Pediatrics

ORLANDO – Reviewing patient charts for asthma risk factors using natural language processing can be done 8 times faster than reviewing the charts by hand, and with high levels of accuracy, researchers reported here.

Thomas R. Collins/Frontline Medical News

Natural language processing (NLP) is a kind of artificial intelligence in which computers are “trained” through a reiterative process to understand human language.

Researchers at Mayo Clinic previously have shown that a program created in-house can successfully and quickly determine patients’ asthma status. In this study, they turned to assessment of asthma risk factors, Chung-Il Wi, MD, assistant professor of pediatrics at Mayo said in a presentation at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

They used a convenience sample of 177 patient charts to train the NLP system. The system extracted – from key terms and sentences in the electronic health record (EHR) – data such as breastfeeding history and history of atopic conditions such as allergic rhinitis, eczema, and food allergy. From parent charts, the system extracted terms related to family history of asthma and other atopic conditions. The performance of the NLP algorithm was assessed by comparison with results of a manual chart review in a test cohort of 220 patient charts.

 

Researchers found a high level of agreement between the NLP analysis and the manual review. For breastfeeding, the positive predictive value (PPV) of the NLP was 98% and the negative predictive value (NPV) was 86%. For history of atopic conditions the PPV was at or near 100%, with a NPV of 97% to 99%, depending on the condition.

For family history of atopic conditions, the PPV was 91% to 100%, depending on the condition, and the NPV was 96% to 99%.

“Childhood asthma risk factors identified (an) NLP algorithm using EHR has excellent concordance with chart review,” researchers wrote.

Using an average time per chart, researchers found that it would take 7 hours to complete a manual review for the information presented in the study, compared to 50 minutes for the NLP.

 

The findings, thought to be the first demonstrating NLP’s value for this purpose, suggest “the huge potential of leveraging NLP for asthma care and research,” researchers said.

Dr. Wi said the system can be applied to any EHR system. He said it only makes sense to put an algorithm to use in this way – it saves both clinical time and time in doing research projects.

“Whenever we do asthma research we need to collect asthma risk factors anyway, but we don’t want to do manual chart review anymore in this EMR era,” he said. “Now, the computer can do it.”

SOURCE: Wi C AAAAI/WAO Joint Congress 2018 abstract 637.

ORLANDO – Reviewing patient charts for asthma risk factors using natural language processing can be done 8 times faster than reviewing the charts by hand, and with high levels of accuracy, researchers reported here.

Thomas R. Collins/Frontline Medical News

Natural language processing (NLP) is a kind of artificial intelligence in which computers are “trained” through a reiterative process to understand human language.

Researchers at Mayo Clinic previously have shown that a program created in-house can successfully and quickly determine patients’ asthma status. In this study, they turned to assessment of asthma risk factors, Chung-Il Wi, MD, assistant professor of pediatrics at Mayo said in a presentation at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

They used a convenience sample of 177 patient charts to train the NLP system. The system extracted – from key terms and sentences in the electronic health record (EHR) – data such as breastfeeding history and history of atopic conditions such as allergic rhinitis, eczema, and food allergy. From parent charts, the system extracted terms related to family history of asthma and other atopic conditions. The performance of the NLP algorithm was assessed by comparison with results of a manual chart review in a test cohort of 220 patient charts.

 

Researchers found a high level of agreement between the NLP analysis and the manual review. For breastfeeding, the positive predictive value (PPV) of the NLP was 98% and the negative predictive value (NPV) was 86%. For history of atopic conditions the PPV was at or near 100%, with a NPV of 97% to 99%, depending on the condition.

For family history of atopic conditions, the PPV was 91% to 100%, depending on the condition, and the NPV was 96% to 99%.

“Childhood asthma risk factors identified (an) NLP algorithm using EHR has excellent concordance with chart review,” researchers wrote.

Using an average time per chart, researchers found that it would take 7 hours to complete a manual review for the information presented in the study, compared to 50 minutes for the NLP.

 

The findings, thought to be the first demonstrating NLP’s value for this purpose, suggest “the huge potential of leveraging NLP for asthma care and research,” researchers said.

Dr. Wi said the system can be applied to any EHR system. He said it only makes sense to put an algorithm to use in this way – it saves both clinical time and time in doing research projects.

“Whenever we do asthma research we need to collect asthma risk factors anyway, but we don’t want to do manual chart review anymore in this EMR era,” he said. “Now, the computer can do it.”

SOURCE: Wi C AAAAI/WAO Joint Congress 2018 abstract 637.

ORLANDO – Reviewing patient charts for asthma risk factors using natural language processing can be done 8 times faster than reviewing the charts by hand, and with high levels of accuracy, researchers reported here.

Thomas R. Collins/Frontline Medical News

Natural language processing (NLP) is a kind of artificial intelligence in which computers are “trained” through a reiterative process to understand human language.

Researchers at Mayo Clinic previously have shown that a program created in-house can successfully and quickly determine patients’ asthma status. In this study, they turned to assessment of asthma risk factors, Chung-Il Wi, MD, assistant professor of pediatrics at Mayo said in a presentation at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

They used a convenience sample of 177 patient charts to train the NLP system. The system extracted – from key terms and sentences in the electronic health record (EHR) – data such as breastfeeding history and history of atopic conditions such as allergic rhinitis, eczema, and food allergy. From parent charts, the system extracted terms related to family history of asthma and other atopic conditions. The performance of the NLP algorithm was assessed by comparison with results of a manual chart review in a test cohort of 220 patient charts.

 

Researchers found a high level of agreement between the NLP analysis and the manual review. For breastfeeding, the positive predictive value (PPV) of the NLP was 98% and the negative predictive value (NPV) was 86%. For history of atopic conditions the PPV was at or near 100%, with a NPV of 97% to 99%, depending on the condition.

For family history of atopic conditions, the PPV was 91% to 100%, depending on the condition, and the NPV was 96% to 99%.

“Childhood asthma risk factors identified (an) NLP algorithm using EHR has excellent concordance with chart review,” researchers wrote.

Using an average time per chart, researchers found that it would take 7 hours to complete a manual review for the information presented in the study, compared to 50 minutes for the NLP.

 

The findings, thought to be the first demonstrating NLP’s value for this purpose, suggest “the huge potential of leveraging NLP for asthma care and research,” researchers said.

Dr. Wi said the system can be applied to any EHR system. He said it only makes sense to put an algorithm to use in this way – it saves both clinical time and time in doing research projects.

“Whenever we do asthma research we need to collect asthma risk factors anyway, but we don’t want to do manual chart review anymore in this EMR era,” he said. “Now, the computer can do it.”

SOURCE: Wi C AAAAI/WAO Joint Congress 2018 abstract 637.

 

ORLANDO – At schools in a Texas district, unassigned epinephrine injectors were used more often than assigned ones were, according to results of an analysis.

In the 2015-2016 and 2016-2017 school years, epinephrine was administered to students 31 times at schools in the Austin Independent School District, which began to stock unassigned epinephrine in its schools after state legislators approved a law in 2015 allowing, but not requiring, school districts to do so. In 21 of those cases, or 68% of the time, it was the unassigned stockpile that was used, Kathryn Neupert, MD, said at the joint congress of the American Academy of Allergy, Asthma, and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical News

Dr. Neupert and her associates said the findings underscore the public health value of stocking unassigned epinephrine in schools. According to the Food Allergy Research and Education organization, all states but Hawaii had laws at least allowing unassigned epinephrine injectors to be stocked in schools as of 2016, with 12 states requiring it.

She drew attention to the finding that 22% of the time in the study, the epinephrine use involved the unassigned stock for children with no history of anaphylaxis or allergy.

 

“It kind of argues that, for those people that we don’t know have anaphylaxis to anything, it’s definitely important to have it available to them,” reported Dr. Neupert, a resident at the University of Texas, Austin.

In 45% of the cases, epinephrine was administered to children who had a known history of allergic reactions but, for whatever reason, did not have an epinephrine injector assigned to them.

Food was the most common trigger for use of epinephrine, accounting for 48% of the injections, with fragrance at 16%, insect stings at 10%, and exercise and overheating at 7%.

Dr. Neupert and her associates said the frequency with which epinephrine was administered after exposure to a fragrance – seen in about the same amount for unassigned and assigned epinephrine – might signal a need for greater education.

 

She emphasized the importance of follow-up after the use of unassigned injectors. If children get epinephrine that was unassigned and they have known allergies, “be sure that they subsequently get back in with their pediatrician or their allergist [and] that they’re getting their up-to-date epinephrine pens renewed,” she noted.

Carla Davis, MD, a pediatrician at Baylor College of Medicine, Houston, said she hopes more school districts adopt the stock epinephrine policy, despite barriers of cost and education. “I do think that it’s important for these schools to have these, because 20% of children will have their first episode of food allergic reaction – and in some of those cases it will be anaphylaxis – in schools.”

Dr. Neupert had no relevant financial disclosures.

SOURCE: AAAAI/WAO Joint Congress Abstract 465.

 

ORLANDO – At schools in a Texas district, unassigned epinephrine injectors were used more often than assigned ones were, according to results of an analysis.

In the 2015-2016 and 2016-2017 school years, epinephrine was administered to students 31 times at schools in the Austin Independent School District, which began to stock unassigned epinephrine in its schools after state legislators approved a law in 2015 allowing, but not requiring, school districts to do so. In 21 of those cases, or 68% of the time, it was the unassigned stockpile that was used, Kathryn Neupert, MD, said at the joint congress of the American Academy of Allergy, Asthma, and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical News

Dr. Neupert and her associates said the findings underscore the public health value of stocking unassigned epinephrine in schools. According to the Food Allergy Research and Education organization, all states but Hawaii had laws at least allowing unassigned epinephrine injectors to be stocked in schools as of 2016, with 12 states requiring it.

She drew attention to the finding that 22% of the time in the study, the epinephrine use involved the unassigned stock for children with no history of anaphylaxis or allergy.

 

“It kind of argues that, for those people that we don’t know have anaphylaxis to anything, it’s definitely important to have it available to them,” reported Dr. Neupert, a resident at the University of Texas, Austin.

In 45% of the cases, epinephrine was administered to children who had a known history of allergic reactions but, for whatever reason, did not have an epinephrine injector assigned to them.

Food was the most common trigger for use of epinephrine, accounting for 48% of the injections, with fragrance at 16%, insect stings at 10%, and exercise and overheating at 7%.

Dr. Neupert and her associates said the frequency with which epinephrine was administered after exposure to a fragrance – seen in about the same amount for unassigned and assigned epinephrine – might signal a need for greater education.

 

She emphasized the importance of follow-up after the use of unassigned injectors. If children get epinephrine that was unassigned and they have known allergies, “be sure that they subsequently get back in with their pediatrician or their allergist [and] that they’re getting their up-to-date epinephrine pens renewed,” she noted.

