User login
ASCO reports progress, challenges in cancer care
The US cancer care delivery system is undergoing changes to better meet the needs of cancer patients, but persistent hurdles threaten to slow progress, according to the American Society of Clinical Oncology (ASCO).
ASCO’s “The State of Cancer Care in America, 2017” report describes areas of progress, including new approaches for cancer diagnosis and treatment, improved data sharing to drive innovation, and an increased focus on value-based healthcare.
However, the report also suggests that access and affordability challenges, along with increased practice burdens, continue to pose barriers to high-value, high-quality cancer care.
The report was published in the Journal of Oncology Practice.
Challenges
The report notes that the US population is growing rapidly, changing demographically, and living longer. And all of these factors contribute to a record number of cancer cases/survivors.
It has been estimated that the number of cancer survivors in the US will grow from 15.5 million to 20.3 million by 2026.
Unfortunately, the report says, cancer care is unaffordable for many patients, even those with health insurance.
And significant health disparities persist that are independent of insurance status. Socioeconomic status, geography, and race/ethnicity all impact patient health outcomes.
The report also suggests that oncology practices are facing increased administrative burdens that divert time and resources from their patients.
Progress
Despite the aforementioned challenges, the report paints an optimistic vision about the future of cancer care and highlights activity in the past year aimed at improving care.
For instance, the Food and Drug Administration approved 5 new anticancer therapies, expanded the use of 13, and approved several diagnostic tests in 2016.
In addition, overall cancer incidence and mortality rates were lower in 2016 than in previous decades.
“Since 1991, we’ve been able to save 2.1 million lives because of significant advances in prevention, diagnosis, and treatment—something unimaginable even a decade ago,” said ASCO President Daniel F. Hayes, MD.
“But there’s still more work to be done to ensure that every patient with cancer, no matter who they are or where they live, has access to high-quality, high-value cancer care.”
The report includes a list of recommendations that, ASCO believes, could help bring the US closer to achieving that goal. 
The US cancer care delivery system is undergoing changes to better meet the needs of cancer patients, but persistent hurdles threaten to slow progress, according to the American Society of Clinical Oncology (ASCO).
ASCO’s “The State of Cancer Care in America, 2017” report describes areas of progress, including new approaches for cancer diagnosis and treatment, improved data sharing to drive innovation, and an increased focus on value-based healthcare.
However, the report also suggests that access and affordability challenges, along with increased practice burdens, continue to pose barriers to high-value, high-quality cancer care.
The report was published in the Journal of Oncology Practice.
Challenges
The report notes that the US population is growing rapidly, changing demographically, and living longer. And all of these factors contribute to a record number of cancer cases/survivors.
It has been estimated that the number of cancer survivors in the US will grow from 15.5 million to 20.3 million by 2026.
Unfortunately, the report says, cancer care is unaffordable for many patients, even those with health insurance.
And significant health disparities persist that are independent of insurance status. Socioeconomic status, geography, and race/ethnicity all impact patient health outcomes.
The report also suggests that oncology practices are facing increased administrative burdens that divert time and resources from their patients.
Progress
Despite the aforementioned challenges, the report paints an optimistic vision about the future of cancer care and highlights activity in the past year aimed at improving care.
For instance, the Food and Drug Administration approved 5 new anticancer therapies, expanded the use of 13, and approved several diagnostic tests in 2016.
In addition, overall cancer incidence and mortality rates were lower in 2016 than in previous decades.
“Since 1991, we’ve been able to save 2.1 million lives because of significant advances in prevention, diagnosis, and treatment—something unimaginable even a decade ago,” said ASCO President Daniel F. Hayes, MD.
“But there’s still more work to be done to ensure that every patient with cancer, no matter who they are or where they live, has access to high-quality, high-value cancer care.”
The report includes a list of recommendations that, ASCO believes, could help bring the US closer to achieving that goal.
The US cancer care delivery system is undergoing changes to better meet the needs of cancer patients, but persistent hurdles threaten to slow progress, according to the American Society of Clinical Oncology (ASCO).
ASCO’s “The State of Cancer Care in America, 2017” report describes areas of progress, including new approaches for cancer diagnosis and treatment, improved data sharing to drive innovation, and an increased focus on value-based healthcare.
However, the report also suggests that access and affordability challenges, along with increased practice burdens, continue to pose barriers to high-value, high-quality cancer care.
The report was published in the Journal of Oncology Practice.
Challenges
The report notes that the US population is growing rapidly, changing demographically, and living longer. And all of these factors contribute to a record number of cancer cases/survivors.
It has been estimated that the number of cancer survivors in the US will grow from 15.5 million to 20.3 million by 2026.
Unfortunately, the report says, cancer care is unaffordable for many patients, even those with health insurance.
And significant health disparities persist that are independent of insurance status. Socioeconomic status, geography, and race/ethnicity all impact patient health outcomes.
The report also suggests that oncology practices are facing increased administrative burdens that divert time and resources from their patients.
Progress
Despite the aforementioned challenges, the report paints an optimistic vision about the future of cancer care and highlights activity in the past year aimed at improving care.
For instance, the Food and Drug Administration approved 5 new anticancer therapies, expanded the use of 13, and approved several diagnostic tests in 2016.
In addition, overall cancer incidence and mortality rates were lower in 2016 than in previous decades.
“Since 1991, we’ve been able to save 2.1 million lives because of significant advances in prevention, diagnosis, and treatment—something unimaginable even a decade ago,” said ASCO President Daniel F. Hayes, MD.
“But there’s still more work to be done to ensure that every patient with cancer, no matter who they are or where they live, has access to high-quality, high-value cancer care.”
The report includes a list of recommendations that, ASCO believes, could help bring the US closer to achieving that goal.
Unavoidable, random DNA replication errors are the most common cancer drivers
Up to two-thirds of the mutations that drive human cancers may be due to DNA replication errors in normally dividing stem cells, not by inherited or environmentally induced mutations, according to a mathematical modeling study.
The proportion of replication error-driven mutations varied widely among 17 cancers analyzed, but the overall attributable risk of these errors was remarkably consistent among 69 countries included in the study, said Cristian Tomasetti, PhD, a coauthor of the paper and a biostatistician at Johns Hopkins University, Baltimore.
The findings should be a game-changer in the cancer field, Dr. Tomasetti said during a press briefing sponsored by the American Association for the Advancement of Science. Research dogma has long held that most cancers are related to lifestyle and environmental exposure, with a few primarily due to genetic factors.
“We have now determined that there is a third factor, and that it causes most of the mutations that drive cancer,” Dr. Tomasetti said. “We cannot ignore it and pretend it doesn’t exist. This is a complete paradigm shift in how we think of cancer and what causes it.”
The finding that 66% of cancer-driving mutations are based on unavoidable replication errors doesn’t challenge well-established epidemiology, said Dr. Tomasetti and his coauthor, Bert Vogelstein, MD. Rather, it fits perfectly with several key understandings of cancer: that about 40% of cases are preventable, that rapidly dividing tissues are more prone to develop cancers, and that cancer incidence rises exponentially as humans age.
“If we have as our starting point the assumption that 42% of cancers are preventable, we are completely consistent with that,” in finding that about 60% of cancers are unavoidable, Dr. Tomasetti said. “Those two numbers go perfectly together.”
The study also found that replication-error mutations (R) were most likely to drive cancers in tissues with rapid turnover, such as colorectal tissue. This makes intuitive sense, given that basal mutation rates hover at about three errors per cell replication cycle regardless of tissue type.
“The basal mutation rate in all cells is pretty even,” said Dr. Vogelstein, the Clayton Professor of Oncology and Pathology at John Hopkins University, Baltimore. “The difference is the number of stem cells. The more cells, the more divisions, and the more mistakes.”
R-mutations also contribute to age-related cancer incidence. As a person ages, more cell divisions accumulate, thus increasing the risk of a cancer-driving R-error. But these mutations also occur in children, who have rapid cell division in all their tissues. In fact, the colleagues suspect that R-errors are the main drivers of almost all pediatric cancers.
The new study bolsters the duo’s controversial 2015 work.
The theory sparked controversy among scholars and researchers. They challenged it on a number of technical fronts, from stem cell counts and division rates to charges that it didn’t adequately assess the interaction between R-mutations and environmental risks.
Some commentators, perceiving nihilism in the paper, expressed concern that clinicians and patients would get the idea that cancer prevention strategies were useless, since most cancers were simply a case of “bad luck.”
A pervading theme of these counter arguments was one familiar to any researcher: Correlation does not equal causation. The new study was an attempt to expand upon and strengthen the original findings, Dr. Tomasetti said.
“There are well-known environmental risk variations across the world, and there was a question of how our findings might change if we did this analysis in a different country. This paper is also the very first time that someone has ever looked at the proportions of mutations in each cancer type and assigned them to these factors.”
The new study employed a similar mathematical model, but comprised data from 423 cancer registries in 69 countries. The researchers examined the relationship between the lifetime risk of 17 cancers (including breast and prostate, which were not included in the 2015 study) and lifetime stem cell divisions for each tissue. The median correlation coefficient was 0.80; 89% of the countries examined had a correlation of greater than 0.70. This was “remarkably similar” to the correlation determined in the 2015 U.S.-only study.
The team’s next step was to determine what fraction of cancer-driving mutations arose from R-errors, from environmental factors (E), and from hereditary factors (H). They examined these proportions in 32 different cancers in which environmental, lifestyle, and genetic factors have been thoroughly studied. Overall, 29% of the driver mutations were due to environment, 5% to heredity, and 66% to R-errors.
The proportions of these drivers did vary widely between the cancer types, the team noted. For example, lung and esophageal cancers and melanoma were primarily driven by environmental factors (more than 60% each). However, they wrote, “even in lung adenocarcinomas, R contributes a third of the total mutations, with tobacco smoke [including secondhand smoke], diet, radiation, and occupational exposures contributing the remainder. In cancers that are less strongly associated with environmental factors, such as those of the pancreas, brain, bone, or prostate, the majority of the mutations are attributable to R.”
During the press briefing, Dr. Tomasetti and Dr. Vogelstein stressed that most of the inevitable R-errors don’t precipitate cancer – and that even if they do increase risk, that risk may not ever trip the disease process.
“Most of the time these replicative mutations do no harm,” Dr Vogelstein said. “They occur in junk DNA genes, or in areas that are unimportant with respect to cancer. That’s the good luck. Occasionally, they occur in a cancer driver gene, and that is bad luck.”
But even a dose of bad luck isn’t enough to cause cancer. Most cancers require multiple hits to develop – which makes primary prevention strategies more important than ever, Dr. Tomasetti said.
“In the case of lung cancer, for instance, three or more mutations are needed. We showed that these mutations are caused by a combination of environment and R-errors. In theory, then, all of these cancers are preventable because if we can prevent even one of the environmentally caused mutations, then that patient won’t develop cancer.”
However, he said, some cancers do appear to be entirely driven by E-errors and, thus, appear entirely unavoidable. This is an extremely difficult area for clinicians and patients to navigate, said Dr. Vogelstein, a former pediatrician.
“We hope that understanding this will offer some comfort to the literally millions of patients who develop cancer despite having lead a near-perfect life,” in terms of managing risk factors. “Cancer develops in people who haven’t smoked, who avoided the sun and wore sunscreen, who eat perfectly healthy diets and exercise regularly. This is a particularly important concept for parents of children who have cancer, who think ‘I either transmitted a bad gene or unknowingly exposed my child to an environmental agent that caused their cancer.’ They need to understand that these cancers would have occurred no matter what they did.”
Dr. Tomasetti had no disclosures. Dr. Vogelstein is on the scientific advisory boards of Morphotek, Exelixis GP, and Sysmex Inostics, and is a founder of PapGene and Personal Genome Diagnostics.
msullivan@frontlinemedcom.com
On Twitter @Alz_gal
Up to two-thirds of the mutations that drive human cancers may be due to DNA replication errors in normally dividing stem cells, not by inherited or environmentally induced mutations, according to a mathematical modeling study.
The proportion of replication error-driven mutations varied widely among 17 cancers analyzed, but the overall attributable risk of these errors was remarkably consistent among 69 countries included in the study, said Cristian Tomasetti, PhD, a coauthor of the paper and a biostatistician at Johns Hopkins University, Baltimore.
