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Primary care needs pile up for sickle cell patients
BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.
People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.
And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.
“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.
People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.
Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.
She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).
It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.
“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.
One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.
“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”
Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).
Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.
“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.
Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.
BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.
People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.
And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.
“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.
People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.
Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.
She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).
It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.
“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.
One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.
“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”
Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).
Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.
“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.
Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.
BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.
People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.
And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.
“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.
People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.
Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.
She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).
It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.
“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.
One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.
“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”
Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).
Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.
“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.
Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.
EXPERT ANALYSIS FROM SICKLE CELL IN FOCUS
Sickle cell disease gene therapy seen advancing
BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health. 
Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.
Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).
Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.
“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.
Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.
The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.
Dr. Bauer provided an update on the BCL11A research that seeks to block what amounts to a genetic “off switch” for production of fetal hemoglobin. It’s long been known that erythrocytes of newborns with sickle cell disease are protected from sickling by high levels of fetal hemoglobin. Clinical manifestations of sickle cell disease then emerge in the first year of life as fetal hemoglobin levels decline.
“A main goal in hematology has been to understand how is it that these alternative fetal hemoglobin genes get silenced and how can we turn them back on,” said Dr. Bauer, a staff physician in pediatric hematology/oncology.
The gene BCL11A also plays key roles in the development of the central nervous system, B lymphatic lymphocyte maturation, and hematopoietic stem cell self-renewal. That led the researchers to hone in on targeting sequences around the BCL11A gene that act as erythroid enhancers, intending to limit potential complications by creating a very specific therapy for sickle cell disease.
In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.
The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.
Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”
Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.
“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.
She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”
The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.
Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.
BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.
Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.
Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).
Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.
“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.
Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.
The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.
Dr. Bauer provided an update on the BCL11A research that seeks to block what amounts to a genetic “off switch” for production of fetal hemoglobin. It’s long been known that erythrocytes of newborns with sickle cell disease are protected from sickling by high levels of fetal hemoglobin. Clinical manifestations of sickle cell disease then emerge in the first year of life as fetal hemoglobin levels decline.
“A main goal in hematology has been to understand how is it that these alternative fetal hemoglobin genes get silenced and how can we turn them back on,” said Dr. Bauer, a staff physician in pediatric hematology/oncology.
The gene BCL11A also plays key roles in the development of the central nervous system, B lymphatic lymphocyte maturation, and hematopoietic stem cell self-renewal. That led the researchers to hone in on targeting sequences around the BCL11A gene that act as erythroid enhancers, intending to limit potential complications by creating a very specific therapy for sickle cell disease.
In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.
The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.
Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”
Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.
“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.
She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”
The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.
Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.
BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.
Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.
Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).
Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.
“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.
Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.
The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.
Dr. Bauer provided an update on the BCL11A research that seeks to block what amounts to a genetic “off switch” for production of fetal hemoglobin. It’s long been known that erythrocytes of newborns with sickle cell disease are protected from sickling by high levels of fetal hemoglobin. Clinical manifestations of sickle cell disease then emerge in the first year of life as fetal hemoglobin levels decline.
“A main goal in hematology has been to understand how is it that these alternative fetal hemoglobin genes get silenced and how can we turn them back on,” said Dr. Bauer, a staff physician in pediatric hematology/oncology.
The gene BCL11A also plays key roles in the development of the central nervous system, B lymphatic lymphocyte maturation, and hematopoietic stem cell self-renewal. That led the researchers to hone in on targeting sequences around the BCL11A gene that act as erythroid enhancers, intending to limit potential complications by creating a very specific therapy for sickle cell disease.
In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.
The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.
Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”
Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.
“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.
She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”
The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.
Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.
REPORTING FROM SICKLE CELL IN FOCUS
App tied to reducing insomnia, depression in adults
ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.
Previous research has shown that CBT for insomnia (CBT-I) that’s conducted in person is not only effective for insomnia but also can reduce co-occurring depression. In fact, the treatment effects for depression are roughly the same magnitude as antidepressants but with fewer side effects and contraindications, said Philip C. Cheng, PhD, of the Sleep Disorders & Research Center at the Henry Ford Health System in Detroit. He cited a systematic literature review published recently that found CBT-I to be a promising treatment for depression comorbid with insomnia (J Psychosom Res. 2018 Mar;106:1-2).
“We’ve got a two-birds-with-one-stone kind of a deal,” Dr. Cheng said, referring to the ability of CBT-I to address both disorders. “It’s hard to resist the impulse to say: ‘This is great. Let’s get this out to everyone who has insomnia.’ ”
But there’s only a limited pool of about 1,200 health care professionals experienced in CBT-I to serve a much larger pool of people who might need help warding off depression, he said. The NIMH estimates that 16.2 million adults in the United States had at least one major depressive episode in 2016, which is about 6.7% of all U.S. adults. To address the shortage, developers have created digital apps such as Sleepio, which Dr. Cheng and his colleagues used in their research.
For this study, they recruited people with insomnia who did not at the time have depression. Patients were assigned to either use the Sleepio app or follow a more traditional sleep education program. The latter consisted of six weekly emails with tips on sleep hygiene, Dr. Cheng said. These contained the typical messages that a patient would receive from a physician to address a sleep disorder, he added.
“The doctor says: ‘Don’t drink caffeine; make sure you sleep in a dark room,’ things like that,” Dr. Cheng said. “A lot of evidence has shown that this is not an effective stand-alone treatment for insomnia.”
With Sleepio, users get online assistance in addressing their challenges with sleep. Dr. Cheng said he and his colleagues used Sleepio because of its grounding in CBT methods. He presented the results seen in 166 people who used the digital CBT-I approach and 146 who received sleep education.
An interim analysis of results showed that within a period of 12 months after treatment, 20% of those in the sleep education control group developed incident depression, whereas only 10% of those in the CBT-I did, Dr. Cheng said. Analyzing the results, Dr. Cheng and his colleagues said those numbers indicate that the number needed to treat to prevent one case of depression was 10.
The subscription for this online tool costs about $400 a year, so it would cost $4,000 to prevent one case of depression, Dr. Cheng said.
Sleepio, also available on web-based platforms, is compatible with personal tracking devices, such as Fitbit. Sleepio users also have online community members who can share their experiences and support (Cogn Behav Pract. 2018 Aug;25[3]:442-8). It was created by Colin Espie, PhD, DSc, of the University of Oxford (England) and Peter Hames. Dr. Espie and Mr. Hames are cofounders of Big Health, a company that creates automated behavioral programs.
The Robert Wood Johnson Foundation funded the study. Dr. Cheng also is funded by a National Institutes of Health grant (K23HL138166). Sleepio provided its product for the study free of charge. Dr. Cheng said that he has no relevant conflicts of interest and that he has funding from Harmony Biosciences for a study unrelated to this work.
ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.
Previous research has shown that CBT for insomnia (CBT-I) that’s conducted in person is not only effective for insomnia but also can reduce co-occurring depression. In fact, the treatment effects for depression are roughly the same magnitude as antidepressants but with fewer side effects and contraindications, said Philip C. Cheng, PhD, of the Sleep Disorders & Research Center at the Henry Ford Health System in Detroit. He cited a systematic literature review published recently that found CBT-I to be a promising treatment for depression comorbid with insomnia (J Psychosom Res. 2018 Mar;106:1-2).
