Diabetes tied to risk of long COVID, too

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Thu, 12/15/2022 - 14:30

Individuals with diabetes who experience COVID-19 are at increased risk for long COVID compared to individuals without diabetes, according to data from a literature review of seven studies.

Diabetes remains a risk factor for severe COVID-19, but whether it is a risk factor for postacute sequelae of COVID-19 (PASC), also known as long COVID, remains unclear, Jessica L. Harding, PhD, of Emory University, said in a late-breaking poster session at the annual scientific sessions of the American Diabetes Association.

Long COVID is generally defined as “sequelae that extend beyond the 4 weeks after initial infection” and may include a range of symptoms that affect multiple organs, Dr. Harding said. A study conducted in January of 2022 suggested that type 2 diabetes was one of several strong risk factors for long COVID, she noted.

Dr. Harding and colleagues reviewed data from seven studies published from Jan. 1, 2020, to Jan. 27, 2022, on the risk of PASC in people with and without diabetes. The studies included patients with a minimum of 4 weeks’ follow-up after COVID-19 diagnosis. All seven studies had a longitudinal cohort design, and included adults from high-income countries, with study populations ranging from 104 to 4,182.

Across the studies, long COVID definitions varied, but included ongoing symptoms of fatigue, cough, and dyspnea, with follow-up periods of 4 weeks to 7 months.

Overall, three of the seven studies indicated that diabetes was a risk factor for long COVID (odds ratio [OR] greater than 4 for all) and four studies indicated that diabetes was not a risk factor for long COVID (OR, 0.5-2.2).

One of the three studies showing increased risk included 2,334 individuals hospitalized with COVID-19; of these about 5% had diabetes. The odds ratio for PASC for individuals with diabetes was 4.18. In another study of 209 persons with COVID-19, of whom 22% had diabetes, diabetes was significantly correlated with respiratory viral disease (meaning at least two respiratory symptoms). The third study showing an increased risk of long COVID in diabetes patients included 104 kidney transplant patients, of whom 20% had diabetes; the odds ratio for PASC was 4.42.

The findings were limited by several factors, including the relatively small number of studies and the heterogeneity of studies regarding definitions of long COVID, specific populations at risk, follow-up times, and risk adjustment, Dr. Harding noted.

More high-quality studies across multiple populations and settings are needed to determine if diabetes is indeed a risk factor for long COVID, she said.

In the meantime, “careful monitoring of people with diabetes for development of PASC may be advised,” Dr. Harding concluded.
 

Findings support need for screening

“Given the devastating impact of COVID on people with diabetes, it’s important to know what data has been accumulated on long COVID for future research and discoveries in this area,” Robert A. Gabbay, MD, chief science and medical officer for the American Diabetes Association, said in an interview. “The more information we have, the better we can understand the implications.”

Courtesy Joslin Diabetes Center
Dr. Robert A. Gabbay

Dr. Gabbay said he was surprised by the current study findings. “We know very little on this subject, so yes, I am surprised to see just how significant the risk of long COVID for people with diabetes seems to be, but clearly, more research needs to be done to understand long COVID,” he emphasized.

The take-home message for clinicians is the importance of screening patients for PASC; also “ask your patients if they had COVID, to better understand any symptoms they might have that could be related to PACS,” he noted. 

“It is crucial that we confirm these results and then look at risk factors in people with diabetes that might explain who is at highest risk and ultimately understand the causes and potential cure,” Dr. Gabbay added.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Harding and Dr. Gabbay had no financial conflicts to disclose.

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Individuals with diabetes who experience COVID-19 are at increased risk for long COVID compared to individuals without diabetes, according to data from a literature review of seven studies.

Diabetes remains a risk factor for severe COVID-19, but whether it is a risk factor for postacute sequelae of COVID-19 (PASC), also known as long COVID, remains unclear, Jessica L. Harding, PhD, of Emory University, said in a late-breaking poster session at the annual scientific sessions of the American Diabetes Association.

Long COVID is generally defined as “sequelae that extend beyond the 4 weeks after initial infection” and may include a range of symptoms that affect multiple organs, Dr. Harding said. A study conducted in January of 2022 suggested that type 2 diabetes was one of several strong risk factors for long COVID, she noted.

Dr. Harding and colleagues reviewed data from seven studies published from Jan. 1, 2020, to Jan. 27, 2022, on the risk of PASC in people with and without diabetes. The studies included patients with a minimum of 4 weeks’ follow-up after COVID-19 diagnosis. All seven studies had a longitudinal cohort design, and included adults from high-income countries, with study populations ranging from 104 to 4,182.

Across the studies, long COVID definitions varied, but included ongoing symptoms of fatigue, cough, and dyspnea, with follow-up periods of 4 weeks to 7 months.

Overall, three of the seven studies indicated that diabetes was a risk factor for long COVID (odds ratio [OR] greater than 4 for all) and four studies indicated that diabetes was not a risk factor for long COVID (OR, 0.5-2.2).

One of the three studies showing increased risk included 2,334 individuals hospitalized with COVID-19; of these about 5% had diabetes. The odds ratio for PASC for individuals with diabetes was 4.18. In another study of 209 persons with COVID-19, of whom 22% had diabetes, diabetes was significantly correlated with respiratory viral disease (meaning at least two respiratory symptoms). The third study showing an increased risk of long COVID in diabetes patients included 104 kidney transplant patients, of whom 20% had diabetes; the odds ratio for PASC was 4.42.

The findings were limited by several factors, including the relatively small number of studies and the heterogeneity of studies regarding definitions of long COVID, specific populations at risk, follow-up times, and risk adjustment, Dr. Harding noted.

More high-quality studies across multiple populations and settings are needed to determine if diabetes is indeed a risk factor for long COVID, she said.

In the meantime, “careful monitoring of people with diabetes for development of PASC may be advised,” Dr. Harding concluded.
 

Findings support need for screening

“Given the devastating impact of COVID on people with diabetes, it’s important to know what data has been accumulated on long COVID for future research and discoveries in this area,” Robert A. Gabbay, MD, chief science and medical officer for the American Diabetes Association, said in an interview. “The more information we have, the better we can understand the implications.”

Courtesy Joslin Diabetes Center
Dr. Robert A. Gabbay

Dr. Gabbay said he was surprised by the current study findings. “We know very little on this subject, so yes, I am surprised to see just how significant the risk of long COVID for people with diabetes seems to be, but clearly, more research needs to be done to understand long COVID,” he emphasized.

The take-home message for clinicians is the importance of screening patients for PASC; also “ask your patients if they had COVID, to better understand any symptoms they might have that could be related to PACS,” he noted. 

“It is crucial that we confirm these results and then look at risk factors in people with diabetes that might explain who is at highest risk and ultimately understand the causes and potential cure,” Dr. Gabbay added.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Harding and Dr. Gabbay had no financial conflicts to disclose.

Individuals with diabetes who experience COVID-19 are at increased risk for long COVID compared to individuals without diabetes, according to data from a literature review of seven studies.

Diabetes remains a risk factor for severe COVID-19, but whether it is a risk factor for postacute sequelae of COVID-19 (PASC), also known as long COVID, remains unclear, Jessica L. Harding, PhD, of Emory University, said in a late-breaking poster session at the annual scientific sessions of the American Diabetes Association.

Long COVID is generally defined as “sequelae that extend beyond the 4 weeks after initial infection” and may include a range of symptoms that affect multiple organs, Dr. Harding said. A study conducted in January of 2022 suggested that type 2 diabetes was one of several strong risk factors for long COVID, she noted.

Dr. Harding and colleagues reviewed data from seven studies published from Jan. 1, 2020, to Jan. 27, 2022, on the risk of PASC in people with and without diabetes. The studies included patients with a minimum of 4 weeks’ follow-up after COVID-19 diagnosis. All seven studies had a longitudinal cohort design, and included adults from high-income countries, with study populations ranging from 104 to 4,182.

Across the studies, long COVID definitions varied, but included ongoing symptoms of fatigue, cough, and dyspnea, with follow-up periods of 4 weeks to 7 months.

Overall, three of the seven studies indicated that diabetes was a risk factor for long COVID (odds ratio [OR] greater than 4 for all) and four studies indicated that diabetes was not a risk factor for long COVID (OR, 0.5-2.2).

One of the three studies showing increased risk included 2,334 individuals hospitalized with COVID-19; of these about 5% had diabetes. The odds ratio for PASC for individuals with diabetes was 4.18. In another study of 209 persons with COVID-19, of whom 22% had diabetes, diabetes was significantly correlated with respiratory viral disease (meaning at least two respiratory symptoms). The third study showing an increased risk of long COVID in diabetes patients included 104 kidney transplant patients, of whom 20% had diabetes; the odds ratio for PASC was 4.42.

The findings were limited by several factors, including the relatively small number of studies and the heterogeneity of studies regarding definitions of long COVID, specific populations at risk, follow-up times, and risk adjustment, Dr. Harding noted.

More high-quality studies across multiple populations and settings are needed to determine if diabetes is indeed a risk factor for long COVID, she said.

In the meantime, “careful monitoring of people with diabetes for development of PASC may be advised,” Dr. Harding concluded.
 

Findings support need for screening

“Given the devastating impact of COVID on people with diabetes, it’s important to know what data has been accumulated on long COVID for future research and discoveries in this area,” Robert A. Gabbay, MD, chief science and medical officer for the American Diabetes Association, said in an interview. “The more information we have, the better we can understand the implications.”

Courtesy Joslin Diabetes Center
Dr. Robert A. Gabbay

Dr. Gabbay said he was surprised by the current study findings. “We know very little on this subject, so yes, I am surprised to see just how significant the risk of long COVID for people with diabetes seems to be, but clearly, more research needs to be done to understand long COVID,” he emphasized.

The take-home message for clinicians is the importance of screening patients for PASC; also “ask your patients if they had COVID, to better understand any symptoms they might have that could be related to PACS,” he noted. 

“It is crucial that we confirm these results and then look at risk factors in people with diabetes that might explain who is at highest risk and ultimately understand the causes and potential cure,” Dr. Gabbay added.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Harding and Dr. Gabbay had no financial conflicts to disclose.

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Eosinophils may predict outcomes in acute COPD exacerbations

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Thu, 06/23/2022 - 16:57

High levels of eosinophils had a protective effect for individuals who experienced acute exacerbations of chronic obstructive pulmonary disease, based on data from nearly 1,000 patients.

Several blood biomarkers are under investigation for links to acute exacerbation of chronic obstructive pulmonary disease (AECOPD), which remains one of the top three causes of death worldwide, wrote Riuying Wang, MD, of Third Hospital of Shanxi Medical University, Taiyuan, China, and colleagues.

“Numerous studies have shown the relationship between eosinophilia and clinical outcomes of patients with AECOPD. However, the evidence lacks consensus, and the research thresholds are controversial,” they said.

In a study published in Heart & Lung, the researchers reviewed data from 984 adults with AECOPD over a 3-year follow-up period. The mean age of the patients was 71 years, and 78% were men. The patients’ blood eosinophil levels were grouped into three categories: EOS < 2%, EOS from 2% to < 3%, and 3% or higher. The researchers examined the association between eosinophilia and various comorbidities, treatment, and mortality.

Eosinophilia occurred in 477 cases. The prevalence of eosinophilia in the three groups was 36.48%, 22.87%, and 48.48% respectively, with eosinophilia defined as eosinophil counts of at least 100 cells per microliter, according to the report in Heart & Lung.

An EOS of 2% or higher was associated with significantly fewer cases of complicated pulmonary heart disease and atrial fibrillation than the lower EOS group. Similarly, patients in the EOS group of 2% or higher were less likely to use ventilators and systemic glucocorticoids and those in the EOS less than 2% group had significantly heavier airflow limitation, higher D-dimer, higher burden of infectious inflammation, and higher prevalence of respiratory failure than the other groups.

In addition, significantly fewer deaths occurred during the study period among patients with EOS of 2% or higher, compared with the lower EOS group (P < .01). The findings suggest that “Eosinophils can be used as a prognostic indicator of mortality in AECOPD,” the researchers said.

The researchers also used the area under the curve to examine the predictive value of EOS. The ROC curve showed that the indicators of AUC 0.5 included chest CT imaging, osteoporosis, mental illness, dust exposure, and being a former smoker; however, “the predictive value of EOS by the ROC curve was unstable. Further validation in large samples is needed,” the researchers wrote in their discussion.

The study findings were limited by several factors including the retrospective design and use of data from a single center, the researchers noted. Other limitations included the relatively small sample size and a lack of data on some clinical features and performance metrics, as well as lack of evaluation of chest CT subtypes.