The findings should be a game-changer in the cancer field, Dr. Tomasetti said during a press briefing sponsored by the American Association for the Advancement of Science. Research dogma has long held that most cancers are related to lifestyle and environmental exposure, with a few primarily due to genetic factors.
“We have now determined that there is a third factor, and that it causes most of the mutations that drive cancer,” Dr. Tomasetti said. “We cannot ignore it and pretend it doesn’t exist. This is a complete paradigm shift in how we think of cancer and what causes it.”
The finding that 66% of cancer-driving mutations are based on unavoidable replication errors doesn’t challenge well-established epidemiology, said Dr. Tomasetti and his coauthor, Bert Vogelstein, MD. Rather, it fits perfectly with several key understandings of cancer: that about 40% of cases are preventable, that rapidly dividing tissues are more prone to develop cancers, and that cancer incidence rises exponentially as humans age.
“If we have as our starting point the assumption that 42% of cancers are preventable, we are completely consistent with that,” in finding that about 60% of cancers are unavoidable, Dr. Tomasetti said. “Those two numbers go perfectly together.”
The study also found that replication-error mutations (R) were most likely to drive cancers in tissues with rapid turnover, such as colorectal tissue. This makes intuitive sense, given that basal mutation rates hover at about three errors per cell replication cycle regardless of tissue type.
“The basal mutation rate in all cells is pretty even,” said Dr. Vogelstein, the Clayton Professor of Oncology and Pathology at John Hopkins University, Baltimore. “The difference is the number of stem cells. The more cells, the more divisions, and the more mistakes.”
R-mutations also contribute to age-related cancer incidence. As a person ages, more cell divisions accumulate, thus increasing the risk of a cancer-driving R-error. But these mutations also occur in children, who have rapid cell division in all their tissues. In fact, the colleagues suspect that R-errors are the main drivers of almost all pediatric cancers.
The new study bolsters the duo’s controversial 2015 work.
The theory sparked controversy among scholars and researchers. They challenged it on a number of technical fronts, from stem cell counts and division rates to charges that it didn’t adequately assess the interaction between R-mutations and environmental risks.
Some commentators, perceiving nihilism in the paper, expressed concern that clinicians and patients would get the idea that cancer prevention strategies were useless, since most cancers were simply a case of “bad luck.”
A pervading theme of these counter arguments was one familiar to any researcher: Correlation does not equal causation. The new study was an attempt to expand upon and strengthen the original findings, Dr. Tomasetti said.
“There are well-known environmental risk variations across the world, and there was a question of how our findings might change if we did this analysis in a different country. This paper is also the very first time that someone has ever looked at the proportions of mutations in each cancer type and assigned them to these factors.”
The new study employed a similar mathematical model, but comprised data from 423 cancer registries in 69 countries. The researchers examined the relationship between the lifetime risk of 17 cancers (including breast and prostate, which were not included in the 2015 study) and lifetime stem cell divisions for each tissue. The median correlation coefficient was 0.80; 89% of the countries examined had a correlation of greater than 0.70. This was “remarkably similar” to the correlation determined in the 2015 U.S.-only study.
The team’s next step was to determine what fraction of cancer-driving mutations arose from R-errors, from environmental factors (E), and from hereditary factors (H). They examined these proportions in 32 different cancers in which environmental, lifestyle, and genetic factors have been thoroughly studied. Overall, 29% of the driver mutations were due to environment, 5% to heredity, and 66% to R-errors.
The proportions of these drivers did vary widely between the cancer types, the team noted. For example, lung and esophageal cancers and melanoma were primarily driven by environmental factors (more than 60% each). However, they wrote, “even in lung adenocarcinomas, R contributes a third of the total mutations, with tobacco smoke [including secondhand smoke], diet, radiation, and occupational exposures contributing the remainder. In cancers that are less strongly associated with environmental factors, such as those of the pancreas, brain, bone, or prostate, the majority of the mutations are attributable to R.”
During the press briefing, Dr. Tomasetti and Dr. Vogelstein stressed that most of the inevitable R-errors don’t precipitate cancer – and that even if they do increase risk, that risk may not ever trip the disease process.
“Most of the time these replicative mutations do no harm,” Dr Vogelstein said. “They occur in junk DNA genes, or in areas that are unimportant with respect to cancer. That’s the good luck. Occasionally, they occur in a cancer driver gene, and that is bad luck.”
But even a dose of bad luck isn’t enough to cause cancer. Most cancers require multiple hits to develop – which makes primary prevention strategies more important than ever, Dr. Tomasetti said.
“In the case of lung cancer, for instance, three or more mutations are needed. We showed that these mutations are caused by a combination of environment and R-errors. In theory, then, all of these cancers are preventable because if we can prevent even one of the environmentally caused mutations, then that patient won’t develop cancer.”
However, he said, some cancers do appear to be entirely driven by E-errors and, thus, appear entirely unavoidable. This is an extremely difficult area for clinicians and patients to navigate, said Dr. Vogelstein, a former pediatrician.
“We hope that understanding this will offer some comfort to the literally millions of patients who develop cancer despite having lead a near-perfect life,” in terms of managing risk factors. “Cancer develops in people who haven’t smoked, who avoided the sun and wore sunscreen, who eat perfectly healthy diets and exercise regularly. This is a particularly important concept for parents of children who have cancer, who think ‘I either transmitted a bad gene or unknowingly exposed my child to an environmental agent that caused their cancer.’ They need to understand that these cancers would have occurred no matter what they did.”
Dr. Tomasetti had no disclosures. Dr. Vogelstein is on the scientific advisory boards of Morphotek, Exelixis GP, and Sysmex Inostics, and is a founder of PapGene and Personal Genome Diagnostics.
msullivan@frontlinemedcom.com
On Twitter @Alz_gal
Up to two-thirds of the mutations that drive human cancers may be due to DNA replication errors in normally dividing stem cells, not by inherited or environmentally induced mutations, according to a mathematical modeling study.
The proportion of replication error-driven mutations varied widely among 17 cancers analyzed, but the overall attributable risk of these errors was remarkably consistent among 69 countries included in the study, said Cristian Tomasetti, PhD, a coauthor of the paper and a biostatistician at Johns Hopkins University, Baltimore.
The findings should be a game-changer in the cancer field, Dr. Tomasetti said during a press briefing sponsored by the American Association for the Advancement of Science. Research dogma has long held that most cancers are related to lifestyle and environmental exposure, with a few primarily due to genetic factors.
“We have now determined that there is a third factor, and that it causes most of the mutations that drive cancer,” Dr. Tomasetti said. “We cannot ignore it and pretend it doesn’t exist. This is a complete paradigm shift in how we think of cancer and what causes it.”
The finding that 66% of cancer-driving mutations are based on unavoidable replication errors doesn’t challenge well-established epidemiology, said Dr. Tomasetti and his coauthor, Bert Vogelstein, MD. Rather, it fits perfectly with several key understandings of cancer: that about 40% of cases are preventable, that rapidly dividing tissues are more prone to develop cancers, and that cancer incidence rises exponentially as humans age.
“If we have as our starting point the assumption that 42% of cancers are preventable, we are completely consistent with that,” in finding that about 60% of cancers are unavoidable, Dr. Tomasetti said. “Those two numbers go perfectly together.”
The study also found that replication-error mutations (R) were most likely to drive cancers in tissues with rapid turnover, such as colorectal tissue. This makes intuitive sense, given that basal mutation rates hover at about three errors per cell replication cycle regardless of tissue type.
“The basal mutation rate in all cells is pretty even,” said Dr. Vogelstein, the Clayton Professor of Oncology and Pathology at John Hopkins University, Baltimore. “The difference is the number of stem cells. The more cells, the more divisions, and the more mistakes.”
R-mutations also contribute to age-related cancer incidence. As a person ages, more cell divisions accumulate, thus increasing the risk of a cancer-driving R-error. But these mutations also occur in children, who have rapid cell division in all their tissues. In fact, the colleagues suspect that R-errors are the main drivers of almost all pediatric cancers.
The new study bolsters the duo’s controversial 2015 work.
The theory sparked controversy among scholars and researchers. They challenged it on a number of technical fronts, from stem cell counts and division rates to charges that it didn’t adequately assess the interaction between R-mutations and environmental risks.
Some commentators, perceiving nihilism in the paper, expressed concern that clinicians and patients would get the idea that cancer prevention strategies were useless, since most cancers were simply a case of “bad luck.”
A pervading theme of these counter arguments was one familiar to any researcher: Correlation does not equal causation. The new study was an attempt to expand upon and strengthen the original findings, Dr. Tomasetti said.
“There are well-known environmental risk variations across the world, and there was a question of how our findings might change if we did this analysis in a different country. This paper is also the very first time that someone has ever looked at the proportions of mutations in each cancer type and assigned them to these factors.”
The new study employed a similar mathematical model, but comprised data from 423 cancer registries in 69 countries. The researchers examined the relationship between the lifetime risk of 17 cancers (including breast and prostate, which were not included in the 2015 study) and lifetime stem cell divisions for each tissue. The median correlation coefficient was 0.80; 89% of the countries examined had a correlation of greater than 0.70. This was “remarkably similar” to the correlation determined in the 2015 U.S.-only study.
The team’s next step was to determine what fraction of cancer-driving mutations arose from R-errors, from environmental factors (E), and from hereditary factors (H). They examined these proportions in 32 different cancers in which environmental, lifestyle, and genetic factors have been thoroughly studied. Overall, 29% of the driver mutations were due to environment, 5% to heredity, and 66% to R-errors.
The proportions of these drivers did vary widely between the cancer types, the team noted. For example, lung and esophageal cancers and melanoma were primarily driven by environmental factors (more than 60% each). However, they wrote, “even in lung adenocarcinomas, R contributes a third of the total mutations, with tobacco smoke [including secondhand smoke], diet, radiation, and occupational exposures contributing the remainder. In cancers that are less strongly associated with environmental factors, such as those of the pancreas, brain, bone, or prostate, the majority of the mutations are attributable to R.”
During the press briefing, Dr. Tomasetti and Dr. Vogelstein stressed that most of the inevitable R-errors don’t precipitate cancer – and that even if they do increase risk, that risk may not ever trip the disease process.
“Most of the time these replicative mutations do no harm,” Dr Vogelstein said. “They occur in junk DNA genes, or in areas that are unimportant with respect to cancer. That’s the good luck. Occasionally, they occur in a cancer driver gene, and that is bad luck.”
But even a dose of bad luck isn’t enough to cause cancer. Most cancers require multiple hits to develop – which makes primary prevention strategies more important than ever, Dr. Tomasetti said.
“In the case of lung cancer, for instance, three or more mutations are needed. We showed that these mutations are caused by a combination of environment and R-errors. In theory, then, all of these cancers are preventable because if we can prevent even one of the environmentally caused mutations, then that patient won’t develop cancer.”
However, he said, some cancers do appear to be entirely driven by E-errors and, thus, appear entirely unavoidable. This is an extremely difficult area for clinicians and patients to navigate, said Dr. Vogelstein, a former pediatrician.
“We hope that understanding this will offer some comfort to the literally millions of patients who develop cancer despite having lead a near-perfect life,” in terms of managing risk factors. “Cancer develops in people who haven’t smoked, who avoided the sun and wore sunscreen, who eat perfectly healthy diets and exercise regularly. This is a particularly important concept for parents of children who have cancer, who think ‘I either transmitted a bad gene or unknowingly exposed my child to an environmental agent that caused their cancer.’ They need to understand that these cancers would have occurred no matter what they did.”
Dr. Tomasetti had no disclosures. Dr. Vogelstein is on the scientific advisory boards of Morphotek, Exelixis GP, and Sysmex Inostics, and is a founder of PapGene and Personal Genome Diagnostics.
msullivan@frontlinemedcom.com
On Twitter @Alz_gal
Key clinical point:
Major finding: Two-thirds (66%) of cancer drivers are replication errors, 29% are environmentally induced, and 5% are hereditary.
Data source: The researchers examined cancer mutation drivers in two cohorts that spanned 69 countries.
Disclosures: Dr. Tomasetti had no disclosures. Dr. Vogelstein is on the scientific advisory boards of Morphotek, Exelixis GP, and Sysmex Inostics, and is a founder of PapGene and Personal Genome Diagnostics.