“We’ve got a two-birds-with-one-stone kind of a deal,” Dr. Cheng said, referring to the ability of CBT-I to address both disorders. “It’s hard to resist the impulse to say: ‘This is great. Let’s get this out to everyone who has insomnia.’ ”
But there’s only a limited pool of about 1,200 health care professionals experienced in CBT-I to serve a much larger pool of people who might need help warding off depression, he said. The NIMH estimates that 16.2 million adults in the United States had at least one major depressive episode in 2016, which is about 6.7% of all U.S. adults. To address the shortage, developers have created digital apps such as Sleepio, which Dr. Cheng and his colleagues used in their research.
For this study, they recruited people with insomnia who did not at the time have depression. Patients were assigned to either use the Sleepio app or follow a more traditional sleep education program. The latter consisted of six weekly emails with tips on sleep hygiene, Dr. Cheng said. These contained the typical messages that a patient would receive from a physician to address a sleep disorder, he added.
“The doctor says: ‘Don’t drink caffeine; make sure you sleep in a dark room,’ things like that,” Dr. Cheng said. “A lot of evidence has shown that this is not an effective stand-alone treatment for insomnia.”
With Sleepio, users get online assistance in addressing their challenges with sleep. Dr. Cheng said he and his colleagues used Sleepio because of its grounding in CBT methods. He presented the results seen in 166 people who used the digital CBT-I approach and 146 who received sleep education.
An interim analysis of results showed that within a period of 12 months after treatment, 20% of those in the sleep education control group developed incident depression, whereas only 10% of those in the CBT-I did, Dr. Cheng said. Analyzing the results, Dr. Cheng and his colleagues said those numbers indicate that the number needed to treat to prevent one case of depression was 10.
The subscription for this online tool costs about $400 a year, so it would cost $4,000 to prevent one case of depression, Dr. Cheng said.
Sleepio, also available on web-based platforms, is compatible with personal tracking devices, such as Fitbit. Sleepio users also have online community members who can share their experiences and support (Cogn Behav Pract. 2018 Aug;25[3]:442-8). It was created by Colin Espie, PhD, DSc, of the University of Oxford (England) and Peter Hames. Dr. Espie and Mr. Hames are cofounders of Big Health, a company that creates automated behavioral programs.
The Robert Wood Johnson Foundation funded the study. Dr. Cheng also is funded by a National Institutes of Health grant (K23HL138166). Sleepio provided its product for the study free of charge. Dr. Cheng said that he has no relevant conflicts of interest and that he has funding from Harmony Biosciences for a study unrelated to this work.
ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.
Previous research has shown that CBT for insomnia (CBT-I) that’s conducted in person is not only effective for insomnia but also can reduce co-occurring depression. In fact, the treatment effects for depression are roughly the same magnitude as antidepressants but with fewer side effects and contraindications, said Philip C. Cheng, PhD, of the Sleep Disorders & Research Center at the Henry Ford Health System in Detroit. He cited a systematic literature review published recently that found CBT-I to be a promising treatment for depression comorbid with insomnia (J Psychosom Res. 2018 Mar;106:1-2).
“We’ve got a two-birds-with-one-stone kind of a deal,” Dr. Cheng said, referring to the ability of CBT-I to address both disorders. “It’s hard to resist the impulse to say: ‘This is great. Let’s get this out to everyone who has insomnia.’ ”
But there’s only a limited pool of about 1,200 health care professionals experienced in CBT-I to serve a much larger pool of people who might need help warding off depression, he said. The NIMH estimates that 16.2 million adults in the United States had at least one major depressive episode in 2016, which is about 6.7% of all U.S. adults. To address the shortage, developers have created digital apps such as Sleepio, which Dr. Cheng and his colleagues used in their research.
For this study, they recruited people with insomnia who did not at the time have depression. Patients were assigned to either use the Sleepio app or follow a more traditional sleep education program. The latter consisted of six weekly emails with tips on sleep hygiene, Dr. Cheng said. These contained the typical messages that a patient would receive from a physician to address a sleep disorder, he added.
“The doctor says: ‘Don’t drink caffeine; make sure you sleep in a dark room,’ things like that,” Dr. Cheng said. “A lot of evidence has shown that this is not an effective stand-alone treatment for insomnia.”
With Sleepio, users get online assistance in addressing their challenges with sleep. Dr. Cheng said he and his colleagues used Sleepio because of its grounding in CBT methods. He presented the results seen in 166 people who used the digital CBT-I approach and 146 who received sleep education.
An interim analysis of results showed that within a period of 12 months after treatment, 20% of those in the sleep education control group developed incident depression, whereas only 10% of those in the CBT-I did, Dr. Cheng said. Analyzing the results, Dr. Cheng and his colleagues said those numbers indicate that the number needed to treat to prevent one case of depression was 10.
The subscription for this online tool costs about $400 a year, so it would cost $4,000 to prevent one case of depression, Dr. Cheng said.
Sleepio, also available on web-based platforms, is compatible with personal tracking devices, such as Fitbit. Sleepio users also have online community members who can share their experiences and support (Cogn Behav Pract. 2018 Aug;25[3]:442-8). It was created by Colin Espie, PhD, DSc, of the University of Oxford (England) and Peter Hames. Dr. Espie and Mr. Hames are cofounders of Big Health, a company that creates automated behavioral programs.
The Robert Wood Johnson Foundation funded the study. Dr. Cheng also is funded by a National Institutes of Health grant (K23HL138166). Sleepio provided its product for the study free of charge. Dr. Cheng said that he has no relevant conflicts of interest and that he has funding from Harmony Biosciences for a study unrelated to this work.
REPORTING FROM AN NIMH CONFERENCE
Key clinical point: The number needed to prevent one case of depression using the app is estimated to be 10.
Major finding: Within a period of 12 months after treatment, 20% of those in the sleep education control group developed incident depression, whereas only 10% of those in the CBT-I group did.
Study details: An interim analysis of results for 312 patients.
Disclosures: The Robert Wood Johnson Foundation funded the study. Dr. Cheng also is funded by a National Institutes of Health grant (K23HL138166). Sleepio provided its product for the study free of charge. Dr. Cheng said that he has no relevant conflicts of interest and that he receives funding from Harmony Biosciences for a study unrelated to this work.
Address physical health risks of people with SMI
ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.
The researchers discussed strategies aimed at combating obesity and diabetes, as well as behaviors such as smoking and sedentary lifestyle.
“One of the things that jumps out is the tremendous need for evidence-based strategies to address these physical health problems that are common in general population but even more of a burden for people with serious mental illness,” said Susan T. Azrin, PhD, of the NIMH, in an interview.
A study published in 2015, estimated that people with schizophrenia, for example, might lose almost 30 years of life because of premature death. Individuals with serious mental illness also experience elevated morbidity from cardiovascular disease and cancer. The NIMH and other federal agencies have in recent years looked for ways to help people with SMI quit smoking, and better control their weight and cholesterol.
But approaches that sound promising for boosting physical fitness in this group of patients have not always proven successful. Joshua Breslau, PhD, ScD, of the Rand Corp. discussed findings from a 2014 paper where he and his colleagues reported somewhat disappointing results from a study of federal Primary and Behavioral Health Care Integration grants.
The researchers matched clinics receiving this funding with similar ones that did not. They found that people with mental illness treated at the clinics receiving the grants showed improvements in some indicators of physical health (diastolic blood pressure, total cholesterol, LDL cholesterol, and fasting plasma glucose) but not in others (systolic blood pressure, body mass index, HDL cholesterol, hemoglobin A1c, triglycerides, self-reported smoking). Dr. Breslau said he and his colleagues also found only limited benefits in quality of care for physical health conditions associated with the grant program. Still, he remains hopeful.
