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Treating Obesity May Reduce Pelvic Organ Prolapse Risk
TOPLINE:
People with central obesity (CO), characterized by excess fat around the abdomen, are at a greater risk for pelvic organ prolapse (POP), particularly those who are younger than 60 years or without a history of hysterectomy. Also, women who have overweight but do not have CO are at greater risk.
METHODOLOGY:
- Researchers conducted a prospective cohort study to estimate the association between CO and general obesity and the risk for POP in individuals using the UK Biobank.
- A total of 251,143 participants (median age, 57 years) without preexisting POP were included, of whom 60.9% were postmenopausal and 17.2% had undergone hysterectomy before enrollment.
- Participants were followed for a median duration of 13.8 years, and POP cases were identified using International Classification of Diseases, 10th Revision (ICD-10) codes.
- Waist circumference, height, and body weight were measured at enrollment for the calculation of waist/height ratio and body mass index (BMI); CO was defined as a waist/height ratio ≥ 0.5.
- The relative risk of POP for the various combinations of waist/height ratio and BMI was evaluated against the reference group (waist/height ratio < 0.5; BMI < 25) using Cox proportional hazards models.
TAKEAWAY:
- During the follow-up period, 9781 cases of POP were identified, of which 71.2% occurred in a single pelvic compartment.
- Around 21.7% of all POP cases were attributable to CO; 2% were attributable to being overweight without CO.
- The risk for POP was 48% higher in individuals with CO regardless of BMI (hazard ratio [HR], 1.48; 95% CI, 1.41-1.56) and 23% higher in those who had overweight without CO (HR, 1.23; 95% CI, 1.14-1.34).
- The association between POP and CO was further strengthened in individuals who were younger than 60 years and those without a history of hysterectomy.
IN PRACTICE:
“We found that waist/height ratio combined with BMI could help differentiate individuals with varying risks of prolapse more accurately. Among individuals within the same BMI category, waist/height ratio can vary, with those having a higher ratio generally facing a greater risk of POP, compared with those with a normal ratio. Therefore, they should not be grouped together based solely on a single measure of obesity. In addition, this combination can help identify more individuals at high risk for POP, compared with using either alone,” the study authors wrote.
SOURCE:
This study was led by Keyi Si, PhD, of Tongji University in Shanghai, China, and was published online in Obstetrics & Gynecology.
LIMITATIONS:
Differences in healthcare-seeking behavior could have biased the association between obesity and risk for POP, as individuals with obesity may have been less likely to notice or report symptoms of POP. The diagnosis of POP was according to ICD-10 codes rather than physical examination, which may have affected accuracy. Other limitations included missing data on delivery mode and history of constipation.
DISCLOSURES:
This study was supported by grants from the National Natural Science Foundation of China, the Science and Technology Commission of Shanghai Municipality, the Shanghai Hospital Development Center, and the Shanghai First Maternity and Infant Hospital. The authors reported no conflicts of interest.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
People with central obesity (CO), characterized by excess fat around the abdomen, are at a greater risk for pelvic organ prolapse (POP), particularly those who are younger than 60 years or without a history of hysterectomy. Also, women who have overweight but do not have CO are at greater risk.
METHODOLOGY:
- Researchers conducted a prospective cohort study to estimate the association between CO and general obesity and the risk for POP in individuals using the UK Biobank.
- A total of 251,143 participants (median age, 57 years) without preexisting POP were included, of whom 60.9% were postmenopausal and 17.2% had undergone hysterectomy before enrollment.
- Participants were followed for a median duration of 13.8 years, and POP cases were identified using International Classification of Diseases, 10th Revision (ICD-10) codes.
- Waist circumference, height, and body weight were measured at enrollment for the calculation of waist/height ratio and body mass index (BMI); CO was defined as a waist/height ratio ≥ 0.5.
- The relative risk of POP for the various combinations of waist/height ratio and BMI was evaluated against the reference group (waist/height ratio < 0.5; BMI < 25) using Cox proportional hazards models.
TAKEAWAY:
- During the follow-up period, 9781 cases of POP were identified, of which 71.2% occurred in a single pelvic compartment.
- Around 21.7% of all POP cases were attributable to CO; 2% were attributable to being overweight without CO.
- The risk for POP was 48% higher in individuals with CO regardless of BMI (hazard ratio [HR], 1.48; 95% CI, 1.41-1.56) and 23% higher in those who had overweight without CO (HR, 1.23; 95% CI, 1.14-1.34).
- The association between POP and CO was further strengthened in individuals who were younger than 60 years and those without a history of hysterectomy.
IN PRACTICE:
“We found that waist/height ratio combined with BMI could help differentiate individuals with varying risks of prolapse more accurately. Among individuals within the same BMI category, waist/height ratio can vary, with those having a higher ratio generally facing a greater risk of POP, compared with those with a normal ratio. Therefore, they should not be grouped together based solely on a single measure of obesity. In addition, this combination can help identify more individuals at high risk for POP, compared with using either alone,” the study authors wrote.
SOURCE:
This study was led by Keyi Si, PhD, of Tongji University in Shanghai, China, and was published online in Obstetrics & Gynecology.
LIMITATIONS:
Differences in healthcare-seeking behavior could have biased the association between obesity and risk for POP, as individuals with obesity may have been less likely to notice or report symptoms of POP. The diagnosis of POP was according to ICD-10 codes rather than physical examination, which may have affected accuracy. Other limitations included missing data on delivery mode and history of constipation.
DISCLOSURES:
This study was supported by grants from the National Natural Science Foundation of China, the Science and Technology Commission of Shanghai Municipality, the Shanghai Hospital Development Center, and the Shanghai First Maternity and Infant Hospital. The authors reported no conflicts of interest.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
People with central obesity (CO), characterized by excess fat around the abdomen, are at a greater risk for pelvic organ prolapse (POP), particularly those who are younger than 60 years or without a history of hysterectomy. Also, women who have overweight but do not have CO are at greater risk.
METHODOLOGY:
- Researchers conducted a prospective cohort study to estimate the association between CO and general obesity and the risk for POP in individuals using the UK Biobank.
- A total of 251,143 participants (median age, 57 years) without preexisting POP were included, of whom 60.9% were postmenopausal and 17.2% had undergone hysterectomy before enrollment.
- Participants were followed for a median duration of 13.8 years, and POP cases were identified using International Classification of Diseases, 10th Revision (ICD-10) codes.
- Waist circumference, height, and body weight were measured at enrollment for the calculation of waist/height ratio and body mass index (BMI); CO was defined as a waist/height ratio ≥ 0.5.
- The relative risk of POP for the various combinations of waist/height ratio and BMI was evaluated against the reference group (waist/height ratio < 0.5; BMI < 25) using Cox proportional hazards models.
TAKEAWAY:
- During the follow-up period, 9781 cases of POP were identified, of which 71.2% occurred in a single pelvic compartment.
- Around 21.7% of all POP cases were attributable to CO; 2% were attributable to being overweight without CO.
- The risk for POP was 48% higher in individuals with CO regardless of BMI (hazard ratio [HR], 1.48; 95% CI, 1.41-1.56) and 23% higher in those who had overweight without CO (HR, 1.23; 95% CI, 1.14-1.34).
- The association between POP and CO was further strengthened in individuals who were younger than 60 years and those without a history of hysterectomy.
IN PRACTICE:
“We found that waist/height ratio combined with BMI could help differentiate individuals with varying risks of prolapse more accurately. Among individuals within the same BMI category, waist/height ratio can vary, with those having a higher ratio generally facing a greater risk of POP, compared with those with a normal ratio. Therefore, they should not be grouped together based solely on a single measure of obesity. In addition, this combination can help identify more individuals at high risk for POP, compared with using either alone,” the study authors wrote.
SOURCE:
This study was led by Keyi Si, PhD, of Tongji University in Shanghai, China, and was published online in Obstetrics & Gynecology.
LIMITATIONS:
Differences in healthcare-seeking behavior could have biased the association between obesity and risk for POP, as individuals with obesity may have been less likely to notice or report symptoms of POP. The diagnosis of POP was according to ICD-10 codes rather than physical examination, which may have affected accuracy. Other limitations included missing data on delivery mode and history of constipation.
DISCLOSURES:
This study was supported by grants from the National Natural Science Foundation of China, the Science and Technology Commission of Shanghai Municipality, the Shanghai Hospital Development Center, and the Shanghai First Maternity and Infant Hospital. The authors reported no conflicts of interest.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
Knowledge Gaps About Obesity Medicine Seen in Primary Care
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
FROM OBESITY WEEK 2024
AI-Assisted Colonoscopy Linked to Higher Rate of Benign Lesion Removal
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
FROM ACG 2024
Short-Course Vasoconstrictors After EVL: Time for a New Standard of Care?