Carla Davis, MD, a pediatrician at Baylor College of Medicine, Houston, said she hopes more school districts adopt the stock epinephrine policy, despite barriers of cost and education. “I do think that it’s important for these schools to have these, because 20% of children will have their first episode of food allergic reaction – and in some of those cases it will be anaphylaxis – in schools.”

Dr. Neupert had no relevant financial disclosures.

SOURCE: AAAAI/WAO Joint Congress Abstract 465.

 

ORLANDO – At schools in a Texas district, unassigned epinephrine injectors were used more often than assigned ones were, according to results of an analysis.

In the 2015-2016 and 2016-2017 school years, epinephrine was administered to students 31 times at schools in the Austin Independent School District, which began to stock unassigned epinephrine in its schools after state legislators approved a law in 2015 allowing, but not requiring, school districts to do so. In 21 of those cases, or 68% of the time, it was the unassigned stockpile that was used, Kathryn Neupert, MD, said at the joint congress of the American Academy of Allergy, Asthma, and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical News

Dr. Neupert and her associates said the findings underscore the public health value of stocking unassigned epinephrine in schools. According to the Food Allergy Research and Education organization, all states but Hawaii had laws at least allowing unassigned epinephrine injectors to be stocked in schools as of 2016, with 12 states requiring it.

She drew attention to the finding that 22% of the time in the study, the epinephrine use involved the unassigned stock for children with no history of anaphylaxis or allergy.

 

“It kind of argues that, for those people that we don’t know have anaphylaxis to anything, it’s definitely important to have it available to them,” reported Dr. Neupert, a resident at the University of Texas, Austin.

In 45% of the cases, epinephrine was administered to children who had a known history of allergic reactions but, for whatever reason, did not have an epinephrine injector assigned to them.

Food was the most common trigger for use of epinephrine, accounting for 48% of the injections, with fragrance at 16%, insect stings at 10%, and exercise and overheating at 7%.

Dr. Neupert and her associates said the frequency with which epinephrine was administered after exposure to a fragrance – seen in about the same amount for unassigned and assigned epinephrine – might signal a need for greater education.

 

She emphasized the importance of follow-up after the use of unassigned injectors. If children get epinephrine that was unassigned and they have known allergies, “be sure that they subsequently get back in with their pediatrician or their allergist [and] that they’re getting their up-to-date epinephrine pens renewed,” she noted.

Carla Davis, MD, a pediatrician at Baylor College of Medicine, Houston, said she hopes more school districts adopt the stock epinephrine policy, despite barriers of cost and education. “I do think that it’s important for these schools to have these, because 20% of children will have their first episode of food allergic reaction – and in some of those cases it will be anaphylaxis – in schools.”

Dr. Neupert had no relevant financial disclosures.

SOURCE: AAAAI/WAO Joint Congress Abstract 465.

 

ORLANDO – Food was found to be the most commonly identified trigger, with peanuts the most prevalent food cause, in what researchers say is the largest comprehensive review of anaphylaxis episodes in North America that led to pediatric intensive-care unit stays.

Researchers examined the Virtual Pediatrics Systems database, an international database of pediatric intensive care unit (PICU) information, said Carla M. Davis, MD, a pediatrician at Baylor College of Medicine, Houston. During 2010-2015, there were 1,989 pediatric anaphylaxis admissions to these units in North America, she reported at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical Communications

Dr. Davis said the study is intended to give a much-needed broad look at what is causing the most severe cases of child anaphylaxis.

“Because anaphylaxis is one of the most severe consequences of allergic disease, we decided that this study needed to be done to see really what the landscape was in the most critically ill children,” she said.

 

Peanuts accounted for 45% of the food triggers, followed by tree nuts and seeds at 19%, and milk at 10%.

Common causes aside from food included drug, blood products, and venom, Dr. Davis said.

Anaphylaxis accounted for 0.3% of all PICU admissions over the 5-year period, researchers found. Dr. Davis said this was “higher than what we anticipated.”

The overall mortality rate was 1%, and researchers found that peanuts and dairy were main causes of death of all the food-induced cases.

Anaphylaxis occurred more often in children ages 6-18 years than in kids of other ages and was least common among those aged 2-5 years. Asian children were disproportionately represented among the PICU anaphylaxis patients, but the mortality rate didn’t vary by any demographic factors.

 

Admissions were most likely to happen in the fall and were more common in the Northeast and Western regions of the United States, Dr. Davis reported.

She said the deep look at the causes of these severe cases should help drive home the importance of counseling patients and families about prevention.

“For patients that have had a history of an allergic reaction to food or medication, but specifically food, I think really stressing avoidance measures will be something that will be very helpful, as well as counseling about epinephrine injectors and carrying them is going to help,” she said. “I think having a little more knowledge, pediatricians should be able to counsel and refer to allergists when they don’t feel they have all the necessary skills.”

Dr. Davis reports financial relationships with Aimmune Therapeutics and DBV Technologies.

pdnews@frontlinemedcom.com

SOURCE: Davis CM et al. 2018 AAAAI/WAO Joint Congress Abstract 775.

 

ORLANDO – Food was found to be the most commonly identified trigger, with peanuts the most prevalent food cause, in what researchers say is the largest comprehensive review of anaphylaxis episodes in North America that led to pediatric intensive-care unit stays.

Researchers examined the Virtual Pediatrics Systems database, an international database of pediatric intensive care unit (PICU) information, said Carla M. Davis, MD, a pediatrician at Baylor College of Medicine, Houston. During 2010-2015, there were 1,989 pediatric anaphylaxis admissions to these units in North America, she reported at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical Communications

Dr. Davis said the study is intended to give a much-needed broad look at what is causing the most severe cases of child anaphylaxis.

“Because anaphylaxis is one of the most severe consequences of allergic disease, we decided that this study needed to be done to see really what the landscape was in the most critically ill children,” she said.

 

Peanuts accounted for 45% of the food triggers, followed by tree nuts and seeds at 19%, and milk at 10%.

Common causes aside from food included drug, blood products, and venom, Dr. Davis said.

Anaphylaxis accounted for 0.3% of all PICU admissions over the 5-year period, researchers found. Dr. Davis said this was “higher than what we anticipated.”

The overall mortality rate was 1%, and researchers found that peanuts and dairy were main causes of death of all the food-induced cases.

Anaphylaxis occurred more often in children ages 6-18 years than in kids of other ages and was least common among those aged 2-5 years. Asian children were disproportionately represented among the PICU anaphylaxis patients, but the mortality rate didn’t vary by any demographic factors.

 

Admissions were most likely to happen in the fall and were more common in the Northeast and Western regions of the United States, Dr. Davis reported.

She said the deep look at the causes of these severe cases should help drive home the importance of counseling patients and families about prevention.

“For patients that have had a history of an allergic reaction to food or medication, but specifically food, I think really stressing avoidance measures will be something that will be very helpful, as well as counseling about epinephrine injectors and carrying them is going to help,” she said. “I think having a little more knowledge, pediatricians should be able to counsel and refer to allergists when they don’t feel they have all the necessary skills.”

Dr. Davis reports financial relationships with Aimmune Therapeutics and DBV Technologies.

pdnews@frontlinemedcom.com

SOURCE: Davis CM et al. 2018 AAAAI/WAO Joint Congress Abstract 775.

 

ORLANDO – Food was found to be the most commonly identified trigger, with peanuts the most prevalent food cause, in what researchers say is the largest comprehensive review of anaphylaxis episodes in North America that led to pediatric intensive-care unit stays.

Researchers examined the Virtual Pediatrics Systems database, an international database of pediatric intensive care unit (PICU) information, said Carla M. Davis, MD, a pediatrician at Baylor College of Medicine, Houston. During 2010-2015, there were 1,989 pediatric anaphylaxis admissions to these units in North America, she reported at the joint congress of the American Academy of Allergy, Asthma and Immunology and the World Asthma Organization.

Thomas R. Collins/Frontline Medical Communications

Dr. Davis said the study is intended to give a much-needed broad look at what is causing the most severe cases of child anaphylaxis.

“Because anaphylaxis is one of the most severe consequences of allergic disease, we decided that this study needed to be done to see really what the landscape was in the most critically ill children,” she said.

 

Peanuts accounted for 45% of the food triggers, followed by tree nuts and seeds at 19%, and milk at 10%.

Common causes aside from food included drug, blood products, and venom, Dr. Davis said.

Anaphylaxis accounted for 0.3% of all PICU admissions over the 5-year period, researchers found. Dr. Davis said this was “higher than what we anticipated.”

The overall mortality rate was 1%, and researchers found that peanuts and dairy were main causes of death of all the food-induced cases.

Anaphylaxis occurred more often in children ages 6-18 years than in kids of other ages and was least common among those aged 2-5 years. Asian children were disproportionately represented among the PICU anaphylaxis patients, but the mortality rate didn’t vary by any demographic factors.

 

Admissions were most likely to happen in the fall and were more common in the Northeast and Western regions of the United States, Dr. Davis reported.

She said the deep look at the causes of these severe cases should help drive home the importance of counseling patients and families about prevention.

“For patients that have had a history of an allergic reaction to food or medication, but specifically food, I think really stressing avoidance measures will be something that will be very helpful, as well as counseling about epinephrine injectors and carrying them is going to help,” she said. “I think having a little more knowledge, pediatricians should be able to counsel and refer to allergists when they don’t feel they have all the necessary skills.”

Dr. Davis reports financial relationships with Aimmune Therapeutics and DBV Technologies.

pdnews@frontlinemedcom.com

SOURCE: Davis CM et al. 2018 AAAAI/WAO Joint Congress Abstract 775.

 

SAN DIEGO – U.S. incidence rates of malignant melanoma in adolescents fell by nearly half during 2000-2014, based on information from a National Cancer Institute database.

The substantial drop in new cases of malignant melanoma in Americans aged 10-19 years over the most recent 15-year period with data available contrasts with a stable rate among children aged 0-9 years, and a steadily rising rate among adults during the same period, Ryan C. Kelm said at the annual meeting of the American Academy of Dermatology.

Mitchel L. Zoler/Frontline Medical News

The findings also confirmed that malignant melanoma is significantly more common in girls, regardless of whether they are aged 0-9 years, or 10-19 years. In addition, girls aged 10-19 years have significantly better 5-year relative survival, compared with boys that age, with rates of 97% and 93%, respectively, said Mr. Kelm, a researcher in the department of dermatology at Northwestern University, Chicago. Relative survival after 5 years among girls and boys aged 0-9 years was 94%. These rates were up from those reported for 2001 in the SEER (Surveillance, Epidemiology, and End Results) Program, when 5-year relative survival was 89% among all children and 92% among all adolescents (J Clin Oncol. 2005 July 20;23[21]:4735-41).