“There is some potential here,” Dr. Breslau said. “Sometimes, we are seeing positive effects, but it’s certainly not a slam-dunk.”
He noted that opening a new setting for primary care services could strain a workforce that’s already in short supply. In addition, he said, attempts to fold primary care services into mental health programs could, in some cases, result in replication of care of chronic conditions for certain patients with SMI.
We “may still not reach that portion of the target population that has the greatest need,” Dr. Breslau said in an interview. “The new services may turn out to be duplicative rather than filling a gap.”
In another session, Gail L. Daumit, MD, MHS, of Johns Hopkins University, Baltimore, discussed her plan to build on a past success in helping people with SMI lose weight.
In the ACHIEVE (Achieving Healthy Lifestyles In Psych Rehabilitation) trial, Dr. Daumit and her colleagues found that people enrolled in an intervention group lost an average of 3.2 kg more than did a control group after 18 months (N Engl J Med. 2013;368:1594-602). The intervention steps included alternating group and individual weight management sessions, on-site group physical activity three times weekly, and weigh-ins. The study had 291 patients who were randomized between the control and intervention groups.
that can be used more broadly. She’s seeking to scale up effective interventions to address cardiovascular risk factors in people with SMI.
“Our goal is not just to get process-of-care measures like ‘counseling was delivered,’ ‘a medicine was started,’ but to actually show impact on health outcomes,” Dr. Daumit said.
Risks tied to antipsychotics
People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.
Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.
“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.
A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.
Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.
Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.
When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”
Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.
ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.
The researchers discussed strategies aimed at combating obesity and diabetes, as well as behaviors such as smoking and sedentary lifestyle.
“One of the things that jumps out is the tremendous need for evidence-based strategies to address these physical health problems that are common in general population but even more of a burden for people with serious mental illness,” said Susan T. Azrin, PhD, of the NIMH, in an interview.
A study published in 2015, estimated that people with schizophrenia, for example, might lose almost 30 years of life because of premature death. Individuals with serious mental illness also experience elevated morbidity from cardiovascular disease and cancer. The NIMH and other federal agencies have in recent years looked for ways to help people with SMI quit smoking, and better control their weight and cholesterol.
But approaches that sound promising for boosting physical fitness in this group of patients have not always proven successful. Joshua Breslau, PhD, ScD, of the Rand Corp. discussed findings from a 2014 paper where he and his colleagues reported somewhat disappointing results from a study of federal Primary and Behavioral Health Care Integration grants.
The researchers matched clinics receiving this funding with similar ones that did not. They found that people with mental illness treated at the clinics receiving the grants showed improvements in some indicators of physical health (diastolic blood pressure, total cholesterol, LDL cholesterol, and fasting plasma glucose) but not in others (systolic blood pressure, body mass index, HDL cholesterol, hemoglobin A1c, triglycerides, self-reported smoking). Dr. Breslau said he and his colleagues also found only limited benefits in quality of care for physical health conditions associated with the grant program. Still, he remains hopeful.
“There is some potential here,” Dr. Breslau said. “Sometimes, we are seeing positive effects, but it’s certainly not a slam-dunk.”
He noted that opening a new setting for primary care services could strain a workforce that’s already in short supply. In addition, he said, attempts to fold primary care services into mental health programs could, in some cases, result in replication of care of chronic conditions for certain patients with SMI.
We “may still not reach that portion of the target population that has the greatest need,” Dr. Breslau said in an interview. “The new services may turn out to be duplicative rather than filling a gap.”
In another session, Gail L. Daumit, MD, MHS, of Johns Hopkins University, Baltimore, discussed her plan to build on a past success in helping people with SMI lose weight.
In the ACHIEVE (Achieving Healthy Lifestyles In Psych Rehabilitation) trial, Dr. Daumit and her colleagues found that people enrolled in an intervention group lost an average of 3.2 kg more than did a control group after 18 months (N Engl J Med. 2013;368:1594-602). The intervention steps included alternating group and individual weight management sessions, on-site group physical activity three times weekly, and weigh-ins. The study had 291 patients who were randomized between the control and intervention groups.
that can be used more broadly. She’s seeking to scale up effective interventions to address cardiovascular risk factors in people with SMI.
“Our goal is not just to get process-of-care measures like ‘counseling was delivered,’ ‘a medicine was started,’ but to actually show impact on health outcomes,” Dr. Daumit said.
Risks tied to antipsychotics
People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.
Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.
“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.
A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.
Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.
Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.
When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”
Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.
ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.
The researchers discussed strategies aimed at combating obesity and diabetes, as well as behaviors such as smoking and sedentary lifestyle.
“One of the things that jumps out is the tremendous need for evidence-based strategies to address these physical health problems that are common in general population but even more of a burden for people with serious mental illness,” said Susan T. Azrin, PhD, of the NIMH, in an interview.
A study published in 2015, estimated that people with schizophrenia, for example, might lose almost 30 years of life because of premature death. Individuals with serious mental illness also experience elevated morbidity from cardiovascular disease and cancer. The NIMH and other federal agencies have in recent years looked for ways to help people with SMI quit smoking, and better control their weight and cholesterol.
But approaches that sound promising for boosting physical fitness in this group of patients have not always proven successful. Joshua Breslau, PhD, ScD, of the Rand Corp. discussed findings from a 2014 paper where he and his colleagues reported somewhat disappointing results from a study of federal Primary and Behavioral Health Care Integration grants.
The researchers matched clinics receiving this funding with similar ones that did not. They found that people with mental illness treated at the clinics receiving the grants showed improvements in some indicators of physical health (diastolic blood pressure, total cholesterol, LDL cholesterol, and fasting plasma glucose) but not in others (systolic blood pressure, body mass index, HDL cholesterol, hemoglobin A1c, triglycerides, self-reported smoking). Dr. Breslau said he and his colleagues also found only limited benefits in quality of care for physical health conditions associated with the grant program. Still, he remains hopeful.
“There is some potential here,” Dr. Breslau said. “Sometimes, we are seeing positive effects, but it’s certainly not a slam-dunk.”
He noted that opening a new setting for primary care services could strain a workforce that’s already in short supply. In addition, he said, attempts to fold primary care services into mental health programs could, in some cases, result in replication of care of chronic conditions for certain patients with SMI.
We “may still not reach that portion of the target population that has the greatest need,” Dr. Breslau said in an interview. “The new services may turn out to be duplicative rather than filling a gap.”
In another session, Gail L. Daumit, MD, MHS, of Johns Hopkins University, Baltimore, discussed her plan to build on a past success in helping people with SMI lose weight.
In the ACHIEVE (Achieving Healthy Lifestyles In Psych Rehabilitation) trial, Dr. Daumit and her colleagues found that people enrolled in an intervention group lost an average of 3.2 kg more than did a control group after 18 months (N Engl J Med. 2013;368:1594-602). The intervention steps included alternating group and individual weight management sessions, on-site group physical activity three times weekly, and weigh-ins. The study had 291 patients who were randomized between the control and intervention groups.
that can be used more broadly. She’s seeking to scale up effective interventions to address cardiovascular risk factors in people with SMI.