However, the results are consistent with previous studies on infection and antibiotics and reviewed the optimal threshold of AECOPD, the researchers wrote. Based on their findings, “Eosinophils can not only guide clinical treatment but also be used as an index to predict the clinical outcome and prognosis of AECOPD patients,” they concluded.

The study received no outside funding. The researchers had no financial conflicts to disclose.

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High levels of eosinophils had a protective effect for individuals who experienced acute exacerbations of chronic obstructive pulmonary disease, based on data from nearly 1,000 patients.

Several blood biomarkers are under investigation for links to acute exacerbation of chronic obstructive pulmonary disease (AECOPD), which remains one of the top three causes of death worldwide, wrote Riuying Wang, MD, of Third Hospital of Shanxi Medical University, Taiyuan, China, and colleagues.

“Numerous studies have shown the relationship between eosinophilia and clinical outcomes of patients with AECOPD. However, the evidence lacks consensus, and the research thresholds are controversial,” they said.

In a study published in Heart & Lung, the researchers reviewed data from 984 adults with AECOPD over a 3-year follow-up period. The mean age of the patients was 71 years, and 78% were men. The patients’ blood eosinophil levels were grouped into three categories: EOS < 2%, EOS from 2% to < 3%, and 3% or higher. The researchers examined the association between eosinophilia and various comorbidities, treatment, and mortality.

Eosinophilia occurred in 477 cases. The prevalence of eosinophilia in the three groups was 36.48%, 22.87%, and 48.48% respectively, with eosinophilia defined as eosinophil counts of at least 100 cells per microliter, according to the report in Heart & Lung.

An EOS of 2% or higher was associated with significantly fewer cases of complicated pulmonary heart disease and atrial fibrillation than the lower EOS group. Similarly, patients in the EOS group of 2% or higher were less likely to use ventilators and systemic glucocorticoids and those in the EOS less than 2% group had significantly heavier airflow limitation, higher D-dimer, higher burden of infectious inflammation, and higher prevalence of respiratory failure than the other groups.

In addition, significantly fewer deaths occurred during the study period among patients with EOS of 2% or higher, compared with the lower EOS group (P < .01). The findings suggest that “Eosinophils can be used as a prognostic indicator of mortality in AECOPD,” the researchers said.

The researchers also used the area under the curve to examine the predictive value of EOS. The ROC curve showed that the indicators of AUC 0.5 included chest CT imaging, osteoporosis, mental illness, dust exposure, and being a former smoker; however, “the predictive value of EOS by the ROC curve was unstable. Further validation in large samples is needed,” the researchers wrote in their discussion.

The study findings were limited by several factors including the retrospective design and use of data from a single center, the researchers noted. Other limitations included the relatively small sample size and a lack of data on some clinical features and performance metrics, as well as lack of evaluation of chest CT subtypes.

However, the results are consistent with previous studies on infection and antibiotics and reviewed the optimal threshold of AECOPD, the researchers wrote. Based on their findings, “Eosinophils can not only guide clinical treatment but also be used as an index to predict the clinical outcome and prognosis of AECOPD patients,” they concluded.

The study received no outside funding. The researchers had no financial conflicts to disclose.

High levels of eosinophils had a protective effect for individuals who experienced acute exacerbations of chronic obstructive pulmonary disease, based on data from nearly 1,000 patients.

Several blood biomarkers are under investigation for links to acute exacerbation of chronic obstructive pulmonary disease (AECOPD), which remains one of the top three causes of death worldwide, wrote Riuying Wang, MD, of Third Hospital of Shanxi Medical University, Taiyuan, China, and colleagues.

“Numerous studies have shown the relationship between eosinophilia and clinical outcomes of patients with AECOPD. However, the evidence lacks consensus, and the research thresholds are controversial,” they said.

In a study published in Heart & Lung, the researchers reviewed data from 984 adults with AECOPD over a 3-year follow-up period. The mean age of the patients was 71 years, and 78% were men. The patients’ blood eosinophil levels were grouped into three categories: EOS < 2%, EOS from 2% to < 3%, and 3% or higher. The researchers examined the association between eosinophilia and various comorbidities, treatment, and mortality.

Eosinophilia occurred in 477 cases. The prevalence of eosinophilia in the three groups was 36.48%, 22.87%, and 48.48% respectively, with eosinophilia defined as eosinophil counts of at least 100 cells per microliter, according to the report in Heart & Lung.

An EOS of 2% or higher was associated with significantly fewer cases of complicated pulmonary heart disease and atrial fibrillation than the lower EOS group. Similarly, patients in the EOS group of 2% or higher were less likely to use ventilators and systemic glucocorticoids and those in the EOS less than 2% group had significantly heavier airflow limitation, higher D-dimer, higher burden of infectious inflammation, and higher prevalence of respiratory failure than the other groups.

In addition, significantly fewer deaths occurred during the study period among patients with EOS of 2% or higher, compared with the lower EOS group (P < .01). The findings suggest that “Eosinophils can be used as a prognostic indicator of mortality in AECOPD,” the researchers said.

The researchers also used the area under the curve to examine the predictive value of EOS. The ROC curve showed that the indicators of AUC 0.5 included chest CT imaging, osteoporosis, mental illness, dust exposure, and being a former smoker; however, “the predictive value of EOS by the ROC curve was unstable. Further validation in large samples is needed,” the researchers wrote in their discussion.

The study findings were limited by several factors including the retrospective design and use of data from a single center, the researchers noted. Other limitations included the relatively small sample size and a lack of data on some clinical features and performance metrics, as well as lack of evaluation of chest CT subtypes.

However, the results are consistent with previous studies on infection and antibiotics and reviewed the optimal threshold of AECOPD, the researchers wrote. Based on their findings, “Eosinophils can not only guide clinical treatment but also be used as an index to predict the clinical outcome and prognosis of AECOPD patients,” they concluded.

The study received no outside funding. The researchers had no financial conflicts to disclose.

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Exercise of any type boosts type 1 diabetes time in range

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Thu, 06/16/2022 - 15:05

Adults with type 1 diabetes had significantly better glycemic control on days they exercised, regardless of exercise type, compared to days when they were inactive, according to a prospective study in nearly 500 individuals.

Different types of exercise, such as aerobic workouts, interval training, or resistance training, may have different immediate glycemic effects in adults with type 1 diabetes (T1D), but the impact of exercise type on the percentage of time diabetes patients maintain glucose in the 70-180 mg/dL range on days when they are active vs. inactive has not been well studied, Zoey Li said in a presentation at the annual scientific sessions of the American Diabetes Association.

Yuri Nunes / EyeEm / Getty Images

In the Type 1 Diabetes Exercise Initiative (T1DEXI) study, Ms. Li and colleagues examined continuous glucose monitoring (CGM) data from 497 adults with T1D. The observational study included self-referred adults aged 18 years and older who had been living with T1D for at least 2 years. Participants were assigned to programs of aerobic exercise (defined as a target heart rate of 70%-80% of age-predicted maximum), interval exercise (defined as an interval heart rate of 80%-90% of age-predicted maximum), or resistance exercise (defined as muscle group fatigue after three sets of eight repetitions).

Participants completed the workouts at home via 30-minute videos at least six times over the 4-week study period. The study design involved an activity goal of at least 150 minutes per week, including the videos and self-reported usual activity, such as walking. The data were collected through an app designed for the study, a heart rate monitor, and a CGM.

The researchers compared glucose levels on days when the participants reported being active compared to days when they were sedentary. The goal of the study was to assess the effect of exercise type on time spent with glucose in the range of 70-180 mg/dL, defined as time in range (TIR).

The mean age of the participants was 37 years; 89% were White. The mean duration of diabetes was 18 years, and the mean hemoglobin A1c was 6.6%. “An astounding 95% were current continuous glucose monitoring [CGM] users,” said Ms. Li, a statistician at the Jaeb Center for Health Research in Tampa, Fla.

A total of 398 participants reported at least one exercise day and one sedentary day, for a total of 1,302 exercise days and 2,470 sedentary days.

Overall, the mean TIR was significantly higher on exercise days compared to sedentary days (75% vs. 70%, P < .001). The median time above 180 mg/dL also was significantly lower on exercise days compared to sedentary days (17% vs. 23%, P < .001), and mean glucose levels were 10 mg/dL lower on exercise days (145 mg/dL vs. 155 mg/dL)

“This all came with a slight hit to their time below range,” Ms. Li noted. The median time below 70 mg/dL was 1.1% on exercise days compared to 0.4% on sedentary days (P < .001). The percentage of days with hypoglycemic events was higher on exercise days compared to sedentary days (47% vs. 40%, P < .001), as they are related to time below 70 mg/dL, she added.

The differences for mean glucose level and TIR between exercise days and sedentary days were significant for each of the three exercise types, Ms. Li said.

“After establishing these glycemic trends, we looked at whether there were any factors that influenced the glycemic differences on exercise vs. sedentary days,” Ms. Li said.

Regardless of exercise type, age, sex, baseline A1c, diabetes duration, body mass index, insulin modality, CGM use, and percentage of time below range in the past 24 hours, there was higher TIR and higher hypoglycemia on exercise days compared to sedentary days.

Although the study was limited in part by the observational design, “with these data, we can better understand the glycemic benefits and disadvantages of exercise in adults with type 1 diabetes,” Ms. Li said.
 

 

 

Don’t forget the negative effects of exercise

“It is well known that the three types of exercise can modulate glucose levels. This can be very useful when attempting to reduce excessively high glucose levels, and when encouraging people to engage in frequent, regular, and consistent physical activity and exercise for general cardiovascular pulmonary and musculoskeletal health,” Helena W. Rodbard, MD, an endocrinologist in private practice in Rockville, Md., said in an interview.

“However, it was not known what effects various types of exercise would have on time in range (70-180 mg/dL) and time below range (< 70 mg/dL) measured over a full 24-hour period in people with type 1 diabetes,” said Dr. Rodbard, who was not involved with the study.

“I was surprised to see that the effect of the three different types of exercise were so similar,” Dr. Rodbard noted. “There had been previous reports suggesting that the time course of glucose could be different for these three types of exercise.”

The current study confirms prior knowledge that exercise can help reduce blood glucose, and increase TIR, said Dr. Rodbard. The study shows that TIR increases by roughly 5-7 percentage points (about 1 hour per day) and reduces mean glucose by 9-13 mg/dL irrespective of the three types of exercise,” she said. “There was a suggestion that the risk of increasing hypoglycemia below 70 mg/dL was less likely for resistance exercise than for the interval or aerobic types of exercise,” she noted.

As for additional research, “This study did not address the various ways in which one can mitigate the potentially deleterious effects of exercise, specifically with reference to rates of hypoglycemia, even mild symptomatic biochemical hypoglycemia,” said Dr. Rodbard. “Since the actual amount of time below 70 mg/dL is usually so small (0.3%-0.7% of the 1,440 minutes in the day, or about 5-10 minutes per day on average), it is difficult to measure and there is considerable variability between different people,” she emphasized. “Finding optimal and robust ways to achieve consistency in the reduction of glucose, between days within subjects, and between subjects, will need further examination of various types of protocols for diet, exercise and insulin administration, and of various methods for education of the patient,” she said.

The study was supported in part by the Leona M. and Harry B. Helmsley Charitable Trust. Ms. Li and Dr. Rodbard had no financial conflicts to disclose. Dr. Rodbard serves on the editorial advisory board of Clinical Endocrinology News.

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Adults with type 1 diabetes had significantly better glycemic control on days they exercised, regardless of exercise type, compared to days when they were inactive, according to a prospective study in nearly 500 individuals.

Different types of exercise, such as aerobic workouts, interval training, or resistance training, may have different immediate glycemic effects in adults with type 1 diabetes (T1D), but the impact of exercise type on the percentage of time diabetes patients maintain glucose in the 70-180 mg/dL range on days when they are active vs. inactive has not been well studied, Zoey Li said in a presentation at the annual scientific sessions of the American Diabetes Association.

Yuri Nunes / EyeEm / Getty Images

In the Type 1 Diabetes Exercise Initiative (T1DEXI) study, Ms. Li and colleagues examined continuous glucose monitoring (CGM) data from 497 adults with T1D. The observational study included self-referred adults aged 18 years and older who had been living with T1D for at least 2 years. Participants were assigned to programs of aerobic exercise (defined as a target heart rate of 70%-80% of age-predicted maximum), interval exercise (defined as an interval heart rate of 80%-90% of age-predicted maximum), or resistance exercise (defined as muscle group fatigue after three sets of eight repetitions).