Mr. Kelm and his associates studied U.S. data compiled from 2000 to 2014 by the SEER Program, maintained by the National Cancer Institute. They identified 1,796 patients aged 0-19 years diagnosed with malignant melanoma (218 children and 1,578 adolescents). The overall incidence rate for the entire 15-year period was just over 1 case per million among children and just under 9 cases per million among adolescents. In contrast, the adult U.S. incidence rate estimates for 2018 are pegged at 260 per million among non-Hispanic whites, 40 per million among Hispanics, and 10 per million among black Americans, according to the American Cancer Society.

 

An additional analysis showed a notable difference in incidence rates over the 15-year period studied, depending on age. In children aged 0-9 years, the annual incidence rate held roughly steady at just under 2 cases per million throughout the 15 years. But among adolescents, the rate fell over time, from about 10-12 cases per million during 2000-2004 to about 5-7 cases per million during 2010-2014. In 2001, the rate was about 11 cases per million, and in 2013, the rate was about 6 cases per million. This contrasts with the adult rate, which has “risen rapidly over the past 30 years,” according to the American Cancer Society’s 2018 report.

The SEER data also showed that distribution of melanoma histologic types differed by age. Among adolescents the most common identified form was “superficial spreading,” in 32%, with nodular in 6%, mixed epithelioid and spindle cell in 2%, and “not otherwise specified” in 54%. In children, the most commonly identified form was mixed epithelioid and spindle cell, in 10%, followed by nodular in 9%, and superficial spreading in 9%, with 63% not otherwise specified.

mzoler@frontlinemedcom.com

SOURCE: Kelm RC et al. AAD 18, Abstract 6722.
 

 

SAN DIEGO – U.S. incidence rates of malignant melanoma in adolescents fell by nearly half during 2000-2014, based on information from a National Cancer Institute database.

The substantial drop in new cases of malignant melanoma in Americans aged 10-19 years over the most recent 15-year period with data available contrasts with a stable rate among children aged 0-9 years, and a steadily rising rate among adults during the same period, Ryan C. Kelm said at the annual meeting of the American Academy of Dermatology.

Mitchel L. Zoler/Frontline Medical News

The findings also confirmed that malignant melanoma is significantly more common in girls, regardless of whether they are aged 0-9 years, or 10-19 years. In addition, girls aged 10-19 years have significantly better 5-year relative survival, compared with boys that age, with rates of 97% and 93%, respectively, said Mr. Kelm, a researcher in the department of dermatology at Northwestern University, Chicago. Relative survival after 5 years among girls and boys aged 0-9 years was 94%. These rates were up from those reported for 2001 in the SEER (Surveillance, Epidemiology, and End Results) Program, when 5-year relative survival was 89% among all children and 92% among all adolescents (J Clin Oncol. 2005 July 20;23[21]:4735-41).

Mr. Kelm and his associates studied U.S. data compiled from 2000 to 2014 by the SEER Program, maintained by the National Cancer Institute. They identified 1,796 patients aged 0-19 years diagnosed with malignant melanoma (218 children and 1,578 adolescents). The overall incidence rate for the entire 15-year period was just over 1 case per million among children and just under 9 cases per million among adolescents. In contrast, the adult U.S. incidence rate estimates for 2018 are pegged at 260 per million among non-Hispanic whites, 40 per million among Hispanics, and 10 per million among black Americans, according to the American Cancer Society.

 

An additional analysis showed a notable difference in incidence rates over the 15-year period studied, depending on age. In children aged 0-9 years, the annual incidence rate held roughly steady at just under 2 cases per million throughout the 15 years. But among adolescents, the rate fell over time, from about 10-12 cases per million during 2000-2004 to about 5-7 cases per million during 2010-2014. In 2001, the rate was about 11 cases per million, and in 2013, the rate was about 6 cases per million. This contrasts with the adult rate, which has “risen rapidly over the past 30 years,” according to the American Cancer Society’s 2018 report.

The SEER data also showed that distribution of melanoma histologic types differed by age. Among adolescents the most common identified form was “superficial spreading,” in 32%, with nodular in 6%, mixed epithelioid and spindle cell in 2%, and “not otherwise specified” in 54%. In children, the most commonly identified form was mixed epithelioid and spindle cell, in 10%, followed by nodular in 9%, and superficial spreading in 9%, with 63% not otherwise specified.

mzoler@frontlinemedcom.com

SOURCE: Kelm RC et al. AAD 18, Abstract 6722.
 

 

SAN DIEGO – U.S. incidence rates of malignant melanoma in adolescents fell by nearly half during 2000-2014, based on information from a National Cancer Institute database.

The substantial drop in new cases of malignant melanoma in Americans aged 10-19 years over the most recent 15-year period with data available contrasts with a stable rate among children aged 0-9 years, and a steadily rising rate among adults during the same period, Ryan C. Kelm said at the annual meeting of the American Academy of Dermatology.

Mitchel L. Zoler/Frontline Medical News

The findings also confirmed that malignant melanoma is significantly more common in girls, regardless of whether they are aged 0-9 years, or 10-19 years. In addition, girls aged 10-19 years have significantly better 5-year relative survival, compared with boys that age, with rates of 97% and 93%, respectively, said Mr. Kelm, a researcher in the department of dermatology at Northwestern University, Chicago. Relative survival after 5 years among girls and boys aged 0-9 years was 94%. These rates were up from those reported for 2001 in the SEER (Surveillance, Epidemiology, and End Results) Program, when 5-year relative survival was 89% among all children and 92% among all adolescents (J Clin Oncol. 2005 July 20;23[21]:4735-41).

Mr. Kelm and his associates studied U.S. data compiled from 2000 to 2014 by the SEER Program, maintained by the National Cancer Institute. They identified 1,796 patients aged 0-19 years diagnosed with malignant melanoma (218 children and 1,578 adolescents). The overall incidence rate for the entire 15-year period was just over 1 case per million among children and just under 9 cases per million among adolescents. In contrast, the adult U.S. incidence rate estimates for 2018 are pegged at 260 per million among non-Hispanic whites, 40 per million among Hispanics, and 10 per million among black Americans, according to the American Cancer Society.

 

An additional analysis showed a notable difference in incidence rates over the 15-year period studied, depending on age. In children aged 0-9 years, the annual incidence rate held roughly steady at just under 2 cases per million throughout the 15 years. But among adolescents, the rate fell over time, from about 10-12 cases per million during 2000-2004 to about 5-7 cases per million during 2010-2014. In 2001, the rate was about 11 cases per million, and in 2013, the rate was about 6 cases per million. This contrasts with the adult rate, which has “risen rapidly over the past 30 years,” according to the American Cancer Society’s 2018 report.

The SEER data also showed that distribution of melanoma histologic types differed by age. Among adolescents the most common identified form was “superficial spreading,” in 32%, with nodular in 6%, mixed epithelioid and spindle cell in 2%, and “not otherwise specified” in 54%. In children, the most commonly identified form was mixed epithelioid and spindle cell, in 10%, followed by nodular in 9%, and superficial spreading in 9%, with 63% not otherwise specified.

mzoler@frontlinemedcom.com

SOURCE: Kelm RC et al. AAD 18, Abstract 6722.
 

Photo by Bill Branson

Childhood cancer survivors (CCSs) have an increased risk of premature cardiovascular disease in adulthood, according to a new study.

Researchers found a nearly 2-fold increased risk of cardiovascular diseases in CCSs compared to the general population.

Cardiovascular disease was identified in 4.5% of CCSs and occurred in most before they reached the age of 40, nearly 8 years earlier than in the general population.

“Our results show that these survivors of childhood cancer have a substantially elevated burden of prematurely occurring traditional cardiovascular risk factors and cardiovascular diseases,” said study author Joerg Faber, MD, PhD, of the Johannes Gutenberg University Mainz in Germany.

Dr Faber and his colleagues reported these results in the European Heart Journal.

The researchers evaluated 951 adult CCSs (ages 23 to 48), referred to as the CVSS cohort (Cardiac and Vascular Late Sequelae in Long-Term Survivors of Childhood Cancer Study).

The patients had been diagnosed with cancer between 1980 and 1990. The most common diagnoses were leukemia (43.5%), central nervous system tumors (12.8%), and lymphoma (9.9%).

For this study, the patients underwent standardized clinical and laboratory cardiovascular screening. The mean time from cancer diagnosis to cardiovascular screening was 28.4 years (range, 23–36).

The researchers compared the incidence of cardiovascular risk factors and cardiovascular disease in the CVSS cohort and subjects from the Gutenberg Health Study (GHS), a population-based study including more than 15,000 subjects.

Risk factors

The CVSS cohort had a greater risk of 2 cardiovascular risk factors—arterial hypertension and dyslipidemia—than the GHS cohort. In the CVSS cohort, the incidence of dyslipidemia was 28.3%, and the incidence of hypertension was 23.0%.

CVSS subjects had an age-adjusted 38% increase in risk for hypertension (relative risk [RR]=1.38) and a 26% increase in risk for dyslipidemia (RR=1.26).

Hypertension occurred about 6 years earlier in CVSS subjects than GHS subjects (rate advancement period estimator [RAP]=5.75). And dyslipidemia occurred about 8 years earlier in the CVSS cohort (RAP=8.16).

“[T]he premature onset of high blood pressure and blood lipid disorders may play an important role in the development of severe cardiovascular conditions, such as heart disease and stroke, in the long-term,” said study author Philipp S. Wild, MD, of the German Center for Cardiovascular Research (DZHK) in Mainz, Germany.

“We also found that a remarkable number [of CVSS subjects] attended their clinical examination for this study with previously unidentified cardiovascular risk factors and cardiovascular disease. For example, only 62 out of 269 were aware of having dyslipidemia. Consequently, 207, approximately 80%, were only diagnosed at that point.”

Disease

In the CVSS cohort, 4.5% of patients had at least 1 type of cardiovascular disease. This included venous thromboembolism (2.0%), congestive heart failure (1.2%), stroke (0.5%), peripheral artery disease (0.5%), atrial fibrillation (0.4%), and coronary heart disease (0.3%).

CVSS subjects had nearly twice the risk of cardiovascular disease as GHS subjects. The age-adjusted RR was 1.89. And CVSS subjects developed cardiovascular disease roughly 8 years earlier than GHS subjects (RAP=7.9).

In the CVSS cohort, the probability of developing cardiovascular disease was estimated as 2.9% at age 30 and 9.6% at age 45.

The researchers said these findings show that CCSs have a greater risk of cardiovascular disease that continues to increase with age. This, in turn, means CCSs may be more likely to die earlier. However, this might be preventable, according to Dr Wild.

“Early systematic screening, particularly focusing on blood pressure and lipid measurements, might be suggested in all childhood cancer survivors irrespective of the type of cancer or treatment they had had,” Dr Wild said. “This might help to prevent long-term cardiovascular diseases by intervening early—for instance, by modifying lifestyles and having treatment for high blood pressure.”

 

“Usually, survivors are followed for only 5 to 10 years after completion of therapy, and this is focused on the risk of the cancer returning and the acute adverse effects of their treatment, rather than on other conditions,” added Dr Faber.