“Our goal is not just to get process-of-care measures like ‘counseling was delivered,’ ‘a medicine was started,’ but to actually show impact on health outcomes,” Dr. Daumit said.
Risks tied to antipsychotics
People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.
Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.
“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.
A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.
Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.
Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.
When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”
Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.
REPORTING FROM AN NIMH CONFERENCE
For people with SMI, disclosure still challenging
ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.
The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.
One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.
Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).
People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.
Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.
“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.
An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.
Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”
“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”
Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.
About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.
“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”
Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).
She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.
“The 6th time was the charm with NIH,” Dr. Baldwin said.
An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.
“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”
Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.
Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.
“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.
Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.
ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.
The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.
One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.
Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).
People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.
Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.
“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.
An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.
Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”
“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”
Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.
About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.
“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”
Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).
She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.
“The 6th time was the charm with NIH,” Dr. Baldwin said.
An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.
“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”
Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.
Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.
“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.
Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.
ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.
The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.
One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.
Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).
People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.
Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.
“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.
An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.
Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”
“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”
Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.
About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.
“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”
Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).
She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.
“The 6th time was the charm with NIH,” Dr. Baldwin said.
An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.
“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”
Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.
Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.
“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.
Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.
EXPERT ANALYSIS FROM AN NIMH CONFERENCE
NIMH urged to shift priorities toward children’s mental health
ROCKVILLE, MD. – A researcher is calling for a major shift in funding and priorities within the National Institute of Mental Health to seize on ripe opportunities to better understand how social and environmental factors affect the development of children’s brains.
“This new agenda that I’m suggesting would prioritize child and family health over other populations,” Kimberly E. Hoagwood, PhD, of NYU Langone Health, said at a National Institute of Mental Health conference on mental health services research. “But if we want science to maximize the public health impact and we want our services implementation research to have the biggest impact, then I think we have to think about rebalancing the portfolio.”
Dr. Hoagwood made her argument during a well-received presentation at the meeting. In an interview afterward, Dr. Hoagwood confirmed that she’s advocating for a potential shifting of funds from basic neuroscience.
She previously argued a case for rebalancing priorities within mental health research in a paper published in the Journal of the American Academy of Child and Adolescent Psychiatry (2018 Jan;57[1]:10-3). In the paper, Dr. Hoagwood and her coauthors said the NIMH’s annual funding for child and adolescent services and intervention research decreased 42%, to $30.2 million, from fiscal 2005 to fiscal 2015.
“The NIMH made an explicit decision to invest in basic neuroscience in part because of concerns about the inadequacy of the diagnostic classification systems and limited understanding of the etiology of mental illness,” Dr. Hoagwood and her colleagues wrote in the paper. “This investment could well pay off in the future. However, at least 20% of children now suffer from mental health problems. They cannot be ignored.”
The share of NIMH’s annual budget dedicated to child and adolescent services and intervention research has hovered around 2%-3% in recent years, according to Dr. Hoagwood.
She argued that increased investment in child and adolescent services and intervention research is needed in part because of a flourishing atmosphere outside of the NIMH. At least two dozen notable initiatives looking at social and environmental factors are underway that could contribute greatly to the understanding of factors outside of genetics that influence early brain development, Dr. Hoagwood said.
Those initiatives include about a dozen Medicaid accountable care organizations that are identifying social risks such as poverty, homelessness, food insecurity, and unemployment. Efforts aimed at addressing the effects of poverty through the adoption of strategies such as living wage laws also are underway, she said.
Dr. Hoagwood highlighted the importance of work on the exposome, which the NIMH has described as a new approach to understanding the mechanisms by which environmental factors alter brain and behavior – starting from prenatal development. Dr. Hoagwood said this approach would collect samples to systematically monitor a range of broad-spectrum environmental exposures. She described it as the “complement to the genomic sequencing.”
She called for launching multisite studies to look at how such factors affect brain development, and where and how early interventions can improve children’s healthy development. Data from community efforts and some of those experiments might, at least initially, be “messy beyond belief,” she said.
“We have to not shy away from it. The genomic sequencing has not shied away from messy data,” Dr. Hoagwood said. “We don’t need to do that, either. We need new methods. We need small experiments of novel payment approaches. We need to use our data systems better.”
Dr. Hoagwood had no financial disclosures to report.
ROCKVILLE, MD. – A researcher is calling for a major shift in funding and priorities within the National Institute of Mental Health to seize on ripe opportunities to better understand how social and environmental factors affect the development of children’s brains.
“This new agenda that I’m suggesting would prioritize child and family health over other populations,” Kimberly E. Hoagwood, PhD, of NYU Langone Health, said at a National Institute of Mental Health conference on mental health services research. “But if we want science to maximize the public health impact and we want our services implementation research to have the biggest impact, then I think we have to think about rebalancing the portfolio.”
Dr. Hoagwood made her argument during a well-received presentation at the meeting. In an interview afterward, Dr. Hoagwood confirmed that she’s advocating for a potential shifting of funds from basic neuroscience.
She previously argued a case for rebalancing priorities within mental health research in a paper published in the Journal of the American Academy of Child and Adolescent Psychiatry (2018 Jan;57[1]:10-3). In the paper, Dr. Hoagwood and her coauthors said the NIMH’s annual funding for child and adolescent services and intervention research decreased 42%, to $30.2 million, from fiscal 2005 to fiscal 2015.
“The NIMH made an explicit decision to invest in basic neuroscience in part because of concerns about the inadequacy of the diagnostic classification systems and limited understanding of the etiology of mental illness,” Dr. Hoagwood and her colleagues wrote in the paper. “This investment could well pay off in the future. However, at least 20% of children now suffer from mental health problems. They cannot be ignored.”
The share of NIMH’s annual budget dedicated to child and adolescent services and intervention research has hovered around 2%-3% in recent years, according to Dr. Hoagwood.
She argued that increased investment in child and adolescent services and intervention research is needed in part because of a flourishing atmosphere outside of the NIMH. At least two dozen notable initiatives looking at social and environmental factors are underway that could contribute greatly to the understanding of factors outside of genetics that influence early brain development, Dr. Hoagwood said.
Those initiatives include about a dozen Medicaid accountable care organizations that are identifying social risks such as poverty, homelessness, food insecurity, and unemployment. Efforts aimed at addressing the effects of poverty through the adoption of strategies such as living wage laws also are underway, she said.
Dr. Hoagwood highlighted the importance of work on the exposome, which the NIMH has described as a new approach to understanding the mechanisms by which environmental factors alter brain and behavior – starting from prenatal development. Dr. Hoagwood said this approach would collect samples to systematically monitor a range of broad-spectrum environmental exposures. She described it as the “complement to the genomic sequencing.”
She called for launching multisite studies to look at how such factors affect brain development, and where and how early interventions can improve children’s healthy development. Data from community efforts and some of those experiments might, at least initially, be “messy beyond belief,” she said.
“We have to not shy away from it. The genomic sequencing has not shied away from messy data,” Dr. Hoagwood said. “We don’t need to do that, either. We need new methods. We need small experiments of novel payment approaches. We need to use our data systems better.”
Dr. Hoagwood had no financial disclosures to report.
ROCKVILLE, MD. – A researcher is calling for a major shift in funding and priorities within the National Institute of Mental Health to seize on ripe opportunities to better understand how social and environmental factors affect the development of children’s brains.