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
FROM ACG 2024
Sea Buckthorn
A member of the Elaeagnaceae family, Hippophae rhamnoides, better known as sea buckthorn, is a high-altitude wild shrub endemic to Europe and Asia with edible fruits and a lengthy record of use in traditional Chinese medicine.1-6 Used as a health supplement and consumed in the diet throughout the world,5 sea buckthorn berries, seeds, and leaves have been used in traditional medicine to treat burns/injuries, edema, hypertension, inflammation, skin grafts, ulcers, and wounds.4,7
This hardy plant is associated with a wide range of biologic activities, including anti-atherogenic, anti-atopic dermatitis, antibacterial, anticancer, antifungal, anti-inflammatory, antimicrobial, antioxidant, anti-psoriasis, anti-sebum, anti-stress, anti-tumor, cytoprotective, hepatoprotective, immunomodulatory, neuroprotective, radioprotective, and tissue regenerative functions.4,5,8-11
Key Constituents
Functional constituents identified in sea buckthorn include alkaloids, carotenoids, flavonoids, lignans, organic acids, phenolic acids, proanthocyanidins, polyunsaturated acids (including omega-3, -6, -7, and -9), steroids, tannins, terpenoids, and volatile oils, as well as nutritional compounds such as minerals, proteins, and vitamins.4,5,11 Sea buckthorn pericarp oil contains copious amounts of saturated palmitic acid (29%-36%) and omega-7 unsaturated palmitoleic acid (36%-48%), which fosters cutaneous and mucosal epithelialization, as well as linoleic (10%-12%) and oleic (4%-6%) acids.12,6 Significant amounts of carotenoids as well as alpha‐linolenic fatty acid (38%), linoleic (36%), oleic (13%), and palmitic (7%) acids are present in sea buckthorn seed oil.6
Polysaccharides
In an expansive review on the pharmacological activities of sea buckthorn polysaccharides, Teng and colleagues reported in April 2024 that 20 diverse polysaccharides have been culled from sea buckthorn and exhibited various healthy activities, including antioxidant, anti-fatigue, anti-inflammatory, anti-obesity, anti-tumor, hepatoprotective, hypoglycemic, and immunoregulation, and regulation of intestinal flora activities.1
Proanthocyanidins and Anti-Aging
In 2023, Liu and colleagues investigated the anti–skin aging impact of sea buckthorn proanthocyanidins in D-galactose-induced aging in mice given the known free radical scavenging activity of these compounds. They found the proanthocyanidins mitigated D-galactose-induced aging and can augment the total antioxidant capacity of the body. Sea buckthorn proanthocyanidins can further attenuate the effects of skin aging by regulating the TGF-beta1/Smads pathway and MMPs/TIMP system, thus amplifying collagen I and tropoelastin content.13
A year earlier, many of the same investigators assessed the possible protective activity of sea buckthorn proanthocyanidins against cutaneous aging engendered by oxidative stress from hydrogen peroxide. The compounds amplified superoxide dismutase and glutathione antioxidant functions. The extracts also fostered collagen I production in aging human skin fibroblasts via the TGF-beta1/Smads pathway and hindered collagen I degradation by regulating the MMPs/TIMPs system, which maintained extracellular matrix integrity. Senescent cell migration was also promoted with 100 mcg/mL of sea buckthorn proanthocyanidins. The researchers concluded that this sets the stage for investigating how sea buckthorn proanthocyanidins can be incorporated in cosmetic formulations.14 In a separate study, Liu and colleagues demonstrated that sea buckthorn proanthocyanidins can attenuate oxidative damage and protect mitochondrial function.9
Acne and Barrier Functions
The extracts of H rhamnoides and Cassia fistula in a combined formulation were found to be effective in lowering skin sebum content in humans with grade I and grade II acne vulgaris in a 2014 single-blind, randomized, placebo-controlled, split-face study with two groups of 25 patients each (aged 18-37 years).15 Khan and colleagues have also reported that a sea buckthorn oil-in-water emulsion improved barrier function in human skin as tested by a tewameter and corneometer (noninvasive probes) in 13 healthy males with a mean age of 27 ± 4.8 years.16
Anti-Aging, Antioxidant, Antibacterial, Skin-Whitening Activity
Zaman and colleagues reported in 2011 that results from an in vivo study of the effects of a sea buckthorn fruit extract topical cream on stratum corneum water content and transepidermal water loss indicated that the formulation enhanced cell surface integrin expression thus facilitating collagen contraction.17
In 2012, Khan and colleagues reported amelioration in skin elasticity, thus achieving an anti-aging result, from the use of a water-in-oil–based hydroalcoholic cream loaded with fruit extract of H rhamnoides, as measured with a Cutometer.18 The previous year, some of the same researchers reported that the antioxidants and flavonoids found in a topical sea buckthorn formulation could decrease cutaneous melanin and erythema levels.
More recently, Gęgotek and colleagues found that sea buckthorn seed oil prevented redox balance and lipid metabolism disturbances in skin fibroblasts and keratinocytes caused by UVA or UVB. They suggested that such findings point to the potential of this natural agent to confer anti-inflammatory properties and photoprotection to the skin.19
In 2020, Ivanišová and colleagues investigated the antioxidant and antimicrobial activities of H rhamnoides 100% oil, 100% juice, dry berries, and tea (dry berries, leaves, and twigs). They found that all of the studied sea buckthorn products displayed high antioxidant activity (identified through DPPH radical scavenging and molybdenum reducing antioxidant power tests). Sea buckthorn juice contained the highest total content of polyphenols, flavonoids, and carotenoids. All of the tested products also exhibited substantial antibacterial activity against the tested microbes.20
Burns and Wound Healing
In a preclinical study of the effects of sea buckthorn leaf extracts on wound healing in albino rats using an excision-punch wound model in 2005, Gupta and colleagues found that twice daily topical application of the aqueous leaf extract fostered wound healing. This was indicated by higher hydroxyproline and protein levels, a diminished wound area, and lower lipid peroxide levels. The investigators suggested that sea buckthorn may facilitate wound healing at least in part because of elevated antioxidant activity in the granulation tissue.3
A year later, Wang and colleagues reported on observations of using H rhamnoides oil, a traditional Chinese herbal medicine derived from sea buckthorn fruit, as a burn treatment. In the study, 151 burn patients received an H rhamnoides oil dressing (changed every other day until wound healing) that was covered with a disinfecting dressing. The dressing reduced swelling and effusion, and alleviated pain, with patients receiving the sea buckthorn dressing experiencing greater apparent exudation reduction, pain reduction, and more rapid epithelial cell growth and wound healing than controls (treated only with Vaseline gauze). The difference between the two groups was statistically significant.21
Conclusion
Sea buckthorn has been used for hundreds if not thousands of years in traditional medical applications, including for dermatologic purposes. Emerging data appear to support the use of this dynamic plant for consideration in dermatologic applications. As is often the case, much more work is necessary in the form of randomized controlled trials to determine the effectiveness of sea buckthorn formulations as well as the most appropriate avenues of research or uses for dermatologic application of this traditionally used botanical agent.
Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the division of cosmetic dermatology at the University of Miami in 1997. The third edition of her bestselling textbook, “Cosmetic Dermatology,” was published in 2022. Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Galderma, Johnson & Johnson, and Burt’s Bees. She is the CEO of Skin Type Solutions, a SaaS company used to generate skin care routines in office and as a e-commerce solution. Write to her at [email protected].