Participants completed the workouts at home via 30-minute videos at least six times over the 4-week study period. The study design involved an activity goal of at least 150 minutes per week, including the videos and self-reported usual activity, such as walking. The data were collected through an app designed for the study, a heart rate monitor, and a CGM.

The researchers compared glucose levels on days when the participants reported being active compared to days when they were sedentary. The goal of the study was to assess the effect of exercise type on time spent with glucose in the range of 70-180 mg/dL, defined as time in range (TIR).

The mean age of the participants was 37 years; 89% were White. The mean duration of diabetes was 18 years, and the mean hemoglobin A1c was 6.6%. “An astounding 95% were current continuous glucose monitoring [CGM] users,” said Ms. Li, a statistician at the Jaeb Center for Health Research in Tampa, Fla.

A total of 398 participants reported at least one exercise day and one sedentary day, for a total of 1,302 exercise days and 2,470 sedentary days.

Overall, the mean TIR was significantly higher on exercise days compared to sedentary days (75% vs. 70%, P < .001). The median time above 180 mg/dL also was significantly lower on exercise days compared to sedentary days (17% vs. 23%, P < .001), and mean glucose levels were 10 mg/dL lower on exercise days (145 mg/dL vs. 155 mg/dL)

“This all came with a slight hit to their time below range,” Ms. Li noted. The median time below 70 mg/dL was 1.1% on exercise days compared to 0.4% on sedentary days (P < .001). The percentage of days with hypoglycemic events was higher on exercise days compared to sedentary days (47% vs. 40%, P < .001), as they are related to time below 70 mg/dL, she added.

The differences for mean glucose level and TIR between exercise days and sedentary days were significant for each of the three exercise types, Ms. Li said.

“After establishing these glycemic trends, we looked at whether there were any factors that influenced the glycemic differences on exercise vs. sedentary days,” Ms. Li said.

Regardless of exercise type, age, sex, baseline A1c, diabetes duration, body mass index, insulin modality, CGM use, and percentage of time below range in the past 24 hours, there was higher TIR and higher hypoglycemia on exercise days compared to sedentary days.

Although the study was limited in part by the observational design, “with these data, we can better understand the glycemic benefits and disadvantages of exercise in adults with type 1 diabetes,” Ms. Li said.
 

 

 

Don’t forget the negative effects of exercise

“It is well known that the three types of exercise can modulate glucose levels. This can be very useful when attempting to reduce excessively high glucose levels, and when encouraging people to engage in frequent, regular, and consistent physical activity and exercise for general cardiovascular pulmonary and musculoskeletal health,” Helena W. Rodbard, MD, an endocrinologist in private practice in Rockville, Md., said in an interview.

“However, it was not known what effects various types of exercise would have on time in range (70-180 mg/dL) and time below range (< 70 mg/dL) measured over a full 24-hour period in people with type 1 diabetes,” said Dr. Rodbard, who was not involved with the study.

“I was surprised to see that the effect of the three different types of exercise were so similar,” Dr. Rodbard noted. “There had been previous reports suggesting that the time course of glucose could be different for these three types of exercise.”

The current study confirms prior knowledge that exercise can help reduce blood glucose, and increase TIR, said Dr. Rodbard. The study shows that TIR increases by roughly 5-7 percentage points (about 1 hour per day) and reduces mean glucose by 9-13 mg/dL irrespective of the three types of exercise,” she said. “There was a suggestion that the risk of increasing hypoglycemia below 70 mg/dL was less likely for resistance exercise than for the interval or aerobic types of exercise,” she noted.

As for additional research, “This study did not address the various ways in which one can mitigate the potentially deleterious effects of exercise, specifically with reference to rates of hypoglycemia, even mild symptomatic biochemical hypoglycemia,” said Dr. Rodbard. “Since the actual amount of time below 70 mg/dL is usually so small (0.3%-0.7% of the 1,440 minutes in the day, or about 5-10 minutes per day on average), it is difficult to measure and there is considerable variability between different people,” she emphasized. “Finding optimal and robust ways to achieve consistency in the reduction of glucose, between days within subjects, and between subjects, will need further examination of various types of protocols for diet, exercise and insulin administration, and of various methods for education of the patient,” she said.

The study was supported in part by the Leona M. and Harry B. Helmsley Charitable Trust. Ms. Li and Dr. Rodbard had no financial conflicts to disclose. Dr. Rodbard serves on the editorial advisory board of Clinical Endocrinology News.

Adults with type 1 diabetes had significantly better glycemic control on days they exercised, regardless of exercise type, compared to days when they were inactive, according to a prospective study in nearly 500 individuals.

Different types of exercise, such as aerobic workouts, interval training, or resistance training, may have different immediate glycemic effects in adults with type 1 diabetes (T1D), but the impact of exercise type on the percentage of time diabetes patients maintain glucose in the 70-180 mg/dL range on days when they are active vs. inactive has not been well studied, Zoey Li said in a presentation at the annual scientific sessions of the American Diabetes Association.

Yuri Nunes / EyeEm / Getty Images

In the Type 1 Diabetes Exercise Initiative (T1DEXI) study, Ms. Li and colleagues examined continuous glucose monitoring (CGM) data from 497 adults with T1D. The observational study included self-referred adults aged 18 years and older who had been living with T1D for at least 2 years. Participants were assigned to programs of aerobic exercise (defined as a target heart rate of 70%-80% of age-predicted maximum), interval exercise (defined as an interval heart rate of 80%-90% of age-predicted maximum), or resistance exercise (defined as muscle group fatigue after three sets of eight repetitions).

Participants completed the workouts at home via 30-minute videos at least six times over the 4-week study period. The study design involved an activity goal of at least 150 minutes per week, including the videos and self-reported usual activity, such as walking. The data were collected through an app designed for the study, a heart rate monitor, and a CGM.

The researchers compared glucose levels on days when the participants reported being active compared to days when they were sedentary. The goal of the study was to assess the effect of exercise type on time spent with glucose in the range of 70-180 mg/dL, defined as time in range (TIR).

The mean age of the participants was 37 years; 89% were White. The mean duration of diabetes was 18 years, and the mean hemoglobin A1c was 6.6%. “An astounding 95% were current continuous glucose monitoring [CGM] users,” said Ms. Li, a statistician at the Jaeb Center for Health Research in Tampa, Fla.

A total of 398 participants reported at least one exercise day and one sedentary day, for a total of 1,302 exercise days and 2,470 sedentary days.

Overall, the mean TIR was significantly higher on exercise days compared to sedentary days (75% vs. 70%, P < .001). The median time above 180 mg/dL also was significantly lower on exercise days compared to sedentary days (17% vs. 23%, P < .001), and mean glucose levels were 10 mg/dL lower on exercise days (145 mg/dL vs. 155 mg/dL)

“This all came with a slight hit to their time below range,” Ms. Li noted. The median time below 70 mg/dL was 1.1% on exercise days compared to 0.4% on sedentary days (P < .001). The percentage of days with hypoglycemic events was higher on exercise days compared to sedentary days (47% vs. 40%, P < .001), as they are related to time below 70 mg/dL, she added.

The differences for mean glucose level and TIR between exercise days and sedentary days were significant for each of the three exercise types, Ms. Li said.

“After establishing these glycemic trends, we looked at whether there were any factors that influenced the glycemic differences on exercise vs. sedentary days,” Ms. Li said.

Regardless of exercise type, age, sex, baseline A1c, diabetes duration, body mass index, insulin modality, CGM use, and percentage of time below range in the past 24 hours, there was higher TIR and higher hypoglycemia on exercise days compared to sedentary days.

Although the study was limited in part by the observational design, “with these data, we can better understand the glycemic benefits and disadvantages of exercise in adults with type 1 diabetes,” Ms. Li said.
 

 

 

Don’t forget the negative effects of exercise

“It is well known that the three types of exercise can modulate glucose levels. This can be very useful when attempting to reduce excessively high glucose levels, and when encouraging people to engage in frequent, regular, and consistent physical activity and exercise for general cardiovascular pulmonary and musculoskeletal health,” Helena W. Rodbard, MD, an endocrinologist in private practice in Rockville, Md., said in an interview.

“However, it was not known what effects various types of exercise would have on time in range (70-180 mg/dL) and time below range (< 70 mg/dL) measured over a full 24-hour period in people with type 1 diabetes,” said Dr. Rodbard, who was not involved with the study.

“I was surprised to see that the effect of the three different types of exercise were so similar,” Dr. Rodbard noted. “There had been previous reports suggesting that the time course of glucose could be different for these three types of exercise.”

The current study confirms prior knowledge that exercise can help reduce blood glucose, and increase TIR, said Dr. Rodbard. The study shows that TIR increases by roughly 5-7 percentage points (about 1 hour per day) and reduces mean glucose by 9-13 mg/dL irrespective of the three types of exercise,” she said. “There was a suggestion that the risk of increasing hypoglycemia below 70 mg/dL was less likely for resistance exercise than for the interval or aerobic types of exercise,” she noted.

As for additional research, “This study did not address the various ways in which one can mitigate the potentially deleterious effects of exercise, specifically with reference to rates of hypoglycemia, even mild symptomatic biochemical hypoglycemia,” said Dr. Rodbard. “Since the actual amount of time below 70 mg/dL is usually so small (0.3%-0.7% of the 1,440 minutes in the day, or about 5-10 minutes per day on average), it is difficult to measure and there is considerable variability between different people,” she emphasized. “Finding optimal and robust ways to achieve consistency in the reduction of glucose, between days within subjects, and between subjects, will need further examination of various types of protocols for diet, exercise and insulin administration, and of various methods for education of the patient,” she said.

The study was supported in part by the Leona M. and Harry B. Helmsley Charitable Trust. Ms. Li and Dr. Rodbard had no financial conflicts to disclose. Dr. Rodbard serves on the editorial advisory board of Clinical Endocrinology News.

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Top children’s hospitals report includes rankings by region to aid families

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Thu, 06/16/2022 - 09:11

Boston Children’s Hospital led the list of 10 children’s hospitals across the United States named to the Best Children’s Hospitals Honor Roll for 2022-2023, issued by U.S. News & World Report.

The 16th annual Best Children’s Hospitals rankings were published on June 14.

Rounding out the top 10 on the Honor Roll were Children’s Hospital of Philadelphia; Texas Children’s Hospital, Houston; Cincinnati Children’s Hospital Medical Center; Children’s Hospital Los Angeles; Children’s Hospital Colorado, Aurora; Children’s National Hospital, Washington, D.C.; Nationwide Children’s Hospital, Columbus, Ohio; UPMC Children’s Hospital of Pittsburgh; and Lucile Packard Children’s Hospital, Palo Alto, Calif.

The Honor Roll hospitals were chosen based on being highly ranked in multiple specialties, such as cancer, cardiology, and orthopedics.

For the second time, the rankings included top hospitals not only in each state, but also in seven multistate regions. The goal of the regional rankings is to help families identify the high-quality pediatric care centers closest to them, according to the U.S. News press release accompanying the rankings.

The top-ranked hospitals for the seven regions were Children’s Hospital Los Angeles (Pacific); Children’s Hospital Colorado, Aurora (Rocky Mountains); Texas Children’s Hospital, Houston (Southwest); Children’s Healthcare of Atlanta and Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, Tenn. (tie for Southeast); Cincinnati Children’s Hospital Medical Center (Midwest); Children’s Hospital of Philadelphia (Mid-Atlantic); and Boston Children’s Hospital (New England).

The 2022-2023 U.S. News rankings identify the top 50 centers across the United States in each of 10 pediatric specialties: cancer, cardiology/ heart surgery, diabetes/endocrinology, gastroenterology/gastrointestinal surgery, neonatology, nephrology, neurology/neurosurgery, orthopedics, pulmonology/lung surgery, and urology.

For the 2022-2023 rankings, U.S. News requested medical data and other information from 200 pediatric facilities across the United States; 119 responded and were evaluated in at least one specialty, and 90 were ranked in one or more specialties.

Approximately one-third of each hospital’s score was based on outcomes such as survival, infections, and surgical complications (although outcomes counted for 38.3% of scores for cardiology and heart surgery). Approximately 13% of the score was based on reputation/expert opinion, determined by an annual survey of experts in the 10 specialties (8% of scores for cardiology and heart surgery), and nearly 60% was based on patient safety, excellence, and family centeredness, according to a statement from U.S. News.

“The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care, offering parents and their pediatricians a helpful starting point in choosing the facility that’s best for their child,” said Ben Harder, chief of health analysis and managing editor at U.S. News, in a press release accompanying the rankings.