The power of interaction – Supporting language and play development
Engaging caregivers in the management and treatment of early childhood developmental challenges is a critical component of effective intervention.1 Family-centered care helps to promote positive outcomes with early intervention (across developmental domains), and there’s increasing evidence that parent-training programs can be effective in promoting skill generalization and targeting core impairments in toddlers with autism.2
Furthermore, a 2014 randomized controlled trial revealed that individual Early Social Interaction (ESI) with home coaching using the SCERTS (Social Communication, Emotional Regulation, and Transactional Support) curriculum was associated with improvement of a range of child outcomes, compared with group ESI. The authors commented on the importance of individualized parent coaching in natural environments as a way to improve social components of communication and receptive language for toddlers with autism.3
For many parents and at-home caregivers, however, engaging in home-based and parent-delivered interventions can be overwhelming and anxiety-provoking, as well as complicated by other barriers (competing responsibilities, cultural beliefs, and so on). Additionally, these interventions can themselves be a source of stress for some families.
Case
Jake is a 3-year-old boy with a history of global developmental delays, who presents with particular struggles: relating his expressive communication, ability to engage peers in an age-appropriate manner, and capacity to self-regulate when frustrated. He and his family participated in an comprehensive autism diagnostic assessment. In reviewing the history and presentation, considerable challenges in the two core symptom domains that characterize an autism spectrum disorder were noted. A diagnosis of autism was provided, and treatment recommendations were discussed. “What can I do at home to help Jake learn?” his mother asked, noting that, with one-on-one attention, he does seem to demonstrate increased responsiveness, less use of echolalic language, and improved eye contact.
Discussion
To complement the autism services that Jake would likely qualify for through an Early Education program, in-home interaction and play to ensure skill development was discussed at length with his mother, who readily acknowledged her own care-giving struggles that, in part, are informed by her own mental health troubles.
We openly explored Jake’s mother’s perceived challenges in engaging with her son at home and developed initial recommendations for interaction that didn’t risk overwhelming her. We impressed upon Jake’s mother that, regardless of a child’s developmental profile, toddlers use play to learn and she can be Jake’s “favorite toy.” After all, “play is really the work of childhood,” as Fred Rogers said.
With all children, back-and-forth interactions serve as the foundation for future development. Using scaffolding techniques, parent support is a primary driver of “how children develop cognitive, language, social-emotional, and higher-level thinking skills.”4 In particular, the quality of parental interaction can influence language development, and, when considering children with autism, there are several recommendations for what parents can do to help build social, play, and communication skills.5 The Hanen Program is a great resource for providers and parents to learn more about parent engagement in early learning, the power of building communication through everyday experiences and attention to responsiveness, and the use of a child’s strengths to help make family interactions more meaningful and enjoyable. Additionally, the 2012 book “An Early Start for Your Child with Autism: Using Everyday Activities to Help Kids Connect, Communicate, and Learn” by Sally Rogers, PhD, et al. is an easy-to-read text for parents and caregivers for learning effective and practical strategies for engaging their child with autism.
With Jake and his mother, our team offered the following in-home recommendations:
- Try to keep interaction fun. Be enthusiastic when encouraging Jake’s attempts to communicate.
- Teach Jake song-gesture games. Encourage him to produce routine, predictable gestures to keep the song going (in imitation of mom). Using songs with vowel emphasis is encouraged (for example: Farmer in the Dell with “E I E I OOOOO”).
- Encourage Jake to produce responsive gestures in play and daily routines not involving songs, such as open arms to receive a ball, reaching to mom when about to be tickled, or having his arms up to have his shirt taken off.
- Capitalize on Jake’s natural desires and personal preferences. Activate a wind-up toy, let it deactivate, and then hand it to Jake.
- Initiate a familiar social game with Jake until he expresses pleasure. Then stop the game and wait for him to initiate continuance.
- Adapt the environment so that Jake will need to frequently request objects of assistance to make choices (place favorite toys in clear containers which may be difficult to open so that he must request help).
Clinical pearl
The United States Department of Education recognizes the importance of family engagement in a child’s early years. Their 2015 policy statement notes that “families are their children’s first and most important teachers, advocates, and nurturers. As such, strong family engagement is central – not supplemental – to the success of early childhood systems and programs that promote children’s healthy development, learning, and wellness.”
By recognizing this principle, primary care providers are in a position to talk with parents about how much youth learn through play and regular interaction. This especially holds true for children with autism. Developing in-home strategies to facilitate active engagement, even strategies that may not be a formal component of a home-based intervention program, are instrumental in fostering positive family- and child-based outcomes and wellness.
Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at pdnews@frontlinemedcom.com.
References
1. Annu Rev Clin Psychol. 2010;6:447-68.
2. J Autism Dev Disord. 2010 Sep;40(9):1045-56.
3. Pediatrics. 2014 Dec;134(6):1084-93.
4. JAMA Pediatr. 2016 Feb;170(2):112-3.
5. Child Dev. 2012 Sep-Oct;83(5):1762-74.
Engaging caregivers in the management and treatment of early childhood developmental challenges is a critical component of effective intervention.1 Family-centered care helps to promote positive outcomes with early intervention (across developmental domains), and there’s increasing evidence that parent-training programs can be effective in promoting skill generalization and targeting core impairments in toddlers with autism.2
Furthermore, a 2014 randomized controlled trial revealed that individual Early Social Interaction (ESI) with home coaching using the SCERTS (Social Communication, Emotional Regulation, and Transactional Support) curriculum was associated with improvement of a range of child outcomes, compared with group ESI. The authors commented on the importance of individualized parent coaching in natural environments as a way to improve social components of communication and receptive language for toddlers with autism.3
For many parents and at-home caregivers, however, engaging in home-based and parent-delivered interventions can be overwhelming and anxiety-provoking, as well as complicated by other barriers (competing responsibilities, cultural beliefs, and so on). Additionally, these interventions can themselves be a source of stress for some families.
Case
Jake is a 3-year-old boy with a history of global developmental delays, who presents with particular struggles: relating his expressive communication, ability to engage peers in an age-appropriate manner, and capacity to self-regulate when frustrated. He and his family participated in an comprehensive autism diagnostic assessment. In reviewing the history and presentation, considerable challenges in the two core symptom domains that characterize an autism spectrum disorder were noted. A diagnosis of autism was provided, and treatment recommendations were discussed. “What can I do at home to help Jake learn?” his mother asked, noting that, with one-on-one attention, he does seem to demonstrate increased responsiveness, less use of echolalic language, and improved eye contact.
Discussion
To complement the autism services that Jake would likely qualify for through an Early Education program, in-home interaction and play to ensure skill development was discussed at length with his mother, who readily acknowledged her own care-giving struggles that, in part, are informed by her own mental health troubles.
We openly explored Jake’s mother’s perceived challenges in engaging with her son at home and developed initial recommendations for interaction that didn’t risk overwhelming her. We impressed upon Jake’s mother that, regardless of a child’s developmental profile, toddlers use play to learn and she can be Jake’s “favorite toy.” After all, “play is really the work of childhood,” as Fred Rogers said.
With all children, back-and-forth interactions serve as the foundation for future development. Using scaffolding techniques, parent support is a primary driver of “how children develop cognitive, language, social-emotional, and higher-level thinking skills.”4 In particular, the quality of parental interaction can influence language development, and, when considering children with autism, there are several recommendations for what parents can do to help build social, play, and communication skills.5 The Hanen Program is a great resource for providers and parents to learn more about parent engagement in early learning, the power of building communication through everyday experiences and attention to responsiveness, and the use of a child’s strengths to help make family interactions more meaningful and enjoyable. Additionally, the 2012 book “An Early Start for Your Child with Autism: Using Everyday Activities to Help Kids Connect, Communicate, and Learn” by Sally Rogers, PhD, et al. is an easy-to-read text for parents and caregivers for learning effective and practical strategies for engaging their child with autism.
With Jake and his mother, our team offered the following in-home recommendations:
- Try to keep interaction fun. Be enthusiastic when encouraging Jake’s attempts to communicate.
- Teach Jake song-gesture games. Encourage him to produce routine, predictable gestures to keep the song going (in imitation of mom). Using songs with vowel emphasis is encouraged (for example: Farmer in the Dell with “E I E I OOOOO”).
- Encourage Jake to produce responsive gestures in play and daily routines not involving songs, such as open arms to receive a ball, reaching to mom when about to be tickled, or having his arms up to have his shirt taken off.
- Capitalize on Jake’s natural desires and personal preferences. Activate a wind-up toy, let it deactivate, and then hand it to Jake.
- Initiate a familiar social game with Jake until he expresses pleasure. Then stop the game and wait for him to initiate continuance.
- Adapt the environment so that Jake will need to frequently request objects of assistance to make choices (place favorite toys in clear containers which may be difficult to open so that he must request help).
Clinical pearl
The United States Department of Education recognizes the importance of family engagement in a child’s early years. Their 2015 policy statement notes that “families are their children’s first and most important teachers, advocates, and nurturers. As such, strong family engagement is central – not supplemental – to the success of early childhood systems and programs that promote children’s healthy development, learning, and wellness.”
By recognizing this principle, primary care providers are in a position to talk with parents about how much youth learn through play and regular interaction. This especially holds true for children with autism. Developing in-home strategies to facilitate active engagement, even strategies that may not be a formal component of a home-based intervention program, are instrumental in fostering positive family- and child-based outcomes and wellness.
Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at pdnews@frontlinemedcom.com.
References
1. Annu Rev Clin Psychol. 2010;6:447-68.
2. J Autism Dev Disord. 2010 Sep;40(9):1045-56.
3. Pediatrics. 2014 Dec;134(6):1084-93.
4. JAMA Pediatr. 2016 Feb;170(2):112-3.
5. Child Dev. 2012 Sep-Oct;83(5):1762-74.
Engaging caregivers in the management and treatment of early childhood developmental challenges is a critical component of effective intervention.1 Family-centered care helps to promote positive outcomes with early intervention (across developmental domains), and there’s increasing evidence that parent-training programs can be effective in promoting skill generalization and targeting core impairments in toddlers with autism.2
Furthermore, a 2014 randomized controlled trial revealed that individual Early Social Interaction (ESI) with home coaching using the SCERTS (Social Communication, Emotional Regulation, and Transactional Support) curriculum was associated with improvement of a range of child outcomes, compared with group ESI. The authors commented on the importance of individualized parent coaching in natural environments as a way to improve social components of communication and receptive language for toddlers with autism.3
For many parents and at-home caregivers, however, engaging in home-based and parent-delivered interventions can be overwhelming and anxiety-provoking, as well as complicated by other barriers (competing responsibilities, cultural beliefs, and so on). Additionally, these interventions can themselves be a source of stress for some families.
Case
Jake is a 3-year-old boy with a history of global developmental delays, who presents with particular struggles: relating his expressive communication, ability to engage peers in an age-appropriate manner, and capacity to self-regulate when frustrated. He and his family participated in an comprehensive autism diagnostic assessment. In reviewing the history and presentation, considerable challenges in the two core symptom domains that characterize an autism spectrum disorder were noted. A diagnosis of autism was provided, and treatment recommendations were discussed. “What can I do at home to help Jake learn?” his mother asked, noting that, with one-on-one attention, he does seem to demonstrate increased responsiveness, less use of echolalic language, and improved eye contact.
Discussion
To complement the autism services that Jake would likely qualify for through an Early Education program, in-home interaction and play to ensure skill development was discussed at length with his mother, who readily acknowledged her own care-giving struggles that, in part, are informed by her own mental health troubles.
We openly explored Jake’s mother’s perceived challenges in engaging with her son at home and developed initial recommendations for interaction that didn’t risk overwhelming her. We impressed upon Jake’s mother that, regardless of a child’s developmental profile, toddlers use play to learn and she can be Jake’s “favorite toy.” After all, “play is really the work of childhood,” as Fred Rogers said.
