that these findings highlight the importance of patient counseling before prostate cancer screening and treatment.
IN PRACTICE:
“We found that, after accounting for baseline population rates, most patients with PCA undergoing treatment experience complications associated with worse quality of life and/or new health risks. The magnitude of these risks, compared with the relatively small benefit found by randomized clinical trials of PCA screening and treatment, should be explicitly reflected in national cancer screening and treatment guidelines and be integral to shared decision-making with patients before initiation of PSA screening, biopsy, or PCA treatment,” wrote the authors of the study.
SOURCE:
The study was led by Joseph M. Unger, PhD, SWOG Statistics and Data Management Center, Fred Hutchinson Cancer Center in Seattle, Washington. It was published online on November 7, 2024, in JAMA Oncology.
LIMITATIONS:
The study did not account for multiple comparisons, which may affect the statistical significance of some findings. Claims data are subject to misclassification and may underreport complications that are not reported to a physician. The study did not differentiate among strategies of prostatectomy or radiotherapy, which may result in different patterns of complications. The cohort comprised men enrolled in large, randomized prevention trials, which may limit the generalizability of the incidence estimates. Confounding by unknown factors cannot be ruled out, affecting the attribution of risks to prostate cancer treatment alone.
DISCLOSURES:
Unger disclosed consulting fees from AstraZeneca and Loxo/Lilly outside the submitted work. One coauthor reported grants from the US National Cancer Institute during the conduct of the study. Another coauthor reported employment with Flatiron Health at the time of manuscript submission and review. Additional disclosures are noted in the original article.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
TOPLINE:
Radiotherapy increases the risk for bladder cancer and radiation-specific complications, according to the new cohort study.
METHODOLOGY:
- Researchers conducted a cohort study to try to characterize long-term treatment-related adverse effects and complications in patients treated for prostate cancer, compared with a general population of older males.
- They used data from the Prostate Cancer Prevention Trial and the Selenium and Vitamin E Cancer Prevention Trial, linked with Medicare claims. A total of 29,196 participants were included in the study’s control group. Of 3946 patients diagnosed with prostate cancer, 655 were treated with prostatectomy, and 1056 were treated with radiotherapy.
- Participants were followed for a median of 10.2 years, with specific follow-up durations being 10.5 years and 8.5 years for the prostatectomy and radiotherapy groups, respectively.
- The study analyzed ten potential treatment-related complications using Medicare claims data, including urinary incontinence, erectile dysfunction, and secondary cancers.
- Multivariable Cox regression was used to adjust for age, race, and year of time-at-risk initiation, with stratification by study and intervention arm.
TAKEAWAY:
- At 12 years, there was a 7.23 increase in hazard risk for urinary or sexual complications for patients who had prostatectomy, compared with controls (P < .001).
- Radiotherapy-treated patients had a nearly three times greater hazard risk for bladder cancer and a 100-fold increased hazard risk for radiation-specific complications, such as radiation cystitis and radiation proctitis (P < .001).
- The incidence of any treatment-related complication per 1000 person-years was 124.26 for prostatectomy, 62.15 for radiotherapy, and 23.61 for untreated participants.
- The authors stated that these findings highlight the importance of patient counseling before prostate cancer screening and treatment.
IN PRACTICE:
“We found that, after accounting for baseline population rates, most patients with PCA undergoing treatment experience complications associated with worse quality of life and/or new health risks. The magnitude of these risks, compared with the relatively small benefit found by randomized clinical trials of PCA screening and treatment, should be explicitly reflected in national cancer screening and treatment guidelines and be integral to shared decision-making with patients before initiation of PSA screening, biopsy, or PCA treatment,” wrote the authors of the study.
SOURCE:
The study was led by Joseph M. Unger, PhD, SWOG Statistics and Data Management Center, Fred Hutchinson Cancer Center in Seattle, Washington. It was published online on November 7, 2024, in JAMA Oncology.
LIMITATIONS:
The study did not account for multiple comparisons, which may affect the statistical significance of some findings. Claims data are subject to misclassification and may underreport complications that are not reported to a physician. The study did not differentiate among strategies of prostatectomy or radiotherapy, which may result in different patterns of complications. The cohort comprised men enrolled in large, randomized prevention trials, which may limit the generalizability of the incidence estimates. Confounding by unknown factors cannot be ruled out, affecting the attribution of risks to prostate cancer treatment alone.