Also new to the ranking system this year was a measure to assess hospitals’ efforts to improve equity of care and to promote diversity and inclusion, which accounts for 2% of each hospital’s score in each specialty, according to U.S. News.

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Boston Children’s Hospital led the list of 10 children’s hospitals across the United States named to the Best Children’s Hospitals Honor Roll for 2022-2023, issued by U.S. News & World Report.

The 16th annual Best Children’s Hospitals rankings were published on June 14.

Rounding out the top 10 on the Honor Roll were Children’s Hospital of Philadelphia; Texas Children’s Hospital, Houston; Cincinnati Children’s Hospital Medical Center; Children’s Hospital Los Angeles; Children’s Hospital Colorado, Aurora; Children’s National Hospital, Washington, D.C.; Nationwide Children’s Hospital, Columbus, Ohio; UPMC Children’s Hospital of Pittsburgh; and Lucile Packard Children’s Hospital, Palo Alto, Calif.

The Honor Roll hospitals were chosen based on being highly ranked in multiple specialties, such as cancer, cardiology, and orthopedics.

For the second time, the rankings included top hospitals not only in each state, but also in seven multistate regions. The goal of the regional rankings is to help families identify the high-quality pediatric care centers closest to them, according to the U.S. News press release accompanying the rankings.

The top-ranked hospitals for the seven regions were Children’s Hospital Los Angeles (Pacific); Children’s Hospital Colorado, Aurora (Rocky Mountains); Texas Children’s Hospital, Houston (Southwest); Children’s Healthcare of Atlanta and Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, Tenn. (tie for Southeast); Cincinnati Children’s Hospital Medical Center (Midwest); Children’s Hospital of Philadelphia (Mid-Atlantic); and Boston Children’s Hospital (New England).

The 2022-2023 U.S. News rankings identify the top 50 centers across the United States in each of 10 pediatric specialties: cancer, cardiology/ heart surgery, diabetes/endocrinology, gastroenterology/gastrointestinal surgery, neonatology, nephrology, neurology/neurosurgery, orthopedics, pulmonology/lung surgery, and urology.

For the 2022-2023 rankings, U.S. News requested medical data and other information from 200 pediatric facilities across the United States; 119 responded and were evaluated in at least one specialty, and 90 were ranked in one or more specialties.

Approximately one-third of each hospital’s score was based on outcomes such as survival, infections, and surgical complications (although outcomes counted for 38.3% of scores for cardiology and heart surgery). Approximately 13% of the score was based on reputation/expert opinion, determined by an annual survey of experts in the 10 specialties (8% of scores for cardiology and heart surgery), and nearly 60% was based on patient safety, excellence, and family centeredness, according to a statement from U.S. News.

“The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care, offering parents and their pediatricians a helpful starting point in choosing the facility that’s best for their child,” said Ben Harder, chief of health analysis and managing editor at U.S. News, in a press release accompanying the rankings.

Also new to the ranking system this year was a measure to assess hospitals’ efforts to improve equity of care and to promote diversity and inclusion, which accounts for 2% of each hospital’s score in each specialty, according to U.S. News.

Boston Children’s Hospital led the list of 10 children’s hospitals across the United States named to the Best Children’s Hospitals Honor Roll for 2022-2023, issued by U.S. News & World Report.

The 16th annual Best Children’s Hospitals rankings were published on June 14.

Rounding out the top 10 on the Honor Roll were Children’s Hospital of Philadelphia; Texas Children’s Hospital, Houston; Cincinnati Children’s Hospital Medical Center; Children’s Hospital Los Angeles; Children’s Hospital Colorado, Aurora; Children’s National Hospital, Washington, D.C.; Nationwide Children’s Hospital, Columbus, Ohio; UPMC Children’s Hospital of Pittsburgh; and Lucile Packard Children’s Hospital, Palo Alto, Calif.

The Honor Roll hospitals were chosen based on being highly ranked in multiple specialties, such as cancer, cardiology, and orthopedics.

For the second time, the rankings included top hospitals not only in each state, but also in seven multistate regions. The goal of the regional rankings is to help families identify the high-quality pediatric care centers closest to them, according to the U.S. News press release accompanying the rankings.

The top-ranked hospitals for the seven regions were Children’s Hospital Los Angeles (Pacific); Children’s Hospital Colorado, Aurora (Rocky Mountains); Texas Children’s Hospital, Houston (Southwest); Children’s Healthcare of Atlanta and Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, Tenn. (tie for Southeast); Cincinnati Children’s Hospital Medical Center (Midwest); Children’s Hospital of Philadelphia (Mid-Atlantic); and Boston Children’s Hospital (New England).

The 2022-2023 U.S. News rankings identify the top 50 centers across the United States in each of 10 pediatric specialties: cancer, cardiology/ heart surgery, diabetes/endocrinology, gastroenterology/gastrointestinal surgery, neonatology, nephrology, neurology/neurosurgery, orthopedics, pulmonology/lung surgery, and urology.

For the 2022-2023 rankings, U.S. News requested medical data and other information from 200 pediatric facilities across the United States; 119 responded and were evaluated in at least one specialty, and 90 were ranked in one or more specialties.

Approximately one-third of each hospital’s score was based on outcomes such as survival, infections, and surgical complications (although outcomes counted for 38.3% of scores for cardiology and heart surgery). Approximately 13% of the score was based on reputation/expert opinion, determined by an annual survey of experts in the 10 specialties (8% of scores for cardiology and heart surgery), and nearly 60% was based on patient safety, excellence, and family centeredness, according to a statement from U.S. News.

“The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care, offering parents and their pediatricians a helpful starting point in choosing the facility that’s best for their child,” said Ben Harder, chief of health analysis and managing editor at U.S. News, in a press release accompanying the rankings.

Also new to the ranking system this year was a measure to assess hospitals’ efforts to improve equity of care and to promote diversity and inclusion, which accounts for 2% of each hospital’s score in each specialty, according to U.S. News.

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Momelotinib hits the mark for deadly bone marrow cancer

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The investigational drug momelotinib has shown benefits in myelofibrosis in a new phase 3 trial, which could now lead to a Food and Drug Administration approval.

This drug had previously shown mixed results in a phase 3 trial funded by Gilead, which stopped development of the product; it was acquired by Sierra Oncology, which conducted the latest positive phase 3 trial and now plans to use it to apply for FDA approval.

Momelotinib, an oral Janus kinase 1 and JAK2 inhibitor, significantly outperformed danazol on symptoms, spleen size, and anemia in adults with anemic myelofibrosis in the randomized trial of 195 patients from 21 countries presented at the annual meeting of the American Society of Clinical Oncology.

“The current state for the treatment of myelofibrosis relies on JAK2,” said Ruben Mesa, MD, of the Mays Cancer Center at the UT Health San Antonio MD Anderson Cancer Center.

“Momelotinib is a JAK1 and JAK2 inhibitor.” However, in the early days of studying momelotinib,“it became clear that there was also potentially an improvement in anemia,” which may be related to the additional inhibition of ACVR1, he explained.

Data suggest that the ability to curb anemia in anemic myelofibrosis patients prolongs their lives for up to 8 years, Dr. Mesa added.

Previous studies, notably the phase 3 SIMPLIFY study, showed that momelotinib was associated with comparable effects on spleen volume, transfusion, and total symptom scores from baseline that were similar to ruxolitinib.

In the current study, known as MOMENTUM, a daily dose of momelotinib was compared to danazol for treatment of symptomatic and anemic myelofibrosis (MF) patients who had previously received standard JAK-inhibitor therapy.

In the study, the researchers randomized 130 patients to momelotinib and 65 to danazol. After 24 weeks, those in the danazol group were allowed to cross over to momelotinib. The primary endpoint of the study was total symptom score (TSS) response after 24 weeks. Secondary endpoints included transfusion independence and splenic response at 24 weeks. The median age of the patients in the momelotinib group was 71 years, 60.8% were male, and 82% were white. The baseline demographics were not significantly different in the danazol group.

Overall, 24.6% of momelotinib patients responded with improved total symptom scores at 24 weeks vs. 9.2% of the danazol group. Spleen response also was significantly higher in the momelotinib group; 40% of patients showed a 25% reduction and 23% showed a 35% reduction, compared with 6.2% and 3.1%, respectively, of patients in the danazol group. Transfusion independence at week 24 also was higher for momelotinib patients, compared with danazol patients (31% vs. 20%, respectively, P = 0064).

Adverse events of grade 3 or higher occurred in 53.8% of momelotinib patients and 64.6% of danazol patients, and serious adverse events occurred in 34.6% and 40.0%, respectively. Nearly all patients had anemia, but only 27.7% and 26.2% of the momelotinib and danazol groups, respectively, had thrombocytopenia of grade 3 or higher. The most common nonhematologic adverse events were diarrhea, nausea, and increased blood creatinine. A total of 27.7% of the patients in the momelotinib group discontinued treatment; 16 of whom did so because of an adverse event.

Also, at 24 weeks, patients in the momelotinib group showed a trend towards increased overall survival, compared with danazol (HR, 0.506, P = 0.719).

With momelotinib, there is a consistent thrombocytopenic profile across subgroups, the data on which were presented separately at ASCO (poster 7061), Dr. Mesa added.

“We feel that these findings support the future use of momelotinib as an effective treatment in MF patients, especially those with anemia,” he concluded.
 

Cytopenia data are exciting

The key finding in the current study is that “momelotinib leads to important endpoints including significant improvement in symptoms and spleen reduction,” said Dr. Gabriela Hobbs of Harvard Medical School, Boston, who served as the discussant for the study.

“I think a novel finding of momelotinib that is definitely exciting from the treatment perspective is that momelotinib can also lead to improvement in cytopenias,” she said. “We often have to decide between treating the symptoms of the spleen at the expense of blood counts,” in MF patients, she noted.

The study was sponsored by Sierra Oncology. Dr. Mesa disclosed relationships with companies including Constellation Pharmaceutical, La Jolla Pharma, and study sponsor Sierra Oncology, as well as funding from AbbVie, Celgene, Constellation Pharmaceuticals, CTI, Genentech, Incyte, Mays Cancer Center, NCI, Promedior, and Samus. Dr. Hobbs had no financial conflicts to disclose.

This article was updated 06/14/2022.

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The investigational drug momelotinib has shown benefits in myelofibrosis in a new phase 3 trial, which could now lead to a Food and Drug Administration approval.

This drug had previously shown mixed results in a phase 3 trial funded by Gilead, which stopped development of the product; it was acquired by Sierra Oncology, which conducted the latest positive phase 3 trial and now plans to use it to apply for FDA approval.

Momelotinib, an oral Janus kinase 1 and JAK2 inhibitor, significantly outperformed danazol on symptoms, spleen size, and anemia in adults with anemic myelofibrosis in the randomized trial of 195 patients from 21 countries presented at the annual meeting of the American Society of Clinical Oncology.

“The current state for the treatment of myelofibrosis relies on JAK2,” said Ruben Mesa, MD, of the Mays Cancer Center at the UT Health San Antonio MD Anderson Cancer Center.

“Momelotinib is a JAK1 and JAK2 inhibitor.” However, in the early days of studying momelotinib,“it became clear that there was also potentially an improvement in anemia,” which may be related to the additional inhibition of ACVR1, he explained.

Data suggest that the ability to curb anemia in anemic myelofibrosis patients prolongs their lives for up to 8 years, Dr. Mesa added.

Previous studies, notably the phase 3 SIMPLIFY study, showed that momelotinib was associated with comparable effects on spleen volume, transfusion, and total symptom scores from baseline that were similar to ruxolitinib.

In the current study, known as MOMENTUM, a daily dose of momelotinib was compared to danazol for treatment of symptomatic and anemic myelofibrosis (MF) patients who had previously received standard JAK-inhibitor therapy.

In the study, the researchers randomized 130 patients to momelotinib and 65 to danazol. After 24 weeks, those in the danazol group were allowed to cross over to momelotinib. The primary endpoint of the study was total symptom score (TSS) response after 24 weeks. Secondary endpoints included transfusion independence and splenic response at 24 weeks. The median age of the patients in the momelotinib group was 71 years, 60.8% were male, and 82% were white. The baseline demographics were not significantly different in the danazol group.

Overall, 24.6% of momelotinib patients responded with improved total symptom scores at 24 weeks vs. 9.2% of the danazol group. Spleen response also was significantly higher in the momelotinib group; 40% of patients showed a 25% reduction and 23% showed a 35% reduction, compared with 6.2% and 3.1%, respectively, of patients in the danazol group. Transfusion independence at week 24 also was higher for momelotinib patients, compared with danazol patients (31% vs. 20%, respectively, P = 0064).