With all children, back-and-forth interactions serve as the foundation for future development. Using scaffolding techniques, parent support is a primary driver of “how children develop cognitive, language, social-emotional, and higher-level thinking skills.”4 In particular, the quality of parental interaction can influence language development, and, when considering children with autism, there are several recommendations for what parents can do to help build social, play, and communication skills.5 The Hanen Program is a great resource for providers and parents to learn more about parent engagement in early learning, the power of building communication through everyday experiences and attention to responsiveness, and the use of a child’s strengths to help make family interactions more meaningful and enjoyable. Additionally, the 2012 book “An Early Start for Your Child with Autism: Using Everyday Activities to Help Kids Connect, Communicate, and Learn” by Sally Rogers, PhD, et al. is an easy-to-read text for parents and caregivers for learning effective and practical strategies for engaging their child with autism.
With Jake and his mother, our team offered the following in-home recommendations:
- Try to keep interaction fun. Be enthusiastic when encouraging Jake’s attempts to communicate.
- Teach Jake song-gesture games. Encourage him to produce routine, predictable gestures to keep the song going (in imitation of mom). Using songs with vowel emphasis is encouraged (for example: Farmer in the Dell with “E I E I OOOOO”).
- Encourage Jake to produce responsive gestures in play and daily routines not involving songs, such as open arms to receive a ball, reaching to mom when about to be tickled, or having his arms up to have his shirt taken off.
- Capitalize on Jake’s natural desires and personal preferences. Activate a wind-up toy, let it deactivate, and then hand it to Jake.
- Initiate a familiar social game with Jake until he expresses pleasure. Then stop the game and wait for him to initiate continuance.
- Adapt the environment so that Jake will need to frequently request objects of assistance to make choices (place favorite toys in clear containers which may be difficult to open so that he must request help).
Clinical pearl
The United States Department of Education recognizes the importance of family engagement in a child’s early years. Their 2015 policy statement notes that “families are their children’s first and most important teachers, advocates, and nurturers. As such, strong family engagement is central – not supplemental – to the success of early childhood systems and programs that promote children’s healthy development, learning, and wellness.”
By recognizing this principle, primary care providers are in a position to talk with parents about how much youth learn through play and regular interaction. This especially holds true for children with autism. Developing in-home strategies to facilitate active engagement, even strategies that may not be a formal component of a home-based intervention program, are instrumental in fostering positive family- and child-based outcomes and wellness.
Dr. Dickerson, a child and adolescent psychiatrist, is assistant professor of psychiatry at the University of Vermont, Burlington, where he is director of the autism diagnostic clinic. Email him at pdnews@frontlinemedcom.com.
References
1. Annu Rev Clin Psychol. 2010;6:447-68.
2. J Autism Dev Disord. 2010 Sep;40(9):1045-56.
3. Pediatrics. 2014 Dec;134(6):1084-93.
4. JAMA Pediatr. 2016 Feb;170(2):112-3.
5. Child Dev. 2012 Sep-Oct;83(5):1762-74.
New self-persuasion app to promote HPV vaccine appears effective
A new tablet application using a self-persuasion method has been successful in convincing parents in underserved communities to have their children vaccinated for human papillomavirus (HPV), according to a study.
Of 45 participating parents, 27 of the 33 (82%) parents whose adolescents were not vaccinated reported that they decided to get their children vaccinated after completing the application. The remaining 12 already had an HPV-vaccinated adolescent. Children of participating parents were aged 11-17 years (Patient Educ Couns. 2016. doi: 10.1016/j.pec.2016.11.014).
Of the 45 parents, 31 (69%) were Hispanic and 29 (64%) held a high school education or less, according to the study.
To test the effectiveness of self-persuasion, researchers developed a tablet application which started with a 5 minute video on HPV and vaccine efficacy, and then asked parents to complete two tasks: answer questions about the HPV vaccine that prompt thought on its benefits and come up with personal reasons for why having their child vaccinated is important.
Parents then participated in a 45-60 minute interview with one of six research assistants, who prompted participants to address four research points: Did they like the application? Which questions generated interest in the HPV vaccine without raising concerns? Were they able to communicate reasons for vaccination? Were they convinced to have their children vaccinated?
After watching the video, 18 (55%) of the 33 parents whose children were not vaccinated changed their minds, and, after participating in the self-persuasion questionnaire, an additional 9 parents decided to vaccinate their children, according to the study. Five parents remained undecided, and one decided against HPV vaccination.
Overall, participants reported that the application questions were helpful in their decision to vaccinate their children, with question ratings ranging from 4.33 to 4.98 on a scale of 1 to 5.
Mr. Baldwin and his colleagues said that, while the initial test showed promise, further research must be done on actual vaccine behavior, as these tests only studied verbally reported decisions by parents.
One of the limitations to the study was that the research was primarily conducted at a research facility. While some was conducted in local clinics, Mr. Baldwin and his colleagues consider that further studies should be conducted in more public areas. All but one of the participants were female, which may make it hard to generalize about the effects of this application with male parents.
The researchers reported no relevant financial disclosures.
ezimmerman@frontlinemedcom.com
On Twitter @EAZTweets
The media has not been kind when it comes to HPV vaccinations. When most parents are approached with the option to have their children vaccinated, they are hesitant because of what they have heard from television, news articles, or online mom chat rooms about the dangerous side effects of the vaccine. When a mother hears about other mothers’ daughters fainting or being subjected to pain, she usually wonders if it is really worth the risk.
The same goes for parents who expect their daughters to practice celibacy until marriage. After all, why expose a child to negative side effects when she won’t be having sex anyway? The problem with this thinking, however, is that, often, it is not based in reality. When I approach families about HPV vaccine, or any other vaccine for that matter, I make sure to present studies that explain the lack of evidence on negative vaccine side effects, as well as information pertaining to how common HPV is.
However, simply handing out a fact sheet is not enough. Parents who are convinced that their children do not need to be vaccinated will not be persuaded through statistics but through personal anecdotes. I like to tell mothers to ask their girlfriends about being vaccinated, to not only see how common HPV is but to break the stigma as well. I explain to parents who do not want their child having sex now that, at some point, they might want their child to grow their family, and, when that time comes, don’t they want their child to be safe? Even if it is just one time, shouldn’t their child be protected? From that point on, the language of conversation has changed, and the channels of communication are more open.
As pediatricians, we are the medical advocates for our patients and their families, not just someone to prescribe medication for them. Establish that role through knowledge and conversation, and persuasion will soon follow.
Francine Pearce, MD, is a pediatrician in Frankfort, Ill. She writes the Pediatric News teen column, Pearce-ings .
The media has not been kind when it comes to HPV vaccinations. When most parents are approached with the option to have their children vaccinated, they are hesitant because of what they have heard from television, news articles, or online mom chat rooms about the dangerous side effects of the vaccine. When a mother hears about other mothers’ daughters fainting or being subjected to pain, she usually wonders if it is really worth the risk.
The same goes for parents who expect their daughters to practice celibacy until marriage. After all, why expose a child to negative side effects when she won’t be having sex anyway? The problem with this thinking, however, is that, often, it is not based in reality. When I approach families about HPV vaccine, or any other vaccine for that matter, I make sure to present studies that explain the lack of evidence on negative vaccine side effects, as well as information pertaining to how common HPV is.
However, simply handing out a fact sheet is not enough. Parents who are convinced that their children do not need to be vaccinated will not be persuaded through statistics but through personal anecdotes. I like to tell mothers to ask their girlfriends about being vaccinated, to not only see how common HPV is but to break the stigma as well. I explain to parents who do not want their child having sex now that, at some point, they might want their child to grow their family, and, when that time comes, don’t they want their child to be safe? Even if it is just one time, shouldn’t their child be protected? From that point on, the language of conversation has changed, and the channels of communication are more open.
As pediatricians, we are the medical advocates for our patients and their families, not just someone to prescribe medication for them. Establish that role through knowledge and conversation, and persuasion will soon follow.
Francine Pearce, MD, is a pediatrician in Frankfort, Ill. She writes the Pediatric News teen column, Pearce-ings .
The media has not been kind when it comes to HPV vaccinations. When most parents are approached with the option to have their children vaccinated, they are hesitant because of what they have heard from television, news articles, or online mom chat rooms about the dangerous side effects of the vaccine. When a mother hears about other mothers’ daughters fainting or being subjected to pain, she usually wonders if it is really worth the risk.
The same goes for parents who expect their daughters to practice celibacy until marriage. After all, why expose a child to negative side effects when she won’t be having sex anyway? The problem with this thinking, however, is that, often, it is not based in reality. When I approach families about HPV vaccine, or any other vaccine for that matter, I make sure to present studies that explain the lack of evidence on negative vaccine side effects, as well as information pertaining to how common HPV is.
However, simply handing out a fact sheet is not enough. Parents who are convinced that their children do not need to be vaccinated will not be persuaded through statistics but through personal anecdotes. I like to tell mothers to ask their girlfriends about being vaccinated, to not only see how common HPV is but to break the stigma as well. I explain to parents who do not want their child having sex now that, at some point, they might want their child to grow their family, and, when that time comes, don’t they want their child to be safe? Even if it is just one time, shouldn’t their child be protected? From that point on, the language of conversation has changed, and the channels of communication are more open.
As pediatricians, we are the medical advocates for our patients and their families, not just someone to prescribe medication for them. Establish that role through knowledge and conversation, and persuasion will soon follow.
Francine Pearce, MD, is a pediatrician in Frankfort, Ill. She writes the Pediatric News teen column, Pearce-ings .
A new tablet application using a self-persuasion method has been successful in convincing parents in underserved communities to have their children vaccinated for human papillomavirus (HPV), according to a study.
Of 45 participating parents, 27 of the 33 (82%) parents whose adolescents were not vaccinated reported that they decided to get their children vaccinated after completing the application. The remaining 12 already had an HPV-vaccinated adolescent. Children of participating parents were aged 11-17 years (Patient Educ Couns. 2016. doi: 10.1016/j.pec.2016.11.014).
Of the 45 parents, 31 (69%) were Hispanic and 29 (64%) held a high school education or less, according to the study.
To test the effectiveness of self-persuasion, researchers developed a tablet application which started with a 5 minute video on HPV and vaccine efficacy, and then asked parents to complete two tasks: answer questions about the HPV vaccine that prompt thought on its benefits and come up with personal reasons for why having their child vaccinated is important.
Parents then participated in a 45-60 minute interview with one of six research assistants, who prompted participants to address four research points: Did they like the application? Which questions generated interest in the HPV vaccine without raising concerns? Were they able to communicate reasons for vaccination? Were they convinced to have their children vaccinated?
After watching the video, 18 (55%) of the 33 parents whose children were not vaccinated changed their minds, and, after participating in the self-persuasion questionnaire, an additional 9 parents decided to vaccinate their children, according to the study. Five parents remained undecided, and one decided against HPV vaccination.
Overall, participants reported that the application questions were helpful in their decision to vaccinate their children, with question ratings ranging from 4.33 to 4.98 on a scale of 1 to 5.
Mr. Baldwin and his colleagues said that, while the initial test showed promise, further research must be done on actual vaccine behavior, as these tests only studied verbally reported decisions by parents.
One of the limitations to the study was that the research was primarily conducted at a research facility. While some was conducted in local clinics, Mr. Baldwin and his colleagues consider that further studies should be conducted in more public areas. All but one of the participants were female, which may make it hard to generalize about the effects of this application with male parents.
The researchers reported no relevant financial disclosures.
ezimmerman@frontlinemedcom.com
On Twitter @EAZTweets
A new tablet application using a self-persuasion method has been successful in convincing parents in underserved communities to have their children vaccinated for human papillomavirus (HPV), according to a study.
Of 45 participating parents, 27 of the 33 (82%) parents whose adolescents were not vaccinated reported that they decided to get their children vaccinated after completing the application. The remaining 12 already had an HPV-vaccinated adolescent. Children of participating parents were aged 11-17 years (Patient Educ Couns. 2016. doi: 10.1016/j.pec.2016.11.014).
Of the 45 parents, 31 (69%) were Hispanic and 29 (64%) held a high school education or less, according to the study.
To test the effectiveness of self-persuasion, researchers developed a tablet application which started with a 5 minute video on HPV and vaccine efficacy, and then asked parents to complete two tasks: answer questions about the HPV vaccine that prompt thought on its benefits and come up with personal reasons for why having their child vaccinated is important.
Parents then participated in a 45-60 minute interview with one of six research assistants, who prompted participants to address four research points: Did they like the application? Which questions generated interest in the HPV vaccine without raising concerns? Were they able to communicate reasons for vaccination? Were they convinced to have their children vaccinated?