DISCLOSURES:
Unger disclosed consulting fees from AstraZeneca and Loxo/Lilly outside the submitted work. One coauthor reported grants from the US National Cancer Institute during the conduct of the study. Another coauthor reported employment with Flatiron Health at the time of manuscript submission and review. Additional disclosures are noted in the original article.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
TOPLINE:
Radiotherapy increases the risk for bladder cancer and radiation-specific complications, according to the new cohort study.
METHODOLOGY:
- Researchers conducted a cohort study to try to characterize long-term treatment-related adverse effects and complications in patients treated for prostate cancer, compared with a general population of older males.
- They used data from the Prostate Cancer Prevention Trial and the Selenium and Vitamin E Cancer Prevention Trial, linked with Medicare claims. A total of 29,196 participants were included in the study’s control group. Of 3946 patients diagnosed with prostate cancer, 655 were treated with prostatectomy, and 1056 were treated with radiotherapy.
- Participants were followed for a median of 10.2 years, with specific follow-up durations being 10.5 years and 8.5 years for the prostatectomy and radiotherapy groups, respectively.
- The study analyzed ten potential treatment-related complications using Medicare claims data, including urinary incontinence, erectile dysfunction, and secondary cancers.
- Multivariable Cox regression was used to adjust for age, race, and year of time-at-risk initiation, with stratification by study and intervention arm.
TAKEAWAY:
- At 12 years, there was a 7.23 increase in hazard risk for urinary or sexual complications for patients who had prostatectomy, compared with controls (P < .001).
- Radiotherapy-treated patients had a nearly three times greater hazard risk for bladder cancer and a 100-fold increased hazard risk for radiation-specific complications, such as radiation cystitis and radiation proctitis (P < .001).
- The incidence of any treatment-related complication per 1000 person-years was 124.26 for prostatectomy, 62.15 for radiotherapy, and 23.61 for untreated participants.
- The authors stated that these findings highlight the importance of patient counseling before prostate cancer screening and treatment.
IN PRACTICE:
“We found that, after accounting for baseline population rates, most patients with PCA undergoing treatment experience complications associated with worse quality of life and/or new health risks. The magnitude of these risks, compared with the relatively small benefit found by randomized clinical trials of PCA screening and treatment, should be explicitly reflected in national cancer screening and treatment guidelines and be integral to shared decision-making with patients before initiation of PSA screening, biopsy, or PCA treatment,” wrote the authors of the study.
SOURCE:
The study was led by Joseph M. Unger, PhD, SWOG Statistics and Data Management Center, Fred Hutchinson Cancer Center in Seattle, Washington. It was published online on November 7, 2024, in JAMA Oncology.
LIMITATIONS:
The study did not account for multiple comparisons, which may affect the statistical significance of some findings. Claims data are subject to misclassification and may underreport complications that are not reported to a physician. The study did not differentiate among strategies of prostatectomy or radiotherapy, which may result in different patterns of complications. The cohort comprised men enrolled in large, randomized prevention trials, which may limit the generalizability of the incidence estimates. Confounding by unknown factors cannot be ruled out, affecting the attribution of risks to prostate cancer treatment alone.
DISCLOSURES:
Unger disclosed consulting fees from AstraZeneca and Loxo/Lilly outside the submitted work. One coauthor reported grants from the US National Cancer Institute during the conduct of the study. Another coauthor reported employment with Flatiron Health at the time of manuscript submission and review. Additional disclosures are noted in the original article.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Outpatient CAR T: Safe, Effective, Accessible
In one recent study, an industry-funded phase 2 trial, researchers found similar outcomes from outpatient and inpatient CAR T-cell therapy for relapsed/refractory large B-cell lymphoma with lisocabtagene maraleucel (Breyanzi).
Another recent study reported that outpatient treatment of B cell non-Hodgkin lymphoma with tisagenlecleucel (Kymriah) had similar efficacy to inpatient treatment. Meanwhile, a 2023 review of CAR T-cell therapy in various settings found similar outcomes in outpatient and inpatient treatment.
“The future of CAR T-cell therapy lies in balancing safety with accessibility,” said Rayne Rouce, MD, a pediatric oncologist at Texas Children’s Cancer Center in Houston, Texas, in an interview. “Expanding CAR T-cell therapy beyond large medical centers is a critical next step.”
Great Outcomes, Low Access
Since 2017, the FDA has approved six CAR T-cell therapies, which target cancer by harnessing the power of a patient’s own T cells. As an Oregon Health & Sciences University/Knight Cancer Center website explains, T cells are removed from the patient’s body, “genetically modified to make the chimeric antigen receptor, or CAR, [which] protein binds to specific proteins on the surface of cancer cells.”