“This new agenda that I’m suggesting would prioritize child and family health over other populations,” Kimberly E. Hoagwood, PhD, of NYU Langone Health, said at a National Institute of Mental Health conference on mental health services research. “But if we want science to maximize the public health impact and we want our services implementation research to have the biggest impact, then I think we have to think about rebalancing the portfolio.”
Dr. Hoagwood made her argument during a well-received presentation at the meeting. In an interview afterward, Dr. Hoagwood confirmed that she’s advocating for a potential shifting of funds from basic neuroscience.
She previously argued a case for rebalancing priorities within mental health research in a paper published in the Journal of the American Academy of Child and Adolescent Psychiatry (2018 Jan;57[1]:10-3). In the paper, Dr. Hoagwood and her coauthors said the NIMH’s annual funding for child and adolescent services and intervention research decreased 42%, to $30.2 million, from fiscal 2005 to fiscal 2015.
“The NIMH made an explicit decision to invest in basic neuroscience in part because of concerns about the inadequacy of the diagnostic classification systems and limited understanding of the etiology of mental illness,” Dr. Hoagwood and her colleagues wrote in the paper. “This investment could well pay off in the future. However, at least 20% of children now suffer from mental health problems. They cannot be ignored.”
The share of NIMH’s annual budget dedicated to child and adolescent services and intervention research has hovered around 2%-3% in recent years, according to Dr. Hoagwood.
She argued that increased investment in child and adolescent services and intervention research is needed in part because of a flourishing atmosphere outside of the NIMH. At least two dozen notable initiatives looking at social and environmental factors are underway that could contribute greatly to the understanding of factors outside of genetics that influence early brain development, Dr. Hoagwood said.
Those initiatives include about a dozen Medicaid accountable care organizations that are identifying social risks such as poverty, homelessness, food insecurity, and unemployment. Efforts aimed at addressing the effects of poverty through the adoption of strategies such as living wage laws also are underway, she said.
Dr. Hoagwood highlighted the importance of work on the exposome, which the NIMH has described as a new approach to understanding the mechanisms by which environmental factors alter brain and behavior – starting from prenatal development. Dr. Hoagwood said this approach would collect samples to systematically monitor a range of broad-spectrum environmental exposures. She described it as the “complement to the genomic sequencing.”
She called for launching multisite studies to look at how such factors affect brain development, and where and how early interventions can improve children’s healthy development. Data from community efforts and some of those experiments might, at least initially, be “messy beyond belief,” she said.
“We have to not shy away from it. The genomic sequencing has not shied away from messy data,” Dr. Hoagwood said. “We don’t need to do that, either. We need new methods. We need small experiments of novel payment approaches. We need to use our data systems better.”
Dr. Hoagwood had no financial disclosures to report.
REPORTING FROM AN NIMH CONFERENCE
Groups release guidelines for CAR T treatment in children
emphasize the need for a flexible approach to detect early signs of serious complications for younger patients treated with this emerging class of medicines.
Researchers at the University of Texas MD Anderson Cancer Center, Houston, and the Pediatric Acute Lung Injury and Sepsis Investigators Network (PALISI) developed the guidelines, which were published in Nature Reviews Clinical Oncology. The recommendations build on the guidelines for more general use of these medicines from MD Anderson’s CARTOX Program, which Nature Reviews Clinical Oncology published in 2017.
Among the chief concerns with this new class of medicines are cytokine-release syndrome (CRS) and CAR T cell-related encephalopathy syndrome (CRES), according to Kris Michael Mahadeo, MD MPH, of the MD Anderson Cancer Center and his coauthors of the new paper.
Some of the tools used for older patients in screening for complications with CAR T drugs don’t work as well with younger ones, Dr. Mahadeo said in an interview. For instance, at MD Anderson, a handwriting sample is used to monitor patients for CAR T cell-related encephalopathy syndrome, which has symptoms of confusion and delirium. Patients provide a baseline handwriting sample of a single sentence that’s scanned into the medical record, and then they are asked to write this again during their time in the hospital, he said. But this tool may not work for children too young to write well.
The new guidelines suggest using the Cornell Assessment of Pediatric Delirium (CAPD) or to evaluate a child’s mental state, asking questions about eye contact, and level of awareness and mood, Dr. Mahadeo said. An alternative for patients aged 12 years and older with greater cognitive ability is the CARTOX-10 grading system.
“The nurses who spent most of the day with these patients will observe them over their shift and kind of get an idea of what was normal and answer a series of questions” through the CAPD tool, which is already used in ICUs, Dr. Mahadeo said. “It takes into consideration both the nurses’ perception and the parents, or whoever is at the bedside with the child. So that if they have a concern, it gives them a point that actually escalates things upward.”
The newly published recommendations also remind physicians and others caring for young patients to pay attention to these reports.
“Parent and/or caregiver concerns should be addressed because early signs or symptoms of CRS can be subtle and best recognized by those who know the child best,” Dr. Mahadeo and his colleagues wrote in a summary of key recommendations in the paper.
The recommendations also noted a need for close monitoring for complications such as hypotension, hypocalcemia, and catheter-related pain in young patients who require a leukapheresis catheter for cell collection. Infant and younger children “might not verbalize these symptoms,” according to the researchers.
Other recommendations include:
- Obtaining the child’s assent when appropriate, with psychological services often aiding in this goal. Dr. Mahadeo and his colleagues recommend considering “age-appropriate advance directives.”
- Maintaining high vigilance for sinus tachycardia as an early sign of CRS, using age-specific normal range or baseline values.
- Giving pediatric dosing of tocilizumab, with patients weighing less than 30 kg receiving 12 mg/kg, and those weighing 30 kg or greater receiving 8 mg/kg.
- Considering participation with a prospective collaboration with intensive-care registries that could allow accurate data entry of cell-therapy variables into the Center for International Blood and Marrow Transplant Research registry by cell-therapy programs.
The Food and Drug Administration approved the first two CAR T-cell therapies in the United States in 2017: Novartis’ tisagenlecleucel (Kymriah) for children and young adults with B-cell precursor acute lymphoblastic leukemia and later for adults with large B-cell lymphoma; and axicabtagene ciloleucel (Yescarta), sold by Gilead, for adults with large B-cell lymphoma. The therapies involve reengineering a patient’s T cells such that they recognize the threat of cancer, and then introducing them back into the body. The European Medicines Agency’s Committee for Medicinal Products for Human Use in June recommended granting marketing authorization to these drugs.
In the new pediatric guidelines, Dr. Mahadeo and his colleagues noted the use of CAR T-cell therapies for treatment of solid tumors and other malignancies in children already “is being explored.” “Moreover, consideration of earlier or upfront use of CAR T-cell therapy might spare patients the acute and long-term toxicities associated with traditional chemotherapy and/or radiation regimens,” they wrote.
There’s been great interest in learning how to most safely use the CAR T cell therapies, said Helen Heslop, MD, of Baylor College of Medicine.
She pointed to a 2014 publication in the journal Blood from Daniel W. Lee and his colleagues as an earlier example of this research. By now, cancer centers will have worked out their own procedures for pediatric use of CAR T therapies, hewing to standards set by the Foundation for the Accreditation of Cellular Therapy (FACT), Dr. Heslop said.
Dr. Heslop also stressed the role of the FDA in requiring risk evaluation and management strategy programs for these drugs. All of this, including the new guidelines from Dr. Mahadeo and his colleagues, is part of a growing body of research into safe use of CAR T therapies, Dr. Heslop said.