After watching the video, 18 (55%) of the 33 parents whose children were not vaccinated changed their minds, and, after participating in the self-persuasion questionnaire, an additional 9 parents decided to vaccinate their children, according to the study. Five parents remained undecided, and one decided against HPV vaccination.
Overall, participants reported that the application questions were helpful in their decision to vaccinate their children, with question ratings ranging from 4.33 to 4.98 on a scale of 1 to 5.
Mr. Baldwin and his colleagues said that, while the initial test showed promise, further research must be done on actual vaccine behavior, as these tests only studied verbally reported decisions by parents.
One of the limitations to the study was that the research was primarily conducted at a research facility. While some was conducted in local clinics, Mr. Baldwin and his colleagues consider that further studies should be conducted in more public areas. All but one of the participants were female, which may make it hard to generalize about the effects of this application with male parents.
The researchers reported no relevant financial disclosures.
ezimmerman@frontlinemedcom.com
On Twitter @EAZTweets
FROM PATIENT EDUCATION & COUNSELING
Key clinical point:
Major finding: Of the 33 parents whose children were not vaccinated, 27 (818%) decided to have their children vaccinated after using the application.
Data source: A study of 45 parents in low-income communities, evaluated through self-reporting questionnaires and in-person interviews conducted by research assistants.
Disclosures: Researchers reported no relevant financial disclosures.
AYAs struggle socially in early years after cancer diagnosis
A new study indicates that adolescent and young adult (AYA) cancer survivors continue to face social difficulties for more than 2 years after their diagnosis.
The research, published in Cancer, suggests these patients may see some improvement in their social lives during the first year after diagnosis.
However, their social functioning tends to remain constant after that, leaving them socially impaired relative to their cancer-free peers.
Previous studies have shown that AYAs with cancer experience greater challenges in social functioning than their cancer-free peers or even compared to older cancer patients.
But few studies have examined this phenomenon by following the same patients over time.
Olga Husson, PhD, of the Radboud University Medical Center in The Netherlands, and her colleagues set out to examine changes in social functioning among AYAs in the early years after a cancer diagnosis.
The researchers asked AYA cancer patients at 5 US medical institutions to complete a survey about social functioning within 4 months of their diagnosis, 12 months later, and 24 months later.
There were 141 patients (ages 14 to 39 at diagnosis) who completed the surveys.
The researchers found that, when compared to population norms, the cancer patients had inferior social functioning at all the time points studied.
Among the cancer patients, the mean social functioning score from the Medical Outcomes Study Short Form 36 Health Survey (version 2) was 52.0 around the time of cancer diagnosis, 73.1 at the 12-month follow-up, and 69.2 at the 24-month follow-up. In comparison, the population norm (for people ages 18 to 44) is 85.1 (P<0.001 for all time points).
The researchers did note that cancer patients experienced significant improvements in social functioning from baseline to the 12-month follow-up, but there was no further improvement after that.
The researchers also examined the different trajectories of social functioning over time. They found that social functioning improved over time for 47% of the cancer patients but worsened for 13%. In addition, 32% of patients had consistently low social functioning, and 9% had consistently high social functioning.
The cancer patients with consistently low social functioning were more likely to be off treatment at the time of follow-up, report more physical symptoms and higher levels of psychological distress (at both baseline and follow-up), and perceive themselves to receive less social support.
“Reducing physical symptoms and psychological distress and enhancing social support by interventions in the period after treatment may potentially help these young survivors to better reintegrate into society,” Dr Husson said.
A new study indicates that adolescent and young adult (AYA) cancer survivors continue to face social difficulties for more than 2 years after their diagnosis.
The research, published in Cancer, suggests these patients may see some improvement in their social lives during the first year after diagnosis.
However, their social functioning tends to remain constant after that, leaving them socially impaired relative to their cancer-free peers.
Previous studies have shown that AYAs with cancer experience greater challenges in social functioning than their cancer-free peers or even compared to older cancer patients.
But few studies have examined this phenomenon by following the same patients over time.
Olga Husson, PhD, of the Radboud University Medical Center in The Netherlands, and her colleagues set out to examine changes in social functioning among AYAs in the early years after a cancer diagnosis.
The researchers asked AYA cancer patients at 5 US medical institutions to complete a survey about social functioning within 4 months of their diagnosis, 12 months later, and 24 months later.
There were 141 patients (ages 14 to 39 at diagnosis) who completed the surveys.
The researchers found that, when compared to population norms, the cancer patients had inferior social functioning at all the time points studied.
Among the cancer patients, the mean social functioning score from the Medical Outcomes Study Short Form 36 Health Survey (version 2) was 52.0 around the time of cancer diagnosis, 73.1 at the 12-month follow-up, and 69.2 at the 24-month follow-up. In comparison, the population norm (for people ages 18 to 44) is 85.1 (P<0.001 for all time points).
The researchers did note that cancer patients experienced significant improvements in social functioning from baseline to the 12-month follow-up, but there was no further improvement after that.
The researchers also examined the different trajectories of social functioning over time. They found that social functioning improved over time for 47% of the cancer patients but worsened for 13%. In addition, 32% of patients had consistently low social functioning, and 9% had consistently high social functioning.
The cancer patients with consistently low social functioning were more likely to be off treatment at the time of follow-up, report more physical symptoms and higher levels of psychological distress (at both baseline and follow-up), and perceive themselves to receive less social support.
“Reducing physical symptoms and psychological distress and enhancing social support by interventions in the period after treatment may potentially help these young survivors to better reintegrate into society,” Dr Husson said.
A new study indicates that adolescent and young adult (AYA) cancer survivors continue to face social difficulties for more than 2 years after their diagnosis.
The research, published in Cancer, suggests these patients may see some improvement in their social lives during the first year after diagnosis.
However, their social functioning tends to remain constant after that, leaving them socially impaired relative to their cancer-free peers.
Previous studies have shown that AYAs with cancer experience greater challenges in social functioning than their cancer-free peers or even compared to older cancer patients.
But few studies have examined this phenomenon by following the same patients over time.
Olga Husson, PhD, of the Radboud University Medical Center in The Netherlands, and her colleagues set out to examine changes in social functioning among AYAs in the early years after a cancer diagnosis.
The researchers asked AYA cancer patients at 5 US medical institutions to complete a survey about social functioning within 4 months of their diagnosis, 12 months later, and 24 months later.
There were 141 patients (ages 14 to 39 at diagnosis) who completed the surveys.
The researchers found that, when compared to population norms, the cancer patients had inferior social functioning at all the time points studied.
Among the cancer patients, the mean social functioning score from the Medical Outcomes Study Short Form 36 Health Survey (version 2) was 52.0 around the time of cancer diagnosis, 73.1 at the 12-month follow-up, and 69.2 at the 24-month follow-up. In comparison, the population norm (for people ages 18 to 44) is 85.1 (P<0.001 for all time points).
The researchers did note that cancer patients experienced significant improvements in social functioning from baseline to the 12-month follow-up, but there was no further improvement after that.
The researchers also examined the different trajectories of social functioning over time. They found that social functioning improved over time for 47% of the cancer patients but worsened for 13%. In addition, 32% of patients had consistently low social functioning, and 9% had consistently high social functioning.
The cancer patients with consistently low social functioning were more likely to be off treatment at the time of follow-up, report more physical symptoms and higher levels of psychological distress (at both baseline and follow-up), and perceive themselves to receive less social support.
“Reducing physical symptoms and psychological distress and enhancing social support by interventions in the period after treatment may potentially help these young survivors to better reintegrate into society,” Dr Husson said.
New insight into high-hyperdiploid ALL
New research appears to explain how 10q21.2 influences the risk of high-hyperdiploid acute lymphoblastic leukemia (HD-ALL).
Previous research indicated that variation in the gene ARID5B at 10q21.2 is associated with HD-ALL.
Now, researchers have reported that the 10q21.2 risk locus for HD-ALL is mediated through the single nucleotide polymorphism (SNP) rs7090445, which disrupts RUNX3 transcription factor binding.
Specifically, the rs7090445-C allele confers an increased risk of HD-ALL through reduced RUNX3-mediated expression of ARID5B.
The researchers described these findings in Nature Communications.
“This study expands our understanding of how genetic risk factors can influence the development of acute lymphoblastic leukemia . . .,” said study author Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK.
Dr Houlston and his colleagues focused this research on 10q21.2 because it had previously been implicated in HD-ALL, but it wasn’t clear how the region affects the risk of HD-ALL.
The team said they found that a SNP in the region, rs7090445, is “highly associated” with HD-ALL.
Further investigation revealed that variation at rs7090445 disrupts RUNX3 binding and reduces the expression of ARID5B, as RUNX3 regulates ARID5B expression.
The researchers also discovered that the rs7090445-C risk allele, which is associated with reduced ARID5B expression, is amplified in HD-ALL. The risk allele is “preferentially retained” on additional copies of chromosome 10 in HD-ALL blasts.
“We implicate reduced expression of a gene called ARID5B in the production and release of the immature ‘blast’ cells that characterize [HD-ALL],” Dr Houlston said. “Our study gives a new insight into the causes of the disease and may open up new strategies for prevention.”
New research appears to explain how 10q21.2 influences the risk of high-hyperdiploid acute lymphoblastic leukemia (HD-ALL).
Previous research indicated that variation in the gene ARID5B at 10q21.2 is associated with HD-ALL.
Now, researchers have reported that the 10q21.2 risk locus for HD-ALL is mediated through the single nucleotide polymorphism (SNP) rs7090445, which disrupts RUNX3 transcription factor binding.
Specifically, the rs7090445-C allele confers an increased risk of HD-ALL through reduced RUNX3-mediated expression of ARID5B.
The researchers described these findings in Nature Communications.
“This study expands our understanding of how genetic risk factors can influence the development of acute lymphoblastic leukemia . . .,” said study author Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK.
Dr Houlston and his colleagues focused this research on 10q21.2 because it had previously been implicated in HD-ALL, but it wasn’t clear how the region affects the risk of HD-ALL.
The team said they found that a SNP in the region, rs7090445, is “highly associated” with HD-ALL.
Further investigation revealed that variation at rs7090445 disrupts RUNX3 binding and reduces the expression of ARID5B, as RUNX3 regulates ARID5B expression.
The researchers also discovered that the rs7090445-C risk allele, which is associated with reduced ARID5B expression, is amplified in HD-ALL. The risk allele is “preferentially retained” on additional copies of chromosome 10 in HD-ALL blasts.
“We implicate reduced expression of a gene called ARID5B in the production and release of the immature ‘blast’ cells that characterize [HD-ALL],” Dr Houlston said. “Our study gives a new insight into the causes of the disease and may open up new strategies for prevention.”
New research appears to explain how 10q21.2 influences the risk of high-hyperdiploid acute lymphoblastic leukemia (HD-ALL).
Previous research indicated that variation in the gene ARID5B at 10q21.2 is associated with HD-ALL.
Now, researchers have reported that the 10q21.2 risk locus for HD-ALL is mediated through the single nucleotide polymorphism (SNP) rs7090445, which disrupts RUNX3 transcription factor binding.
Specifically, the rs7090445-C allele confers an increased risk of HD-ALL through reduced RUNX3-mediated expression of ARID5B.
The researchers described these findings in Nature Communications.
“This study expands our understanding of how genetic risk factors can influence the development of acute lymphoblastic leukemia . . .,” said study author Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK.
Dr Houlston and his colleagues focused this research on 10q21.2 because it had previously been implicated in HD-ALL, but it wasn’t clear how the region affects the risk of HD-ALL.
The team said they found that a SNP in the region, rs7090445, is “highly associated” with HD-ALL.
Further investigation revealed that variation at rs7090445 disrupts RUNX3 binding and reduces the expression of ARID5B, as RUNX3 regulates ARID5B expression.
The researchers also discovered that the rs7090445-C risk allele, which is associated with reduced ARID5B expression, is amplified in HD-ALL. The risk allele is “preferentially retained” on additional copies of chromosome 10 in HD-ALL blasts.
“We implicate reduced expression of a gene called ARID5B in the production and release of the immature ‘blast’ cells that characterize [HD-ALL],” Dr Houlston said. “Our study gives a new insight into the causes of the disease and may open up new strategies for prevention.”