Modified cells are grown and then infused back into the body, where they “multiply and may be able to destroy all the cancer cells.”
As Rouce puts it, “CAR T-cells have revolutionized the treatment of relapsed or refractory blood cancers.” One or more of the therapies have been approved to treat types of lymphoblastic leukemia, B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, and multiple myeloma.
A 2023 review of clinical trial data reported complete response rates of 40%-54% in aggressive B-cell lymphoma, 67% in mantle cell lymphoma, and 69%-74% in indolent B cell lymphoma.
“Commercialization of CAR T-cell therapy brought hope that access would expand beyond the major academic medical centers with the highly specialized infrastructure and advanced laboratories required to manufacture and ultimately treat patients,” Rouce said. “However, it quickly became clear that patients who are underinsured or uninsured — or who live outside the network of the well-resourced institutions that house these therapies — are still unable to access these potentially life-saving therapies.”
A 2024 report estimated the cost of CAR T-cell therapy as $700,000-$1 million and said only a small percentage of those who could benefit from the treatment actually get it. For example, an estimated 10,000 patients with diffuse large B-cell lymphoma alone could benefit from CAR T therapy annually, but a survey of 200 US healthcare centers in 2021 found that 1900 procedures were performed overall for all indications.
Distance to Treatment Is a Major Obstacle
Even if patients have insurance plans willing to cover CAR T-cell therapy, they may not be able get care. While more than 150 US centers are certified to administer the therapy, “distance to major medical centers with CAR T capabilities is a major obstacle,” Yuliya Linhares, MD, chief of lymphoma at Miami Cancer Institute in Miami, Florida, said in an interview.
“I have had patients who chose to not proceed with CAR T therapy due to inability to travel the distance to the medical center for pre-CAR T appointments and assessments and a lack of caretakers who are available to stay nearby,” Linhares said.
Indeed, the challenges facing patients in rural and underserved urban areas can be overwhelming, Hoda Badr, PhD, professor of medicine at Baylor College of Medicine in Houston, Texas, said in an interview.
“They must take time off work, arrange accommodations near treatment sites, and manage travel costs, all of which strain limited financial resources. The inability to afford these additional expenses can lead to delays in receiving care or patients forgoing the treatment altogether,” Badr said. She added that “the psychological and social burden of being away from family and community support systems during treatment can intensify the stress of an already difficult situation.”
A statistic tells the story of the urban/community divide. CAR T-cell therapy administration at academic centers after leukapheresis — the separation and collection of white blood cells — is reported to be at around 90%, while it’s only 47% in community-based practices that have to refer patients elsewhere, Linhares noted.
Researchers Explore CAR T-Cell Therapy in the Community
Linhares is lead author of the phase 2 trial that explored administration of lisocabtagene maraleucel in 82 patients with relapsed/refractory large B-cell lymphoma. The findings were published Sept. 30 in Blood Advances.
The OUTREACH trial, funded by Juno/Bristol-Myers Squibb, treated patients in the third line and beyond at community medical centers (outpatient-monitored, 70%; inpatient-monitored, 30%). The trial didn’t require facilities to be certified by the Foundation for the Accreditation of Cellular Therapy (FACT); all had to be non-tertiary cancer centers that weren’t associated with a university. In order to administer therapy on the outpatient basis, the centers had to have phase 1 or hematopoietic stem cell transplant capabilities.
As Linhares explained, 72% of participating centers hadn’t provided CAR T-cell therapy before, and 44% did not have FACT accreditation. “About 32% of patients received CAR T at CAR T naive sites, while 70% of patients received CAR T as outpatients. Investigators had to decide whether patients qualified for the outpatient observation or had to be admitted for the inpatient observation,” she noted.
Community Outcomes Were Comparable to Major Trial
As for the results, grade 3 or higher adverse events occurred at a similar frequency among outpatients and inpatients at 74% and 76%, Linhares said. There were no grade 5 adverse events, and 25% of patients treated as outpatients were never hospitalized.
Response rates were similar to those in the major TRANSCEND trial with the objective response rates rate of 80% and complete response rates of 54%.
“Overall,” Linhares said, “our study demonstrated that with the availability of standard operating procedures, specially trained staff and a multidisciplinary team trained in CAR T toxicity management, inpatient and outpatient CAR T administration is feasible at specialized community medical centers.”
In 2023, another study examined patients with B-cell non-Hodgkin lymphoma who were treated on an outpatient basis with tisagenlecleucel. Researchers reported that outpatient therapy was “feasible and associated with similar efficacy outcomes as inpatient treatment.”