References
1. Teng H et al. J Ethnopharmacol. 2024 Apr 24;324:117809. doi: 10.1016/j.jep.2024.117809.
2. Wang Z et al. Int J Biol Macromol. 2024 Apr;263(Pt 1):130206. doi: 10.1016/j.ijbiomac.2024.130206.
3. Gupta A et al. Int J Low Extrem Wounds. 2005 Jun;4(2):88-92. doi: 10.1177/1534734605277401.
4. Pundir S et al. J Ethnopharmacol. 2021 Feb 10;266:113434. doi: 10.1016/j.jep.2020.113434.
5. Ma QG et al. J Agric Food Chem. 2023 Mar 29;71(12):4769-4788. doi: 10.1021/acs.jafc.2c06916.
6. Poljšak N et al. Phytother Res. 2020 Feb;34(2):254-269. doi: 10.1002/ptr.6524. doi: 10.1002/ptr.6524.
7. Upadhyay NK et al. Evid Based Complement Alternat Med. 2011;2011:659705. doi: 10.1093/ecam/nep189.
8. Suryakumar G, Gupta A. J Ethnopharmacol. 2011 Nov 18;138(2):268-78. doi: 10.1016/j.jep.2011.09.024.
9. Liu K et al. Front Pharmacol. 2022 Jul 8;13:914146. doi: 10.3389/fphar.2022.914146.
10. Akhtar N et al. J Pharm Bioallied Sci. 2010 Jan;2(1):13-7. doi: 10.4103/0975-7406.62698.
11. Ren R et al. RSC Adv. 2020 Dec 17;10(73):44654-44671. doi: 10.1039/d0ra06488b.
12. Ito H et al. Burns. 2014 May;40(3):511-9. doi: 10.1016/j.burns.2013.08.011.
13. Liu X et al. Food Sci Nutr. 2023 Dec 7;12(2):1082-1094. doi: 10.1002/fsn3.3823.
14. Liu X at al. Antioxidants (Basel). 2022 Sep 25;11(10):1900. doi: 10.3390/antiox11101900.
15. Khan BA, Akhtar N. Postepy Dermatol Alergol. 2014 Aug;31(4):229-234. doi: 10.5114/pdia.2014.40934.
16. Khan BA, Akhtar N. Pak J Pharm Sci. 2014 Nov;27(6):1919-22.
17. Khan AB et al. African J Pharm Pharmacol. 2011 Aug;5(8):1092-5.
18. Khan BA, Akhtar N, Braga VA. Trop J Pharm Res. 2012;11(6):955-62.
19. Gęgotek A et al. Antioxidants (Basel). 2018 Aug 23;7(9):110. doi: 10.3390/antiox7090110.
20. Ivanišová E et al. Acta Sci Pol Technol Aliment. 2020 Apr-Jun;19(2):195-205. doi: 10.17306/J.AFS.0809.
21. Wang ZY, Luo XL, He CP. Nan Fang Yi Ke Da Xue Xue Bao. 2006 Jan;26(1):124-5.
A member of the Elaeagnaceae family, Hippophae rhamnoides, better known as sea buckthorn, is a high-altitude wild shrub endemic to Europe and Asia with edible fruits and a lengthy record of use in traditional Chinese medicine.1-6 Used as a health supplement and consumed in the diet throughout the world,5 sea buckthorn berries, seeds, and leaves have been used in traditional medicine to treat burns/injuries, edema, hypertension, inflammation, skin grafts, ulcers, and wounds.4,7
This hardy plant is associated with a wide range of biologic activities, including anti-atherogenic, anti-atopic dermatitis, antibacterial, anticancer, antifungal, anti-inflammatory, antimicrobial, antioxidant, anti-psoriasis, anti-sebum, anti-stress, anti-tumor, cytoprotective, hepatoprotective, immunomodulatory, neuroprotective, radioprotective, and tissue regenerative functions.4,5,8-11
Key Constituents
Functional constituents identified in sea buckthorn include alkaloids, carotenoids, flavonoids, lignans, organic acids, phenolic acids, proanthocyanidins, polyunsaturated acids (including omega-3, -6, -7, and -9), steroids, tannins, terpenoids, and volatile oils, as well as nutritional compounds such as minerals, proteins, and vitamins.4,5,11 Sea buckthorn pericarp oil contains copious amounts of saturated palmitic acid (29%-36%) and omega-7 unsaturated palmitoleic acid (36%-48%), which fosters cutaneous and mucosal epithelialization, as well as linoleic (10%-12%) and oleic (4%-6%) acids.12,6 Significant amounts of carotenoids as well as alpha‐linolenic fatty acid (38%), linoleic (36%), oleic (13%), and palmitic (7%) acids are present in sea buckthorn seed oil.6
Polysaccharides
In an expansive review on the pharmacological activities of sea buckthorn polysaccharides, Teng and colleagues reported in April 2024 that 20 diverse polysaccharides have been culled from sea buckthorn and exhibited various healthy activities, including antioxidant, anti-fatigue, anti-inflammatory, anti-obesity, anti-tumor, hepatoprotective, hypoglycemic, and immunoregulation, and regulation of intestinal flora activities.1
Proanthocyanidins and Anti-Aging
In 2023, Liu and colleagues investigated the anti–skin aging impact of sea buckthorn proanthocyanidins in D-galactose-induced aging in mice given the known free radical scavenging activity of these compounds. They found the proanthocyanidins mitigated D-galactose-induced aging and can augment the total antioxidant capacity of the body. Sea buckthorn proanthocyanidins can further attenuate the effects of skin aging by regulating the TGF-beta1/Smads pathway and MMPs/TIMP system, thus amplifying collagen I and tropoelastin content.13
A year earlier, many of the same investigators assessed the possible protective activity of sea buckthorn proanthocyanidins against cutaneous aging engendered by oxidative stress from hydrogen peroxide. The compounds amplified superoxide dismutase and glutathione antioxidant functions. The extracts also fostered collagen I production in aging human skin fibroblasts via the TGF-beta1/Smads pathway and hindered collagen I degradation by regulating the MMPs/TIMPs system, which maintained extracellular matrix integrity. Senescent cell migration was also promoted with 100 mcg/mL of sea buckthorn proanthocyanidins. The researchers concluded that this sets the stage for investigating how sea buckthorn proanthocyanidins can be incorporated in cosmetic formulations.14 In a separate study, Liu and colleagues demonstrated that sea buckthorn proanthocyanidins can attenuate oxidative damage and protect mitochondrial function.9
Acne and Barrier Functions
The extracts of H rhamnoides and Cassia fistula in a combined formulation were found to be effective in lowering skin sebum content in humans with grade I and grade II acne vulgaris in a 2014 single-blind, randomized, placebo-controlled, split-face study with two groups of 25 patients each (aged 18-37 years).15 Khan and colleagues have also reported that a sea buckthorn oil-in-water emulsion improved barrier function in human skin as tested by a tewameter and corneometer (noninvasive probes) in 13 healthy males with a mean age of 27 ± 4.8 years.16
Anti-Aging, Antioxidant, Antibacterial, Skin-Whitening Activity
Zaman and colleagues reported in 2011 that results from an in vivo study of the effects of a sea buckthorn fruit extract topical cream on stratum corneum water content and transepidermal water loss indicated that the formulation enhanced cell surface integrin expression thus facilitating collagen contraction.17
In 2012, Khan and colleagues reported amelioration in skin elasticity, thus achieving an anti-aging result, from the use of a water-in-oil–based hydroalcoholic cream loaded with fruit extract of H rhamnoides, as measured with a Cutometer.18 The previous year, some of the same researchers reported that the antioxidants and flavonoids found in a topical sea buckthorn formulation could decrease cutaneous melanin and erythema levels.
More recently, Gęgotek and colleagues found that sea buckthorn seed oil prevented redox balance and lipid metabolism disturbances in skin fibroblasts and keratinocytes caused by UVA or UVB. They suggested that such findings point to the potential of this natural agent to confer anti-inflammatory properties and photoprotection to the skin.19
In 2020, Ivanišová and colleagues investigated the antioxidant and antimicrobial activities of H rhamnoides 100% oil, 100% juice, dry berries, and tea (dry berries, leaves, and twigs). They found that all of the studied sea buckthorn products displayed high antioxidant activity (identified through DPPH radical scavenging and molybdenum reducing antioxidant power tests). Sea buckthorn juice contained the highest total content of polyphenols, flavonoids, and carotenoids. All of the tested products also exhibited substantial antibacterial activity against the tested microbes.20
Burns and Wound Healing
In a preclinical study of the effects of sea buckthorn leaf extracts on wound healing in albino rats using an excision-punch wound model in 2005, Gupta and colleagues found that twice daily topical application of the aqueous leaf extract fostered wound healing. This was indicated by higher hydroxyproline and protein levels, a diminished wound area, and lower lipid peroxide levels. The investigators suggested that sea buckthorn may facilitate wound healing at least in part because of elevated antioxidant activity in the granulation tissue.3
A year later, Wang and colleagues reported on observations of using H rhamnoides oil, a traditional Chinese herbal medicine derived from sea buckthorn fruit, as a burn treatment. In the study, 151 burn patients received an H rhamnoides oil dressing (changed every other day until wound healing) that was covered with a disinfecting dressing. The dressing reduced swelling and effusion, and alleviated pain, with patients receiving the sea buckthorn dressing experiencing greater apparent exudation reduction, pain reduction, and more rapid epithelial cell growth and wound healing than controls (treated only with Vaseline gauze). The difference between the two groups was statistically significant.21
Conclusion
Sea buckthorn has been used for hundreds if not thousands of years in traditional medical applications, including for dermatologic purposes. Emerging data appear to support the use of this dynamic plant for consideration in dermatologic applications. As is often the case, much more work is necessary in the form of randomized controlled trials to determine the effectiveness of sea buckthorn formulations as well as the most appropriate avenues of research or uses for dermatologic application of this traditionally used botanical agent.
Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the division of cosmetic dermatology at the University of Miami in 1997. The third edition of her bestselling textbook, “Cosmetic Dermatology,” was published in 2022. Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Galderma, Johnson & Johnson, and Burt’s Bees. She is the CEO of Skin Type Solutions, a SaaS company used to generate skin care routines in office and as a e-commerce solution. Write to her at [email protected].