LVADs achieve cardiac palliation in muscular dystrophies
At one time, respiratory failure was the primary cause of death in young men and boys with muscular dystrophies, but since improvements in ventilator support have addressed this problem, cardiac complications such as cardiomyopathy have become the main cause of death in this group, with the highest risk of death in people with Duchenne muscular dystrophy (DMD). Researchers from Rome have reported that the novel use of ventricular assist devices in this population can prolong life.
Gianluigi Perri, MD, PhD, of University Hospital and Bambino Gesù Children Hospital in Rome, and his coauthors, shared their experience treating seven patients with dystrophinopathies and dilated cardiomyopathy (DCM) with left ventricular assist devices (LVADs) from February 2011 to February 2016 (J Thorac Cardiovasc Surg. 2017 March;153:669-74). “Our experience indicates that the use of an LVAD as destination therapy in patients with dystrophinopathies with end-stage DCM is feasible, suggesting that it may be suitable as a palliative therapy for the treatment of these patients with no other therapeutic options,” Dr. Perri and his coauthors said.
Heart transplantation is considered the procedure of choice for children with severe advanced heart failure, but transplantation is contraindicated for children with dystrophinopathies because of the risk of respiratory failure and progression of skeletal myopathy leads to limited functional capacity. Hence, Dr. Perri and his coauthors developed their alternative treatment for end-stage heart failure in these children. They used the Jarvik 2000 LVAD (Jarvik Heart Inc., New York) as destination therapy.
Six of the seven patients they operated on had DMD and one had beta-2 sarcoglycan deficit. Their ages ranged from 14.2 to 23.4 years. Two patients had early complications: retropharyngeal bleeding and cholecystectomy; and abdominal bleeding and splenectomy. Two different patients had late complications: gastrostomy; and osteolysis and infection at the pedestal site. Three patients died after the operation: one of stroke at 15 months; one of severe bleeding about 28 months later; and one of lung infection 45 months afterward. Follow-up for the surviving patients ranged from about 2 months to 40 months. Median hospital stay was 77 days.
Dr. Perri and his coauthors noted that the DMD Care Considerations Working Group expanded acceptable therapies for DMD cardiomyopathy to include novel treatments such as mechanical circulatory support and implantable cardioverter-defibrillators.
“Although the best approach remains unclear, it does seem clear that treatment should be more aggressive,” the researchers said. The limited life expectancy of these patients makes transplantation a complicated choice when a shortage of donors is a concern. “Therefore, the alternative therapeutic option is the use of LVAD,” Dr. Perri and his coauthors said.
These patients need care at centers “with a high level of experience of patients with DMD,” the researchers stated. Common comorbidities such as severe kyphoscoliosis and respiratory muscle weakness in this population increase surgical risks.
Dr. Perri and his coauthors used a surgical technique that involved avoiding the left thoracotomy approach common in adults who undergo VAD implantation, because of respiratory insufficiency in these younger patients. They also used cardiopulmonary bypass in all but one patient who had a minimally invasive off-pump procedure through a left anterior minithoracotomy.
The researchers “strongly suggest” noninvasive ventilation after surgery to assist in pulmonary function often compromised by scoliosis and muscle weakness. “Our experience shows that postoperative care can be extremely challenging and is often burdened by unexpected complications,” they noted.
Kyphoscoliosis poses challenges when placing drains, and complications of these patients should be treated only in a specialized center. “Indeed, one of our patients died in a peripheral hospital because they underwent bronchoscopic examination with an endoscope that caused severe and intractable retropharyngeal bleeding,” they said.
The researchers no relevant financial relationships to disclose.
Almost all young men living with Duchenne muscular dystrophy will develop heart failure, but for many of these patients, continuous-flow left ventricular assist devices can provide “reliable support” for up to a decade, David L. S. Morales, MD, of the Heart Institute at Cincinnati Children’s Hospital Medical Center, said in his invited commentary (J Thorac Cardiovasc Surg. 2017;153:675-6)
“The current series demonstrates, as has been shown at our institute as well as others, that one can provide an effective therapy for certain patients with DMD and heart failure,” Dr. Morales said of the work of Dr. Perri and coauthors. Dr. Morales added that maximizing outcomes in this population hinges on finding the appropriate time point for intervention in the disease process.
While “there is still much to be learned,” Dr. Morales said, Dr. Perri and his coauthors have shown that LVAD therapy is an option in patients with DMD and heart failure who have failed other treatments. “These young men may, therefore, have the option to extend their lives and possibly have the opportunity to benefit from the impressive medical advances being made,” he said. “Perhaps they and their families have been provided hope.”
Dr. Morales disclosed relationships with Berlin Heart, HeartWare and Oregon Total Artificial Heart.
Almost all young men living with Duchenne muscular dystrophy will develop heart failure, but for many of these patients, continuous-flow left ventricular assist devices can provide “reliable support” for up to a decade, David L. S. Morales, MD, of the Heart Institute at Cincinnati Children’s Hospital Medical Center, said in his invited commentary (J Thorac Cardiovasc Surg. 2017;153:675-6)
“The current series demonstrates, as has been shown at our institute as well as others, that one can provide an effective therapy for certain patients with DMD and heart failure,” Dr. Morales said of the work of Dr. Perri and coauthors. Dr. Morales added that maximizing outcomes in this population hinges on finding the appropriate time point for intervention in the disease process.
While “there is still much to be learned,” Dr. Morales said, Dr. Perri and his coauthors have shown that LVAD therapy is an option in patients with DMD and heart failure who have failed other treatments. “These young men may, therefore, have the option to extend their lives and possibly have the opportunity to benefit from the impressive medical advances being made,” he said. “Perhaps they and their families have been provided hope.”
Dr. Morales disclosed relationships with Berlin Heart, HeartWare and Oregon Total Artificial Heart.
Almost all young men living with Duchenne muscular dystrophy will develop heart failure, but for many of these patients, continuous-flow left ventricular assist devices can provide “reliable support” for up to a decade, David L. S. Morales, MD, of the Heart Institute at Cincinnati Children’s Hospital Medical Center, said in his invited commentary (J Thorac Cardiovasc Surg. 2017;153:675-6)
“The current series demonstrates, as has been shown at our institute as well as others, that one can provide an effective therapy for certain patients with DMD and heart failure,” Dr. Morales said of the work of Dr. Perri and coauthors. Dr. Morales added that maximizing outcomes in this population hinges on finding the appropriate time point for intervention in the disease process.
While “there is still much to be learned,” Dr. Morales said, Dr. Perri and his coauthors have shown that LVAD therapy is an option in patients with DMD and heart failure who have failed other treatments. “These young men may, therefore, have the option to extend their lives and possibly have the opportunity to benefit from the impressive medical advances being made,” he said. “Perhaps they and their families have been provided hope.”
Dr. Morales disclosed relationships with Berlin Heart, HeartWare and Oregon Total Artificial Heart.
At one time, respiratory failure was the primary cause of death in young men and boys with muscular dystrophies, but since improvements in ventilator support have addressed this problem, cardiac complications such as cardiomyopathy have become the main cause of death in this group, with the highest risk of death in people with Duchenne muscular dystrophy (DMD). Researchers from Rome have reported that the novel use of ventricular assist devices in this population can prolong life.
Gianluigi Perri, MD, PhD, of University Hospital and Bambino Gesù Children Hospital in Rome, and his coauthors, shared their experience treating seven patients with dystrophinopathies and dilated cardiomyopathy (DCM) with left ventricular assist devices (LVADs) from February 2011 to February 2016 (J Thorac Cardiovasc Surg. 2017 March;153:669-74). “Our experience indicates that the use of an LVAD as destination therapy in patients with dystrophinopathies with end-stage DCM is feasible, suggesting that it may be suitable as a palliative therapy for the treatment of these patients with no other therapeutic options,” Dr. Perri and his coauthors said.
Heart transplantation is considered the procedure of choice for children with severe advanced heart failure, but transplantation is contraindicated for children with dystrophinopathies because of the risk of respiratory failure and progression of skeletal myopathy leads to limited functional capacity. Hence, Dr. Perri and his coauthors developed their alternative treatment for end-stage heart failure in these children. They used the Jarvik 2000 LVAD (Jarvik Heart Inc., New York) as destination therapy.
Six of the seven patients they operated on had DMD and one had beta-2 sarcoglycan deficit. Their ages ranged from 14.2 to 23.4 years. Two patients had early complications: retropharyngeal bleeding and cholecystectomy; and abdominal bleeding and splenectomy. Two different patients had late complications: gastrostomy; and osteolysis and infection at the pedestal site. Three patients died after the operation: one of stroke at 15 months; one of severe bleeding about 28 months later; and one of lung infection 45 months afterward. Follow-up for the surviving patients ranged from about 2 months to 40 months. Median hospital stay was 77 days.
Dr. Perri and his coauthors noted that the DMD Care Considerations Working Group expanded acceptable therapies for DMD cardiomyopathy to include novel treatments such as mechanical circulatory support and implantable cardioverter-defibrillators.
“Although the best approach remains unclear, it does seem clear that treatment should be more aggressive,” the researchers said. The limited life expectancy of these patients makes transplantation a complicated choice when a shortage of donors is a concern. “Therefore, the alternative therapeutic option is the use of LVAD,” Dr. Perri and his coauthors said.
These patients need care at centers “with a high level of experience of patients with DMD,” the researchers stated. Common comorbidities such as severe kyphoscoliosis and respiratory muscle weakness in this population increase surgical risks.
Dr. Perri and his coauthors used a surgical technique that involved avoiding the left thoracotomy approach common in adults who undergo VAD implantation, because of respiratory insufficiency in these younger patients. They also used cardiopulmonary bypass in all but one patient who had a minimally invasive off-pump procedure through a left anterior minithoracotomy.
The researchers “strongly suggest” noninvasive ventilation after surgery to assist in pulmonary function often compromised by scoliosis and muscle weakness. “Our experience shows that postoperative care can be extremely challenging and is often burdened by unexpected complications,” they noted.
Kyphoscoliosis poses challenges when placing drains, and complications of these patients should be treated only in a specialized center. “Indeed, one of our patients died in a peripheral hospital because they underwent bronchoscopic examination with an endoscope that caused severe and intractable retropharyngeal bleeding,” they said.
The researchers no relevant financial relationships to disclose.
At one time, respiratory failure was the primary cause of death in young men and boys with muscular dystrophies, but since improvements in ventilator support have addressed this problem, cardiac complications such as cardiomyopathy have become the main cause of death in this group, with the highest risk of death in people with Duchenne muscular dystrophy (DMD). Researchers from Rome have reported that the novel use of ventricular assist devices in this population can prolong life.
Gianluigi Perri, MD, PhD, of University Hospital and Bambino Gesù Children Hospital in Rome, and his coauthors, shared their experience treating seven patients with dystrophinopathies and dilated cardiomyopathy (DCM) with left ventricular assist devices (LVADs) from February 2011 to February 2016 (J Thorac Cardiovasc Surg. 2017 March;153:669-74). “Our experience indicates that the use of an LVAD as destination therapy in patients with dystrophinopathies with end-stage DCM is feasible, suggesting that it may be suitable as a palliative therapy for the treatment of these patients with no other therapeutic options,” Dr. Perri and his coauthors said.
Heart transplantation is considered the procedure of choice for children with severe advanced heart failure, but transplantation is contraindicated for children with dystrophinopathies because of the risk of respiratory failure and progression of skeletal myopathy leads to limited functional capacity. Hence, Dr. Perri and his coauthors developed their alternative treatment for end-stage heart failure in these children. They used the Jarvik 2000 LVAD (Jarvik Heart Inc., New York) as destination therapy.
Six of the seven patients they operated on had DMD and one had beta-2 sarcoglycan deficit. Their ages ranged from 14.2 to 23.4 years. Two patients had early complications: retropharyngeal bleeding and cholecystectomy; and abdominal bleeding and splenectomy. Two different patients had late complications: gastrostomy; and osteolysis and infection at the pedestal site. Three patients died after the operation: one of stroke at 15 months; one of severe bleeding about 28 months later; and one of lung infection 45 months afterward. Follow-up for the surviving patients ranged from about 2 months to 40 months. Median hospital stay was 77 days.