“Current guidelines recommend cardiovascular assessments only for subgroups known to be at risk, such as for patients who were treated with anthracycline therapy and/or radiation therapy. However, further investigations are needed to answer questions about the best follow-up care.”

Photo by Bill Branson

Childhood cancer survivors (CCSs) have an increased risk of premature cardiovascular disease in adulthood, according to a new study.

Researchers found a nearly 2-fold increased risk of cardiovascular diseases in CCSs compared to the general population.

Cardiovascular disease was identified in 4.5% of CCSs and occurred in most before they reached the age of 40, nearly 8 years earlier than in the general population.

“Our results show that these survivors of childhood cancer have a substantially elevated burden of prematurely occurring traditional cardiovascular risk factors and cardiovascular diseases,” said study author Joerg Faber, MD, PhD, of the Johannes Gutenberg University Mainz in Germany.

Dr Faber and his colleagues reported these results in the European Heart Journal.

The researchers evaluated 951 adult CCSs (ages 23 to 48), referred to as the CVSS cohort (Cardiac and Vascular Late Sequelae in Long-Term Survivors of Childhood Cancer Study).

The patients had been diagnosed with cancer between 1980 and 1990. The most common diagnoses were leukemia (43.5%), central nervous system tumors (12.8%), and lymphoma (9.9%).

For this study, the patients underwent standardized clinical and laboratory cardiovascular screening. The mean time from cancer diagnosis to cardiovascular screening was 28.4 years (range, 23–36).

The researchers compared the incidence of cardiovascular risk factors and cardiovascular disease in the CVSS cohort and subjects from the Gutenberg Health Study (GHS), a population-based study including more than 15,000 subjects.

Risk factors

The CVSS cohort had a greater risk of 2 cardiovascular risk factors—arterial hypertension and dyslipidemia—than the GHS cohort. In the CVSS cohort, the incidence of dyslipidemia was 28.3%, and the incidence of hypertension was 23.0%.

CVSS subjects had an age-adjusted 38% increase in risk for hypertension (relative risk [RR]=1.38) and a 26% increase in risk for dyslipidemia (RR=1.26).

Hypertension occurred about 6 years earlier in CVSS subjects than GHS subjects (rate advancement period estimator [RAP]=5.75). And dyslipidemia occurred about 8 years earlier in the CVSS cohort (RAP=8.16).

“[T]he premature onset of high blood pressure and blood lipid disorders may play an important role in the development of severe cardiovascular conditions, such as heart disease and stroke, in the long-term,” said study author Philipp S. Wild, MD, of the German Center for Cardiovascular Research (DZHK) in Mainz, Germany.

“We also found that a remarkable number [of CVSS subjects] attended their clinical examination for this study with previously unidentified cardiovascular risk factors and cardiovascular disease. For example, only 62 out of 269 were aware of having dyslipidemia. Consequently, 207, approximately 80%, were only diagnosed at that point.”

Disease

In the CVSS cohort, 4.5% of patients had at least 1 type of cardiovascular disease. This included venous thromboembolism (2.0%), congestive heart failure (1.2%), stroke (0.5%), peripheral artery disease (0.5%), atrial fibrillation (0.4%), and coronary heart disease (0.3%).

CVSS subjects had nearly twice the risk of cardiovascular disease as GHS subjects. The age-adjusted RR was 1.89. And CVSS subjects developed cardiovascular disease roughly 8 years earlier than GHS subjects (RAP=7.9).

In the CVSS cohort, the probability of developing cardiovascular disease was estimated as 2.9% at age 30 and 9.6% at age 45.

The researchers said these findings show that CCSs have a greater risk of cardiovascular disease that continues to increase with age. This, in turn, means CCSs may be more likely to die earlier. However, this might be preventable, according to Dr Wild.

“Early systematic screening, particularly focusing on blood pressure and lipid measurements, might be suggested in all childhood cancer survivors irrespective of the type of cancer or treatment they had had,” Dr Wild said. “This might help to prevent long-term cardiovascular diseases by intervening early—for instance, by modifying lifestyles and having treatment for high blood pressure.”

 

“Usually, survivors are followed for only 5 to 10 years after completion of therapy, and this is focused on the risk of the cancer returning and the acute adverse effects of their treatment, rather than on other conditions,” added Dr Faber.

“Current guidelines recommend cardiovascular assessments only for subgroups known to be at risk, such as for patients who were treated with anthracycline therapy and/or radiation therapy. However, further investigations are needed to answer questions about the best follow-up care.”

Photo by Bill Branson

Childhood cancer survivors (CCSs) have an increased risk of premature cardiovascular disease in adulthood, according to a new study.

Researchers found a nearly 2-fold increased risk of cardiovascular diseases in CCSs compared to the general population.

Cardiovascular disease was identified in 4.5% of CCSs and occurred in most before they reached the age of 40, nearly 8 years earlier than in the general population.

“Our results show that these survivors of childhood cancer have a substantially elevated burden of prematurely occurring traditional cardiovascular risk factors and cardiovascular diseases,” said study author Joerg Faber, MD, PhD, of the Johannes Gutenberg University Mainz in Germany.

Dr Faber and his colleagues reported these results in the European Heart Journal.

The researchers evaluated 951 adult CCSs (ages 23 to 48), referred to as the CVSS cohort (Cardiac and Vascular Late Sequelae in Long-Term Survivors of Childhood Cancer Study).

The patients had been diagnosed with cancer between 1980 and 1990. The most common diagnoses were leukemia (43.5%), central nervous system tumors (12.8%), and lymphoma (9.9%).

For this study, the patients underwent standardized clinical and laboratory cardiovascular screening. The mean time from cancer diagnosis to cardiovascular screening was 28.4 years (range, 23–36).

The researchers compared the incidence of cardiovascular risk factors and cardiovascular disease in the CVSS cohort and subjects from the Gutenberg Health Study (GHS), a population-based study including more than 15,000 subjects.

Risk factors

The CVSS cohort had a greater risk of 2 cardiovascular risk factors—arterial hypertension and dyslipidemia—than the GHS cohort. In the CVSS cohort, the incidence of dyslipidemia was 28.3%, and the incidence of hypertension was 23.0%.

CVSS subjects had an age-adjusted 38% increase in risk for hypertension (relative risk [RR]=1.38) and a 26% increase in risk for dyslipidemia (RR=1.26).

Hypertension occurred about 6 years earlier in CVSS subjects than GHS subjects (rate advancement period estimator [RAP]=5.75). And dyslipidemia occurred about 8 years earlier in the CVSS cohort (RAP=8.16).

“[T]he premature onset of high blood pressure and blood lipid disorders may play an important role in the development of severe cardiovascular conditions, such as heart disease and stroke, in the long-term,” said study author Philipp S. Wild, MD, of the German Center for Cardiovascular Research (DZHK) in Mainz, Germany.

“We also found that a remarkable number [of CVSS subjects] attended their clinical examination for this study with previously unidentified cardiovascular risk factors and cardiovascular disease. For example, only 62 out of 269 were aware of having dyslipidemia. Consequently, 207, approximately 80%, were only diagnosed at that point.”

Disease

In the CVSS cohort, 4.5% of patients had at least 1 type of cardiovascular disease. This included venous thromboembolism (2.0%), congestive heart failure (1.2%), stroke (0.5%), peripheral artery disease (0.5%), atrial fibrillation (0.4%), and coronary heart disease (0.3%).

CVSS subjects had nearly twice the risk of cardiovascular disease as GHS subjects. The age-adjusted RR was 1.89. And CVSS subjects developed cardiovascular disease roughly 8 years earlier than GHS subjects (RAP=7.9).

In the CVSS cohort, the probability of developing cardiovascular disease was estimated as 2.9% at age 30 and 9.6% at age 45.

The researchers said these findings show that CCSs have a greater risk of cardiovascular disease that continues to increase with age. This, in turn, means CCSs may be more likely to die earlier. However, this might be preventable, according to Dr Wild.

“Early systematic screening, particularly focusing on blood pressure and lipid measurements, might be suggested in all childhood cancer survivors irrespective of the type of cancer or treatment they had had,” Dr Wild said. “This might help to prevent long-term cardiovascular diseases by intervening early—for instance, by modifying lifestyles and having treatment for high blood pressure.”

 

“Usually, survivors are followed for only 5 to 10 years after completion of therapy, and this is focused on the risk of the cancer returning and the acute adverse effects of their treatment, rather than on other conditions,” added Dr Faber.

“Current guidelines recommend cardiovascular assessments only for subgroups known to be at risk, such as for patients who were treated with anthracycline therapy and/or radiation therapy. However, further investigations are needed to answer questions about the best follow-up care.”

 

SAN ANTONIO – Use of a pediatric early warning system reduced the incidence of late ICU admissions among hospitalized pediatric patients, but did not reduce the rate of all-cause hospital mortality, according to results of a large, multicenter trial.

Taken together, the findings of the trial do not support the use of the Bedside Pediatric Early Warning System (BedsidePEWS) to reduce hospital mortality, noted investigator Christopher S. Parshuram, MBChB, DPhil, during a presentation at the Critical Care Congress sponsored by the Society of Critical Care Medicine.

©drpnncpp/thinkstockphotos.com

BedsidePEWS is a documentation-based care system that combines a validated severity of illness score, a specialized documentation record, and specific recommendations for care escalation.

The multicenter randomized cluster study, called the EPOCH trial, included 21 hospitals in seven countries that provided inpatient pediatric care. Ten of the hospitals delivered the BedsidePEWS intervention, while the remaining 11 provided usual care. The study data included 144,539 patient discharges comprising 559,443 patient days. Enrollment began Feb. 28, 2011, and ended on June 21, 2015.

 

For the BedsidePEWS group, all-cause hospital mortality was 1.93 per 1,000 patient discharges, versus 1.56 per 1,000 patient discharges for usual care (adjusted odds ratio, 1.01; 95% confidence interval, 0.61-1.69; P = .96), according to a report on this study that was published in JAMA.

However, the BedsidePEWS group had a significant improvement in the secondary outcome of significant clinical deterioration events, a composite outcome reflecting late ICU admissions.

In the BedsidePEWS group, the rate of significant clinical deterioration events was 0.50 per 1,000 patient-days, compared with 0.84 per 1,000 patient-days at hospitals with usual care (adjusted rate ratio, 0.77; 95% CI, 0.61-0.97; P = .03), the investigators wrote.

The goal of the EPOCH trial was to determine whether BedsidePEWS could reduce rates of all-cause hospital mortality and significant clinical deterioration among hospitalized children, according to the researchers.

 

“The BedsidePEWS versus usual care did improve processes of care and early detection of critical illness, aligned with the notion of providing the right care, right now,” Dr. Parshuram, associate professor of critical care medicine and pediatrics at the University of Toronto, said during his presentation at the meeting. “Certainly more vital signs were documented, and anecdotally there were reports of culture change.

“However, when we looked further, there was no difference in hospital mortality, nor hospital resource utilization,” Dr. Parshuram added.