References
1. Teng H et al. J Ethnopharmacol. 2024 Apr 24;324:117809. doi: 10.1016/j.jep.2024.117809.
2. Wang Z et al. Int J Biol Macromol. 2024 Apr;263(Pt 1):130206. doi: 10.1016/j.ijbiomac.2024.130206.
3. Gupta A et al. Int J Low Extrem Wounds. 2005 Jun;4(2):88-92. doi: 10.1177/1534734605277401.
4. Pundir S et al. J Ethnopharmacol. 2021 Feb 10;266:113434. doi: 10.1016/j.jep.2020.113434.
5. Ma QG et al. J Agric Food Chem. 2023 Mar 29;71(12):4769-4788. doi: 10.1021/acs.jafc.2c06916.
6. Poljšak N et al. Phytother Res. 2020 Feb;34(2):254-269. doi: 10.1002/ptr.6524. doi: 10.1002/ptr.6524.
7. Upadhyay NK et al. Evid Based Complement Alternat Med. 2011;2011:659705. doi: 10.1093/ecam/nep189.
8. Suryakumar G, Gupta A. J Ethnopharmacol. 2011 Nov 18;138(2):268-78. doi: 10.1016/j.jep.2011.09.024.
9. Liu K et al. Front Pharmacol. 2022 Jul 8;13:914146. doi: 10.3389/fphar.2022.914146.
10. Akhtar N et al. J Pharm Bioallied Sci. 2010 Jan;2(1):13-7. doi: 10.4103/0975-7406.62698.
11. Ren R et al. RSC Adv. 2020 Dec 17;10(73):44654-44671. doi: 10.1039/d0ra06488b.
12. Ito H et al. Burns. 2014 May;40(3):511-9. doi: 10.1016/j.burns.2013.08.011.
13. Liu X et al. Food Sci Nutr. 2023 Dec 7;12(2):1082-1094. doi: 10.1002/fsn3.3823.
14. Liu X at al. Antioxidants (Basel). 2022 Sep 25;11(10):1900. doi: 10.3390/antiox11101900.
15. Khan BA, Akhtar N. Postepy Dermatol Alergol. 2014 Aug;31(4):229-234. doi: 10.5114/pdia.2014.40934.
16. Khan BA, Akhtar N. Pak J Pharm Sci. 2014 Nov;27(6):1919-22.
17. Khan AB et al. African J Pharm Pharmacol. 2011 Aug;5(8):1092-5.
18. Khan BA, Akhtar N, Braga VA. Trop J Pharm Res. 2012;11(6):955-62.
19. Gęgotek A et al. Antioxidants (Basel). 2018 Aug 23;7(9):110. doi: 10.3390/antiox7090110.
20. Ivanišová E et al. Acta Sci Pol Technol Aliment. 2020 Apr-Jun;19(2):195-205. doi: 10.17306/J.AFS.0809.
21. Wang ZY, Luo XL, He CP. Nan Fang Yi Ke Da Xue Xue Bao. 2006 Jan;26(1):124-5.
A member of the Elaeagnaceae family, Hippophae rhamnoides, better known as sea buckthorn, is a high-altitude wild shrub endemic to Europe and Asia with edible fruits and a lengthy record of use in traditional Chinese medicine.1-6 Used as a health supplement and consumed in the diet throughout the world,5 sea buckthorn berries, seeds, and leaves have been used in traditional medicine to treat burns/injuries, edema, hypertension, inflammation, skin grafts, ulcers, and wounds.4,7
This hardy plant is associated with a wide range of biologic activities, including anti-atherogenic, anti-atopic dermatitis, antibacterial, anticancer, antifungal, anti-inflammatory, antimicrobial, antioxidant, anti-psoriasis, anti-sebum, anti-stress, anti-tumor, cytoprotective, hepatoprotective, immunomodulatory, neuroprotective, radioprotective, and tissue regenerative functions.4,5,8-11
Key Constituents
Functional constituents identified in sea buckthorn include alkaloids, carotenoids, flavonoids, lignans, organic acids, phenolic acids, proanthocyanidins, polyunsaturated acids (including omega-3, -6, -7, and -9), steroids, tannins, terpenoids, and volatile oils, as well as nutritional compounds such as minerals, proteins, and vitamins.4,5,11 Sea buckthorn pericarp oil contains copious amounts of saturated palmitic acid (29%-36%) and omega-7 unsaturated palmitoleic acid (36%-48%), which fosters cutaneous and mucosal epithelialization, as well as linoleic (10%-12%) and oleic (4%-6%) acids.12,6 Significant amounts of carotenoids as well as alpha‐linolenic fatty acid (38%), linoleic (36%), oleic (13%), and palmitic (7%) acids are present in sea buckthorn seed oil.6
Polysaccharides
In an expansive review on the pharmacological activities of sea buckthorn polysaccharides, Teng and colleagues reported in April 2024 that 20 diverse polysaccharides have been culled from sea buckthorn and exhibited various healthy activities, including antioxidant, anti-fatigue, anti-inflammatory, anti-obesity, anti-tumor, hepatoprotective, hypoglycemic, and immunoregulation, and regulation of intestinal flora activities.1
Proanthocyanidins and Anti-Aging
In 2023, Liu and colleagues investigated the anti–skin aging impact of sea buckthorn proanthocyanidins in D-galactose-induced aging in mice given the known free radical scavenging activity of these compounds. They found the proanthocyanidins mitigated D-galactose-induced aging and can augment the total antioxidant capacity of the body. Sea buckthorn proanthocyanidins can further attenuate the effects of skin aging by regulating the TGF-beta1/Smads pathway and MMPs/TIMP system, thus amplifying collagen I and tropoelastin content.13
A year earlier, many of the same investigators assessed the possible protective activity of sea buckthorn proanthocyanidins against cutaneous aging engendered by oxidative stress from hydrogen peroxide. The compounds amplified superoxide dismutase and glutathione antioxidant functions. The extracts also fostered collagen I production in aging human skin fibroblasts via the TGF-beta1/Smads pathway and hindered collagen I degradation by regulating the MMPs/TIMPs system, which maintained extracellular matrix integrity. Senescent cell migration was also promoted with 100 mcg/mL of sea buckthorn proanthocyanidins. The researchers concluded that this sets the stage for investigating how sea buckthorn proanthocyanidins can be incorporated in cosmetic formulations.14 In a separate study, Liu and colleagues demonstrated that sea buckthorn proanthocyanidins can attenuate oxidative damage and protect mitochondrial function.9
Acne and Barrier Functions
The extracts of H rhamnoides and Cassia fistula in a combined formulation were found to be effective in lowering skin sebum content in humans with grade I and grade II acne vulgaris in a 2014 single-blind, randomized, placebo-controlled, split-face study with two groups of 25 patients each (aged 18-37 years).15 Khan and colleagues have also reported that a sea buckthorn oil-in-water emulsion improved barrier function in human skin as tested by a tewameter and corneometer (noninvasive probes) in 13 healthy males with a mean age of 27 ± 4.8 years.16
Anti-Aging, Antioxidant, Antibacterial, Skin-Whitening Activity
Zaman and colleagues reported in 2011 that results from an in vivo study of the effects of a sea buckthorn fruit extract topical cream on stratum corneum water content and transepidermal water loss indicated that the formulation enhanced cell surface integrin expression thus facilitating collagen contraction.17
In 2012, Khan and colleagues reported amelioration in skin elasticity, thus achieving an anti-aging result, from the use of a water-in-oil–based hydroalcoholic cream loaded with fruit extract of H rhamnoides, as measured with a Cutometer.18 The previous year, some of the same researchers reported that the antioxidants and flavonoids found in a topical sea buckthorn formulation could decrease cutaneous melanin and erythema levels.
More recently, Gęgotek and colleagues found that sea buckthorn seed oil prevented redox balance and lipid metabolism disturbances in skin fibroblasts and keratinocytes caused by UVA or UVB. They suggested that such findings point to the potential of this natural agent to confer anti-inflammatory properties and photoprotection to the skin.19
In 2020, Ivanišová and colleagues investigated the antioxidant and antimicrobial activities of H rhamnoides 100% oil, 100% juice, dry berries, and tea (dry berries, leaves, and twigs). They found that all of the studied sea buckthorn products displayed high antioxidant activity (identified through DPPH radical scavenging and molybdenum reducing antioxidant power tests). Sea buckthorn juice contained the highest total content of polyphenols, flavonoids, and carotenoids. All of the tested products also exhibited substantial antibacterial activity against the tested microbes.20
Burns and Wound Healing
In a preclinical study of the effects of sea buckthorn leaf extracts on wound healing in albino rats using an excision-punch wound model in 2005, Gupta and colleagues found that twice daily topical application of the aqueous leaf extract fostered wound healing. This was indicated by higher hydroxyproline and protein levels, a diminished wound area, and lower lipid peroxide levels. The investigators suggested that sea buckthorn may facilitate wound healing at least in part because of elevated antioxidant activity in the granulation tissue.3
A year later, Wang and colleagues reported on observations of using H rhamnoides oil, a traditional Chinese herbal medicine derived from sea buckthorn fruit, as a burn treatment. In the study, 151 burn patients received an H rhamnoides oil dressing (changed every other day until wound healing) that was covered with a disinfecting dressing. The dressing reduced swelling and effusion, and alleviated pain, with patients receiving the sea buckthorn dressing experiencing greater apparent exudation reduction, pain reduction, and more rapid epithelial cell growth and wound healing than controls (treated only with Vaseline gauze). The difference between the two groups was statistically significant.21
Conclusion
Sea buckthorn has been used for hundreds if not thousands of years in traditional medical applications, including for dermatologic purposes. Emerging data appear to support the use of this dynamic plant for consideration in dermatologic applications. As is often the case, much more work is necessary in the form of randomized controlled trials to determine the effectiveness of sea buckthorn formulations as well as the most appropriate avenues of research or uses for dermatologic application of this traditionally used botanical agent.
Dr. Baumann is a private practice dermatologist, researcher, author, and entrepreneur in Miami. She founded the division of cosmetic dermatology at the University of Miami in 1997. The third edition of her bestselling textbook, “Cosmetic Dermatology,” was published in 2022. Dr. Baumann has received funding for advisory boards and/or clinical research trials from Allergan, Galderma, Johnson & Johnson, and Burt’s Bees. She is the CEO of Skin Type Solutions, a SaaS company used to generate skin care routines in office and as a e-commerce solution. Write to her at [email protected].
References
1. Teng H et al. J Ethnopharmacol. 2024 Apr 24;324:117809. doi: 10.1016/j.jep.2024.117809.
2. Wang Z et al. Int J Biol Macromol. 2024 Apr;263(Pt 1):130206. doi: 10.1016/j.ijbiomac.2024.130206.
3. Gupta A et al. Int J Low Extrem Wounds. 2005 Jun;4(2):88-92. doi: 10.1177/1534734605277401.
4. Pundir S et al. J Ethnopharmacol. 2021 Feb 10;266:113434. doi: 10.1016/j.jep.2020.113434.
5. Ma QG et al. J Agric Food Chem. 2023 Mar 29;71(12):4769-4788. doi: 10.1021/acs.jafc.2c06916.
6. Poljšak N et al. Phytother Res. 2020 Feb;34(2):254-269. doi: 10.1002/ptr.6524. doi: 10.1002/ptr.6524.
7. Upadhyay NK et al. Evid Based Complement Alternat Med. 2011;2011:659705. doi: 10.1093/ecam/nep189.
8. Suryakumar G, Gupta A. J Ethnopharmacol. 2011 Nov 18;138(2):268-78. doi: 10.1016/j.jep.2011.09.024.
9. Liu K et al. Front Pharmacol. 2022 Jul 8;13:914146. doi: 10.3389/fphar.2022.914146.
10. Akhtar N et al. J Pharm Bioallied Sci. 2010 Jan;2(1):13-7. doi: 10.4103/0975-7406.62698.
11. Ren R et al. RSC Adv. 2020 Dec 17;10(73):44654-44671. doi: 10.1039/d0ra06488b.
12. Ito H et al. Burns. 2014 May;40(3):511-9. doi: 10.1016/j.burns.2013.08.011.
13. Liu X et al. Food Sci Nutr. 2023 Dec 7;12(2):1082-1094. doi: 10.1002/fsn3.3823.
14. Liu X at al. Antioxidants (Basel). 2022 Sep 25;11(10):1900. doi: 10.3390/antiox11101900.
15. Khan BA, Akhtar N. Postepy Dermatol Alergol. 2014 Aug;31(4):229-234. doi: 10.5114/pdia.2014.40934.
16. Khan BA, Akhtar N. Pak J Pharm Sci. 2014 Nov;27(6):1919-22.
17. Khan AB et al. African J Pharm Pharmacol. 2011 Aug;5(8):1092-5.
18. Khan BA, Akhtar N, Braga VA. Trop J Pharm Res. 2012;11(6):955-62.
19. Gęgotek A et al. Antioxidants (Basel). 2018 Aug 23;7(9):110. doi: 10.3390/antiox7090110.
20. Ivanišová E et al. Acta Sci Pol Technol Aliment. 2020 Apr-Jun;19(2):195-205. doi: 10.17306/J.AFS.0809.
21. Wang ZY, Luo XL, He CP. Nan Fang Yi Ke Da Xue Xue Bao. 2006 Jan;26(1):124-5.
Experts Challenge New Diagnostic Criteria for Alzheimer’s disease
In a paper published online in JAMA Neurology, the International Working Group (IWG), which includes 46 experts from 17 countries, is recommending that the diagnosis of Alzheimer’s disease be limited to individuals with mild cognitive impairment or dementia and not be applied to cognitively normal individuals with Alzheimer’s disease biomarkers such as amyloid-beta 42/40 or p-tau.