And a 2023 systematic literature review identified 11 studies that reported outpatient vs inpatient outcomes in CAR T-cell therapy and found “comparable response rates (80-82% in outpatient and 72-80% in inpatient).” Costs were cheaper in the outpatient setting.
Research findings like these are good news, Baylor College of Medicine’s Badr said. “Outpatient administration could help to scale the availability of this therapy to a broader range of healthcare settings, including those serving underserved populations. Findings indicate promising safety profiles, which is encouraging for expanding access.”
Not Every Patient Can Tolerate Outpatient Care
Linhares noted that the patients who received outpatient care in the lisocabtagene maraleucel study were in better shape than those in the inpatient group. Those selected for inpatient care had “higher disease risk characteristics, including high grade B cell lymphoma histology, higher disease burden, and having received bridging therapy. This points to the fact that the investigators properly selected patients who were at a higher risk of complications for inpatient observation. Additionally, some patients stayed as inpatient due to social factors, which increases length of stay independently of disease characteristics.”
Specifically, reasons for inpatient monitoring were disease characteristics (48%) including tumor burden and risk of adverse events; psychosocial factors (32%) including lack of caregiver support or transportation; COVID-19 precautions (8%); pre-infusion adverse events (8%) of fever and vasovagal reaction; and principal investigator decision (4%) due to limited hospital experience with CAR T-cell therapy.
Texas Children’s Cancer Center’s Rouce said “certain patients, particularly those with higher risk for complications or those who require intensive monitoring, may not be suited for outpatient CAR T-cell therapy. This may be due to other comorbidities or baseline factors known to predispose to CAR T-related toxicities. However, evidence-based risk mitigation algorithms may still allow closely monitored outpatient treatment, with recognition that hospital admission for incipient side effects may be necessary.”
What’s Next for Access to Therapy?
Rouce noted that her institution, like many others, is offering CAR T-cell therapy on an outpatient basis. “Additionally, continued scientific innovation, such as immediately available, off-the-shelf cell therapies and inducible safety switches, will ultimately improve access,” she said.
Linhares noted a recent advance and highlighted research that’s now in progress. “CAR Ts now have an indication as a second-line therapy in relapsed/refractory large B-cell lymphoma, and there are ongoing clinical trials that will potentially move CAR Ts into the first line,” she said. “Some trials are exploring allogeneic, readily available off-the-shelf CAR T for the treatment of minimal residual disease positive large B-cell lymphoma after completion of first-line therapy.”
These potential advances “are increasing the need for CAR T-capable medical centers,” Linhares noted. “More and more medical centers with expert hematology teams are becoming CAR T-certified, with more patients having access to CAR T.”
Still, she said, “I don’t think access is nearly as good as it should be. Many patients in rural areas are still unable to get this life-saving treatment. “However, “it is very possible that other novel targeted therapies, such as bispecific antibodies, will be used in place of CAR T in areas with poor CAR T access. Bispecific antibody efficacy in various B cell lymphoma histologies are being currently explored.”
Rouce discloses relationships with Novartis and Pfizer. Linhares reports ties with Kyowa Kirin, AbbVie, ADC, BeiGene, Genentech, Gilead, GlaxoSmithKline, Seagen, and TG. Badr has no disclosures.
A version of this article appeared on Medscape.com.
In one recent study, an industry-funded phase 2 trial, researchers found similar outcomes from outpatient and inpatient CAR T-cell therapy for relapsed/refractory large B-cell lymphoma with lisocabtagene maraleucel (Breyanzi).
Another recent study reported that outpatient treatment of B cell non-Hodgkin lymphoma with tisagenlecleucel (Kymriah) had similar efficacy to inpatient treatment. Meanwhile, a 2023 review of CAR T-cell therapy in various settings found similar outcomes in outpatient and inpatient treatment.
“The future of CAR T-cell therapy lies in balancing safety with accessibility,” said Rayne Rouce, MD, a pediatric oncologist at Texas Children’s Cancer Center in Houston, Texas, in an interview. “Expanding CAR T-cell therapy beyond large medical centers is a critical next step.”
Great Outcomes, Low Access
Since 2017, the FDA has approved six CAR T-cell therapies, which target cancer by harnessing the power of a patient’s own T cells. As an Oregon Health & Sciences University/Knight Cancer Center website explains, T cells are removed from the patient’s body, “genetically modified to make the chimeric antigen receptor, or CAR, [which] protein binds to specific proteins on the surface of cancer cells.”
Modified cells are grown and then infused back into the body, where they “multiply and may be able to destroy all the cancer cells.”