Dr. Perri and his coauthors noted that the DMD Care Considerations Working Group expanded acceptable therapies for DMD cardiomyopathy to include novel treatments such as mechanical circulatory support and implantable cardioverter-defibrillators.
“Although the best approach remains unclear, it does seem clear that treatment should be more aggressive,” the researchers said. The limited life expectancy of these patients makes transplantation a complicated choice when a shortage of donors is a concern. “Therefore, the alternative therapeutic option is the use of LVAD,” Dr. Perri and his coauthors said.
These patients need care at centers “with a high level of experience of patients with DMD,” the researchers stated. Common comorbidities such as severe kyphoscoliosis and respiratory muscle weakness in this population increase surgical risks.
Dr. Perri and his coauthors used a surgical technique that involved avoiding the left thoracotomy approach common in adults who undergo VAD implantation, because of respiratory insufficiency in these younger patients. They also used cardiopulmonary bypass in all but one patient who had a minimally invasive off-pump procedure through a left anterior minithoracotomy.
The researchers “strongly suggest” noninvasive ventilation after surgery to assist in pulmonary function often compromised by scoliosis and muscle weakness. “Our experience shows that postoperative care can be extremely challenging and is often burdened by unexpected complications,” they noted.
Kyphoscoliosis poses challenges when placing drains, and complications of these patients should be treated only in a specialized center. “Indeed, one of our patients died in a peripheral hospital because they underwent bronchoscopic examination with an endoscope that caused severe and intractable retropharyngeal bleeding,” they said.
The researchers no relevant financial relationships to disclose.
FROM THE JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY
Key clinical point: A left ventricular assist device can be used as destination therapy in patients with Duchenne muscular dystrophy dystrophinopathies and end-stage dilated cardiomyopathy.
Major finding: Four of seven patients who had LVAD survived long term, and survival for the three who died ranged from 15 to 44 months.
Data source: Single-center, retrospective review of seven patients with DMD who had LVAD for DCM from February 2011 to February 2016.
Disclosure: Dr. Perri and his coauthors reported having no relevant financial disclosures.
Dupilumab improved eczema scores in children in open label trial
ORLANDO – Treatment with dupilumab in children and adolescents with moderate to severe atopic dermatitis (AD) reduced severity and pruritus scores from baseline and was well tolerated, in a multicenter, open-label trial of 78 children and adolescents.
Dupilumab’s “powerful ability” to block interleukin-4 and interleukin-13 pathways of inflammation is “especially exciting because AD is even more Th-2 cell driven in children,” said Michael J. Cork, MD, PhD, head of dermatologic research at the University of Sheffield (England), who presented the findings during a late-breaking research session at the annual meeting of the American Academy of Dermatology.
The study assessed the pharmacokinetics, safety, and efficacy of dupilumab and was conducted with 38 children, aged 6-11 years, and 40 adolescents, aged 12-17 years, with moderate to severe AD. All had failed topical corticosteroid therapy. Some of the children (16%) and adolescents (22.5%) had failed at least one systemic therapy.
Both age groups were given either a 2 mg/kg or a 4 mg/kg dose of dupilumab (administered subcutaneously), nothing for 8 weeks, followed by 4 weekly doses of their respective regimens. Mean Eczema Area and Severity Index (EASI) scores at baseline were 31.7 among the adolescents and 35.9 among the children.
At week 12, mean scores in the younger cohort given either 2 mg/kg or 4 mg/kg had improved by 76.2% and 63.4%, respectively, from baseline. In the adolescents, EASI scores at week 12 had improved by a mean of 66.4% in the 2-mg/kg group and 69.7% in the 4-mg/kg group.
Itch also improved “dramatically,” according to Dr. Cork. In the younger children, peak pruritus Numerical Rating Scale scores improved from baseline by a mean of 41.6% in the lower-dose group and 39.6% in the higher-dose group. In the older cohort, pruritus scores improved from baseline by a mean of 30.8% in the lower-dose group and 37.6% in the higher-dose group.
Treatment was well tolerated across the study, and adverse events were “mild, transient, and unrelated,” Dr. Cork said. “I would like to emphasize that these were not related to dupilumab, as they occurred during the period of time after the first dose, in weeks 6 and 7,” he commented. He attributed AD flares experienced in the study to the quick clearance of the drug in the first few weeks.
Dr. Cork reported numerous disclosures, including serving as an adviser, consultant, and investigator for Regeneron, the sponsor of the trial, and Sanofi; the companies developing dupilumab.
ORLANDO – Treatment with dupilumab in children and adolescents with moderate to severe atopic dermatitis (AD) reduced severity and pruritus scores from baseline and was well tolerated, in a multicenter, open-label trial of 78 children and adolescents.
Dupilumab’s “powerful ability” to block interleukin-4 and interleukin-13 pathways of inflammation is “especially exciting because AD is even more Th-2 cell driven in children,” said Michael J. Cork, MD, PhD, head of dermatologic research at the University of Sheffield (England), who presented the findings during a late-breaking research session at the annual meeting of the American Academy of Dermatology.
The study assessed the pharmacokinetics, safety, and efficacy of dupilumab and was conducted with 38 children, aged 6-11 years, and 40 adolescents, aged 12-17 years, with moderate to severe AD. All had failed topical corticosteroid therapy. Some of the children (16%) and adolescents (22.5%) had failed at least one systemic therapy.
Both age groups were given either a 2 mg/kg or a 4 mg/kg dose of dupilumab (administered subcutaneously), nothing for 8 weeks, followed by 4 weekly doses of their respective regimens. Mean Eczema Area and Severity Index (EASI) scores at baseline were 31.7 among the adolescents and 35.9 among the children.
At week 12, mean scores in the younger cohort given either 2 mg/kg or 4 mg/kg had improved by 76.2% and 63.4%, respectively, from baseline. In the adolescents, EASI scores at week 12 had improved by a mean of 66.4% in the 2-mg/kg group and 69.7% in the 4-mg/kg group.
Itch also improved “dramatically,” according to Dr. Cork. In the younger children, peak pruritus Numerical Rating Scale scores improved from baseline by a mean of 41.6% in the lower-dose group and 39.6% in the higher-dose group. In the older cohort, pruritus scores improved from baseline by a mean of 30.8% in the lower-dose group and 37.6% in the higher-dose group.
Treatment was well tolerated across the study, and adverse events were “mild, transient, and unrelated,” Dr. Cork said. “I would like to emphasize that these were not related to dupilumab, as they occurred during the period of time after the first dose, in weeks 6 and 7,” he commented. He attributed AD flares experienced in the study to the quick clearance of the drug in the first few weeks.
Dr. Cork reported numerous disclosures, including serving as an adviser, consultant, and investigator for Regeneron, the sponsor of the trial, and Sanofi; the companies developing dupilumab.
ORLANDO – Treatment with dupilumab in children and adolescents with moderate to severe atopic dermatitis (AD) reduced severity and pruritus scores from baseline and was well tolerated, in a multicenter, open-label trial of 78 children and adolescents.
Dupilumab’s “powerful ability” to block interleukin-4 and interleukin-13 pathways of inflammation is “especially exciting because AD is even more Th-2 cell driven in children,” said Michael J. Cork, MD, PhD, head of dermatologic research at the University of Sheffield (England), who presented the findings during a late-breaking research session at the annual meeting of the American Academy of Dermatology.
The study assessed the pharmacokinetics, safety, and efficacy of dupilumab and was conducted with 38 children, aged 6-11 years, and 40 adolescents, aged 12-17 years, with moderate to severe AD. All had failed topical corticosteroid therapy. Some of the children (16%) and adolescents (22.5%) had failed at least one systemic therapy.
Both age groups were given either a 2 mg/kg or a 4 mg/kg dose of dupilumab (administered subcutaneously), nothing for 8 weeks, followed by 4 weekly doses of their respective regimens. Mean Eczema Area and Severity Index (EASI) scores at baseline were 31.7 among the adolescents and 35.9 among the children.
At week 12, mean scores in the younger cohort given either 2 mg/kg or 4 mg/kg had improved by 76.2% and 63.4%, respectively, from baseline. In the adolescents, EASI scores at week 12 had improved by a mean of 66.4% in the 2-mg/kg group and 69.7% in the 4-mg/kg group.
Itch also improved “dramatically,” according to Dr. Cork. In the younger children, peak pruritus Numerical Rating Scale scores improved from baseline by a mean of 41.6% in the lower-dose group and 39.6% in the higher-dose group. In the older cohort, pruritus scores improved from baseline by a mean of 30.8% in the lower-dose group and 37.6% in the higher-dose group.
Treatment was well tolerated across the study, and adverse events were “mild, transient, and unrelated,” Dr. Cork said. “I would like to emphasize that these were not related to dupilumab, as they occurred during the period of time after the first dose, in weeks 6 and 7,” he commented. He attributed AD flares experienced in the study to the quick clearance of the drug in the first few weeks.
Dr. Cork reported numerous disclosures, including serving as an adviser, consultant, and investigator for Regeneron, the sponsor of the trial, and Sanofi; the companies developing dupilumab.
AT AAD 17
Key clinical point: Data on dupilumab for treating moderate to severe atopic dermatitis in children and adolescents are promising.
Major finding: At week 12, treatment with dupilumab at 2 mg/kg and 4 mg/kg doses, across 8 weeks in two pediatric cohorts improved baseline EASI scores by 69.7% and pruritus scores by a third and was well tolerated.
Data source: A phase IIa, multicenter, open label pharmacokinetics, safety, and efficacy trial evaluating two dosing regimens of dupilumab in 78 children with moderate to severe AD.
Disclosures: Dr. Cork reported numerous disclosures, including serving as an adviser, consultant, and investigator for Regeneron, the sponsor of the trial, and Sanofi – the two companies developing dupilumab.
Influenza vaccine is underused in children with heart disease
The influenza vaccine is underused in children with heart disease; approximately one-third were vaccinated in a prospective study of 186 children in September and October 2012.
“Annual influenza vaccination is the most effective and safe means of preventing the disease,” and children with chronic diseases including heart conditions are at increased risk for complications that would require hospitalization, wrote Gilat Livni, MD, of Tel Aviv University and colleagues.
Overall, 59% of parents reported that their primary pediatrician recommended flu vaccination, and 53% of these parents complied. By contrast, only 13% of children whose pediatricians had not recommended vaccination received it.
“The failure of parents to receive information or advice from a physician regarding vaccination was strongly inversely related to vaccination of the child,” the researchers wrote. Parents’ misconceptions included the belief that the vaccine would cause flu (66%, of whom 30% had their child vaccinated); the belief that the vaccine would cause severe side effects (55%, of whom 26% had their child vaccinated), and the belief that the vaccine was unsafe (47%, 21% of whom had their child vaccinated).
“Our results emphasize the need to raise awareness among physicians and other medical health care personnel dealing with children with heart disease of the importance of properly counseling parents regarding influenza vaccination,” the researchers said. “Recommending the vaccine should be made part of routine patient visits in fall and winter.”
The researchers had no financial conflicts to disclose. The findings were published online ahead of print in the Pediatric Infectious Disease Journal (Ped Infect Dis J. 2017. doi: 10.1097/INF.0000000000001579).
The influenza vaccine is underused in children with heart disease; approximately one-third were vaccinated in a prospective study of 186 children in September and October 2012.
“Annual influenza vaccination is the most effective and safe means of preventing the disease,” and children with chronic diseases including heart conditions are at increased risk for complications that would require hospitalization, wrote Gilat Livni, MD, of Tel Aviv University and colleagues.
Overall, 59% of parents reported that their primary pediatrician recommended flu vaccination, and 53% of these parents complied. By contrast, only 13% of children whose pediatricians had not recommended vaccination received it.
“The failure of parents to receive information or advice from a physician regarding vaccination was strongly inversely related to vaccination of the child,” the researchers wrote. Parents’ misconceptions included the belief that the vaccine would cause flu (66%, of whom 30% had their child vaccinated); the belief that the vaccine would cause severe side effects (55%, of whom 26% had their child vaccinated), and the belief that the vaccine was unsafe (47%, 21% of whom had their child vaccinated).
“Our results emphasize the need to raise awareness among physicians and other medical health care personnel dealing with children with heart disease of the importance of properly counseling parents regarding influenza vaccination,” the researchers said. “Recommending the vaccine should be made part of routine patient visits in fall and winter.”