Adverse events of grade 3 or higher occurred in 53.8% of momelotinib patients and 64.6% of danazol patients, and serious adverse events occurred in 34.6% and 40.0%, respectively. Nearly all patients had anemia, but only 27.7% and 26.2% of the momelotinib and danazol groups, respectively, had thrombocytopenia of grade 3 or higher. The most common nonhematologic adverse events were diarrhea, nausea, and increased blood creatinine. A total of 27.7% of the patients in the momelotinib group discontinued treatment; 16 of whom did so because of an adverse event.

Also, at 24 weeks, patients in the momelotinib group showed a trend towards increased overall survival, compared with danazol (HR, 0.506, P = 0.719).

With momelotinib, there is a consistent thrombocytopenic profile across subgroups, the data on which were presented separately at ASCO (poster 7061), Dr. Mesa added.

“We feel that these findings support the future use of momelotinib as an effective treatment in MF patients, especially those with anemia,” he concluded.
 

Cytopenia data are exciting

The key finding in the current study is that “momelotinib leads to important endpoints including significant improvement in symptoms and spleen reduction,” said Dr. Gabriela Hobbs of Harvard Medical School, Boston, who served as the discussant for the study.

“I think a novel finding of momelotinib that is definitely exciting from the treatment perspective is that momelotinib can also lead to improvement in cytopenias,” she said. “We often have to decide between treating the symptoms of the spleen at the expense of blood counts,” in MF patients, she noted.

The study was sponsored by Sierra Oncology. Dr. Mesa disclosed relationships with companies including Constellation Pharmaceutical, La Jolla Pharma, and study sponsor Sierra Oncology, as well as funding from AbbVie, Celgene, Constellation Pharmaceuticals, CTI, Genentech, Incyte, Mays Cancer Center, NCI, Promedior, and Samus. Dr. Hobbs had no financial conflicts to disclose.

This article was updated 06/14/2022.

 

The investigational drug momelotinib has shown benefits in myelofibrosis in a new phase 3 trial, which could now lead to a Food and Drug Administration approval.

This drug had previously shown mixed results in a phase 3 trial funded by Gilead, which stopped development of the product; it was acquired by Sierra Oncology, which conducted the latest positive phase 3 trial and now plans to use it to apply for FDA approval.

Momelotinib, an oral Janus kinase 1 and JAK2 inhibitor, significantly outperformed danazol on symptoms, spleen size, and anemia in adults with anemic myelofibrosis in the randomized trial of 195 patients from 21 countries presented at the annual meeting of the American Society of Clinical Oncology.

“The current state for the treatment of myelofibrosis relies on JAK2,” said Ruben Mesa, MD, of the Mays Cancer Center at the UT Health San Antonio MD Anderson Cancer Center.

“Momelotinib is a JAK1 and JAK2 inhibitor.” However, in the early days of studying momelotinib,“it became clear that there was also potentially an improvement in anemia,” which may be related to the additional inhibition of ACVR1, he explained.

Data suggest that the ability to curb anemia in anemic myelofibrosis patients prolongs their lives for up to 8 years, Dr. Mesa added.

Previous studies, notably the phase 3 SIMPLIFY study, showed that momelotinib was associated with comparable effects on spleen volume, transfusion, and total symptom scores from baseline that were similar to ruxolitinib.

In the current study, known as MOMENTUM, a daily dose of momelotinib was compared to danazol for treatment of symptomatic and anemic myelofibrosis (MF) patients who had previously received standard JAK-inhibitor therapy.

In the study, the researchers randomized 130 patients to momelotinib and 65 to danazol. After 24 weeks, those in the danazol group were allowed to cross over to momelotinib. The primary endpoint of the study was total symptom score (TSS) response after 24 weeks. Secondary endpoints included transfusion independence and splenic response at 24 weeks. The median age of the patients in the momelotinib group was 71 years, 60.8% were male, and 82% were white. The baseline demographics were not significantly different in the danazol group.

Overall, 24.6% of momelotinib patients responded with improved total symptom scores at 24 weeks vs. 9.2% of the danazol group. Spleen response also was significantly higher in the momelotinib group; 40% of patients showed a 25% reduction and 23% showed a 35% reduction, compared with 6.2% and 3.1%, respectively, of patients in the danazol group. Transfusion independence at week 24 also was higher for momelotinib patients, compared with danazol patients (31% vs. 20%, respectively, P = 0064).

Adverse events of grade 3 or higher occurred in 53.8% of momelotinib patients and 64.6% of danazol patients, and serious adverse events occurred in 34.6% and 40.0%, respectively. Nearly all patients had anemia, but only 27.7% and 26.2% of the momelotinib and danazol groups, respectively, had thrombocytopenia of grade 3 or higher. The most common nonhematologic adverse events were diarrhea, nausea, and increased blood creatinine. A total of 27.7% of the patients in the momelotinib group discontinued treatment; 16 of whom did so because of an adverse event.

Also, at 24 weeks, patients in the momelotinib group showed a trend towards increased overall survival, compared with danazol (HR, 0.506, P = 0.719).

With momelotinib, there is a consistent thrombocytopenic profile across subgroups, the data on which were presented separately at ASCO (poster 7061), Dr. Mesa added.

“We feel that these findings support the future use of momelotinib as an effective treatment in MF patients, especially those with anemia,” he concluded.
 

Cytopenia data are exciting

The key finding in the current study is that “momelotinib leads to important endpoints including significant improvement in symptoms and spleen reduction,” said Dr. Gabriela Hobbs of Harvard Medical School, Boston, who served as the discussant for the study.

“I think a novel finding of momelotinib that is definitely exciting from the treatment perspective is that momelotinib can also lead to improvement in cytopenias,” she said. “We often have to decide between treating the symptoms of the spleen at the expense of blood counts,” in MF patients, she noted.

The study was sponsored by Sierra Oncology. Dr. Mesa disclosed relationships with companies including Constellation Pharmaceutical, La Jolla Pharma, and study sponsor Sierra Oncology, as well as funding from AbbVie, Celgene, Constellation Pharmaceuticals, CTI, Genentech, Incyte, Mays Cancer Center, NCI, Promedior, and Samus. Dr. Hobbs had no financial conflicts to disclose.

This article was updated 06/14/2022.

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Just 20 minutes of vigorous activity daily benefits teens

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Vigorous physical activity for 20 minutes a day was enough to maximize cardiorespiratory benefits in adolescents, based on data from more than 300 individuals.

Current recommendations for physical activity in children and adolescents from the World Health Organization call for moderate to vigorous physical activity (MVPA) for an average of 60 minutes a day for physical and mental health; however, guidance on how much physical activity teens need to maximize cardiorespiratory fitness (CRF) has not been determined, Samuel Joseph Burden, BMedSci, of John Radcliffe Hospital Oxford (England), and colleagues wrote.

“Although data in young people are limited, adult studies have shown that regular, brief vigorous physical activity is highly effective at improving health markers, including CRF, which is also an important marker of health in youth,” the researchers wrote.

In a study published in Pediatrics, the researchers examined the associations between physical activity intensity and maximal CRF. The study population included 339 adolescents aged 13-14 years who were evaluated during the 2018-2019 and 2019-2020 school years. Participants wore wrist accelerometers to measure the intensity of physical activity and participated in 20-meter shuttle runs to demonstrate CRF. The researchers used partial multivariable linear regression to assess variables at different intensities including moderate physical activity (MPA), light physical activity (LPA), and sedentary time, as well as vigorous physical activity (VPA).

The wrist monitors measured the intensities of physical activity based on the bandpass-filtered followed by Euclidean norm metric (BFEN), a validated metric. “Previously validated thresholds for BFEN were used to determine the average duration of daily physical activity at each intensity: 0.1 g for LPA, 0.314 g for MPA, and 0.998 g for VPA,” the researchers wrote. Physical activity below the threshold for LPA was categorized as sedentary time.

Participants wore the accelerometers for 1 week; value recording included at least 3 weekdays and 1 weekend day, and each valid day required more than 6 hours of awake time.

Overall, VPA for up to 20 minutes was significantly associated with improved CRF. However, the benefits on CRF plateaued after that time, and longer duration of VPA was not associated with significantly greater improvements in CRF. Neither MPA nor LPA were associated with any improvements in CRF.

Participants who engaged in an average of 14 minutes (range, 12-17 minutes) of VPA per day met the median CRF.

The researchers also conducted independent t tests to assess differences in VPA at different CRF thresholds.

Those in the highest quartile of VPA had CRF z scores 1.03 higher, compared with those in the lower quartiles.

Given that current PA guidelines involve a combination of moderate and vigorous PA that could be met by MPA with no VPA, the current findings have public health implications for improving CRF in adolescents, the researchers wrote.

Even with MPA as an option, most adolescents fail to meet the recommendations of at least 60 minutes of MVPA, they said. “One possible reason is that this duration is quite long, requiring a daily time commitment that some may find difficult to maintain. A shorter target of 20 minutes might be easier to schedule daily and a focus on VPA would simplify messages about the intensity of activity that is likely to improve CRF.”

The study findings were limited by several factors including the use of data from only two schools in the United Kingdom, which may limit generalizability, and future research would ideally include a more direct assessment of VO2 max, the researchers wrote. However, the results were strengthened by the large and diverse study population, including teens with a wide range of body mass index as well as CRF.

Future research is needed to test whether interventions based on a target of 20 minutes of VPA creates significant improvements in adolescent cardiometabolic health, the researchers concluded.
 

 

 

Any activity has value for sedentary teens

The current study suggests that counseling teens about physical activity may be less challenging for clinicians if optimal cardiorespiratory benefits can be reached with shorter bouts of activity, Michele LaBotz, MD, of Intermed Sports Medicine, South Portland, Maine, and Sarah Hoffman, DO, of Tufts University, Boston, wrote in an accompanying editorial.

The results have two key implications for pediatricians, the authors said. First, “optimal CRF can be achieved with much shorter periods of activity than previously recommended.” Second, “current ‘moderate to vigorous’ PA recommendations may not be sufficient to improve CRF in adolescents, if achieved through moderate activity only.”

However, the editorialists emphasized that, although shorter periods of higher-intensity exercise reduce the time burden for teens and families, specific education is needed to explain the extra effort involved in exercising vigorously enough for cardiorespiratory benefits.

Patients can be counseled that activity is vigorous when they start to sweat, their face gets red, and they feel short of breath and unable to talk during activity,” they explained. These sensations may be new and uncomfortable for children and teens who have been quite sedentary or used to low-intensity activity. Dr. LaBotz and Dr. Hoffman advised pediatricians to counsel patients to build intensity gradually, with “exercise snacks,” that involve several minutes of activity that become more challenging over time.

“Exercise snacks can include anything that elevates the heart rate for a minute or more, such as running up and down the stairs a few times; chasing the dog around the backyard; or just putting on some music and dancing hard,” the editorialists wrote.

“Some exercise is better than none, and extrapolating from adult data, the biggest benefit likely occurs when we can help our most sedentary and least fit patients become a bit more active, even if it falls short of currently recommended levels,” they concluded.

The study was supported by grants to various researchers from the British Heart Foundation, the Elizabeth Casson Trust, the U.K. National Institute of Health Research, the Professor Nigel Groome Studentship scheme (Oxford Brookes University), and the U.K. Department of Health. The researchers had no financial conflicts to disclose. The editorialists had no financial conflicts to disclose.

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Vigorous physical activity for 20 minutes a day was enough to maximize cardiorespiratory benefits in adolescents, based on data from more than 300 individuals.

Current recommendations for physical activity in children and adolescents from the World Health Organization call for moderate to vigorous physical activity (MVPA) for an average of 60 minutes a day for physical and mental health; however, guidance on how much physical activity teens need to maximize cardiorespiratory fitness (CRF) has not been determined, Samuel Joseph Burden, BMedSci, of John Radcliffe Hospital Oxford (England), and colleagues wrote.

“Although data in young people are limited, adult studies have shown that regular, brief vigorous physical activity is highly effective at improving health markers, including CRF, which is also an important marker of health in youth,” the researchers wrote.

In a study published in Pediatrics, the researchers examined the associations between physical activity intensity and maximal CRF. The study population included 339 adolescents aged 13-14 years who were evaluated during the 2018-2019 and 2019-2020 school years. Participants wore wrist accelerometers to measure the intensity of physical activity and participated in 20-meter shuttle runs to demonstrate CRF. The researchers used partial multivariable linear regression to assess variables at different intensities including moderate physical activity (MPA), light physical activity (LPA), and sedentary time, as well as vigorous physical activity (VPA).