“It’s an active area of research,” she said. “Most centers will look at all of it and then develop what works best in their own individual center for providing the best care for the patients.”
The newly published guidelines could prove an “important contribution” to managing the risk of CAR T therapies, Phyllis I. Warkentin, MD, chief medical officer for FACT, said in an interview, while stressing that they were not more or less important than other similar efforts. Physicians learning how to use the CAR T therapies may welcome new input, as most of what’s been published has been about adults, she said.
“You don’t have the luxury of a lot of time to be learning on the job, so to speak,” with CAR T therapies, she said. “Many of the toxicities are fairly severe and fairly sudden.”
Dr. Heslop has been on advisory board for Gilead and Novartis. Dr. Warkentin and Dr. Mahadeo each reported having no financial disclosures. Other authors of the guidelines paper reported a patent with applications in the field of gene-modified T cell therapy for cancer, as well as financial ties to Cellectis, NexImmune, Torque Pharma, Kite Pharma (a Gilead company), Poseida Therapeutics, Celgene, Novartis, and Unum Therapeutics.
SOURCE: Mahadeo KM et al. Nat Rev Clin Oncol. 2018 Aug 6. doi: 10.1038/s41571-018-0075-2.
emphasize the need for a flexible approach to detect early signs of serious complications for younger patients treated with this emerging class of medicines.
Researchers at the University of Texas MD Anderson Cancer Center, Houston, and the Pediatric Acute Lung Injury and Sepsis Investigators Network (PALISI) developed the guidelines, which were published in Nature Reviews Clinical Oncology. The recommendations build on the guidelines for more general use of these medicines from MD Anderson’s CARTOX Program, which Nature Reviews Clinical Oncology published in 2017.
Among the chief concerns with this new class of medicines are cytokine-release syndrome (CRS) and CAR T cell-related encephalopathy syndrome (CRES), according to Kris Michael Mahadeo, MD MPH, of the MD Anderson Cancer Center and his coauthors of the new paper.
Some of the tools used for older patients in screening for complications with CAR T drugs don’t work as well with younger ones, Dr. Mahadeo said in an interview. For instance, at MD Anderson, a handwriting sample is used to monitor patients for CAR T cell-related encephalopathy syndrome, which has symptoms of confusion and delirium. Patients provide a baseline handwriting sample of a single sentence that’s scanned into the medical record, and then they are asked to write this again during their time in the hospital, he said. But this tool may not work for children too young to write well.
The new guidelines suggest using the Cornell Assessment of Pediatric Delirium (CAPD) or to evaluate a child’s mental state, asking questions about eye contact, and level of awareness and mood, Dr. Mahadeo said. An alternative for patients aged 12 years and older with greater cognitive ability is the CARTOX-10 grading system.
“The nurses who spent most of the day with these patients will observe them over their shift and kind of get an idea of what was normal and answer a series of questions” through the CAPD tool, which is already used in ICUs, Dr. Mahadeo said. “It takes into consideration both the nurses’ perception and the parents, or whoever is at the bedside with the child. So that if they have a concern, it gives them a point that actually escalates things upward.”
The newly published recommendations also remind physicians and others caring for young patients to pay attention to these reports.
“Parent and/or caregiver concerns should be addressed because early signs or symptoms of CRS can be subtle and best recognized by those who know the child best,” Dr. Mahadeo and his colleagues wrote in a summary of key recommendations in the paper.
The recommendations also noted a need for close monitoring for complications such as hypotension, hypocalcemia, and catheter-related pain in young patients who require a leukapheresis catheter for cell collection. Infant and younger children “might not verbalize these symptoms,” according to the researchers.
Other recommendations include:
- Obtaining the child’s assent when appropriate, with psychological services often aiding in this goal. Dr. Mahadeo and his colleagues recommend considering “age-appropriate advance directives.”
- Maintaining high vigilance for sinus tachycardia as an early sign of CRS, using age-specific normal range or baseline values.
- Giving pediatric dosing of tocilizumab, with patients weighing less than 30 kg receiving 12 mg/kg, and those weighing 30 kg or greater receiving 8 mg/kg.
- Considering participation with a prospective collaboration with intensive-care registries that could allow accurate data entry of cell-therapy variables into the Center for International Blood and Marrow Transplant Research registry by cell-therapy programs.
The Food and Drug Administration approved the first two CAR T-cell therapies in the United States in 2017: Novartis’ tisagenlecleucel (Kymriah) for children and young adults with B-cell precursor acute lymphoblastic leukemia and later for adults with large B-cell lymphoma; and axicabtagene ciloleucel (Yescarta), sold by Gilead, for adults with large B-cell lymphoma. The therapies involve reengineering a patient’s T cells such that they recognize the threat of cancer, and then introducing them back into the body. The European Medicines Agency’s Committee for Medicinal Products for Human Use in June recommended granting marketing authorization to these drugs.
In the new pediatric guidelines, Dr. Mahadeo and his colleagues noted the use of CAR T-cell therapies for treatment of solid tumors and other malignancies in children already “is being explored.” “Moreover, consideration of earlier or upfront use of CAR T-cell therapy might spare patients the acute and long-term toxicities associated with traditional chemotherapy and/or radiation regimens,” they wrote.
There’s been great interest in learning how to most safely use the CAR T cell therapies, said Helen Heslop, MD, of Baylor College of Medicine.
She pointed to a 2014 publication in the journal Blood from Daniel W. Lee and his colleagues as an earlier example of this research. By now, cancer centers will have worked out their own procedures for pediatric use of CAR T therapies, hewing to standards set by the Foundation for the Accreditation of Cellular Therapy (FACT), Dr. Heslop said.
Dr. Heslop also stressed the role of the FDA in requiring risk evaluation and management strategy programs for these drugs. All of this, including the new guidelines from Dr. Mahadeo and his colleagues, is part of a growing body of research into safe use of CAR T therapies, Dr. Heslop said.
“It’s an active area of research,” she said. “Most centers will look at all of it and then develop what works best in their own individual center for providing the best care for the patients.”
The newly published guidelines could prove an “important contribution” to managing the risk of CAR T therapies, Phyllis I. Warkentin, MD, chief medical officer for FACT, said in an interview, while stressing that they were not more or less important than other similar efforts. Physicians learning how to use the CAR T therapies may welcome new input, as most of what’s been published has been about adults, she said.
“You don’t have the luxury of a lot of time to be learning on the job, so to speak,” with CAR T therapies, she said. “Many of the toxicities are fairly severe and fairly sudden.”
Dr. Heslop has been on advisory board for Gilead and Novartis. Dr. Warkentin and Dr. Mahadeo each reported having no financial disclosures. Other authors of the guidelines paper reported a patent with applications in the field of gene-modified T cell therapy for cancer, as well as financial ties to Cellectis, NexImmune, Torque Pharma, Kite Pharma (a Gilead company), Poseida Therapeutics, Celgene, Novartis, and Unum Therapeutics.
SOURCE: Mahadeo KM et al. Nat Rev Clin Oncol. 2018 Aug 6. doi: 10.1038/s41571-018-0075-2.
emphasize the need for a flexible approach to detect early signs of serious complications for younger patients treated with this emerging class of medicines.
Researchers at the University of Texas MD Anderson Cancer Center, Houston, and the Pediatric Acute Lung Injury and Sepsis Investigators Network (PALISI) developed the guidelines, which were published in Nature Reviews Clinical Oncology. The recommendations build on the guidelines for more general use of these medicines from MD Anderson’s CARTOX Program, which Nature Reviews Clinical Oncology published in 2017.