As Rouce puts it, “CAR T-cells have revolutionized the treatment of relapsed or refractory blood cancers.” One or more of the therapies have been approved to treat types of lymphoblastic leukemia, B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, and multiple myeloma.
A 2023 review of clinical trial data reported complete response rates of 40%-54% in aggressive B-cell lymphoma, 67% in mantle cell lymphoma, and 69%-74% in indolent B cell lymphoma.
“Commercialization of CAR T-cell therapy brought hope that access would expand beyond the major academic medical centers with the highly specialized infrastructure and advanced laboratories required to manufacture and ultimately treat patients,” Rouce said. “However, it quickly became clear that patients who are underinsured or uninsured — or who live outside the network of the well-resourced institutions that house these therapies — are still unable to access these potentially life-saving therapies.”
A 2024 report estimated the cost of CAR T-cell therapy as $700,000-$1 million and said only a small percentage of those who could benefit from the treatment actually get it. For example, an estimated 10,000 patients with diffuse large B-cell lymphoma alone could benefit from CAR T therapy annually, but a survey of 200 US healthcare centers in 2021 found that 1900 procedures were performed overall for all indications.
Distance to Treatment Is a Major Obstacle
Even if patients have insurance plans willing to cover CAR T-cell therapy, they may not be able get care. While more than 150 US centers are certified to administer the therapy, “distance to major medical centers with CAR T capabilities is a major obstacle,” Yuliya Linhares, MD, chief of lymphoma at Miami Cancer Institute in Miami, Florida, said in an interview.
“I have had patients who chose to not proceed with CAR T therapy due to inability to travel the distance to the medical center for pre-CAR T appointments and assessments and a lack of caretakers who are available to stay nearby,” Linhares said.
Indeed, the challenges facing patients in rural and underserved urban areas can be overwhelming, Hoda Badr, PhD, professor of medicine at Baylor College of Medicine in Houston, Texas, said in an interview.
“They must take time off work, arrange accommodations near treatment sites, and manage travel costs, all of which strain limited financial resources. The inability to afford these additional expenses can lead to delays in receiving care or patients forgoing the treatment altogether,” Badr said. She added that “the psychological and social burden of being away from family and community support systems during treatment can intensify the stress of an already difficult situation.”
A statistic tells the story of the urban/community divide. CAR T-cell therapy administration at academic centers after leukapheresis — the separation and collection of white blood cells — is reported to be at around 90%, while it’s only 47% in community-based practices that have to refer patients elsewhere, Linhares noted.
Researchers Explore CAR T-Cell Therapy in the Community
Linhares is lead author of the phase 2 trial that explored administration of lisocabtagene maraleucel in 82 patients with relapsed/refractory large B-cell lymphoma. The findings were published Sept. 30 in Blood Advances.
The OUTREACH trial, funded by Juno/Bristol-Myers Squibb, treated patients in the third line and beyond at community medical centers (outpatient-monitored, 70%; inpatient-monitored, 30%). The trial didn’t require facilities to be certified by the Foundation for the Accreditation of Cellular Therapy (FACT); all had to be non-tertiary cancer centers that weren’t associated with a university. In order to administer therapy on the outpatient basis, the centers had to have phase 1 or hematopoietic stem cell transplant capabilities.
As Linhares explained, 72% of participating centers hadn’t provided CAR T-cell therapy before, and 44% did not have FACT accreditation. “About 32% of patients received CAR T at CAR T naive sites, while 70% of patients received CAR T as outpatients. Investigators had to decide whether patients qualified for the outpatient observation or had to be admitted for the inpatient observation,” she noted.
Community Outcomes Were Comparable to Major Trial
As for the results, grade 3 or higher adverse events occurred at a similar frequency among outpatients and inpatients at 74% and 76%, Linhares said. There were no grade 5 adverse events, and 25% of patients treated as outpatients were never hospitalized.
Response rates were similar to those in the major TRANSCEND trial with the objective response rates rate of 80% and complete response rates of 54%.
“Overall,” Linhares said, “our study demonstrated that with the availability of standard operating procedures, specially trained staff and a multidisciplinary team trained in CAR T toxicity management, inpatient and outpatient CAR T administration is feasible at specialized community medical centers.”
In 2023, another study examined patients with B-cell non-Hodgkin lymphoma who were treated on an outpatient basis with tisagenlecleucel. Researchers reported that outpatient therapy was “feasible and associated with similar efficacy outcomes as inpatient treatment.”