The Canadian Institutes of Health Research funded the study. Dr. Parshuram is an inventor of BedsidePEWS and owns shares in a company that is commercializing it.

SOURCE: Parshuram et al. JAMA. 2018 Feb 27. doi: 10.1001/jama.2018.0948.

 

SAN ANTONIO – Use of a pediatric early warning system reduced the incidence of late ICU admissions among hospitalized pediatric patients, but did not reduce the rate of all-cause hospital mortality, according to results of a large, multicenter trial.

Taken together, the findings of the trial do not support the use of the Bedside Pediatric Early Warning System (BedsidePEWS) to reduce hospital mortality, noted investigator Christopher S. Parshuram, MBChB, DPhil, during a presentation at the Critical Care Congress sponsored by the Society of Critical Care Medicine.

©drpnncpp/thinkstockphotos.com

BedsidePEWS is a documentation-based care system that combines a validated severity of illness score, a specialized documentation record, and specific recommendations for care escalation.

The multicenter randomized cluster study, called the EPOCH trial, included 21 hospitals in seven countries that provided inpatient pediatric care. Ten of the hospitals delivered the BedsidePEWS intervention, while the remaining 11 provided usual care. The study data included 144,539 patient discharges comprising 559,443 patient days. Enrollment began Feb. 28, 2011, and ended on June 21, 2015.

 

For the BedsidePEWS group, all-cause hospital mortality was 1.93 per 1,000 patient discharges, versus 1.56 per 1,000 patient discharges for usual care (adjusted odds ratio, 1.01; 95% confidence interval, 0.61-1.69; P = .96), according to a report on this study that was published in JAMA.

However, the BedsidePEWS group had a significant improvement in the secondary outcome of significant clinical deterioration events, a composite outcome reflecting late ICU admissions.

In the BedsidePEWS group, the rate of significant clinical deterioration events was 0.50 per 1,000 patient-days, compared with 0.84 per 1,000 patient-days at hospitals with usual care (adjusted rate ratio, 0.77; 95% CI, 0.61-0.97; P = .03), the investigators wrote.

The goal of the EPOCH trial was to determine whether BedsidePEWS could reduce rates of all-cause hospital mortality and significant clinical deterioration among hospitalized children, according to the researchers.

 

“The BedsidePEWS versus usual care did improve processes of care and early detection of critical illness, aligned with the notion of providing the right care, right now,” Dr. Parshuram, associate professor of critical care medicine and pediatrics at the University of Toronto, said during his presentation at the meeting. “Certainly more vital signs were documented, and anecdotally there were reports of culture change.

“However, when we looked further, there was no difference in hospital mortality, nor hospital resource utilization,” Dr. Parshuram added.

The Canadian Institutes of Health Research funded the study. Dr. Parshuram is an inventor of BedsidePEWS and owns shares in a company that is commercializing it.

SOURCE: Parshuram et al. JAMA. 2018 Feb 27. doi: 10.1001/jama.2018.0948.

 

SAN ANTONIO – Use of a pediatric early warning system reduced the incidence of late ICU admissions among hospitalized pediatric patients, but did not reduce the rate of all-cause hospital mortality, according to results of a large, multicenter trial.

Taken together, the findings of the trial do not support the use of the Bedside Pediatric Early Warning System (BedsidePEWS) to reduce hospital mortality, noted investigator Christopher S. Parshuram, MBChB, DPhil, during a presentation at the Critical Care Congress sponsored by the Society of Critical Care Medicine.

©drpnncpp/thinkstockphotos.com

BedsidePEWS is a documentation-based care system that combines a validated severity of illness score, a specialized documentation record, and specific recommendations for care escalation.

The multicenter randomized cluster study, called the EPOCH trial, included 21 hospitals in seven countries that provided inpatient pediatric care. Ten of the hospitals delivered the BedsidePEWS intervention, while the remaining 11 provided usual care. The study data included 144,539 patient discharges comprising 559,443 patient days. Enrollment began Feb. 28, 2011, and ended on June 21, 2015.

 

For the BedsidePEWS group, all-cause hospital mortality was 1.93 per 1,000 patient discharges, versus 1.56 per 1,000 patient discharges for usual care (adjusted odds ratio, 1.01; 95% confidence interval, 0.61-1.69; P = .96), according to a report on this study that was published in JAMA.

However, the BedsidePEWS group had a significant improvement in the secondary outcome of significant clinical deterioration events, a composite outcome reflecting late ICU admissions.

In the BedsidePEWS group, the rate of significant clinical deterioration events was 0.50 per 1,000 patient-days, compared with 0.84 per 1,000 patient-days at hospitals with usual care (adjusted rate ratio, 0.77; 95% CI, 0.61-0.97; P = .03), the investigators wrote.

The goal of the EPOCH trial was to determine whether BedsidePEWS could reduce rates of all-cause hospital mortality and significant clinical deterioration among hospitalized children, according to the researchers.

 

“The BedsidePEWS versus usual care did improve processes of care and early detection of critical illness, aligned with the notion of providing the right care, right now,” Dr. Parshuram, associate professor of critical care medicine and pediatrics at the University of Toronto, said during his presentation at the meeting. “Certainly more vital signs were documented, and anecdotally there were reports of culture change.

“However, when we looked further, there was no difference in hospital mortality, nor hospital resource utilization,” Dr. Parshuram added.

The Canadian Institutes of Health Research funded the study. Dr. Parshuram is an inventor of BedsidePEWS and owns shares in a company that is commercializing it.

SOURCE: Parshuram et al. JAMA. 2018 Feb 27. doi: 10.1001/jama.2018.0948.

Photo by Rhoda Baer

New research suggests guidelines from the National Comprehensive Cancer Network (NCCN) may sometimes be supported by low-quality evidence or no evidence at all.

Researchers compared NCCN recommendations for cancer drugs to US cancer drug approvals over a 5-year period.

Thirty-nine percent of NCCN’s treatment recommendations did not coincide with uses approved by the US Food and Drug Administration (FDA).

For most of these recommendations (84%), NCCN did not provide supporting data from randomized, phase 3 trials.

For 36% of the recommendations, NCCN gave no supporting evidence.

Vinay Prasad, MD, of Oregon Health & Science University in Portland, Oregon, and his colleagues reported these findings in The BMJ.

Dr Prasad and his colleagues compared FDA approvals of cancer drugs between 2011 and 2015 with NCCN recommendations as of March 25, 2016.

When NCCN made recommendations beyond FDA approvals, the researchers evaluated the evidence used to support those recommendations.

Forty-seven new cancer drugs were approved by the FDA for 69 indications between 2011 and 2015. NCCN recommended the 47 drugs for 113 indications, including the 69 FDA-approved indications.

So 39% (n=44) of NCCN’s recommendations were not approved by the FDA, and NCCN gave the following evidence to support these recommendations:

• No evidence—36% (n=16)
• Phase 2 trial without randomization—30% (n=13)
• Randomized, phase 3 trial—16% (n=7)
• Phase 2 trial with randomization—7% (n=3)
• Case report or series of less than 5 patients—5% (n=2)
• Book chapter or review article—2% (n=1)
• Phase 1 trial—2% (n=1)
• Ongoing trial—2% (n=1).

Dr Prasad and his colleagues did point out that not all FDA approvals are supported by randomized, phase 3 trials.

And when the team followed-up 21 months after their initial analysis, they found that 6 of the 44 (14%) additional recommendations by NCCN had received FDA approval.

The researchers also noted that they did not search for independent evidence to support NCCN recommendations beyond the references NCCN provided. So some of the recommendations may have had more or better supporting evidence than what was provided.

Still, the team said these results suggest NCCN “frequently” makes recommendations that go beyond FDA approvals and “often fails to cite evidence or relies on low levels of evidence.” Therefore, NCCN should cite all evidence used to formulate its recommendations.

NCCN argues that it does provide ample evidence to support the recommendations in its guidelines.

“The NCCN guidelines contain more than 24,500 references to inform users of the evidence used in making its decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

“These data are supplemented by the analysis of the available evidence by expert clinician researchers and patient advocates who evaluate each recommendation and come to consensus. Each recommendation is labeled with a Category of Evidence, and the vast majority of those for systemic therapies are accompanied by Evidence Blocks, which outline, on 1-5 scales, the efficacy, safety, quality of the evidence, consistency of the evidence, and affordability of the treatment.”

Photo by Rhoda Baer

New research suggests guidelines from the National Comprehensive Cancer Network (NCCN) may sometimes be supported by low-quality evidence or no evidence at all.

Researchers compared NCCN recommendations for cancer drugs to US cancer drug approvals over a 5-year period.

Thirty-nine percent of NCCN’s treatment recommendations did not coincide with uses approved by the US Food and Drug Administration (FDA).

For most of these recommendations (84%), NCCN did not provide supporting data from randomized, phase 3 trials.

For 36% of the recommendations, NCCN gave no supporting evidence.

Vinay Prasad, MD, of Oregon Health & Science University in Portland, Oregon, and his colleagues reported these findings in The BMJ.

Dr Prasad and his colleagues compared FDA approvals of cancer drugs between 2011 and 2015 with NCCN recommendations as of March 25, 2016.

When NCCN made recommendations beyond FDA approvals, the researchers evaluated the evidence used to support those recommendations.

Forty-seven new cancer drugs were approved by the FDA for 69 indications between 2011 and 2015. NCCN recommended the 47 drugs for 113 indications, including the 69 FDA-approved indications.

So 39% (n=44) of NCCN’s recommendations were not approved by the FDA, and NCCN gave the following evidence to support these recommendations:

• No evidence—36% (n=16)
• Phase 2 trial without randomization—30% (n=13)
• Randomized, phase 3 trial—16% (n=7)
• Phase 2 trial with randomization—7% (n=3)
• Case report or series of less than 5 patients—5% (n=2)
• Book chapter or review article—2% (n=1)
• Phase 1 trial—2% (n=1)
• Ongoing trial—2% (n=1).

Dr Prasad and his colleagues did point out that not all FDA approvals are supported by randomized, phase 3 trials.

And when the team followed-up 21 months after their initial analysis, they found that 6 of the 44 (14%) additional recommendations by NCCN had received FDA approval.

The researchers also noted that they did not search for independent evidence to support NCCN recommendations beyond the references NCCN provided. So some of the recommendations may have had more or better supporting evidence than what was provided.

Still, the team said these results suggest NCCN “frequently” makes recommendations that go beyond FDA approvals and “often fails to cite evidence or relies on low levels of evidence.” Therefore, NCCN should cite all evidence used to formulate its recommendations.

NCCN argues that it does provide ample evidence to support the recommendations in its guidelines.

“The NCCN guidelines contain more than 24,500 references to inform users of the evidence used in making its decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

“These data are supplemented by the analysis of the available evidence by expert clinician researchers and patient advocates who evaluate each recommendation and come to consensus. Each recommendation is labeled with a Category of Evidence, and the vast majority of those for systemic therapies are accompanied by Evidence Blocks, which outline, on 1-5 scales, the efficacy, safety, quality of the evidence, consistency of the evidence, and affordability of the treatment.”