The wrist monitors measured the intensities of physical activity based on the bandpass-filtered followed by Euclidean norm metric (BFEN), a validated metric. “Previously validated thresholds for BFEN were used to determine the average duration of daily physical activity at each intensity: 0.1 g for LPA, 0.314 g for MPA, and 0.998 g for VPA,” the researchers wrote. Physical activity below the threshold for LPA was categorized as sedentary time.

Participants wore the accelerometers for 1 week; value recording included at least 3 weekdays and 1 weekend day, and each valid day required more than 6 hours of awake time.

Overall, VPA for up to 20 minutes was significantly associated with improved CRF. However, the benefits on CRF plateaued after that time, and longer duration of VPA was not associated with significantly greater improvements in CRF. Neither MPA nor LPA were associated with any improvements in CRF.

Participants who engaged in an average of 14 minutes (range, 12-17 minutes) of VPA per day met the median CRF.

The researchers also conducted independent t tests to assess differences in VPA at different CRF thresholds.

Those in the highest quartile of VPA had CRF z scores 1.03 higher, compared with those in the lower quartiles.

Given that current PA guidelines involve a combination of moderate and vigorous PA that could be met by MPA with no VPA, the current findings have public health implications for improving CRF in adolescents, the researchers wrote.

Even with MPA as an option, most adolescents fail to meet the recommendations of at least 60 minutes of MVPA, they said. “One possible reason is that this duration is quite long, requiring a daily time commitment that some may find difficult to maintain. A shorter target of 20 minutes might be easier to schedule daily and a focus on VPA would simplify messages about the intensity of activity that is likely to improve CRF.”

The study findings were limited by several factors including the use of data from only two schools in the United Kingdom, which may limit generalizability, and future research would ideally include a more direct assessment of VO2 max, the researchers wrote. However, the results were strengthened by the large and diverse study population, including teens with a wide range of body mass index as well as CRF.

Future research is needed to test whether interventions based on a target of 20 minutes of VPA creates significant improvements in adolescent cardiometabolic health, the researchers concluded.
 

 

 

Any activity has value for sedentary teens

The current study suggests that counseling teens about physical activity may be less challenging for clinicians if optimal cardiorespiratory benefits can be reached with shorter bouts of activity, Michele LaBotz, MD, of Intermed Sports Medicine, South Portland, Maine, and Sarah Hoffman, DO, of Tufts University, Boston, wrote in an accompanying editorial.

The results have two key implications for pediatricians, the authors said. First, “optimal CRF can be achieved with much shorter periods of activity than previously recommended.” Second, “current ‘moderate to vigorous’ PA recommendations may not be sufficient to improve CRF in adolescents, if achieved through moderate activity only.”

However, the editorialists emphasized that, although shorter periods of higher-intensity exercise reduce the time burden for teens and families, specific education is needed to explain the extra effort involved in exercising vigorously enough for cardiorespiratory benefits.

Patients can be counseled that activity is vigorous when they start to sweat, their face gets red, and they feel short of breath and unable to talk during activity,” they explained. These sensations may be new and uncomfortable for children and teens who have been quite sedentary or used to low-intensity activity. Dr. LaBotz and Dr. Hoffman advised pediatricians to counsel patients to build intensity gradually, with “exercise snacks,” that involve several minutes of activity that become more challenging over time.

“Exercise snacks can include anything that elevates the heart rate for a minute or more, such as running up and down the stairs a few times; chasing the dog around the backyard; or just putting on some music and dancing hard,” the editorialists wrote.

“Some exercise is better than none, and extrapolating from adult data, the biggest benefit likely occurs when we can help our most sedentary and least fit patients become a bit more active, even if it falls short of currently recommended levels,” they concluded.

The study was supported by grants to various researchers from the British Heart Foundation, the Elizabeth Casson Trust, the U.K. National Institute of Health Research, the Professor Nigel Groome Studentship scheme (Oxford Brookes University), and the U.K. Department of Health. The researchers had no financial conflicts to disclose. The editorialists had no financial conflicts to disclose.

Vigorous physical activity for 20 minutes a day was enough to maximize cardiorespiratory benefits in adolescents, based on data from more than 300 individuals.

Current recommendations for physical activity in children and adolescents from the World Health Organization call for moderate to vigorous physical activity (MVPA) for an average of 60 minutes a day for physical and mental health; however, guidance on how much physical activity teens need to maximize cardiorespiratory fitness (CRF) has not been determined, Samuel Joseph Burden, BMedSci, of John Radcliffe Hospital Oxford (England), and colleagues wrote.

“Although data in young people are limited, adult studies have shown that regular, brief vigorous physical activity is highly effective at improving health markers, including CRF, which is also an important marker of health in youth,” the researchers wrote.

In a study published in Pediatrics, the researchers examined the associations between physical activity intensity and maximal CRF. The study population included 339 adolescents aged 13-14 years who were evaluated during the 2018-2019 and 2019-2020 school years. Participants wore wrist accelerometers to measure the intensity of physical activity and participated in 20-meter shuttle runs to demonstrate CRF. The researchers used partial multivariable linear regression to assess variables at different intensities including moderate physical activity (MPA), light physical activity (LPA), and sedentary time, as well as vigorous physical activity (VPA).

The wrist monitors measured the intensities of physical activity based on the bandpass-filtered followed by Euclidean norm metric (BFEN), a validated metric. “Previously validated thresholds for BFEN were used to determine the average duration of daily physical activity at each intensity: 0.1 g for LPA, 0.314 g for MPA, and 0.998 g for VPA,” the researchers wrote. Physical activity below the threshold for LPA was categorized as sedentary time.

Participants wore the accelerometers for 1 week; value recording included at least 3 weekdays and 1 weekend day, and each valid day required more than 6 hours of awake time.

Overall, VPA for up to 20 minutes was significantly associated with improved CRF. However, the benefits on CRF plateaued after that time, and longer duration of VPA was not associated with significantly greater improvements in CRF. Neither MPA nor LPA were associated with any improvements in CRF.

Participants who engaged in an average of 14 minutes (range, 12-17 minutes) of VPA per day met the median CRF.

The researchers also conducted independent t tests to assess differences in VPA at different CRF thresholds.

Those in the highest quartile of VPA had CRF z scores 1.03 higher, compared with those in the lower quartiles.

Given that current PA guidelines involve a combination of moderate and vigorous PA that could be met by MPA with no VPA, the current findings have public health implications for improving CRF in adolescents, the researchers wrote.

Even with MPA as an option, most adolescents fail to meet the recommendations of at least 60 minutes of MVPA, they said. “One possible reason is that this duration is quite long, requiring a daily time commitment that some may find difficult to maintain. A shorter target of 20 minutes might be easier to schedule daily and a focus on VPA would simplify messages about the intensity of activity that is likely to improve CRF.”

The study findings were limited by several factors including the use of data from only two schools in the United Kingdom, which may limit generalizability, and future research would ideally include a more direct assessment of VO2 max, the researchers wrote. However, the results were strengthened by the large and diverse study population, including teens with a wide range of body mass index as well as CRF.

Future research is needed to test whether interventions based on a target of 20 minutes of VPA creates significant improvements in adolescent cardiometabolic health, the researchers concluded.
 

 

 

Any activity has value for sedentary teens

The current study suggests that counseling teens about physical activity may be less challenging for clinicians if optimal cardiorespiratory benefits can be reached with shorter bouts of activity, Michele LaBotz, MD, of Intermed Sports Medicine, South Portland, Maine, and Sarah Hoffman, DO, of Tufts University, Boston, wrote in an accompanying editorial.

The results have two key implications for pediatricians, the authors said. First, “optimal CRF can be achieved with much shorter periods of activity than previously recommended.” Second, “current ‘moderate to vigorous’ PA recommendations may not be sufficient to improve CRF in adolescents, if achieved through moderate activity only.”

However, the editorialists emphasized that, although shorter periods of higher-intensity exercise reduce the time burden for teens and families, specific education is needed to explain the extra effort involved in exercising vigorously enough for cardiorespiratory benefits.

Patients can be counseled that activity is vigorous when they start to sweat, their face gets red, and they feel short of breath and unable to talk during activity,” they explained. These sensations may be new and uncomfortable for children and teens who have been quite sedentary or used to low-intensity activity. Dr. LaBotz and Dr. Hoffman advised pediatricians to counsel patients to build intensity gradually, with “exercise snacks,” that involve several minutes of activity that become more challenging over time.

“Exercise snacks can include anything that elevates the heart rate for a minute or more, such as running up and down the stairs a few times; chasing the dog around the backyard; or just putting on some music and dancing hard,” the editorialists wrote.

“Some exercise is better than none, and extrapolating from adult data, the biggest benefit likely occurs when we can help our most sedentary and least fit patients become a bit more active, even if it falls short of currently recommended levels,” they concluded.

The study was supported by grants to various researchers from the British Heart Foundation, the Elizabeth Casson Trust, the U.K. National Institute of Health Research, the Professor Nigel Groome Studentship scheme (Oxford Brookes University), and the U.K. Department of Health. The researchers had no financial conflicts to disclose. The editorialists had no financial conflicts to disclose.

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Novel gene therapy offers hope for some lymphomas

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Lisocabtagene maraleucel, a novel chimeric antigen receptor (CAR) T-cell product, evoked a clinical response in patients with relapsed or refractory large B-cell lymphoma in a pilot study presented at the annual meeting of the American Society of Clinical Oncology.

Not all patients with relapsed or refractory large B cell lymphoma (r/r LBCL) are candidates for high-dose chemotherapy or hematopoietic stem cell transplantation (HSCT), and options for second-line therapies for this population are limited, said Dr. Alison Sehgal of the University of Pittsburgh Medical Center in her presentation of the findings.

Lisocabtagene maraleucel (liso-cel) is a CD19-directed CAR T-cell product. In a previous phase 3 randomized trial (the TRANSFORM study), lisocabtagene showed superiority over salvage chemotherapy for LBCL patients who were fit candidates for stem cell transplant, but its use in older, frail patients who are not transplant candidates remains uncertain, wrote Dr. Sehgal and colleagues in their poster at the meeting.

In the study, the researchers identified 74 patients with r/r LBCL. Of these, 61 were treated with liso-cel. The patients ranged in age from 53 to 84 years, with a median age of 74 years, 61% were male, and 89% were white. Approximately half were refractory and half were relapsed.

For the therapy, patients underwent lymphodepletion with cyclophosphamide and fludarabine, followed 2-7 days later by an infusion of liso-cel at a target dose of 100 x 106 CAR+ T cells; all patients had at least 6 months of follow-up from their first response.

The primary endpoint of overall response rate occurred in 80% of the patients, and clinically meaningful complete response occurred in 54% over a median follow-up of 12.3 months.

“Clinically meaningful CRs were observed across all subgroups,” Dr. Sehgal said in her presentation.

The response lasted a median of 21.7 months, and the median follow-up for duration of response was 15.5 months. The median overall survival was not reached, but the median progression-free survival was 9.0 months, with a median follow-up period of 13.0 months.

Responses occurred across all prespecified subgroups, with no significant differences in either safety or efficacy based on hematopoietic cell transplantation–specific comorbidity index (HCT-CI) scores.

“Despite the advanced age and comorbidities of the population, the safety profile was consistent with previous reports,” and no new or increased safety signals appeared, Dr. Sehgal said.

The most common treatment-emergent adverse events of grade 3 or higher were neutropenia (48%), leukopenia (21%), thrombocytopenia (20%), and anemia (11%). Cytokine-release syndrome (CRS) occurred in 23 patients (38%); of these, 1 patient was grade 3 and none were grades 4 or 5.

Approximately one-third of the patients (31%) experienced neurological events during the study; three cases were grade 3, none were grades 4 or 5. Patients with CRS or NE were treated with tocilizumab (10%), corticosteroids (3%), or both (20%). Treatment-emergent adverse events of grade 3 or higher occurred in 79% of patients overall, including grade 5 events in two patients because of COVID-19.

The study findings were limited by the small sample size and lack of controls. However, the results support the potential use of liso-cel as a second-line therapy for r/r LBCL patients who are not candidates for HSCT, Dr. Sehgal concluded.
 

 

 

Addressing an ongoing unmet need

In an interview, study coauthor Dr. Leo I. Gordon of Northwestern University, Chicago, observed, “Patients with relapsed or refractory large B-cell lymphoma who are not considered candidates for stem cell transplant following first-line treatment, based on age, comorbidities, health status, or other prognostic factors, have more difficult-to-treat disease, poor prognosis, and more limited treatment options.”