And a 2023 systematic literature review identified 11 studies that reported outpatient vs inpatient outcomes in CAR T-cell therapy and found “comparable response rates (80-82% in outpatient and 72-80% in inpatient).” Costs were cheaper in the outpatient setting.
Research findings like these are good news, Baylor College of Medicine’s Badr said. “Outpatient administration could help to scale the availability of this therapy to a broader range of healthcare settings, including those serving underserved populations. Findings indicate promising safety profiles, which is encouraging for expanding access.”
Not Every Patient Can Tolerate Outpatient Care
Linhares noted that the patients who received outpatient care in the lisocabtagene maraleucel study were in better shape than those in the inpatient group. Those selected for inpatient care had “higher disease risk characteristics, including high grade B cell lymphoma histology, higher disease burden, and having received bridging therapy. This points to the fact that the investigators properly selected patients who were at a higher risk of complications for inpatient observation. Additionally, some patients stayed as inpatient due to social factors, which increases length of stay independently of disease characteristics.”
Specifically, reasons for inpatient monitoring were disease characteristics (48%) including tumor burden and risk of adverse events; psychosocial factors (32%) including lack of caregiver support or transportation; COVID-19 precautions (8%); pre-infusion adverse events (8%) of fever and vasovagal reaction; and principal investigator decision (4%) due to limited hospital experience with CAR T-cell therapy.
Texas Children’s Cancer Center’s Rouce said “certain patients, particularly those with higher risk for complications or those who require intensive monitoring, may not be suited for outpatient CAR T-cell therapy. This may be due to other comorbidities or baseline factors known to predispose to CAR T-related toxicities. However, evidence-based risk mitigation algorithms may still allow closely monitored outpatient treatment, with recognition that hospital admission for incipient side effects may be necessary.”
What’s Next for Access to Therapy?
Rouce noted that her institution, like many others, is offering CAR T-cell therapy on an outpatient basis. “Additionally, continued scientific innovation, such as immediately available, off-the-shelf cell therapies and inducible safety switches, will ultimately improve access,” she said.
Linhares noted a recent advance and highlighted research that’s now in progress. “CAR Ts now have an indication as a second-line therapy in relapsed/refractory large B-cell lymphoma, and there are ongoing clinical trials that will potentially move CAR Ts into the first line,” she said. “Some trials are exploring allogeneic, readily available off-the-shelf CAR T for the treatment of minimal residual disease positive large B-cell lymphoma after completion of first-line therapy.”
These potential advances “are increasing the need for CAR T-capable medical centers,” Linhares noted. “More and more medical centers with expert hematology teams are becoming CAR T-certified, with more patients having access to CAR T.”
Still, she said, “I don’t think access is nearly as good as it should be. Many patients in rural areas are still unable to get this life-saving treatment. “However, “it is very possible that other novel targeted therapies, such as bispecific antibodies, will be used in place of CAR T in areas with poor CAR T access. Bispecific antibody efficacy in various B cell lymphoma histologies are being currently explored.”
Rouce discloses relationships with Novartis and Pfizer. Linhares reports ties with Kyowa Kirin, AbbVie, ADC, BeiGene, Genentech, Gilead, GlaxoSmithKline, Seagen, and TG. Badr has no disclosures.
A version of this article appeared on Medscape.com.
In one recent study, an industry-funded phase 2 trial, researchers found similar outcomes from outpatient and inpatient CAR T-cell therapy for relapsed/refractory large B-cell lymphoma with lisocabtagene maraleucel (Breyanzi).
Another recent study reported that outpatient treatment of B cell non-Hodgkin lymphoma with tisagenlecleucel (Kymriah) had similar efficacy to inpatient treatment. Meanwhile, a 2023 review of CAR T-cell therapy in various settings found similar outcomes in outpatient and inpatient treatment.
“The future of CAR T-cell therapy lies in balancing safety with accessibility,” said Rayne Rouce, MD, a pediatric oncologist at Texas Children’s Cancer Center in Houston, Texas, in an interview. “Expanding CAR T-cell therapy beyond large medical centers is a critical next step.”
Great Outcomes, Low Access
Since 2017, the FDA has approved six CAR T-cell therapies, which target cancer by harnessing the power of a patient’s own T cells. As an Oregon Health & Sciences University/Knight Cancer Center website explains, T cells are removed from the patient’s body, “genetically modified to make the chimeric antigen receptor, or CAR, [which] protein binds to specific proteins on the surface of cancer cells.”
Modified cells are grown and then infused back into the body, where they “multiply and may be able to destroy all the cancer cells.”