Among the chief concerns with this new class of medicines are cytokine-release syndrome (CRS) and CAR T cell-related encephalopathy syndrome (CRES), according to Kris Michael Mahadeo, MD MPH, of the MD Anderson Cancer Center and his coauthors of the new paper.
Some of the tools used for older patients in screening for complications with CAR T drugs don’t work as well with younger ones, Dr. Mahadeo said in an interview. For instance, at MD Anderson, a handwriting sample is used to monitor patients for CAR T cell-related encephalopathy syndrome, which has symptoms of confusion and delirium. Patients provide a baseline handwriting sample of a single sentence that’s scanned into the medical record, and then they are asked to write this again during their time in the hospital, he said. But this tool may not work for children too young to write well.
The new guidelines suggest using the Cornell Assessment of Pediatric Delirium (CAPD) or to evaluate a child’s mental state, asking questions about eye contact, and level of awareness and mood, Dr. Mahadeo said. An alternative for patients aged 12 years and older with greater cognitive ability is the CARTOX-10 grading system.
“The nurses who spent most of the day with these patients will observe them over their shift and kind of get an idea of what was normal and answer a series of questions” through the CAPD tool, which is already used in ICUs, Dr. Mahadeo said. “It takes into consideration both the nurses’ perception and the parents, or whoever is at the bedside with the child. So that if they have a concern, it gives them a point that actually escalates things upward.”
The newly published recommendations also remind physicians and others caring for young patients to pay attention to these reports.
“Parent and/or caregiver concerns should be addressed because early signs or symptoms of CRS can be subtle and best recognized by those who know the child best,” Dr. Mahadeo and his colleagues wrote in a summary of key recommendations in the paper.
The recommendations also noted a need for close monitoring for complications such as hypotension, hypocalcemia, and catheter-related pain in young patients who require a leukapheresis catheter for cell collection. Infant and younger children “might not verbalize these symptoms,” according to the researchers.
Other recommendations include:
- Obtaining the child’s assent when appropriate, with psychological services often aiding in this goal. Dr. Mahadeo and his colleagues recommend considering “age-appropriate advance directives.”
- Maintaining high vigilance for sinus tachycardia as an early sign of CRS, using age-specific normal range or baseline values.
- Giving pediatric dosing of tocilizumab, with patients weighing less than 30 kg receiving 12 mg/kg, and those weighing 30 kg or greater receiving 8 mg/kg.
- Considering participation with a prospective collaboration with intensive-care registries that could allow accurate data entry of cell-therapy variables into the Center for International Blood and Marrow Transplant Research registry by cell-therapy programs.
The Food and Drug Administration approved the first two CAR T-cell therapies in the United States in 2017: Novartis’ tisagenlecleucel (Kymriah) for children and young adults with B-cell precursor acute lymphoblastic leukemia and later for adults with large B-cell lymphoma; and axicabtagene ciloleucel (Yescarta), sold by Gilead, for adults with large B-cell lymphoma. The therapies involve reengineering a patient’s T cells such that they recognize the threat of cancer, and then introducing them back into the body. The European Medicines Agency’s Committee for Medicinal Products for Human Use in June recommended granting marketing authorization to these drugs.
In the new pediatric guidelines, Dr. Mahadeo and his colleagues noted the use of CAR T-cell therapies for treatment of solid tumors and other malignancies in children already “is being explored.” “Moreover, consideration of earlier or upfront use of CAR T-cell therapy might spare patients the acute and long-term toxicities associated with traditional chemotherapy and/or radiation regimens,” they wrote.
There’s been great interest in learning how to most safely use the CAR T cell therapies, said Helen Heslop, MD, of Baylor College of Medicine.
She pointed to a 2014 publication in the journal Blood from Daniel W. Lee and his colleagues as an earlier example of this research. By now, cancer centers will have worked out their own procedures for pediatric use of CAR T therapies, hewing to standards set by the Foundation for the Accreditation of Cellular Therapy (FACT), Dr. Heslop said.
Dr. Heslop also stressed the role of the FDA in requiring risk evaluation and management strategy programs for these drugs. All of this, including the new guidelines from Dr. Mahadeo and his colleagues, is part of a growing body of research into safe use of CAR T therapies, Dr. Heslop said.
“It’s an active area of research,” she said. “Most centers will look at all of it and then develop what works best in their own individual center for providing the best care for the patients.”
The newly published guidelines could prove an “important contribution” to managing the risk of CAR T therapies, Phyllis I. Warkentin, MD, chief medical officer for FACT, said in an interview, while stressing that they were not more or less important than other similar efforts. Physicians learning how to use the CAR T therapies may welcome new input, as most of what’s been published has been about adults, she said.
“You don’t have the luxury of a lot of time to be learning on the job, so to speak,” with CAR T therapies, she said. “Many of the toxicities are fairly severe and fairly sudden.”
Dr. Heslop has been on advisory board for Gilead and Novartis. Dr. Warkentin and Dr. Mahadeo each reported having no financial disclosures. Other authors of the guidelines paper reported a patent with applications in the field of gene-modified T cell therapy for cancer, as well as financial ties to Cellectis, NexImmune, Torque Pharma, Kite Pharma (a Gilead company), Poseida Therapeutics, Celgene, Novartis, and Unum Therapeutics.
SOURCE: Mahadeo KM et al. Nat Rev Clin Oncol. 2018 Aug 6. doi: 10.1038/s41571-018-0075-2.
FROM NATURE REVIEWS CLINICAL ONCOLOGY
Key clinical point: Multidisciplinary approach aids in managing CAR T-cell therapy’s severe potential toxicities in children.
Major finding: The guideline calls for pediatric dosing of tocilizumab, with patients weighing less than 30 kg receiving 12 mg/kg, and those weighing 30 kg or greater receiving 8 mg/kg.
Study details: Consensus guidelines on the care of children receiving CAR T-cell therapy from the Pediatric Acute Lung Injury and Sepsis Investigators and the MD Anderson Cancer Center CARTOX program.
Disclosures: Dr. Mahadeo reported having no financial disclosures. Other coauthors reported a patent with applications in the field of gene-modified T cell therapy for cancer, as well as financial ties to Cellectis, NexImmune, Torque Pharma, Kite Pharma (a Gilead company), Poseida Therapeutics, Celgene, Novartis, and Unum Therapeutics.
Source: Mahadeo KM et al. Nat Rev Clin Oncol. 2018 Aug 6. doi: 10.1038/s41571-018-0075-2.
Judge seeks replication of efforts to support people with SMI
Florida’s Miami-Dade County reportedly has the largest percentage of residents with serious mental illnesses (SMI) among large U.S. communities. And a Florida judge who helped develop approaches aimed at sparing his state’s residents with mental illness from harmful, avoidable, and expensive bouts of prison time wants to see his strategies replicated.
“There is something terribly wrong with a society that is willing to spend more money to incarcerate people who are ill than to treat them,” Judge Steve Leifman of the 11th judicial circuit court said at a National Institute of Mental Health conference on mental health services research.
Judge Leifman in 2000 created the Criminal Mental Health Project. It’s been recognized for its success in keeping people with SMI from becoming ensnared in the criminal justice system because of minor offenses. It also helps those who do spend time in jail from returning.