Photo by Rhoda Baer

New research suggests guidelines from the National Comprehensive Cancer Network (NCCN) may sometimes be supported by low-quality evidence or no evidence at all.

Researchers compared NCCN recommendations for cancer drugs to US cancer drug approvals over a 5-year period.

Thirty-nine percent of NCCN’s treatment recommendations did not coincide with uses approved by the US Food and Drug Administration (FDA).

For most of these recommendations (84%), NCCN did not provide supporting data from randomized, phase 3 trials.

For 36% of the recommendations, NCCN gave no supporting evidence.

Vinay Prasad, MD, of Oregon Health & Science University in Portland, Oregon, and his colleagues reported these findings in The BMJ.

Dr Prasad and his colleagues compared FDA approvals of cancer drugs between 2011 and 2015 with NCCN recommendations as of March 25, 2016.

When NCCN made recommendations beyond FDA approvals, the researchers evaluated the evidence used to support those recommendations.

Forty-seven new cancer drugs were approved by the FDA for 69 indications between 2011 and 2015. NCCN recommended the 47 drugs for 113 indications, including the 69 FDA-approved indications.

So 39% (n=44) of NCCN’s recommendations were not approved by the FDA, and NCCN gave the following evidence to support these recommendations:

• No evidence—36% (n=16)
• Phase 2 trial without randomization—30% (n=13)
• Randomized, phase 3 trial—16% (n=7)
• Phase 2 trial with randomization—7% (n=3)
• Case report or series of less than 5 patients—5% (n=2)
• Book chapter or review article—2% (n=1)
• Phase 1 trial—2% (n=1)
• Ongoing trial—2% (n=1).

Dr Prasad and his colleagues did point out that not all FDA approvals are supported by randomized, phase 3 trials.

And when the team followed-up 21 months after their initial analysis, they found that 6 of the 44 (14%) additional recommendations by NCCN had received FDA approval.

The researchers also noted that they did not search for independent evidence to support NCCN recommendations beyond the references NCCN provided. So some of the recommendations may have had more or better supporting evidence than what was provided.

Still, the team said these results suggest NCCN “frequently” makes recommendations that go beyond FDA approvals and “often fails to cite evidence or relies on low levels of evidence.” Therefore, NCCN should cite all evidence used to formulate its recommendations.

NCCN argues that it does provide ample evidence to support the recommendations in its guidelines.

“The NCCN guidelines contain more than 24,500 references to inform users of the evidence used in making its decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

“These data are supplemented by the analysis of the available evidence by expert clinician researchers and patient advocates who evaluate each recommendation and come to consensus. Each recommendation is labeled with a Category of Evidence, and the vast majority of those for systemic therapies are accompanied by Evidence Blocks, which outline, on 1-5 scales, the efficacy, safety, quality of the evidence, consistency of the evidence, and affordability of the treatment.”

 

Prenatal tenofovir didn’t reduce the rate of hepatitis B among infants born to women infected with the virus.

Among 322 6-month-olds, the rate of HBV transmission was 0 in those whose mothers received the antiviral during pregnancy and 2% among those whose mothers received placebo – not a statistically significant difference, Gonzague Jourdain, MD, and his colleagues reported in the New England Journal of Medicine.

©sarathsasidharan/Thinkstock

All of the infants in the study, who were born in Thailand, got hepatitis B immune globulin and began a 5-dose HBV vaccination schedule within the first few hours after birth, something that “may have contributed to the low rate of HBV transmission that was observed” in the study, said Dr. Jourdain, a visiting scientist at the Harvard School of Public Health, Boston.

The study randomized 331 pregnant women with proven HBV infections to either tenofovir or placebo from 28 weeks’ gestation to 2 months post partum. All infants received HBV immune globulin at birth, and HBV vaccine at birth and at 1, 2, 4, and 6 months. The primary endpoint was confirmed HBV infection in the infant at 6 months.

 

Women were a mean of 28 weeks pregnant at baseline, with a mean viral load of 8.0 log¹⁰ IU/mL. Most women (about 90% of each group) had an HBV DNA of more than 200,000 IU/mL – a level associated with an increased risk of perinatal HBV infection despite vaccination.

There were 322 deliveries, resulting in 319 singletons, two pairs of twins, and one stillbirth. Postpartum infant treatment was quick, with a median of 1.3 hours from birth to administration of immune globulin and a median of 1.2 hours to administration of the first dose of the vaccine.

At 6 months, there were no HBV infections in the tenofovir-exposed group and 3 (2%) in the placebo group – a nonsignificant difference (P = .12).

Tenofovir was safe for both mother and fetus, with no significant adverse events in either group. The incidence of elevated maternal alanine aminotransferase level (more than 300 IU/L) was 6% in the tenofovir group and 3% in the placebo group, also a nonsignificant finding.

 

Dr. Jourdain and his colleagues noted that the 2% transmission rate in the placebo group is considerably lower than the 7% seen in similar studies and could be related to the rapid postpartum administration of HBV immune globulin and vaccine. If this is the case, prenatal antivirals could be more effective in countries where postpartum treatment is delayed or inconsistent.

“Maternal use of tenofovir may prevent transmissions that would occur when the birth dose is delayed, but its exact timing has not been reported consistently in previous perinatal studies,” the team said.

Another question is whether the stringent, 5-dose infant HBV vaccine series required in Thailand is simply more effective than schedules that have fewer doses or are combined with other vaccines and delivered later.

“It remains unclear whether the administration of more vaccine doses is more efficacious than the administration of the three vaccine doses that is recommended in the United States and by the World Health Organization.”

Dr. Jourdain had no financial disclosures relevant to the study, which was sponsored by the National Institute of Child Health and Human Development.

msullivan@frontlinemedcom.com

SOURCE: Jourdain G et al. N Engl J Med. 2018;378:911-23.
 

 

Prenatal tenofovir didn’t reduce the rate of hepatitis B among infants born to women infected with the virus.

Among 322 6-month-olds, the rate of HBV transmission was 0 in those whose mothers received the antiviral during pregnancy and 2% among those whose mothers received placebo – not a statistically significant difference, Gonzague Jourdain, MD, and his colleagues reported in the New England Journal of Medicine.

©sarathsasidharan/Thinkstock

All of the infants in the study, who were born in Thailand, got hepatitis B immune globulin and began a 5-dose HBV vaccination schedule within the first few hours after birth, something that “may have contributed to the low rate of HBV transmission that was observed” in the study, said Dr. Jourdain, a visiting scientist at the Harvard School of Public Health, Boston.

The study randomized 331 pregnant women with proven HBV infections to either tenofovir or placebo from 28 weeks’ gestation to 2 months post partum. All infants received HBV immune globulin at birth, and HBV vaccine at birth and at 1, 2, 4, and 6 months. The primary endpoint was confirmed HBV infection in the infant at 6 months.

 

Women were a mean of 28 weeks pregnant at baseline, with a mean viral load of 8.0 log¹⁰ IU/mL. Most women (about 90% of each group) had an HBV DNA of more than 200,000 IU/mL – a level associated with an increased risk of perinatal HBV infection despite vaccination.

There were 322 deliveries, resulting in 319 singletons, two pairs of twins, and one stillbirth. Postpartum infant treatment was quick, with a median of 1.3 hours from birth to administration of immune globulin and a median of 1.2 hours to administration of the first dose of the vaccine.

At 6 months, there were no HBV infections in the tenofovir-exposed group and 3 (2%) in the placebo group – a nonsignificant difference (P = .12).

Tenofovir was safe for both mother and fetus, with no significant adverse events in either group. The incidence of elevated maternal alanine aminotransferase level (more than 300 IU/L) was 6% in the tenofovir group and 3% in the placebo group, also a nonsignificant finding.

 

Dr. Jourdain and his colleagues noted that the 2% transmission rate in the placebo group is considerably lower than the 7% seen in similar studies and could be related to the rapid postpartum administration of HBV immune globulin and vaccine. If this is the case, prenatal antivirals could be more effective in countries where postpartum treatment is delayed or inconsistent.

“Maternal use of tenofovir may prevent transmissions that would occur when the birth dose is delayed, but its exact timing has not been reported consistently in previous perinatal studies,” the team said.

Another question is whether the stringent, 5-dose infant HBV vaccine series required in Thailand is simply more effective than schedules that have fewer doses or are combined with other vaccines and delivered later.

“It remains unclear whether the administration of more vaccine doses is more efficacious than the administration of the three vaccine doses that is recommended in the United States and by the World Health Organization.”

Dr. Jourdain had no financial disclosures relevant to the study, which was sponsored by the National Institute of Child Health and Human Development.

msullivan@frontlinemedcom.com

SOURCE: Jourdain G et al. N Engl J Med. 2018;378:911-23.
 

 

Prenatal tenofovir didn’t reduce the rate of hepatitis B among infants born to women infected with the virus.

Among 322 6-month-olds, the rate of HBV transmission was 0 in those whose mothers received the antiviral during pregnancy and 2% among those whose mothers received placebo – not a statistically significant difference, Gonzague Jourdain, MD, and his colleagues reported in the New England Journal of Medicine.

©sarathsasidharan/Thinkstock

All of the infants in the study, who were born in Thailand, got hepatitis B immune globulin and began a 5-dose HBV vaccination schedule within the first few hours after birth, something that “may have contributed to the low rate of HBV transmission that was observed” in the study, said Dr. Jourdain, a visiting scientist at the Harvard School of Public Health, Boston.

The study randomized 331 pregnant women with proven HBV infections to either tenofovir or placebo from 28 weeks’ gestation to 2 months post partum. All infants received HBV immune globulin at birth, and HBV vaccine at birth and at 1, 2, 4, and 6 months. The primary endpoint was confirmed HBV infection in the infant at 6 months.

 

Women were a mean of 28 weeks pregnant at baseline, with a mean viral load of 8.0 log¹⁰ IU/mL. Most women (about 90% of each group) had an HBV DNA of more than 200,000 IU/mL – a level associated with an increased risk of perinatal HBV infection despite vaccination.

There were 322 deliveries, resulting in 319 singletons, two pairs of twins, and one stillbirth. Postpartum infant treatment was quick, with a median of 1.3 hours from birth to administration of immune globulin and a median of 1.2 hours to administration of the first dose of the vaccine.

At 6 months, there were no HBV infections in the tenofovir-exposed group and 3 (2%) in the placebo group – a nonsignificant difference (P = .12).

Tenofovir was safe for both mother and fetus, with no significant adverse events in either group. The incidence of elevated maternal alanine aminotransferase level (more than 300 IU/L) was 6% in the tenofovir group and 3% in the placebo group, also a nonsignificant finding.

 

Dr. Jourdain and his colleagues noted that the 2% transmission rate in the placebo group is considerably lower than the 7% seen in similar studies and could be related to the rapid postpartum administration of HBV immune globulin and vaccine. If this is the case, prenatal antivirals could be more effective in countries where postpartum treatment is delayed or inconsistent.