Clinicians should be “very careful” about using the “A” word (Alzheimer’s) for cognitively unimpaired people with Alzheimer’s disease biomarkers, said the paper’s first author Bruno Dubois, MD, professor of neurology, Sorbonne University and Department of Neurology, Pitié-Salpêtrière Hospital, Paris, France.
Providing an Alzheimer’s disease diagnosis to those who have a high chance of never developing cognitive impairment can be psychologically harmful, said Dubois.
“It’s not something small like telling someone they have a fever. Just imagine you’re 65 years old and are amyloid positive, and you’re told you have Alzheimer’s disease. It affects the decisions you make for the rest of your life and changes your vision of your future, even though you may never develop the disease,” he added.
Divergent View
The IWG’s perspective on Alzheimer’s disease contrasts with a recent proposal from the Alzheimer’s Association. The Alzheimer’s Association criteria suggest that Alzheimer’s disease should be regarded solely as a biological entity, which could include cognitively normal individuals with one core Alzheimer’s disease biomarker.
The IWG noted that its concerns regarding the application of a purely biological definition of Alzheimer’s disease in clinical practice prompted the group to consider updating its guidelines, potentially offering “an alternative definitional view of Alzheimer’s disease as a clinical-biological construct for clinical use.”
The group conducted a PubMed search for relevant Alzheimer’s disease articles, and included references, published between July 2020 and March 2024. The research showed the majority of biomarker-positive, cognitively normal individuals will not become symptomatic during their lifetime.
The risk of a 55-year-old who is amyloid positive developing Alzheimer’s disease is not that much higher than that for an individual of a similar age who is amyloid negative, Dubois noted. “There’s an 83% chance that person will never develop Alzheimer’s disease.”
Disclosing a diagnosis of Alzheimer’s disease to cognitively normal people with only one core Alzheimer’s disease biomarker represents “the most problematic implication of a purely biological definition of the disease,” the authors noted.
“A biomarker is a marker of pathology, not a biomarker of disease,” said Dubois, adding that a person may have markers for several different brain diseases.
The IWG recommends the following nomenclature: At risk for Alzheimer’s disease for those with Alzheimer’s disease biomarkers but low lifetime risk and presymptomatic Alzheimer’s disease for those with Alzheimer’s disease biomarkers with a very high lifetime risk for progression such as individuals with autosomal dominant genetic mutations and other distinct biomarker profiles that put them at extremely high lifetime risk of developing the disease.
Dubois emphasized the difference between those showing typical Alzheimer’s disease symptoms with positive biomarkers who should be considered to have the disease and those with positive biomarkers but no typical Alzheimer’s disease symptoms who should be considered at risk.
This is an important distinction as it affects research approaches and assessment of risks, he said.
For low-risk asymptomatic individuals, the IWG does not recommend routine diagnostic testing outside of the research setting. “There’s no reason to send a 65-year-old cognitively normal subject off to collect biomarker information,” said Dubois.
He reiterated the importance of clinicians using appropriate and sensitive language surrounding Alzheimer’s disease when face to face with patients. This issue “is not purely semantic; this is real life.”
For these patients in the clinical setting, “we have to be very careful about proposing treatments that may have side effects,” he said.
However, this does not mean asymptomatic at-risk people should not be studied to determine what pharmacological interventions might prevent or delay the onset of clinical disease, he noted.
Presymptomatic individuals who are at a high risk of developing Alzheimer’s disease “should be the target for clinical trials in the future” to determine best ways to delay the conversion to Alzheimer’s disease, he said.
The main focus of such research should be to better understand the “biomarker pattern profile” that is associated with a high risk of developing Alzheimer’s disease, said Dubois.
Plea for Unity
In an accompanying editorial, Ronald C. Petersen, PhD, MD, director, Mayo Clinic Alzheimer’s Disease Research Center and Mayo Clinic Study of Aging, Rochester, Minnesota, and colleagues outline the difference between the IWG and Alzheimer’s Association positions.
As the IWG uses Alzheimer’s disease to define those with cognitive impairment and the Alzheimer’s Association group uses Alzheimer’s disease to define those with the pathology of the disease, the field is now at a crossroads. “Do we name the disease before clinical symptoms?” they asked.
They note that Alzheimer’s Association criteria distinguish between a disease and an illness, whereas the IWG does not. “As such, although the primary disagreement between the groups is semantic, the ramifications of the labeling can be significant.”
It is “incumbent” that the field “come together” on an Alzheimer’s disease definition, the editorial concluded. “Neither the Alzheimer’s Association or IWG documents are appropriate to serve as a guide for how to apply biomarkers in a clinical setting. Appropriate-use criteria are needed to form a bridge between biological frameworks and real-world clinical practice so we can all maximally help all of our patients with this disorder.”
In a comment, Reisa Sperling, MD, professor of neurology, Harvard Medical School, and director, Center for Alzheimer Research and Treatment, Brigham and Women’s Hospital and Massachusetts General Hospital, all in Boston, who is part of the Alzheimer’s Association work group that published the revised criteria for diagnosis and staging of Alzheimer’s disease, likened Alzheimer’s disease, which begins in the brain many years before dementia onset, to cardiovascular disease in that it involves multiple processes. She noted the World Health Organization classifies cardiovascular disease as a “disease” prior to clinical manifestations such as stroke and myocardial infarction.
“If someone has Alzheimer’s disease pathology in their brain, they are at risk for dementia or clinical manifestations of the disease — just like vascular disease quantifies the risk of stroke or heart attack, not risk of developing ‘vascular disease’ if the underlying vascular disease is already present,” said Sperling.
A large part of the controversy is related to terminology and the “stigma” of the “A” word in the same way there used to be fear around using the “C” word — cancer, said Sperling.
“Once people began talking about cancer publicly as a potentially treatable disease and began getting screened and diagnosed before symptoms of cancer were manifest, this has had a tremendous impact on public health.”
She clarified that her work group does not recommend screening asymptomatic people with Alzheimer’s disease biomarkers. “We actually need to prove that treating at the preclinical stage of the disease is able to prevent clinical impairment and dementia,” she said, adding “hopefully, we are getting closer to this.”
Dubois reported no relevant disclosures. Petersen reported receiving personal fees from Roche, Genentech, Eli Lilly and Company, Eisai, and Novo Nordisk outside the submitted work and royalties from Oxford University Press, UpToDate, and Medscape educational activities.
A version of this article appeared on Medscape.com.
In a paper published online in JAMA Neurology, the International Working Group (IWG), which includes 46 experts from 17 countries, is recommending that the diagnosis of Alzheimer’s disease be limited to individuals with mild cognitive impairment or dementia and not be applied to cognitively normal individuals with Alzheimer’s disease biomarkers such as amyloid-beta 42/40 or p-tau.
Clinicians should be “very careful” about using the “A” word (Alzheimer’s) for cognitively unimpaired people with Alzheimer’s disease biomarkers, said the paper’s first author Bruno Dubois, MD, professor of neurology, Sorbonne University and Department of Neurology, Pitié-Salpêtrière Hospital, Paris, France.
Providing an Alzheimer’s disease diagnosis to those who have a high chance of never developing cognitive impairment can be psychologically harmful, said Dubois.
“It’s not something small like telling someone they have a fever. Just imagine you’re 65 years old and are amyloid positive, and you’re told you have Alzheimer’s disease. It affects the decisions you make for the rest of your life and changes your vision of your future, even though you may never develop the disease,” he added.
Divergent View
The IWG’s perspective on Alzheimer’s disease contrasts with a recent proposal from the Alzheimer’s Association. The Alzheimer’s Association criteria suggest that Alzheimer’s disease should be regarded solely as a biological entity, which could include cognitively normal individuals with one core Alzheimer’s disease biomarker.
The IWG noted that its concerns regarding the application of a purely biological definition of Alzheimer’s disease in clinical practice prompted the group to consider updating its guidelines, potentially offering “an alternative definitional view of Alzheimer’s disease as a clinical-biological construct for clinical use.”
The group conducted a PubMed search for relevant Alzheimer’s disease articles, and included references, published between July 2020 and March 2024. The research showed the majority of biomarker-positive, cognitively normal individuals will not become symptomatic during their lifetime.
The risk of a 55-year-old who is amyloid positive developing Alzheimer’s disease is not that much higher than that for an individual of a similar age who is amyloid negative, Dubois noted. “There’s an 83% chance that person will never develop Alzheimer’s disease.”
Disclosing a diagnosis of Alzheimer’s disease to cognitively normal people with only one core Alzheimer’s disease biomarker represents “the most problematic implication of a purely biological definition of the disease,” the authors noted.
“A biomarker is a marker of pathology, not a biomarker of disease,” said Dubois, adding that a person may have markers for several different brain diseases.
The IWG recommends the following nomenclature: At risk for Alzheimer’s disease for those with Alzheimer’s disease biomarkers but low lifetime risk and presymptomatic Alzheimer’s disease for those with Alzheimer’s disease biomarkers with a very high lifetime risk for progression such as individuals with autosomal dominant genetic mutations and other distinct biomarker profiles that put them at extremely high lifetime risk of developing the disease.
Dubois emphasized the difference between those showing typical Alzheimer’s disease symptoms with positive biomarkers who should be considered to have the disease and those with positive biomarkers but no typical Alzheimer’s disease symptoms who should be considered at risk.
This is an important distinction as it affects research approaches and assessment of risks, he said.
For low-risk asymptomatic individuals, the IWG does not recommend routine diagnostic testing outside of the research setting. “There’s no reason to send a 65-year-old cognitively normal subject off to collect biomarker information,” said Dubois.