The researchers had no financial conflicts to disclose. The findings were published online ahead of print in the Pediatric Infectious Disease Journal (Ped Infect Dis J. 2017. doi: 10.1097/INF.0000000000001579).
The influenza vaccine is underused in children with heart disease; approximately one-third were vaccinated in a prospective study of 186 children in September and October 2012.
“Annual influenza vaccination is the most effective and safe means of preventing the disease,” and children with chronic diseases including heart conditions are at increased risk for complications that would require hospitalization, wrote Gilat Livni, MD, of Tel Aviv University and colleagues.
Overall, 59% of parents reported that their primary pediatrician recommended flu vaccination, and 53% of these parents complied. By contrast, only 13% of children whose pediatricians had not recommended vaccination received it.
“The failure of parents to receive information or advice from a physician regarding vaccination was strongly inversely related to vaccination of the child,” the researchers wrote. Parents’ misconceptions included the belief that the vaccine would cause flu (66%, of whom 30% had their child vaccinated); the belief that the vaccine would cause severe side effects (55%, of whom 26% had their child vaccinated), and the belief that the vaccine was unsafe (47%, 21% of whom had their child vaccinated).
“Our results emphasize the need to raise awareness among physicians and other medical health care personnel dealing with children with heart disease of the importance of properly counseling parents regarding influenza vaccination,” the researchers said. “Recommending the vaccine should be made part of routine patient visits in fall and winter.”
The researchers had no financial conflicts to disclose. The findings were published online ahead of print in the Pediatric Infectious Disease Journal (Ped Infect Dis J. 2017. doi: 10.1097/INF.0000000000001579).
FROM THE JOURNAL OF PEDIATRICS
How will crisaborole for atopic dermatitis fit into clinical practice?
WAILEA, HAWAII – With the recent approval of crisaborole ointment 2% for the treatment of atopic dermatitis, the question of how to best utilize this novel drug in daily clinical practice was put to two atopic dermatitis experts at the Hawaii Dermatology Seminar sponsored by the Global Academy for Medical Education/Skin Disease Research Foundation.
Two of these experts, Lawrence F. Eichenfield, MD, and Wynnis L. Tom, MD, have garnered extensive clinical trials experience with crisaborole (Eucrisa), which, in December 2016, became the first prescription drug approved by the Food and Drug Administration for atopic dermatitis (AD) in more than 10 years. The topical phosphodiesterase-4 inhibitor is indicated for twice-daily treatment of patients aged 2 years and older with mild to moderate AD.
“Topical steroids are the most cost-effective medicine for the management of acute flares of atopic dermatitis. And if I can then take patients down to only intermittent use of topical corticosteroids of mid-strength or lower, depending on patient age, I’ll probably hang in and do just that,” said Dr. Eichenfield, professor of dermatology and pediatrics at the University of California, San Diego, and chief of pediatric and adolescent dermatology at Rady Children’s Hospital-San Diego.
“But, when I have patients with more persistent disease, frequent flaring where I can’t get by with just twice-a-week topical steroids, or ... sensitive areas where I need to avoid steroid atrophy, that’s when I’m likely to incorporate a nonsteroidal agent into regimens of care. Traditionally, over the past 15-18 years, that’s been either with topical tacrolimus [Protopic] or pimecrolimus [Elidel]. And now crisaborole will fit right in there,” he continued.
As for how long to keep patients on crisaborole, Dr. Eichenfield noted that the package labeling “is pretty open.” It doesn’t address the question of treatment duration, and the patient handout that accompanies the tube of ointment simply states, “Use [crisaborole] Eucrisa exactly as your health care provider tells you to use it.”
“In a no-data situation such as this, I’ll probably try to see if use a few times a week or in a mix/match between corticosteroids, a topical calcineurin inhibitor, and crisaborole will provide decent long-term disease control,” Dr. Eichenfield said.
The important, remaining questions regarding crisaborole include, Is it comparatively efficient? – a question that will require head-to-head clinical trials. Is it cost effective? Will it be effective and safe in children younger than 2 years of age? Is it safe beyond 1 year of use? – a particularly important question, given that the phase III clinical trials were only a month long, the dermatologist added.
Dr. Eichenfield emphasized that he was offering only his personal opinion on the use of crisaborole in response to audience questions. Eventually, guidelines committees will formally address how to incorporate the new drug into a sound treatment strategy.
Dr. Tom, like Dr. Eichenfield, was a coauthor of the two pivotal phase III randomized trials of crisaborole, which found infrequent and minimal side effects along with significant improvement of formal quality of life measures (J Am Acad Dermatol. 2016 Sep;75[3]:494-503.e4).
She said that one way she’ll immediately put the new topical agent to work is in treating disease on the face and other sensitive skin areas. The drug’s steroid-sparing ability is also a major asset, given how many parents of children with AD are steroid phobic.
“I’m certainly not going to give up on topical steroids, but anything that enables me to lessen their use is a comfort for parents. That’s really important,” observed Dr. Tom of the University of California, San Diego, and Rady Children’s Hospital.
Both pediatric dermatologists reported having received research grants from or serving as consultants to roughly half-a-dozen pharmaceutical companies, including Anacor/Pfizer, which markets crisaborole.
SDEF and this news organization are owned by the same parent company.
WAILEA, HAWAII – With the recent approval of crisaborole ointment 2% for the treatment of atopic dermatitis, the question of how to best utilize this novel drug in daily clinical practice was put to two atopic dermatitis experts at the Hawaii Dermatology Seminar sponsored by the Global Academy for Medical Education/Skin Disease Research Foundation.
Two of these experts, Lawrence F. Eichenfield, MD, and Wynnis L. Tom, MD, have garnered extensive clinical trials experience with crisaborole (Eucrisa), which, in December 2016, became the first prescription drug approved by the Food and Drug Administration for atopic dermatitis (AD) in more than 10 years. The topical phosphodiesterase-4 inhibitor is indicated for twice-daily treatment of patients aged 2 years and older with mild to moderate AD.
“Topical steroids are the most cost-effective medicine for the management of acute flares of atopic dermatitis. And if I can then take patients down to only intermittent use of topical corticosteroids of mid-strength or lower, depending on patient age, I’ll probably hang in and do just that,” said Dr. Eichenfield, professor of dermatology and pediatrics at the University of California, San Diego, and chief of pediatric and adolescent dermatology at Rady Children’s Hospital-San Diego.
“But, when I have patients with more persistent disease, frequent flaring where I can’t get by with just twice-a-week topical steroids, or ... sensitive areas where I need to avoid steroid atrophy, that’s when I’m likely to incorporate a nonsteroidal agent into regimens of care. Traditionally, over the past 15-18 years, that’s been either with topical tacrolimus [Protopic] or pimecrolimus [Elidel]. And now crisaborole will fit right in there,” he continued.
As for how long to keep patients on crisaborole, Dr. Eichenfield noted that the package labeling “is pretty open.” It doesn’t address the question of treatment duration, and the patient handout that accompanies the tube of ointment simply states, “Use [crisaborole] Eucrisa exactly as your health care provider tells you to use it.”
“In a no-data situation such as this, I’ll probably try to see if use a few times a week or in a mix/match between corticosteroids, a topical calcineurin inhibitor, and crisaborole will provide decent long-term disease control,” Dr. Eichenfield said.
The important, remaining questions regarding crisaborole include, Is it comparatively efficient? – a question that will require head-to-head clinical trials. Is it cost effective? Will it be effective and safe in children younger than 2 years of age? Is it safe beyond 1 year of use? – a particularly important question, given that the phase III clinical trials were only a month long, the dermatologist added.
Dr. Eichenfield emphasized that he was offering only his personal opinion on the use of crisaborole in response to audience questions. Eventually, guidelines committees will formally address how to incorporate the new drug into a sound treatment strategy.
Dr. Tom, like Dr. Eichenfield, was a coauthor of the two pivotal phase III randomized trials of crisaborole, which found infrequent and minimal side effects along with significant improvement of formal quality of life measures (J Am Acad Dermatol. 2016 Sep;75[3]:494-503.e4).
She said that one way she’ll immediately put the new topical agent to work is in treating disease on the face and other sensitive skin areas. The drug’s steroid-sparing ability is also a major asset, given how many parents of children with AD are steroid phobic.
“I’m certainly not going to give up on topical steroids, but anything that enables me to lessen their use is a comfort for parents. That’s really important,” observed Dr. Tom of the University of California, San Diego, and Rady Children’s Hospital.
Both pediatric dermatologists reported having received research grants from or serving as consultants to roughly half-a-dozen pharmaceutical companies, including Anacor/Pfizer, which markets crisaborole.
SDEF and this news organization are owned by the same parent company.
WAILEA, HAWAII – With the recent approval of crisaborole ointment 2% for the treatment of atopic dermatitis, the question of how to best utilize this novel drug in daily clinical practice was put to two atopic dermatitis experts at the Hawaii Dermatology Seminar sponsored by the Global Academy for Medical Education/Skin Disease Research Foundation.
Two of these experts, Lawrence F. Eichenfield, MD, and Wynnis L. Tom, MD, have garnered extensive clinical trials experience with crisaborole (Eucrisa), which, in December 2016, became the first prescription drug approved by the Food and Drug Administration for atopic dermatitis (AD) in more than 10 years. The topical phosphodiesterase-4 inhibitor is indicated for twice-daily treatment of patients aged 2 years and older with mild to moderate AD.
“Topical steroids are the most cost-effective medicine for the management of acute flares of atopic dermatitis. And if I can then take patients down to only intermittent use of topical corticosteroids of mid-strength or lower, depending on patient age, I’ll probably hang in and do just that,” said Dr. Eichenfield, professor of dermatology and pediatrics at the University of California, San Diego, and chief of pediatric and adolescent dermatology at Rady Children’s Hospital-San Diego.
“But, when I have patients with more persistent disease, frequent flaring where I can’t get by with just twice-a-week topical steroids, or ... sensitive areas where I need to avoid steroid atrophy, that’s when I’m likely to incorporate a nonsteroidal agent into regimens of care. Traditionally, over the past 15-18 years, that’s been either with topical tacrolimus [Protopic] or pimecrolimus [Elidel]. And now crisaborole will fit right in there,” he continued.
As for how long to keep patients on crisaborole, Dr. Eichenfield noted that the package labeling “is pretty open.” It doesn’t address the question of treatment duration, and the patient handout that accompanies the tube of ointment simply states, “Use [crisaborole] Eucrisa exactly as your health care provider tells you to use it.”
“In a no-data situation such as this, I’ll probably try to see if use a few times a week or in a mix/match between corticosteroids, a topical calcineurin inhibitor, and crisaborole will provide decent long-term disease control,” Dr. Eichenfield said.
The important, remaining questions regarding crisaborole include, Is it comparatively efficient? – a question that will require head-to-head clinical trials. Is it cost effective? Will it be effective and safe in children younger than 2 years of age? Is it safe beyond 1 year of use? – a particularly important question, given that the phase III clinical trials were only a month long, the dermatologist added.
Dr. Eichenfield emphasized that he was offering only his personal opinion on the use of crisaborole in response to audience questions. Eventually, guidelines committees will formally address how to incorporate the new drug into a sound treatment strategy.
Dr. Tom, like Dr. Eichenfield, was a coauthor of the two pivotal phase III randomized trials of crisaborole, which found infrequent and minimal side effects along with significant improvement of formal quality of life measures (J Am Acad Dermatol. 2016 Sep;75[3]:494-503.e4).
She said that one way she’ll immediately put the new topical agent to work is in treating disease on the face and other sensitive skin areas. The drug’s steroid-sparing ability is also a major asset, given how many parents of children with AD are steroid phobic.
“I’m certainly not going to give up on topical steroids, but anything that enables me to lessen their use is a comfort for parents. That’s really important,” observed Dr. Tom of the University of California, San Diego, and Rady Children’s Hospital.
Both pediatric dermatologists reported having received research grants from or serving as consultants to roughly half-a-dozen pharmaceutical companies, including Anacor/Pfizer, which markets crisaborole.
SDEF and this news organization are owned by the same parent company.
EXPERT ANALYSIS FROM THE SDEF HAWAII DERMATOLOGY SEMINAR