Dr. Gordon noted that the PILOT study is the only trial to evaluate a CAR T-cell therapy as a second-line treatment for r/r LBCL patients who are not considered candidates for stem cell transplant.

“Data from the primary analysis of the PILOT study further demonstrate the potential value of using CAR T-cell therapies earlier in the treatment paradigm for relapsed or refractory LBCL to help improve clinical outcomes and address ongoing unmet need,” he said.

CAR T-cell therapies have shown benefits in later lines for r/r LBCL and as a second-line treatment for r/r LBCL patients who are deemed candidates for stem cell transplant, “so we were encouraged and not surprised by these data.”

However, Dr. Gordon noted, “There may be some patients with similar presentations that might have a transplant, so one limitation of the trial is how one defines patients where transplant is the intended therapy, and that assessment varies among institutions and clinicians.”

An application for liso-cel as a treatment for patients with r/r LBCL who have failed front-line therapy is currently under Priority Review with the FDA, with a Prescription Drug User Fee Act (PDUFA) goal date of June 24, 2022, he added.
 

Liso-cel may fill treatment gap as second-line therapy

The current study is important because “the long-term outcomes of patients with relapsed or refractory large B-cell lymphoma who are not candidates for stem cell transplantation is very poor,” said Dr. Brian Till of Fred Hutchinson Cancer Research Center, Seattle, in an interview.

“CAR T therapy leads to about a 40% cure rate, but is currently only available in this population after the failure of second-line therapy,” said Dr. Till, who was not involved in the study.

“Given that liso-cel was shown to improve outcomes in the second-line setting among transplant candidates, it is logical to consider it as second-line therapy in nontransplant candidates as well, who are otherwise fit enough to receive CAR T therapy,” Dr. Till explained.

“This study showed a rate of long-term progression-free survival similar to what has been observed in the third-line setting and was reasonably well tolerated in these older patients,” said Dr. Till. The results suggest “that second-line liso-cel may be an attractive treatment strategy for patients who are not candidates for stem cell transplantation due to advanced age or comorbidities,” he noted.

Dr. Till had no relevant financial conflicts to disclose.

The study was funded by Bristol Myers Squibb.

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Lisocabtagene maraleucel, a novel chimeric antigen receptor (CAR) T-cell product, evoked a clinical response in patients with relapsed or refractory large B-cell lymphoma in a pilot study presented at the annual meeting of the American Society of Clinical Oncology.

Not all patients with relapsed or refractory large B cell lymphoma (r/r LBCL) are candidates for high-dose chemotherapy or hematopoietic stem cell transplantation (HSCT), and options for second-line therapies for this population are limited, said Dr. Alison Sehgal of the University of Pittsburgh Medical Center in her presentation of the findings.

Lisocabtagene maraleucel (liso-cel) is a CD19-directed CAR T-cell product. In a previous phase 3 randomized trial (the TRANSFORM study), lisocabtagene showed superiority over salvage chemotherapy for LBCL patients who were fit candidates for stem cell transplant, but its use in older, frail patients who are not transplant candidates remains uncertain, wrote Dr. Sehgal and colleagues in their poster at the meeting.

In the study, the researchers identified 74 patients with r/r LBCL. Of these, 61 were treated with liso-cel. The patients ranged in age from 53 to 84 years, with a median age of 74 years, 61% were male, and 89% were white. Approximately half were refractory and half were relapsed.

For the therapy, patients underwent lymphodepletion with cyclophosphamide and fludarabine, followed 2-7 days later by an infusion of liso-cel at a target dose of 100 x 106 CAR+ T cells; all patients had at least 6 months of follow-up from their first response.

The primary endpoint of overall response rate occurred in 80% of the patients, and clinically meaningful complete response occurred in 54% over a median follow-up of 12.3 months.

“Clinically meaningful CRs were observed across all subgroups,” Dr. Sehgal said in her presentation.

The response lasted a median of 21.7 months, and the median follow-up for duration of response was 15.5 months. The median overall survival was not reached, but the median progression-free survival was 9.0 months, with a median follow-up period of 13.0 months.

Responses occurred across all prespecified subgroups, with no significant differences in either safety or efficacy based on hematopoietic cell transplantation–specific comorbidity index (HCT-CI) scores.

“Despite the advanced age and comorbidities of the population, the safety profile was consistent with previous reports,” and no new or increased safety signals appeared, Dr. Sehgal said.

The most common treatment-emergent adverse events of grade 3 or higher were neutropenia (48%), leukopenia (21%), thrombocytopenia (20%), and anemia (11%). Cytokine-release syndrome (CRS) occurred in 23 patients (38%); of these, 1 patient was grade 3 and none were grades 4 or 5.

Approximately one-third of the patients (31%) experienced neurological events during the study; three cases were grade 3, none were grades 4 or 5. Patients with CRS or NE were treated with tocilizumab (10%), corticosteroids (3%), or both (20%). Treatment-emergent adverse events of grade 3 or higher occurred in 79% of patients overall, including grade 5 events in two patients because of COVID-19.

The study findings were limited by the small sample size and lack of controls. However, the results support the potential use of liso-cel as a second-line therapy for r/r LBCL patients who are not candidates for HSCT, Dr. Sehgal concluded.
 

 

 

Addressing an ongoing unmet need

In an interview, study coauthor Dr. Leo I. Gordon of Northwestern University, Chicago, observed, “Patients with relapsed or refractory large B-cell lymphoma who are not considered candidates for stem cell transplant following first-line treatment, based on age, comorbidities, health status, or other prognostic factors, have more difficult-to-treat disease, poor prognosis, and more limited treatment options.”

Dr. Gordon noted that the PILOT study is the only trial to evaluate a CAR T-cell therapy as a second-line treatment for r/r LBCL patients who are not considered candidates for stem cell transplant.

“Data from the primary analysis of the PILOT study further demonstrate the potential value of using CAR T-cell therapies earlier in the treatment paradigm for relapsed or refractory LBCL to help improve clinical outcomes and address ongoing unmet need,” he said.

CAR T-cell therapies have shown benefits in later lines for r/r LBCL and as a second-line treatment for r/r LBCL patients who are deemed candidates for stem cell transplant, “so we were encouraged and not surprised by these data.”

However, Dr. Gordon noted, “There may be some patients with similar presentations that might have a transplant, so one limitation of the trial is how one defines patients where transplant is the intended therapy, and that assessment varies among institutions and clinicians.”

An application for liso-cel as a treatment for patients with r/r LBCL who have failed front-line therapy is currently under Priority Review with the FDA, with a Prescription Drug User Fee Act (PDUFA) goal date of June 24, 2022, he added.
 

Liso-cel may fill treatment gap as second-line therapy

The current study is important because “the long-term outcomes of patients with relapsed or refractory large B-cell lymphoma who are not candidates for stem cell transplantation is very poor,” said Dr. Brian Till of Fred Hutchinson Cancer Research Center, Seattle, in an interview.

“CAR T therapy leads to about a 40% cure rate, but is currently only available in this population after the failure of second-line therapy,” said Dr. Till, who was not involved in the study.

“Given that liso-cel was shown to improve outcomes in the second-line setting among transplant candidates, it is logical to consider it as second-line therapy in nontransplant candidates as well, who are otherwise fit enough to receive CAR T therapy,” Dr. Till explained.

“This study showed a rate of long-term progression-free survival similar to what has been observed in the third-line setting and was reasonably well tolerated in these older patients,” said Dr. Till. The results suggest “that second-line liso-cel may be an attractive treatment strategy for patients who are not candidates for stem cell transplantation due to advanced age or comorbidities,” he noted.

Dr. Till had no relevant financial conflicts to disclose.

The study was funded by Bristol Myers Squibb.

Lisocabtagene maraleucel, a novel chimeric antigen receptor (CAR) T-cell product, evoked a clinical response in patients with relapsed or refractory large B-cell lymphoma in a pilot study presented at the annual meeting of the American Society of Clinical Oncology.

Not all patients with relapsed or refractory large B cell lymphoma (r/r LBCL) are candidates for high-dose chemotherapy or hematopoietic stem cell transplantation (HSCT), and options for second-line therapies for this population are limited, said Dr. Alison Sehgal of the University of Pittsburgh Medical Center in her presentation of the findings.

Lisocabtagene maraleucel (liso-cel) is a CD19-directed CAR T-cell product. In a previous phase 3 randomized trial (the TRANSFORM study), lisocabtagene showed superiority over salvage chemotherapy for LBCL patients who were fit candidates for stem cell transplant, but its use in older, frail patients who are not transplant candidates remains uncertain, wrote Dr. Sehgal and colleagues in their poster at the meeting.

In the study, the researchers identified 74 patients with r/r LBCL. Of these, 61 were treated with liso-cel. The patients ranged in age from 53 to 84 years, with a median age of 74 years, 61% were male, and 89% were white. Approximately half were refractory and half were relapsed.

For the therapy, patients underwent lymphodepletion with cyclophosphamide and fludarabine, followed 2-7 days later by an infusion of liso-cel at a target dose of 100 x 106 CAR+ T cells; all patients had at least 6 months of follow-up from their first response.

The primary endpoint of overall response rate occurred in 80% of the patients, and clinically meaningful complete response occurred in 54% over a median follow-up of 12.3 months.

“Clinically meaningful CRs were observed across all subgroups,” Dr. Sehgal said in her presentation.

The response lasted a median of 21.7 months, and the median follow-up for duration of response was 15.5 months. The median overall survival was not reached, but the median progression-free survival was 9.0 months, with a median follow-up period of 13.0 months.

Responses occurred across all prespecified subgroups, with no significant differences in either safety or efficacy based on hematopoietic cell transplantation–specific comorbidity index (HCT-CI) scores.

“Despite the advanced age and comorbidities of the population, the safety profile was consistent with previous reports,” and no new or increased safety signals appeared, Dr. Sehgal said.

The most common treatment-emergent adverse events of grade 3 or higher were neutropenia (48%), leukopenia (21%), thrombocytopenia (20%), and anemia (11%). Cytokine-release syndrome (CRS) occurred in 23 patients (38%); of these, 1 patient was grade 3 and none were grades 4 or 5.

Approximately one-third of the patients (31%) experienced neurological events during the study; three cases were grade 3, none were grades 4 or 5. Patients with CRS or NE were treated with tocilizumab (10%), corticosteroids (3%), or both (20%). Treatment-emergent adverse events of grade 3 or higher occurred in 79% of patients overall, including grade 5 events in two patients because of COVID-19.

The study findings were limited by the small sample size and lack of controls. However, the results support the potential use of liso-cel as a second-line therapy for r/r LBCL patients who are not candidates for HSCT, Dr. Sehgal concluded.
 

 

 

Addressing an ongoing unmet need

In an interview, study coauthor Dr. Leo I. Gordon of Northwestern University, Chicago, observed, “Patients with relapsed or refractory large B-cell lymphoma who are not considered candidates for stem cell transplant following first-line treatment, based on age, comorbidities, health status, or other prognostic factors, have more difficult-to-treat disease, poor prognosis, and more limited treatment options.”

Dr. Gordon noted that the PILOT study is the only trial to evaluate a CAR T-cell therapy as a second-line treatment for r/r LBCL patients who are not considered candidates for stem cell transplant.

“Data from the primary analysis of the PILOT study further demonstrate the potential value of using CAR T-cell therapies earlier in the treatment paradigm for relapsed or refractory LBCL to help improve clinical outcomes and address ongoing unmet need,” he said.

CAR T-cell therapies have shown benefits in later lines for r/r LBCL and as a second-line treatment for r/r LBCL patients who are deemed candidates for stem cell transplant, “so we were encouraged and not surprised by these data.”

However, Dr. Gordon noted, “There may be some patients with similar presentations that might have a transplant, so one limitation of the trial is how one defines patients where transplant is the intended therapy, and that assessment varies among institutions and clinicians.”

An application for liso-cel as a treatment for patients with r/r LBCL who have failed front-line therapy is currently under Priority Review with the FDA, with a Prescription Drug User Fee Act (PDUFA) goal date of June 24, 2022, he added.
 

Liso-cel may fill treatment gap as second-line therapy

The current study is important because “the long-term outcomes of patients with relapsed or refractory large B-cell lymphoma who are not candidates for stem cell transplantation is very poor,” said Dr. Brian Till of Fred Hutchinson Cancer Research Center, Seattle, in an interview.

“CAR T therapy leads to about a 40% cure rate, but is currently only available in this population after the failure of second-line therapy,” said Dr. Till, who was not involved in the study.