He reiterated the importance of clinicians using appropriate and sensitive language surrounding Alzheimer’s disease when face to face with patients. This issue “is not purely semantic; this is real life.”
For these patients in the clinical setting, “we have to be very careful about proposing treatments that may have side effects,” he said.
However, this does not mean asymptomatic at-risk people should not be studied to determine what pharmacological interventions might prevent or delay the onset of clinical disease, he noted.
Presymptomatic individuals who are at a high risk of developing Alzheimer’s disease “should be the target for clinical trials in the future” to determine best ways to delay the conversion to Alzheimer’s disease, he said.
The main focus of such research should be to better understand the “biomarker pattern profile” that is associated with a high risk of developing Alzheimer’s disease, said Dubois.
Plea for Unity
In an accompanying editorial, Ronald C. Petersen, PhD, MD, director, Mayo Clinic Alzheimer’s Disease Research Center and Mayo Clinic Study of Aging, Rochester, Minnesota, and colleagues outline the difference between the IWG and Alzheimer’s Association positions.
As the IWG uses Alzheimer’s disease to define those with cognitive impairment and the Alzheimer’s Association group uses Alzheimer’s disease to define those with the pathology of the disease, the field is now at a crossroads. “Do we name the disease before clinical symptoms?” they asked.
They note that Alzheimer’s Association criteria distinguish between a disease and an illness, whereas the IWG does not. “As such, although the primary disagreement between the groups is semantic, the ramifications of the labeling can be significant.”
It is “incumbent” that the field “come together” on an Alzheimer’s disease definition, the editorial concluded. “Neither the Alzheimer’s Association or IWG documents are appropriate to serve as a guide for how to apply biomarkers in a clinical setting. Appropriate-use criteria are needed to form a bridge between biological frameworks and real-world clinical practice so we can all maximally help all of our patients with this disorder.”
In a comment, Reisa Sperling, MD, professor of neurology, Harvard Medical School, and director, Center for Alzheimer Research and Treatment, Brigham and Women’s Hospital and Massachusetts General Hospital, all in Boston, who is part of the Alzheimer’s Association work group that published the revised criteria for diagnosis and staging of Alzheimer’s disease, likened Alzheimer’s disease, which begins in the brain many years before dementia onset, to cardiovascular disease in that it involves multiple processes. She noted the World Health Organization classifies cardiovascular disease as a “disease” prior to clinical manifestations such as stroke and myocardial infarction.
“If someone has Alzheimer’s disease pathology in their brain, they are at risk for dementia or clinical manifestations of the disease — just like vascular disease quantifies the risk of stroke or heart attack, not risk of developing ‘vascular disease’ if the underlying vascular disease is already present,” said Sperling.
A large part of the controversy is related to terminology and the “stigma” of the “A” word in the same way there used to be fear around using the “C” word — cancer, said Sperling.
“Once people began talking about cancer publicly as a potentially treatable disease and began getting screened and diagnosed before symptoms of cancer were manifest, this has had a tremendous impact on public health.”
She clarified that her work group does not recommend screening asymptomatic people with Alzheimer’s disease biomarkers. “We actually need to prove that treating at the preclinical stage of the disease is able to prevent clinical impairment and dementia,” she said, adding “hopefully, we are getting closer to this.”
Dubois reported no relevant disclosures. Petersen reported receiving personal fees from Roche, Genentech, Eli Lilly and Company, Eisai, and Novo Nordisk outside the submitted work and royalties from Oxford University Press, UpToDate, and Medscape educational activities.
A version of this article appeared on Medscape.com.
In a paper published online in JAMA Neurology, the International Working Group (IWG), which includes 46 experts from 17 countries, is recommending that the diagnosis of Alzheimer’s disease be limited to individuals with mild cognitive impairment or dementia and not be applied to cognitively normal individuals with Alzheimer’s disease biomarkers such as amyloid-beta 42/40 or p-tau.
Clinicians should be “very careful” about using the “A” word (Alzheimer’s) for cognitively unimpaired people with Alzheimer’s disease biomarkers, said the paper’s first author Bruno Dubois, MD, professor of neurology, Sorbonne University and Department of Neurology, Pitié-Salpêtrière Hospital, Paris, France.
Providing an Alzheimer’s disease diagnosis to those who have a high chance of never developing cognitive impairment can be psychologically harmful, said Dubois.
“It’s not something small like telling someone they have a fever. Just imagine you’re 65 years old and are amyloid positive, and you’re told you have Alzheimer’s disease. It affects the decisions you make for the rest of your life and changes your vision of your future, even though you may never develop the disease,” he added.
Divergent View
The IWG’s perspective on Alzheimer’s disease contrasts with a recent proposal from the Alzheimer’s Association. The Alzheimer’s Association criteria suggest that Alzheimer’s disease should be regarded solely as a biological entity, which could include cognitively normal individuals with one core Alzheimer’s disease biomarker.
The IWG noted that its concerns regarding the application of a purely biological definition of Alzheimer’s disease in clinical practice prompted the group to consider updating its guidelines, potentially offering “an alternative definitional view of Alzheimer’s disease as a clinical-biological construct for clinical use.”
The group conducted a PubMed search for relevant Alzheimer’s disease articles, and included references, published between July 2020 and March 2024. The research showed the majority of biomarker-positive, cognitively normal individuals will not become symptomatic during their lifetime.
The risk of a 55-year-old who is amyloid positive developing Alzheimer’s disease is not that much higher than that for an individual of a similar age who is amyloid negative, Dubois noted. “There’s an 83% chance that person will never develop Alzheimer’s disease.”
Disclosing a diagnosis of Alzheimer’s disease to cognitively normal people with only one core Alzheimer’s disease biomarker represents “the most problematic implication of a purely biological definition of the disease,” the authors noted.
“A biomarker is a marker of pathology, not a biomarker of disease,” said Dubois, adding that a person may have markers for several different brain diseases.
The IWG recommends the following nomenclature: At risk for Alzheimer’s disease for those with Alzheimer’s disease biomarkers but low lifetime risk and presymptomatic Alzheimer’s disease for those with Alzheimer’s disease biomarkers with a very high lifetime risk for progression such as individuals with autosomal dominant genetic mutations and other distinct biomarker profiles that put them at extremely high lifetime risk of developing the disease.
Dubois emphasized the difference between those showing typical Alzheimer’s disease symptoms with positive biomarkers who should be considered to have the disease and those with positive biomarkers but no typical Alzheimer’s disease symptoms who should be considered at risk.
This is an important distinction as it affects research approaches and assessment of risks, he said.
For low-risk asymptomatic individuals, the IWG does not recommend routine diagnostic testing outside of the research setting. “There’s no reason to send a 65-year-old cognitively normal subject off to collect biomarker information,” said Dubois.
He reiterated the importance of clinicians using appropriate and sensitive language surrounding Alzheimer’s disease when face to face with patients. This issue “is not purely semantic; this is real life.”
For these patients in the clinical setting, “we have to be very careful about proposing treatments that may have side effects,” he said.
However, this does not mean asymptomatic at-risk people should not be studied to determine what pharmacological interventions might prevent or delay the onset of clinical disease, he noted.
Presymptomatic individuals who are at a high risk of developing Alzheimer’s disease “should be the target for clinical trials in the future” to determine best ways to delay the conversion to Alzheimer’s disease, he said.
The main focus of such research should be to better understand the “biomarker pattern profile” that is associated with a high risk of developing Alzheimer’s disease, said Dubois.
Plea for Unity
In an accompanying editorial, Ronald C. Petersen, PhD, MD, director, Mayo Clinic Alzheimer’s Disease Research Center and Mayo Clinic Study of Aging, Rochester, Minnesota, and colleagues outline the difference between the IWG and Alzheimer’s Association positions.
As the IWG uses Alzheimer’s disease to define those with cognitive impairment and the Alzheimer’s Association group uses Alzheimer’s disease to define those with the pathology of the disease, the field is now at a crossroads. “Do we name the disease before clinical symptoms?” they asked.
They note that Alzheimer’s Association criteria distinguish between a disease and an illness, whereas the IWG does not. “As such, although the primary disagreement between the groups is semantic, the ramifications of the labeling can be significant.”
It is “incumbent” that the field “come together” on an Alzheimer’s disease definition, the editorial concluded. “Neither the Alzheimer’s Association or IWG documents are appropriate to serve as a guide for how to apply biomarkers in a clinical setting. Appropriate-use criteria are needed to form a bridge between biological frameworks and real-world clinical practice so we can all maximally help all of our patients with this disorder.”
In a comment, Reisa Sperling, MD, professor of neurology, Harvard Medical School, and director, Center for Alzheimer Research and Treatment, Brigham and Women’s Hospital and Massachusetts General Hospital, all in Boston, who is part of the Alzheimer’s Association work group that published the revised criteria for diagnosis and staging of Alzheimer’s disease, likened Alzheimer’s disease, which begins in the brain many years before dementia onset, to cardiovascular disease in that it involves multiple processes. She noted the World Health Organization classifies cardiovascular disease as a “disease” prior to clinical manifestations such as stroke and myocardial infarction.
“If someone has Alzheimer’s disease pathology in their brain, they are at risk for dementia or clinical manifestations of the disease — just like vascular disease quantifies the risk of stroke or heart attack, not risk of developing ‘vascular disease’ if the underlying vascular disease is already present,” said Sperling.
A large part of the controversy is related to terminology and the “stigma” of the “A” word in the same way there used to be fear around using the “C” word — cancer, said Sperling.
“Once people began talking about cancer publicly as a potentially treatable disease and began getting screened and diagnosed before symptoms of cancer were manifest, this has had a tremendous impact on public health.”
She clarified that her work group does not recommend screening asymptomatic people with Alzheimer’s disease biomarkers. “We actually need to prove that treating at the preclinical stage of the disease is able to prevent clinical impairment and dementia,” she said, adding “hopefully, we are getting closer to this.”