As Rouce puts it, “CAR T-cells have revolutionized the treatment of relapsed or refractory blood cancers.” One or more of the therapies have been approved to treat types of lymphoblastic leukemia, B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, and multiple myeloma.
A 2023 review of clinical trial data reported complete response rates of 40%-54% in aggressive B-cell lymphoma, 67% in mantle cell lymphoma, and 69%-74% in indolent B cell lymphoma.
“Commercialization of CAR T-cell therapy brought hope that access would expand beyond the major academic medical centers with the highly specialized infrastructure and advanced laboratories required to manufacture and ultimately treat patients,” Rouce said. “However, it quickly became clear that patients who are underinsured or uninsured — or who live outside the network of the well-resourced institutions that house these therapies — are still unable to access these potentially life-saving therapies.”
A 2024 report estimated the cost of CAR T-cell therapy as $700,000-$1 million and said only a small percentage of those who could benefit from the treatment actually get it. For example, an estimated 10,000 patients with diffuse large B-cell lymphoma alone could benefit from CAR T therapy annually, but a survey of 200 US healthcare centers in 2021 found that 1900 procedures were performed overall for all indications.
Distance to Treatment Is a Major Obstacle
Even if patients have insurance plans willing to cover CAR T-cell therapy, they may not be able get care. While more than 150 US centers are certified to administer the therapy, “distance to major medical centers with CAR T capabilities is a major obstacle,” Yuliya Linhares, MD, chief of lymphoma at Miami Cancer Institute in Miami, Florida, said in an interview.
“I have had patients who chose to not proceed with CAR T therapy due to inability to travel the distance to the medical center for pre-CAR T appointments and assessments and a lack of caretakers who are available to stay nearby,” Linhares said.
Indeed, the challenges facing patients in rural and underserved urban areas can be overwhelming, Hoda Badr, PhD, professor of medicine at Baylor College of Medicine in Houston, Texas, said in an interview.
“They must take time off work, arrange accommodations near treatment sites, and manage travel costs, all of which strain limited financial resources. The inability to afford these additional expenses can lead to delays in receiving care or patients forgoing the treatment altogether,” Badr said. She added that “the psychological and social burden of being away from family and community support systems during treatment can intensify the stress of an already difficult situation.”
A statistic tells the story of the urban/community divide. CAR T-cell therapy administration at academic centers after leukapheresis — the separation and collection of white blood cells — is reported to be at around 90%, while it’s only 47% in community-based practices that have to refer patients elsewhere, Linhares noted.
Researchers Explore CAR T-Cell Therapy in the Community
Linhares is lead author of the phase 2 trial that explored administration of lisocabtagene maraleucel in 82 patients with relapsed/refractory large B-cell lymphoma. The findings were published Sept. 30 in Blood Advances.
The OUTREACH trial, funded by Juno/Bristol-Myers Squibb, treated patients in the third line and beyond at community medical centers (outpatient-monitored, 70%; inpatient-monitored, 30%). The trial didn’t require facilities to be certified by the Foundation for the Accreditation of Cellular Therapy (FACT); all had to be non-tertiary cancer centers that weren’t associated with a university. In order to administer therapy on the outpatient basis, the centers had to have phase 1 or hematopoietic stem cell transplant capabilities.
As Linhares explained, 72% of participating centers hadn’t provided CAR T-cell therapy before, and 44% did not have FACT accreditation. “About 32% of patients received CAR T at CAR T naive sites, while 70% of patients received CAR T as outpatients. Investigators had to decide whether patients qualified for the outpatient observation or had to be admitted for the inpatient observation,” she noted.
Community Outcomes Were Comparable to Major Trial
As for the results, grade 3 or higher adverse events occurred at a similar frequency among outpatients and inpatients at 74% and 76%, Linhares said. There were no grade 5 adverse events, and 25% of patients treated as outpatients were never hospitalized.
Response rates were similar to those in the major TRANSCEND trial with the objective response rates rate of 80% and complete response rates of 54%.
“Overall,” Linhares said, “our study demonstrated that with the availability of standard operating procedures, specially trained staff and a multidisciplinary team trained in CAR T toxicity management, inpatient and outpatient CAR T administration is feasible at specialized community medical centers.”
In 2023, another study examined patients with B-cell non-Hodgkin lymphoma who were treated on an outpatient basis with tisagenlecleucel. Researchers reported that outpatient therapy was “feasible and associated with similar efficacy outcomes as inpatient treatment.”