In Miami-Dade County, for example, 97 people were a significant driver of costs in the criminal justice system in a study that was completed in 2010, Judge Leifman said. The members of this group were largely men who suffered from schizophrenia spectrum disorders and were homeless with a co-occurring disorder. Combined, the number of arrests for this group was about 2,200 over a 5-year period, Judge Leifman said. They spent 27,000 days in the Miami-Dade County jail – costing taxpayers about $13.7 million.
Judge Leifman said. “They would have had a shot at an education. They would have had housing. They probably would have done a lot better.”
Through the Criminal Mental Health Project, Judge Leifman and his colleagues seek to both prevent people with mental illness from being arrested and jailed for minor offenses, and to provide a support network for those who have reached jail. The project’s “prebooking diversion” efforts are built on a model developed in Memphis, Tenn., in the late 1980s. Through it, police officers get special training in recognizing mental illness and resolving crises in which people who have these disorders are involved.
The project’s “postbooking diversion” techniques require participants to voluntarily consent to mental health treatment and services. The program is open only to those less serious felonies, which can include drug charges and theft. Through participation in the Criminal Mental Health Project, people can have charges dismissed or reduced. The program provides them with connections to community-based treatment, support, and housing services, according to its website.
Participants in the program who were charged with minor felonies had 75% fewer jail bookings and jail days after enrolling in the Criminal Mental Health Project (N Engl J Med. 2016;374:1701-3).
Judge Leifman said the postbooking jail diversion program has, since 2001, served more than 4,000 individuals. Recidivism rates among participants charged with misdemeanors dropped from roughly 75% to 20%, he said.
Still, Judge Leifman describes his role as a judge as making him a “gatekeeper to the largest psychiatric facility in Florida – the Miami-Dade County Jail.” The jail houses about 1,200 people with serious mental illness on any given day, according to the Criminal Mental Health Project’s website.
Judge Leifman said that, ultimately, he wants more communities to devote more resources to providing medical care for people with mental illness.
Florida’s Miami-Dade County reportedly has the largest percentage of residents with serious mental illnesses (SMI) among large U.S. communities. And a Florida judge who helped develop approaches aimed at sparing his state’s residents with mental illness from harmful, avoidable, and expensive bouts of prison time wants to see his strategies replicated.
“There is something terribly wrong with a society that is willing to spend more money to incarcerate people who are ill than to treat them,” Judge Steve Leifman of the 11th judicial circuit court said at a National Institute of Mental Health conference on mental health services research.
Judge Leifman in 2000 created the Criminal Mental Health Project. It’s been recognized for its success in keeping people with SMI from becoming ensnared in the criminal justice system because of minor offenses. It also helps those who do spend time in jail from returning.
In Miami-Dade County, for example, 97 people were a significant driver of costs in the criminal justice system in a study that was completed in 2010, Judge Leifman said. The members of this group were largely men who suffered from schizophrenia spectrum disorders and were homeless with a co-occurring disorder. Combined, the number of arrests for this group was about 2,200 over a 5-year period, Judge Leifman said. They spent 27,000 days in the Miami-Dade County jail – costing taxpayers about $13.7 million.
Judge Leifman said. “They would have had a shot at an education. They would have had housing. They probably would have done a lot better.”
Through the Criminal Mental Health Project, Judge Leifman and his colleagues seek to both prevent people with mental illness from being arrested and jailed for minor offenses, and to provide a support network for those who have reached jail. The project’s “prebooking diversion” efforts are built on a model developed in Memphis, Tenn., in the late 1980s. Through it, police officers get special training in recognizing mental illness and resolving crises in which people who have these disorders are involved.
The project’s “postbooking diversion” techniques require participants to voluntarily consent to mental health treatment and services. The program is open only to those less serious felonies, which can include drug charges and theft. Through participation in the Criminal Mental Health Project, people can have charges dismissed or reduced. The program provides them with connections to community-based treatment, support, and housing services, according to its website.
Participants in the program who were charged with minor felonies had 75% fewer jail bookings and jail days after enrolling in the Criminal Mental Health Project (N Engl J Med. 2016;374:1701-3).
Judge Leifman said the postbooking jail diversion program has, since 2001, served more than 4,000 individuals. Recidivism rates among participants charged with misdemeanors dropped from roughly 75% to 20%, he said.
Still, Judge Leifman describes his role as a judge as making him a “gatekeeper to the largest psychiatric facility in Florida – the Miami-Dade County Jail.” The jail houses about 1,200 people with serious mental illness on any given day, according to the Criminal Mental Health Project’s website.
Judge Leifman said that, ultimately, he wants more communities to devote more resources to providing medical care for people with mental illness.
Florida’s Miami-Dade County reportedly has the largest percentage of residents with serious mental illnesses (SMI) among large U.S. communities. And a Florida judge who helped develop approaches aimed at sparing his state’s residents with mental illness from harmful, avoidable, and expensive bouts of prison time wants to see his strategies replicated.
“There is something terribly wrong with a society that is willing to spend more money to incarcerate people who are ill than to treat them,” Judge Steve Leifman of the 11th judicial circuit court said at a National Institute of Mental Health conference on mental health services research.
Judge Leifman in 2000 created the Criminal Mental Health Project. It’s been recognized for its success in keeping people with SMI from becoming ensnared in the criminal justice system because of minor offenses. It also helps those who do spend time in jail from returning.
In Miami-Dade County, for example, 97 people were a significant driver of costs in the criminal justice system in a study that was completed in 2010, Judge Leifman said. The members of this group were largely men who suffered from schizophrenia spectrum disorders and were homeless with a co-occurring disorder. Combined, the number of arrests for this group was about 2,200 over a 5-year period, Judge Leifman said. They spent 27,000 days in the Miami-Dade County jail – costing taxpayers about $13.7 million.
Judge Leifman said. “They would have had a shot at an education. They would have had housing. They probably would have done a lot better.”
Through the Criminal Mental Health Project, Judge Leifman and his colleagues seek to both prevent people with mental illness from being arrested and jailed for minor offenses, and to provide a support network for those who have reached jail. The project’s “prebooking diversion” efforts are built on a model developed in Memphis, Tenn., in the late 1980s. Through it, police officers get special training in recognizing mental illness and resolving crises in which people who have these disorders are involved.
The project’s “postbooking diversion” techniques require participants to voluntarily consent to mental health treatment and services. The program is open only to those less serious felonies, which can include drug charges and theft. Through participation in the Criminal Mental Health Project, people can have charges dismissed or reduced. The program provides them with connections to community-based treatment, support, and housing services, according to its website.
Participants in the program who were charged with minor felonies had 75% fewer jail bookings and jail days after enrolling in the Criminal Mental Health Project (N Engl J Med. 2016;374:1701-3).
Judge Leifman said the postbooking jail diversion program has, since 2001, served more than 4,000 individuals. Recidivism rates among participants charged with misdemeanors dropped from roughly 75% to 20%, he said.
Still, Judge Leifman describes his role as a judge as making him a “gatekeeper to the largest psychiatric facility in Florida – the Miami-Dade County Jail.” The jail houses about 1,200 people with serious mental illness on any given day, according to the Criminal Mental Health Project’s website.
Judge Leifman said that, ultimately, he wants more communities to devote more resources to providing medical care for people with mental illness.
REPORTING FROM AN NIMH CONFERENCE