Dubois reported no relevant disclosures. Petersen reported receiving personal fees from Roche, Genentech, Eli Lilly and Company, Eisai, and Novo Nordisk outside the submitted work and royalties from Oxford University Press, UpToDate, and Medscape educational activities.
A version of this article appeared on Medscape.com.
From JAMA Neurology
How Extreme Rainfall Amplifies Health Risks
Climate change is intensifying the variability of precipitation caused by extreme daily and overall rainfall events. Awareness of the effects of these events is crucial for understanding the complex health consequences of climate change. Physicians have often advised their patients to move to a better climate, and when they did, the recommendation was rarely based on precise scientific knowledge. However, the benefits of changing environments were often so evident that they were indisputable.
Today, advanced models, satellite imagery, and biological approaches such as environmental epigenetics are enhancing our understanding of health risks related to climate change.
Extreme Rainfall and Health
The increase in precipitation variability is linked to climate warming, which leads to higher atmospheric humidity and extreme rainfall events. These manifestations can cause rapid weather changes, increasing interactions with harmful aerosols and raising the risk for various cardiovascular and respiratory conditions. However, a full understanding of the association between rain and health has been hindered by conflicting results and methodological issues (limited geographical locations and short observation durations) in studies.
The association between rainfall intensity and health effects is likely nonlinear. Moderate precipitation can mitigate summer heat and help reduce air pollution, an effect that may lower some environmental health risks. Conversely, intense, low-frequency, short-duration rainfall events can have particularly harmful effects on health, as such events can trigger rapid weather changes, increased proliferation of pathogens, and a rise in the risk of various pollutants, potentially exacerbating health conditions.
Rain and Mortality
Using an intensity-duration-frequency model of three rainfall indices (high intensity, low frequency, short duration), a study published in October 2024 combined these with mortality data from 34 countries or regions. Researchers estimated associations between mortality (all cause, cardiovascular, and respiratory) and rainfall events with different return periods (the average time expected before an extreme event of a certain magnitude occurs again) and crucial effect modifiers, including climatic, socioeconomic, and urban environmental conditions.
The analysis included 109,954,744 deaths from all causes; 31,164,161 cardiovascular deaths; and 11,817,278 respiratory deaths. During the study period, from 1980 to 2020, a total of 50,913 rainfall events with a 1-year return period, 8362 events with a 2-year return period, and 3301 events with a 5-year return period were identified.
The most significant finding was a global positive association between all-cause mortality and extreme rainfall events with a 5-year return period. One day of extreme rainfall with a 5-year return period was associated with a cumulative relative risk (RRc) of 1.08 (95% CI, 1.05-1.11) for daily mortality from all causes. Rainfall events with a 2-year return period were associated with increased daily respiratory mortality (RRc, 1.14), while no significant effect was observed for cardiovascular mortality during the same period. Rainfall events with a 5-year return period were associated with an increased risk for both cardiovascular mortality (RRc, 1.05) and respiratory mortality (RRc, 1.29), with the respiratory mortality being significantly higher.
Points of Concern
According to the authors, moderate to high rainfall can exert protective effects through two main mechanisms: Improving air quality (rainfall can reduce the concentration of particulate matter 2.5 cm in diameter or less in the atmosphere) and behavioral changes in people (more time spent in enclosed environments, reducing direct exposure to outdoor air pollution and nonoptimal temperatures). As rainfall intensity increases, the initial protective effects may be overshadowed by a cascade of negative impacts including:
- Critical resource disruptions: Intense rainfall can cause severe disruptions to access to healthcare, infrastructure damage including power outages, and compromised water and food quality.
- Physiological effects: Increased humidity levels facilitate the growth of airborne pathogens, potentially triggering allergic reactions and respiratory issues, particularly in vulnerable individuals. Rapid shifts in atmospheric pressure and temperature fluctuations can lead to cardiovascular and respiratory complications.
- Indirect effects: Extreme rainfall can have profound effects on mental health, inducing stress and anxiety that may exacerbate pre-existing mental health conditions and indirectly contribute to increased overall mortality from nonexternal causes.
The intensity-response curves for the health effects of heavy rainfall showed a nonlinear trend, transitioning from a protective effect at moderate levels of rainfall to a risk for severe harm when rainfall intensity became extreme. Additionally, the significant effects of extreme events were modified by various types of climate and were more pronounced in areas characterized by low variability in precipitation or sparse vegetation cover.
The study demonstrated that various local factors, such as climatic conditions, climate type, and vegetation cover, can potentially influence cardiovascular and respiratory mortality and all-cause mortality related to precipitation. The findings may help physicians convey to their patients the impact of climate change on their health.
This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Climate change is intensifying the variability of precipitation caused by extreme daily and overall rainfall events. Awareness of the effects of these events is crucial for understanding the complex health consequences of climate change. Physicians have often advised their patients to move to a better climate, and when they did, the recommendation was rarely based on precise scientific knowledge. However, the benefits of changing environments were often so evident that they were indisputable.
Today, advanced models, satellite imagery, and biological approaches such as environmental epigenetics are enhancing our understanding of health risks related to climate change.
Extreme Rainfall and Health
The increase in precipitation variability is linked to climate warming, which leads to higher atmospheric humidity and extreme rainfall events. These manifestations can cause rapid weather changes, increasing interactions with harmful aerosols and raising the risk for various cardiovascular and respiratory conditions. However, a full understanding of the association between rain and health has been hindered by conflicting results and methodological issues (limited geographical locations and short observation durations) in studies.
The association between rainfall intensity and health effects is likely nonlinear. Moderate precipitation can mitigate summer heat and help reduce air pollution, an effect that may lower some environmental health risks. Conversely, intense, low-frequency, short-duration rainfall events can have particularly harmful effects on health, as such events can trigger rapid weather changes, increased proliferation of pathogens, and a rise in the risk of various pollutants, potentially exacerbating health conditions.
Rain and Mortality
Using an intensity-duration-frequency model of three rainfall indices (high intensity, low frequency, short duration), a study published in October 2024 combined these with mortality data from 34 countries or regions. Researchers estimated associations between mortality (all cause, cardiovascular, and respiratory) and rainfall events with different return periods (the average time expected before an extreme event of a certain magnitude occurs again) and crucial effect modifiers, including climatic, socioeconomic, and urban environmental conditions.
The analysis included 109,954,744 deaths from all causes; 31,164,161 cardiovascular deaths; and 11,817,278 respiratory deaths. During the study period, from 1980 to 2020, a total of 50,913 rainfall events with a 1-year return period, 8362 events with a 2-year return period, and 3301 events with a 5-year return period were identified.
The most significant finding was a global positive association between all-cause mortality and extreme rainfall events with a 5-year return period. One day of extreme rainfall with a 5-year return period was associated with a cumulative relative risk (RRc) of 1.08 (95% CI, 1.05-1.11) for daily mortality from all causes. Rainfall events with a 2-year return period were associated with increased daily respiratory mortality (RRc, 1.14), while no significant effect was observed for cardiovascular mortality during the same period. Rainfall events with a 5-year return period were associated with an increased risk for both cardiovascular mortality (RRc, 1.05) and respiratory mortality (RRc, 1.29), with the respiratory mortality being significantly higher.
Points of Concern
According to the authors, moderate to high rainfall can exert protective effects through two main mechanisms: Improving air quality (rainfall can reduce the concentration of particulate matter 2.5 cm in diameter or less in the atmosphere) and behavioral changes in people (more time spent in enclosed environments, reducing direct exposure to outdoor air pollution and nonoptimal temperatures). As rainfall intensity increases, the initial protective effects may be overshadowed by a cascade of negative impacts including:
- Critical resource disruptions: Intense rainfall can cause severe disruptions to access to healthcare, infrastructure damage including power outages, and compromised water and food quality.
- Physiological effects: Increased humidity levels facilitate the growth of airborne pathogens, potentially triggering allergic reactions and respiratory issues, particularly in vulnerable individuals. Rapid shifts in atmospheric pressure and temperature fluctuations can lead to cardiovascular and respiratory complications.
- Indirect effects: Extreme rainfall can have profound effects on mental health, inducing stress and anxiety that may exacerbate pre-existing mental health conditions and indirectly contribute to increased overall mortality from nonexternal causes.
The intensity-response curves for the health effects of heavy rainfall showed a nonlinear trend, transitioning from a protective effect at moderate levels of rainfall to a risk for severe harm when rainfall intensity became extreme. Additionally, the significant effects of extreme events were modified by various types of climate and were more pronounced in areas characterized by low variability in precipitation or sparse vegetation cover.
The study demonstrated that various local factors, such as climatic conditions, climate type, and vegetation cover, can potentially influence cardiovascular and respiratory mortality and all-cause mortality related to precipitation. The findings may help physicians convey to their patients the impact of climate change on their health.
This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Climate change is intensifying the variability of precipitation caused by extreme daily and overall rainfall events. Awareness of the effects of these events is crucial for understanding the complex health consequences of climate change. Physicians have often advised their patients to move to a better climate, and when they did, the recommendation was rarely based on precise scientific knowledge. However, the benefits of changing environments were often so evident that they were indisputable.
Today, advanced models, satellite imagery, and biological approaches such as environmental epigenetics are enhancing our understanding of health risks related to climate change.
Extreme Rainfall and Health
The increase in precipitation variability is linked to climate warming, which leads to higher atmospheric humidity and extreme rainfall events. These manifestations can cause rapid weather changes, increasing interactions with harmful aerosols and raising the risk for various cardiovascular and respiratory conditions. However, a full understanding of the association between rain and health has been hindered by conflicting results and methodological issues (limited geographical locations and short observation durations) in studies.
The association between rainfall intensity and health effects is likely nonlinear. Moderate precipitation can mitigate summer heat and help reduce air pollution, an effect that may lower some environmental health risks. Conversely, intense, low-frequency, short-duration rainfall events can have particularly harmful effects on health, as such events can trigger rapid weather changes, increased proliferation of pathogens, and a rise in the risk of various pollutants, potentially exacerbating health conditions.