And a 2023 systematic literature review identified 11 studies that reported outpatient vs inpatient outcomes in CAR T-cell therapy and found “comparable response rates (80-82% in outpatient and 72-80% in inpatient).” Costs were cheaper in the outpatient setting.
Research findings like these are good news, Baylor College of Medicine’s Badr said. “Outpatient administration could help to scale the availability of this therapy to a broader range of healthcare settings, including those serving underserved populations. Findings indicate promising safety profiles, which is encouraging for expanding access.”
Not Every Patient Can Tolerate Outpatient Care
Linhares noted that the patients who received outpatient care in the lisocabtagene maraleucel study were in better shape than those in the inpatient group. Those selected for inpatient care had “higher disease risk characteristics, including high grade B cell lymphoma histology, higher disease burden, and having received bridging therapy. This points to the fact that the investigators properly selected patients who were at a higher risk of complications for inpatient observation. Additionally, some patients stayed as inpatient due to social factors, which increases length of stay independently of disease characteristics.”
Specifically, reasons for inpatient monitoring were disease characteristics (48%) including tumor burden and risk of adverse events; psychosocial factors (32%) including lack of caregiver support or transportation; COVID-19 precautions (8%); pre-infusion adverse events (8%) of fever and vasovagal reaction; and principal investigator decision (4%) due to limited hospital experience with CAR T-cell therapy.
Texas Children’s Cancer Center’s Rouce said “certain patients, particularly those with higher risk for complications or those who require intensive monitoring, may not be suited for outpatient CAR T-cell therapy. This may be due to other comorbidities or baseline factors known to predispose to CAR T-related toxicities. However, evidence-based risk mitigation algorithms may still allow closely monitored outpatient treatment, with recognition that hospital admission for incipient side effects may be necessary.”
What’s Next for Access to Therapy?
Rouce noted that her institution, like many others, is offering CAR T-cell therapy on an outpatient basis. “Additionally, continued scientific innovation, such as immediately available, off-the-shelf cell therapies and inducible safety switches, will ultimately improve access,” she said.
Linhares noted a recent advance and highlighted research that’s now in progress. “CAR Ts now have an indication as a second-line therapy in relapsed/refractory large B-cell lymphoma, and there are ongoing clinical trials that will potentially move CAR Ts into the first line,” she said. “Some trials are exploring allogeneic, readily available off-the-shelf CAR T for the treatment of minimal residual disease positive large B-cell lymphoma after completion of first-line therapy.”
These potential advances “are increasing the need for CAR T-capable medical centers,” Linhares noted. “More and more medical centers with expert hematology teams are becoming CAR T-certified, with more patients having access to CAR T.”
Still, she said, “I don’t think access is nearly as good as it should be. Many patients in rural areas are still unable to get this life-saving treatment. “However, “it is very possible that other novel targeted therapies, such as bispecific antibodies, will be used in place of CAR T in areas with poor CAR T access. Bispecific antibody efficacy in various B cell lymphoma histologies are being currently explored.”
Rouce discloses relationships with Novartis and Pfizer. Linhares reports ties with Kyowa Kirin, AbbVie, ADC, BeiGene, Genentech, Gilead, GlaxoSmithKline, Seagen, and TG. Badr has no disclosures.
A version of this article appeared on Medscape.com.
ATA: Updates on Risk, Diagnosis, and Treatment of Thyroid Cancer
The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications.
“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.
The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use.
“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said.
“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
Malignancy in Bethesda III and IV Thyroid Nodules
At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.
Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”
Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.
The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.
She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
RET-Mutated Medullary Thyroid Cancer
For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.
This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.
Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said.
“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
TRK-Fusion Differentiated Thyroid Cancer
Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.
“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said.
“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”
The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.
Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.
Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.
A version of this article appeared on Medscape.com.
The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications.
“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.
The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use.
“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said.
“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
Malignancy in Bethesda III and IV Thyroid Nodules
At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.
Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”
Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.
The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.
She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
RET-Mutated Medullary Thyroid Cancer
For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.
This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.
Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said.
“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
TRK-Fusion Differentiated Thyroid Cancer
Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.
“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said.
“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”
The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.
Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.
Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.
A version of this article appeared on Medscape.com.
The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications.
“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.
The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use.
“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said.
“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
Malignancy in Bethesda III and IV Thyroid Nodules
At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.
Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”
Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.
The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.
She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
RET-Mutated Medullary Thyroid Cancer
For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.
This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.
Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said.
“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
TRK-Fusion Differentiated Thyroid Cancer
Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.
“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said.
“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”
The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.
Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.
Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.
A version of this article appeared on Medscape.com.
FROM ATA 2024