“Maternal use of tenofovir may prevent transmissions that would occur when the birth dose is delayed, but its exact timing has not been reported consistently in previous perinatal studies,” the team said.

Another question is whether the stringent, 5-dose infant HBV vaccine series required in Thailand is simply more effective than schedules that have fewer doses or are combined with other vaccines and delivered later.

“It remains unclear whether the administration of more vaccine doses is more efficacious than the administration of the three vaccine doses that is recommended in the United States and by the World Health Organization.”

Dr. Jourdain had no financial disclosures relevant to the study, which was sponsored by the National Institute of Child Health and Human Development.

msullivan@frontlinemedcom.com

SOURCE: Jourdain G et al. N Engl J Med. 2018;378:911-23.
 

 

Transmasculine youths distressed by breast development who undergo chest reconstruction reported low levels of distress and almost none said they regretted the surgery, according to study results.

This study is one of the first to document the ongoing impact of chest dysphoria in transgender youths, defined as individuals assigned female at birth who have a masculine gender identity.

“Given these findings, professional guidelines and clinical practice should consider patients for chest surgery based on individual need rather than chronologic age,” said Johanna Olson-Kennedy, MD, of the division of adolescent medicine at Children’s Hospital Los Angeles, and her coauthors.

National guidelines on transgender health care are unclear as to whether minors should be referred for chest surgery because of a lack of data documenting effects of chest surgery in individuals younger than 18 years of age, Dr. Olson-Kennedy and her colleagues wrote in the study, published in JAMA Pediatrics.

To evaluate the discomfort and subsequent consequences of chest dysphoria, the researchers developed a 10-minute, 21-item survey based on Dr. Olson-Kennedy’s 11 years of experience providing care for transgender youth. It was reviewed by a small number of transmasculine youth and adults to determine whether the questions contained the elements of chest dysphoria effectively, used appropriate language, and was otherwise acceptable. From the survey, the researchers derived a chest dysphoria composite score of 0-51, with higher scores indicating increased distress.

Some of the items on the chest dysphoria survey included avoiding exercise, not seeking medical care, and not swimming because of “my chest,” and that taking a shower is difficult as is dating and physical intimacy.

The study included surveys completed by nonsurgical (n = 68) and postsurgical (n = 68) cohorts of transmasculine individuals aged 13-25 years.

The chest dysphoria composite score was significantly higher for minors and young adults who had not undergone chest reconstruction, compared with those who had undergone the procedure (29.6 vs. 3.3; P less than .001), the investigators reported.

Among transmasculine youths who had not undergone surgery, 94% perceived the procedure as very important, Dr. Olson-Kennedy and her coauthors noted.

 

Moreover, chest dysphoria increased by 0.33 points for every month that passed between a youth starting testosterone therapy and undergoing surgery, results of a linear regression analysis showed.

Most survey respondents reported that they were currently taking testosterone: 87% of the nonsurgical group and 97% of the postsurgical group.

Of the individuals who had undergone chest reconstruction, one (less than 1%) reported regretting the procedure “sometimes,” said Dr. Olson-Kennedy and her coauthors.

The mean age of survey participants was 17 (13-23 years) and 19 years (14-25 years) in the nonsurgical and postsurgical cohorts, respectively.Based on the results, Dr. Olson-Kennedy and her associates called for changes to clinical practice and to insurance plans, which sometimes require 12 months of continuous testosterone therapy prior to chest surgery. “Individualized, patient-centered care plans should be considered the standard of care for all transgender adolescents, and referrals should be made accordingly.”

Dr. Olson-Kennedy and her coauthors reported no conflicts of interest related to the study, which was funded by the Eunice Kennedy Shriver National Institute for Child Health and Human Development.

pdnews@frontlinemedcom.com

SOURCE: Olson-Kennedy J et al. JAMA Pediatrics. 2018 Mar 5. doi: 10.1001/jamapediatrics.2017.5440.

 

Transmasculine youths distressed by breast development who undergo chest reconstruction reported low levels of distress and almost none said they regretted the surgery, according to study results.

This study is one of the first to document the ongoing impact of chest dysphoria in transgender youths, defined as individuals assigned female at birth who have a masculine gender identity.

“Given these findings, professional guidelines and clinical practice should consider patients for chest surgery based on individual need rather than chronologic age,” said Johanna Olson-Kennedy, MD, of the division of adolescent medicine at Children’s Hospital Los Angeles, and her coauthors.

National guidelines on transgender health care are unclear as to whether minors should be referred for chest surgery because of a lack of data documenting effects of chest surgery in individuals younger than 18 years of age, Dr. Olson-Kennedy and her colleagues wrote in the study, published in JAMA Pediatrics.

To evaluate the discomfort and subsequent consequences of chest dysphoria, the researchers developed a 10-minute, 21-item survey based on Dr. Olson-Kennedy’s 11 years of experience providing care for transgender youth. It was reviewed by a small number of transmasculine youth and adults to determine whether the questions contained the elements of chest dysphoria effectively, used appropriate language, and was otherwise acceptable. From the survey, the researchers derived a chest dysphoria composite score of 0-51, with higher scores indicating increased distress.

Some of the items on the chest dysphoria survey included avoiding exercise, not seeking medical care, and not swimming because of “my chest,” and that taking a shower is difficult as is dating and physical intimacy.

The study included surveys completed by nonsurgical (n = 68) and postsurgical (n = 68) cohorts of transmasculine individuals aged 13-25 years.

The chest dysphoria composite score was significantly higher for minors and young adults who had not undergone chest reconstruction, compared with those who had undergone the procedure (29.6 vs. 3.3; P less than .001), the investigators reported.

Among transmasculine youths who had not undergone surgery, 94% perceived the procedure as very important, Dr. Olson-Kennedy and her coauthors noted.

 

Moreover, chest dysphoria increased by 0.33 points for every month that passed between a youth starting testosterone therapy and undergoing surgery, results of a linear regression analysis showed.

Most survey respondents reported that they were currently taking testosterone: 87% of the nonsurgical group and 97% of the postsurgical group.

Of the individuals who had undergone chest reconstruction, one (less than 1%) reported regretting the procedure “sometimes,” said Dr. Olson-Kennedy and her coauthors.

The mean age of survey participants was 17 (13-23 years) and 19 years (14-25 years) in the nonsurgical and postsurgical cohorts, respectively.Based on the results, Dr. Olson-Kennedy and her associates called for changes to clinical practice and to insurance plans, which sometimes require 12 months of continuous testosterone therapy prior to chest surgery. “Individualized, patient-centered care plans should be considered the standard of care for all transgender adolescents, and referrals should be made accordingly.”

Dr. Olson-Kennedy and her coauthors reported no conflicts of interest related to the study, which was funded by the Eunice Kennedy Shriver National Institute for Child Health and Human Development.

pdnews@frontlinemedcom.com

SOURCE: Olson-Kennedy J et al. JAMA Pediatrics. 2018 Mar 5. doi: 10.1001/jamapediatrics.2017.5440.

 

Transmasculine youths distressed by breast development who undergo chest reconstruction reported low levels of distress and almost none said they regretted the surgery, according to study results.

This study is one of the first to document the ongoing impact of chest dysphoria in transgender youths, defined as individuals assigned female at birth who have a masculine gender identity.

“Given these findings, professional guidelines and clinical practice should consider patients for chest surgery based on individual need rather than chronologic age,” said Johanna Olson-Kennedy, MD, of the division of adolescent medicine at Children’s Hospital Los Angeles, and her coauthors.

National guidelines on transgender health care are unclear as to whether minors should be referred for chest surgery because of a lack of data documenting effects of chest surgery in individuals younger than 18 years of age, Dr. Olson-Kennedy and her colleagues wrote in the study, published in JAMA Pediatrics.

To evaluate the discomfort and subsequent consequences of chest dysphoria, the researchers developed a 10-minute, 21-item survey based on Dr. Olson-Kennedy’s 11 years of experience providing care for transgender youth. It was reviewed by a small number of transmasculine youth and adults to determine whether the questions contained the elements of chest dysphoria effectively, used appropriate language, and was otherwise acceptable. From the survey, the researchers derived a chest dysphoria composite score of 0-51, with higher scores indicating increased distress.

Some of the items on the chest dysphoria survey included avoiding exercise, not seeking medical care, and not swimming because of “my chest,” and that taking a shower is difficult as is dating and physical intimacy.

The study included surveys completed by nonsurgical (n = 68) and postsurgical (n = 68) cohorts of transmasculine individuals aged 13-25 years.

The chest dysphoria composite score was significantly higher for minors and young adults who had not undergone chest reconstruction, compared with those who had undergone the procedure (29.6 vs. 3.3; P less than .001), the investigators reported.

Among transmasculine youths who had not undergone surgery, 94% perceived the procedure as very important, Dr. Olson-Kennedy and her coauthors noted.

 

Moreover, chest dysphoria increased by 0.33 points for every month that passed between a youth starting testosterone therapy and undergoing surgery, results of a linear regression analysis showed.

Most survey respondents reported that they were currently taking testosterone: 87% of the nonsurgical group and 97% of the postsurgical group.

Of the individuals who had undergone chest reconstruction, one (less than 1%) reported regretting the procedure “sometimes,” said Dr. Olson-Kennedy and her coauthors.

The mean age of survey participants was 17 (13-23 years) and 19 years (14-25 years) in the nonsurgical and postsurgical cohorts, respectively.Based on the results, Dr. Olson-Kennedy and her associates called for changes to clinical practice and to insurance plans, which sometimes require 12 months of continuous testosterone therapy prior to chest surgery. “Individualized, patient-centered care plans should be considered the standard of care for all transgender adolescents, and referrals should be made accordingly.”

Dr. Olson-Kennedy and her coauthors reported no conflicts of interest related to the study, which was funded by the Eunice Kennedy Shriver National Institute for Child Health and Human Development.

pdnews@frontlinemedcom.com

SOURCE: Olson-Kennedy J et al. JAMA Pediatrics. 2018 Mar 5. doi: 10.1001/jamapediatrics.2017.5440.

 

A reasonable number of patients with treatment-resistant epilepsy experienced a decrease in the frequency of seizures when treated with pharmaceutical-grade cannabidiol, according to findings from a systematic review.

The review, published online March 6 in the Journal of Neurology, Neurosurgery and Psychiatry, centers on 36 studies testing the use of cannabinoids as adjunctive treatments for treatment-resistant epilepsy, including six randomized controlled trials involving a total of 555 patients and 30 observational studies involving 2,865 patients.

IvelinRadkov/Thinkstock

Two randomized, controlled trials representing a total of 291 patients (one with 120 patients with Dravet syndrome and another with 171 patients with Lennox-Gastaut syndrome) found cannabidiol (CBD) treatment was 74% more likely than placebo to achieve a greater than 50% reduction in seizures. In the observational studies, nearly half (48.5%) of the 970 patients across a range of epilepsy subtypes achieved a 50% or greater reduction in seizures.