Rain and Mortality
Using an intensity-duration-frequency model of three rainfall indices (high intensity, low frequency, short duration), a study published in October 2024 combined these with mortality data from 34 countries or regions. Researchers estimated associations between mortality (all cause, cardiovascular, and respiratory) and rainfall events with different return periods (the average time expected before an extreme event of a certain magnitude occurs again) and crucial effect modifiers, including climatic, socioeconomic, and urban environmental conditions.
The analysis included 109,954,744 deaths from all causes; 31,164,161 cardiovascular deaths; and 11,817,278 respiratory deaths. During the study period, from 1980 to 2020, a total of 50,913 rainfall events with a 1-year return period, 8362 events with a 2-year return period, and 3301 events with a 5-year return period were identified.
The most significant finding was a global positive association between all-cause mortality and extreme rainfall events with a 5-year return period. One day of extreme rainfall with a 5-year return period was associated with a cumulative relative risk (RRc) of 1.08 (95% CI, 1.05-1.11) for daily mortality from all causes. Rainfall events with a 2-year return period were associated with increased daily respiratory mortality (RRc, 1.14), while no significant effect was observed for cardiovascular mortality during the same period. Rainfall events with a 5-year return period were associated with an increased risk for both cardiovascular mortality (RRc, 1.05) and respiratory mortality (RRc, 1.29), with the respiratory mortality being significantly higher.
Points of Concern
According to the authors, moderate to high rainfall can exert protective effects through two main mechanisms: Improving air quality (rainfall can reduce the concentration of particulate matter 2.5 cm in diameter or less in the atmosphere) and behavioral changes in people (more time spent in enclosed environments, reducing direct exposure to outdoor air pollution and nonoptimal temperatures). As rainfall intensity increases, the initial protective effects may be overshadowed by a cascade of negative impacts including:
- Critical resource disruptions: Intense rainfall can cause severe disruptions to access to healthcare, infrastructure damage including power outages, and compromised water and food quality.
- Physiological effects: Increased humidity levels facilitate the growth of airborne pathogens, potentially triggering allergic reactions and respiratory issues, particularly in vulnerable individuals. Rapid shifts in atmospheric pressure and temperature fluctuations can lead to cardiovascular and respiratory complications.
- Indirect effects: Extreme rainfall can have profound effects on mental health, inducing stress and anxiety that may exacerbate pre-existing mental health conditions and indirectly contribute to increased overall mortality from nonexternal causes.
The intensity-response curves for the health effects of heavy rainfall showed a nonlinear trend, transitioning from a protective effect at moderate levels of rainfall to a risk for severe harm when rainfall intensity became extreme. Additionally, the significant effects of extreme events were modified by various types of climate and were more pronounced in areas characterized by low variability in precipitation or sparse vegetation cover.
The study demonstrated that various local factors, such as climatic conditions, climate type, and vegetation cover, can potentially influence cardiovascular and respiratory mortality and all-cause mortality related to precipitation. The findings may help physicians convey to their patients the impact of climate change on their health.
This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Myasthenia Gravis: Where Does Traditional Therapy Fit In?
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
FROM AANEM 2024
New Drug Options Abound for Duchenne Muscular Dystrophy
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
FROM AANEM 2024
Weight Loss Interventions Improve Key Features of PCOS
TOPLINE:
Weight loss interventions using medication or behavioral changes can improve insulin resistance, hormonal markers, and menstrual frequency in women with polycystic ovary syndrome (PCOS), according to a new meta-analysis. Losing weight may not significantly reduce hirsutism or improve quality of life in women with the condition, however.
METHODOLOGY:
- Researchers systematically reviewed randomized controlled trials comparing weight loss interventions to usual care in women with PCOS.
- They focused on 12 studies with behavioral interventions (mainly diets with modest energy deficits), nine trials that used glucagon-like peptide 1 (GLP-1) receptor agonists, and eight studies using other weight loss medications.
- A total of 1529 participants were included in the analysis.
- The investigators synthesized the data using a random-effects meta-analysis with Knapp-Hartung adjustment to examine pooled mean differences.
TAKEAWAY:
- Menstrual frequency increased by 2.64 menses per year (95% CI, 0.65-4.63) with weight loss interventions.
- “To our knowledge, this is the first review to show a clinically significant association in improvement in menstrual frequency with weight loss interventions, an important indicator of subsequent fertility and an important outcome for women,” the researchers wrote.
- Glycemic control also improved, with a mean reduction in homeostatic model assessment of insulin resistance of 0.45 (95% CI, –0.75 to –0.15).
- Free androgen index decreased by an average of 2.03 (95% CI, –3.0 to –1.07).
IN PRACTICE:
“Clinicians may use these findings to counsel women with PCOS on the expected improvements in PCOS markers after weight loss and direct patients toward interventions,” the authors of the study wrote. “Because weight loss programs are cost-effective interventions to improve cardiometabolic risk, they may be particularly valuable for this population at elevated risk.”
SOURCE:
The study was led by Jadine Scragg, PhD, with the Nuffield Department of Primary Care Health Sciences at the University of Oxford in England. It was published online in Annals of Internal Medicine.
LIMITATIONS:
Interventions using GLP-1 agonists were dosed for glycemic control rather than weight management. The studies in the meta-analysis were relatively few and heterogeneous. Data were insufficient to assess ovulation and acne.
DISCLOSURES:
The meta-analysis was supported by grants from the National Institute for Health and Care Research School for Primary Care Research. Authors disclosed ties to Nestlé Health Science and Second Nature.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
Weight loss interventions using medication or behavioral changes can improve insulin resistance, hormonal markers, and menstrual frequency in women with polycystic ovary syndrome (PCOS), according to a new meta-analysis. Losing weight may not significantly reduce hirsutism or improve quality of life in women with the condition, however.
METHODOLOGY:
- Researchers systematically reviewed randomized controlled trials comparing weight loss interventions to usual care in women with PCOS.
- They focused on 12 studies with behavioral interventions (mainly diets with modest energy deficits), nine trials that used glucagon-like peptide 1 (GLP-1) receptor agonists, and eight studies using other weight loss medications.
- A total of 1529 participants were included in the analysis.
- The investigators synthesized the data using a random-effects meta-analysis with Knapp-Hartung adjustment to examine pooled mean differences.
TAKEAWAY:
- Menstrual frequency increased by 2.64 menses per year (95% CI, 0.65-4.63) with weight loss interventions.
- “To our knowledge, this is the first review to show a clinically significant association in improvement in menstrual frequency with weight loss interventions, an important indicator of subsequent fertility and an important outcome for women,” the researchers wrote.
- Glycemic control also improved, with a mean reduction in homeostatic model assessment of insulin resistance of 0.45 (95% CI, –0.75 to –0.15).
- Free androgen index decreased by an average of 2.03 (95% CI, –3.0 to –1.07).
IN PRACTICE:
“Clinicians may use these findings to counsel women with PCOS on the expected improvements in PCOS markers after weight loss and direct patients toward interventions,” the authors of the study wrote. “Because weight loss programs are cost-effective interventions to improve cardiometabolic risk, they may be particularly valuable for this population at elevated risk.”
SOURCE:
The study was led by Jadine Scragg, PhD, with the Nuffield Department of Primary Care Health Sciences at the University of Oxford in England. It was published online in Annals of Internal Medicine.
LIMITATIONS:
Interventions using GLP-1 agonists were dosed for glycemic control rather than weight management. The studies in the meta-analysis were relatively few and heterogeneous. Data were insufficient to assess ovulation and acne.
DISCLOSURES:
The meta-analysis was supported by grants from the National Institute for Health and Care Research School for Primary Care Research. Authors disclosed ties to Nestlé Health Science and Second Nature.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
Weight loss interventions using medication or behavioral changes can improve insulin resistance, hormonal markers, and menstrual frequency in women with polycystic ovary syndrome (PCOS), according to a new meta-analysis. Losing weight may not significantly reduce hirsutism or improve quality of life in women with the condition, however.
METHODOLOGY:
- Researchers systematically reviewed randomized controlled trials comparing weight loss interventions to usual care in women with PCOS.
- They focused on 12 studies with behavioral interventions (mainly diets with modest energy deficits), nine trials that used glucagon-like peptide 1 (GLP-1) receptor agonists, and eight studies using other weight loss medications.
- A total of 1529 participants were included in the analysis.
- The investigators synthesized the data using a random-effects meta-analysis with Knapp-Hartung adjustment to examine pooled mean differences.
TAKEAWAY:
- Menstrual frequency increased by 2.64 menses per year (95% CI, 0.65-4.63) with weight loss interventions.
- “To our knowledge, this is the first review to show a clinically significant association in improvement in menstrual frequency with weight loss interventions, an important indicator of subsequent fertility and an important outcome for women,” the researchers wrote.
- Glycemic control also improved, with a mean reduction in homeostatic model assessment of insulin resistance of 0.45 (95% CI, –0.75 to –0.15).
- Free androgen index decreased by an average of 2.03 (95% CI, –3.0 to –1.07).
IN PRACTICE:
“Clinicians may use these findings to counsel women with PCOS on the expected improvements in PCOS markers after weight loss and direct patients toward interventions,” the authors of the study wrote. “Because weight loss programs are cost-effective interventions to improve cardiometabolic risk, they may be particularly valuable for this population at elevated risk.”
SOURCE:
The study was led by Jadine Scragg, PhD, with the Nuffield Department of Primary Care Health Sciences at the University of Oxford in England. It was published online in Annals of Internal Medicine.
LIMITATIONS:
Interventions using GLP-1 agonists were dosed for glycemic control rather than weight management. The studies in the meta-analysis were relatively few and heterogeneous. Data were insufficient to assess ovulation and acne.
DISCLOSURES:
The meta-analysis was supported by grants from the National Institute for Health and Care Research School for Primary Care Research. Authors disclosed ties to Nestlé Health Science and Second Nature.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.