Emily Stockings, PhD, of the National Drug and Alcohol Research Centre at the University of New South Wales, Sydney, and her coauthors estimated that eight patients would need to receive CBD treatment to achieve a 50% reduction in seizures in one person. However, they also pointed out that the quality of the evidence was mixed.

 

“There is insufficient evidence from moderate-quality or high-quality studies to assess whether there is a treatment effect of Cannabis sativa, CBD:THC combinations, or oral cannabis extracts,” they wrote.

There were three randomized, controlled trials that also looked at complete seizure freedom, finding a sixfold higher likelihood of total seizure freedom with CBD, compared with placebo. However, the number needed to treat to achieve this was 171, and again, the quality of evidence was described as “mixed.”

Just over half of patients treated with CBD reported improved quality of life, and significantly more parents and caregivers of those treated with CBD said the patient’s overall condition had improved. The pooled estimates from observational studies suggested that 55.8% of patients experienced improvements in their quality of life when using cannabinoids.

Studies involving patients with Dravet syndrome reported the greatest improvements in quality of life, compared with studies involving a mix of epilepsy syndromes. However, the authors noted that the studies that involved Dravet syndrome patients were all case series in which every patient responded and suggested they should be interpreted with caution.

 

The authors said they were more confident of the benefits of CBD in children than in adults, because the more recent, larger, and better-conducted randomized, controlled trials focused on children and adolescents.

“In RCTs, and most of the non-RCTs, cannabinoids were used as an adjunctive therapy rather than as a standalone intervention, so at present, there is little evidence to support any recommendation that cannabinoids can be recommended as a replacement for current standard [antiepileptic drugs].”

The review also looked at the number of withdrawals, which they said could serve as an indicator of the tolerability and effectiveness of a treatment. The randomized, controlled trials showed no difference in withdrawal rates between patients on CBD and those on placebo, although CBD patients were more likely to withdraw because of adverse events.

There was a small but significant increase in the risk of adverse events with CBD, compared with placebo: particularly drowsiness, diarrhea, fatigue, and changes in appetite. There also was a higher incidence of serious adverse events (AEs), including status epilepticus and elevated aminotransferase levels.

 

“The fact that more patients withdrew or experienced AEs when receiving CBD than placebo indicates the need for clinicians and patients to weigh the risks and benefits of adding CBD to other AED [antiepileptic drug] treatment,” the authors wrote.

The study was supported by the Commonwealth Department of Health, the New South Wales Government Centre for Medicinal Cannabis Research and Innovation, the Victorian Department of Health and Human Services, and the Queensland Department of Health. Four authors were also supported by National Health and Medical Research Council grants. Three authors declared grants from the pharmaceutical industry, and one author has provided evidence to parliamentary committees on medical uses of cannabis in Australia and the United Kingdom, and is on the Australian Advisory Council on the Medicinal Use of Cannabis. No other conflicts of interest were declared.

SOURCE: Stockings E et al. J Neurol Neurosurg Psychiatry. 2018 Mar 6. doi: 10.1136/jnnp-2017-317168

 

A reasonable number of patients with treatment-resistant epilepsy experienced a decrease in the frequency of seizures when treated with pharmaceutical-grade cannabidiol, according to findings from a systematic review.

The review, published online March 6 in the Journal of Neurology, Neurosurgery and Psychiatry, centers on 36 studies testing the use of cannabinoids as adjunctive treatments for treatment-resistant epilepsy, including six randomized controlled trials involving a total of 555 patients and 30 observational studies involving 2,865 patients.

IvelinRadkov/Thinkstock

Two randomized, controlled trials representing a total of 291 patients (one with 120 patients with Dravet syndrome and another with 171 patients with Lennox-Gastaut syndrome) found cannabidiol (CBD) treatment was 74% more likely than placebo to achieve a greater than 50% reduction in seizures. In the observational studies, nearly half (48.5%) of the 970 patients across a range of epilepsy subtypes achieved a 50% or greater reduction in seizures.

Emily Stockings, PhD, of the National Drug and Alcohol Research Centre at the University of New South Wales, Sydney, and her coauthors estimated that eight patients would need to receive CBD treatment to achieve a 50% reduction in seizures in one person. However, they also pointed out that the quality of the evidence was mixed.

 

“There is insufficient evidence from moderate-quality or high-quality studies to assess whether there is a treatment effect of Cannabis sativa, CBD:THC combinations, or oral cannabis extracts,” they wrote.

There were three randomized, controlled trials that also looked at complete seizure freedom, finding a sixfold higher likelihood of total seizure freedom with CBD, compared with placebo. However, the number needed to treat to achieve this was 171, and again, the quality of evidence was described as “mixed.”

Just over half of patients treated with CBD reported improved quality of life, and significantly more parents and caregivers of those treated with CBD said the patient’s overall condition had improved. The pooled estimates from observational studies suggested that 55.8% of patients experienced improvements in their quality of life when using cannabinoids.

Studies involving patients with Dravet syndrome reported the greatest improvements in quality of life, compared with studies involving a mix of epilepsy syndromes. However, the authors noted that the studies that involved Dravet syndrome patients were all case series in which every patient responded and suggested they should be interpreted with caution.

 

The authors said they were more confident of the benefits of CBD in children than in adults, because the more recent, larger, and better-conducted randomized, controlled trials focused on children and adolescents.

“In RCTs, and most of the non-RCTs, cannabinoids were used as an adjunctive therapy rather than as a standalone intervention, so at present, there is little evidence to support any recommendation that cannabinoids can be recommended as a replacement for current standard [antiepileptic drugs].”

The review also looked at the number of withdrawals, which they said could serve as an indicator of the tolerability and effectiveness of a treatment. The randomized, controlled trials showed no difference in withdrawal rates between patients on CBD and those on placebo, although CBD patients were more likely to withdraw because of adverse events.

There was a small but significant increase in the risk of adverse events with CBD, compared with placebo: particularly drowsiness, diarrhea, fatigue, and changes in appetite. There also was a higher incidence of serious adverse events (AEs), including status epilepticus and elevated aminotransferase levels.

 

“The fact that more patients withdrew or experienced AEs when receiving CBD than placebo indicates the need for clinicians and patients to weigh the risks and benefits of adding CBD to other AED [antiepileptic drug] treatment,” the authors wrote.

The study was supported by the Commonwealth Department of Health, the New South Wales Government Centre for Medicinal Cannabis Research and Innovation, the Victorian Department of Health and Human Services, and the Queensland Department of Health. Four authors were also supported by National Health and Medical Research Council grants. Three authors declared grants from the pharmaceutical industry, and one author has provided evidence to parliamentary committees on medical uses of cannabis in Australia and the United Kingdom, and is on the Australian Advisory Council on the Medicinal Use of Cannabis. No other conflicts of interest were declared.

SOURCE: Stockings E et al. J Neurol Neurosurg Psychiatry. 2018 Mar 6. doi: 10.1136/jnnp-2017-317168

 

A reasonable number of patients with treatment-resistant epilepsy experienced a decrease in the frequency of seizures when treated with pharmaceutical-grade cannabidiol, according to findings from a systematic review.

The review, published online March 6 in the Journal of Neurology, Neurosurgery and Psychiatry, centers on 36 studies testing the use of cannabinoids as adjunctive treatments for treatment-resistant epilepsy, including six randomized controlled trials involving a total of 555 patients and 30 observational studies involving 2,865 patients.

IvelinRadkov/Thinkstock

Two randomized, controlled trials representing a total of 291 patients (one with 120 patients with Dravet syndrome and another with 171 patients with Lennox-Gastaut syndrome) found cannabidiol (CBD) treatment was 74% more likely than placebo to achieve a greater than 50% reduction in seizures. In the observational studies, nearly half (48.5%) of the 970 patients across a range of epilepsy subtypes achieved a 50% or greater reduction in seizures.

Emily Stockings, PhD, of the National Drug and Alcohol Research Centre at the University of New South Wales, Sydney, and her coauthors estimated that eight patients would need to receive CBD treatment to achieve a 50% reduction in seizures in one person. However, they also pointed out that the quality of the evidence was mixed.

 

“There is insufficient evidence from moderate-quality or high-quality studies to assess whether there is a treatment effect of Cannabis sativa, CBD:THC combinations, or oral cannabis extracts,” they wrote.

There were three randomized, controlled trials that also looked at complete seizure freedom, finding a sixfold higher likelihood of total seizure freedom with CBD, compared with placebo. However, the number needed to treat to achieve this was 171, and again, the quality of evidence was described as “mixed.”

Just over half of patients treated with CBD reported improved quality of life, and significantly more parents and caregivers of those treated with CBD said the patient’s overall condition had improved. The pooled estimates from observational studies suggested that 55.8% of patients experienced improvements in their quality of life when using cannabinoids.

Studies involving patients with Dravet syndrome reported the greatest improvements in quality of life, compared with studies involving a mix of epilepsy syndromes. However, the authors noted that the studies that involved Dravet syndrome patients were all case series in which every patient responded and suggested they should be interpreted with caution.

 

The authors said they were more confident of the benefits of CBD in children than in adults, because the more recent, larger, and better-conducted randomized, controlled trials focused on children and adolescents.

“In RCTs, and most of the non-RCTs, cannabinoids were used as an adjunctive therapy rather than as a standalone intervention, so at present, there is little evidence to support any recommendation that cannabinoids can be recommended as a replacement for current standard [antiepileptic drugs].”

The review also looked at the number of withdrawals, which they said could serve as an indicator of the tolerability and effectiveness of a treatment. The randomized, controlled trials showed no difference in withdrawal rates between patients on CBD and those on placebo, although CBD patients were more likely to withdraw because of adverse events.

There was a small but significant increase in the risk of adverse events with CBD, compared with placebo: particularly drowsiness, diarrhea, fatigue, and changes in appetite. There also was a higher incidence of serious adverse events (AEs), including status epilepticus and elevated aminotransferase levels.

 

“The fact that more patients withdrew or experienced AEs when receiving CBD than placebo indicates the need for clinicians and patients to weigh the risks and benefits of adding CBD to other AED [antiepileptic drug] treatment,” the authors wrote.

The study was supported by the Commonwealth Department of Health, the New South Wales Government Centre for Medicinal Cannabis Research and Innovation, the Victorian Department of Health and Human Services, and the Queensland Department of Health. Four authors were also supported by National Health and Medical Research Council grants. Three authors declared grants from the pharmaceutical industry, and one author has provided evidence to parliamentary committees on medical uses of cannabis in Australia and the United Kingdom, and is on the Australian Advisory Council on the Medicinal Use of Cannabis. No other conflicts of interest were declared.

SOURCE: Stockings E et al. J Neurol Neurosurg Psychiatry. 2018 Mar 6. doi: 10.1136/jnnp-2017-317168