“Given that liso-cel was shown to improve outcomes in the second-line setting among transplant candidates, it is logical to consider it as second-line therapy in nontransplant candidates as well, who are otherwise fit enough to receive CAR T therapy,” Dr. Till explained.

“This study showed a rate of long-term progression-free survival similar to what has been observed in the third-line setting and was reasonably well tolerated in these older patients,” said Dr. Till. The results suggest “that second-line liso-cel may be an attractive treatment strategy for patients who are not candidates for stem cell transplantation due to advanced age or comorbidities,” he noted.

Dr. Till had no relevant financial conflicts to disclose.

The study was funded by Bristol Myers Squibb.

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Glofitamab prevails against r/r DLBCL

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Approximately half of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) treated with glofitamab showed clinical response, based on phase 2 data from 154 adults.

“We know that relapsed diffuse large B-cell lymphoma has a particularly poor prognosis,” especially for patients who have undergone at least two therapies, Dr. Michael Dickinson, MBBS, of the University of Melbourne said in a presentation at the annual meeting of the American Society of Clinical Oncology.

Bispecific T cell–recruiting antibodies are emerging as a novel treatment option for B-cell cancers, said Dr. Dickinson.

Glofitamab is distinct among these therapies for its configuration that is bivalent for CD20 and monovalent for CD3, providing more potency than a 1:1 format, Dr. Dickinson explained in his presentation.

The study population included 154 adults aged 21-90 years with DLBCL who had received at least two prior treatments; all had received prior anti-CD20 Ab, and 149 had received anthracycline. The median age of the patients was 66 years, 65% were male, 75% had Ann Arbor stage III or IV disease, and 90% were refractory to any prior therapy.

The patients received intravenous glutamate-pyruvate transaminase, followed by an initial intravenous dose of glofitamab 7 days later. Glofitamab was given as step-up doses starting with 2.5 mg to a target of 30 mg.

The primary endpoint was complete response (CR) during initial treatment using Independent Review Committee (IRC) criteria, with overall response rate, duration of response, duration of complete response, progression-free survival, and overall survival as key secondary outcomes.

At a median of 12.6 months’ follow-up, the CR and overall response rates were 39.4% and 51.6%, respectively.

“Responses were achieved early; the median time to first complete response was 42 days,” Dr. Dickinson reported. Of the 38 patients with CR at the data cutoff point, 33 remained in complete remission (87%) based on IRC criteria. Complete response rates were consistent across prespecified subgroups, notably 42% of patients with no prior chimeric antigen receptor T-cell therapy and 70% and 34%, of those who were relapsed or refractory, respectively, to their last prior treatments.

The median duration of overall response was 18.4 months, and the median duration of complete response had not yet been estimated, Dr. Dickinson said. The median progression-free survival and overall survival rates were 4.9 months and 11.5 months, respectively, and the estimated 12-month overall survival was 49.8%.

“These are highly clinically significant results for this difficult to treat population,” Dr. Dickinson said in his presentation.

The most common adverse event was cytokine release syndrome (CRS), which occurred in 63% of patients. Of these, 3.9% were grade 3 or 4. Patients received corticosteroids (27.8%) or tocilizumab for management of CRS.

“As we have shown before, this is a first-course phenomenon, becoming far less frequent once step up dosing is complete,” Dr. Dickinson said in the presentation. “The median time to CRS is predictable, occurring around 10 hours after the IV infusion,” he said. Overall, 3.2% of the patients discontinued because of an adverse event.

A total of eight deaths occurred during the study; five of these were related to COVID-19 and the remaining three were in patients with manifest progression of disease.

Infections are to be expected in such a heavily treated population, and 14.9% of patients developed infections of grade 3 or higher, said Dr. Dickinson. Neutropenia of any grade occurred in 37.7%, febrile neutropenia in 2.6%. Neurological events occurred in 38.3% of patients; 3.2% were grade 3 or higher.

The study did not prospectively record immune effector cell–associated neurotoxicity syndrome, Dr. Dickinson said, but an estimate suggests a rate of 2.6%, and none of the events were considered to be related to glofitamab, he noted.

The researchers also looked at a supporting cohort of 35 patients with a median follow up of more than 2 years. In this group, the complete remission rate was 35% and the median duration of remission was 34.2 months. “Our six longest patients have been in remission for longer than 3 years,” Dr. Dickinson said.

The latest glofitamab data “reflect routine practice and an area of need for this disease,” said Dr. Dickinson.

“I think these results will prove to be very meaningful for our patients with large cell lymphoma, and this drug will prove to be an important treatment option,” he said.
 

 

 

More follow-up needed, but findings show promise

A number of CD20/CD3-bispecific antibodies are in development for patients with relapsed/refractory B-cell lymphomas, said study discussant Kerry J. Savage, MD, of the University of British Columbia, Vancouver, who served as the discussant for the session.

Glofitamab differs from other treatments in that it is bivalent for CD20 and monovalent for CD3, “which imparts greater potency,” she noted. Glofitamab also has a silent Fc region that is designed to extend half-life and reduce toxicity.

Patients in the current study had at least two prior regimens, and importantly, “CR rates were similar, regardless of prior therapy,” said Dr. Savage. The longer follow-up cohort provides “a hint that the response may be durable.”

Looking ahead, “the important thing will be response durability” with longer follow-up, she added. “We don’t know the curative potential yet, but the results are encouraging so far.”

In the meantime, “the best use of bispecific antibodies is through clinical trials,” Dr. Savage said. “Keep an eye out for bispecific antibody combination trials as well.”

The study was funded by F. Hoffmann–La Roche. Dr. Dickinson disclosed honoraria from or serving as a consultant to companies including Amgen, Bristol Myers-Squibb, Gilead Sciences, Janssen, MSD, Novartis, and Roche. Dr. Savage disclosed relationships, funding, and support from multiple companies including Bristol Myers-Squibb, Janssen Oncology, Kyowa, Merck, Novartis Canada Pharmaceuticals, Seattle Genetics and Roche.

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Approximately half of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) treated with glofitamab showed clinical response, based on phase 2 data from 154 adults.

“We know that relapsed diffuse large B-cell lymphoma has a particularly poor prognosis,” especially for patients who have undergone at least two therapies, Dr. Michael Dickinson, MBBS, of the University of Melbourne said in a presentation at the annual meeting of the American Society of Clinical Oncology.

Bispecific T cell–recruiting antibodies are emerging as a novel treatment option for B-cell cancers, said Dr. Dickinson.

Glofitamab is distinct among these therapies for its configuration that is bivalent for CD20 and monovalent for CD3, providing more potency than a 1:1 format, Dr. Dickinson explained in his presentation.

The study population included 154 adults aged 21-90 years with DLBCL who had received at least two prior treatments; all had received prior anti-CD20 Ab, and 149 had received anthracycline. The median age of the patients was 66 years, 65% were male, 75% had Ann Arbor stage III or IV disease, and 90% were refractory to any prior therapy.

The patients received intravenous glutamate-pyruvate transaminase, followed by an initial intravenous dose of glofitamab 7 days later. Glofitamab was given as step-up doses starting with 2.5 mg to a target of 30 mg.

The primary endpoint was complete response (CR) during initial treatment using Independent Review Committee (IRC) criteria, with overall response rate, duration of response, duration of complete response, progression-free survival, and overall survival as key secondary outcomes.

At a median of 12.6 months’ follow-up, the CR and overall response rates were 39.4% and 51.6%, respectively.

“Responses were achieved early; the median time to first complete response was 42 days,” Dr. Dickinson reported. Of the 38 patients with CR at the data cutoff point, 33 remained in complete remission (87%) based on IRC criteria. Complete response rates were consistent across prespecified subgroups, notably 42% of patients with no prior chimeric antigen receptor T-cell therapy and 70% and 34%, of those who were relapsed or refractory, respectively, to their last prior treatments.

The median duration of overall response was 18.4 months, and the median duration of complete response had not yet been estimated, Dr. Dickinson said. The median progression-free survival and overall survival rates were 4.9 months and 11.5 months, respectively, and the estimated 12-month overall survival was 49.8%.

“These are highly clinically significant results for this difficult to treat population,” Dr. Dickinson said in his presentation.

The most common adverse event was cytokine release syndrome (CRS), which occurred in 63% of patients. Of these, 3.9% were grade 3 or 4. Patients received corticosteroids (27.8%) or tocilizumab for management of CRS.

“As we have shown before, this is a first-course phenomenon, becoming far less frequent once step up dosing is complete,” Dr. Dickinson said in the presentation. “The median time to CRS is predictable, occurring around 10 hours after the IV infusion,” he said. Overall, 3.2% of the patients discontinued because of an adverse event.

A total of eight deaths occurred during the study; five of these were related to COVID-19 and the remaining three were in patients with manifest progression of disease.

Infections are to be expected in such a heavily treated population, and 14.9% of patients developed infections of grade 3 or higher, said Dr. Dickinson. Neutropenia of any grade occurred in 37.7%, febrile neutropenia in 2.6%. Neurological events occurred in 38.3% of patients; 3.2% were grade 3 or higher.

The study did not prospectively record immune effector cell–associated neurotoxicity syndrome, Dr. Dickinson said, but an estimate suggests a rate of 2.6%, and none of the events were considered to be related to glofitamab, he noted.

The researchers also looked at a supporting cohort of 35 patients with a median follow up of more than 2 years. In this group, the complete remission rate was 35% and the median duration of remission was 34.2 months. “Our six longest patients have been in remission for longer than 3 years,” Dr. Dickinson said.

The latest glofitamab data “reflect routine practice and an area of need for this disease,” said Dr. Dickinson.

“I think these results will prove to be very meaningful for our patients with large cell lymphoma, and this drug will prove to be an important treatment option,” he said.
 

 

 

More follow-up needed, but findings show promise

A number of CD20/CD3-bispecific antibodies are in development for patients with relapsed/refractory B-cell lymphomas, said study discussant Kerry J. Savage, MD, of the University of British Columbia, Vancouver, who served as the discussant for the session.

Glofitamab differs from other treatments in that it is bivalent for CD20 and monovalent for CD3, “which imparts greater potency,” she noted. Glofitamab also has a silent Fc region that is designed to extend half-life and reduce toxicity.

Patients in the current study had at least two prior regimens, and importantly, “CR rates were similar, regardless of prior therapy,” said Dr. Savage. The longer follow-up cohort provides “a hint that the response may be durable.”

Looking ahead, “the important thing will be response durability” with longer follow-up, she added. “We don’t know the curative potential yet, but the results are encouraging so far.”

In the meantime, “the best use of bispecific antibodies is through clinical trials,” Dr. Savage said. “Keep an eye out for bispecific antibody combination trials as well.”

The study was funded by F. Hoffmann–La Roche. Dr. Dickinson disclosed honoraria from or serving as a consultant to companies including Amgen, Bristol Myers-Squibb, Gilead Sciences, Janssen, MSD, Novartis, and Roche. Dr. Savage disclosed relationships, funding, and support from multiple companies including Bristol Myers-Squibb, Janssen Oncology, Kyowa, Merck, Novartis Canada Pharmaceuticals, Seattle Genetics and Roche.

Approximately half of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) treated with glofitamab showed clinical response, based on phase 2 data from 154 adults.

“We know that relapsed diffuse large B-cell lymphoma has a particularly poor prognosis,” especially for patients who have undergone at least two therapies, Dr. Michael Dickinson, MBBS, of the University of Melbourne said in a presentation at the annual meeting of the American Society of Clinical Oncology.

Bispecific T cell–recruiting antibodies are emerging as a novel treatment option for B-cell cancers, said Dr. Dickinson.

Glofitamab is distinct among these therapies for its configuration that is bivalent for CD20 and monovalent for CD3, providing more potency than a 1:1 format, Dr. Dickinson explained in his presentation.

The study population included 154 adults aged 21-90 years with DLBCL who had received at least two prior treatments; all had received prior anti-CD20 Ab, and 149 had received anthracycline. The median age of the patients was 66 years, 65% were male, 75% had Ann Arbor stage III or IV disease, and 90% were refractory to any prior therapy.

The patients received intravenous glutamate-pyruvate transaminase, followed by an initial intravenous dose of glofitamab 7 days later. Glofitamab was given as step-up doses starting with 2.5 mg to a target of 30 mg.

The primary endpoint was complete response (CR) during initial treatment using Independent Review Committee (IRC) criteria, with overall response rate, duration of response, duration of complete response, progression-free survival, and overall survival as key secondary outcomes.

At a median of 12.6 months’ follow-up, the CR and overall response rates were 39.4% and 51.6%, respectively.

“Responses were achieved early; the median time to first complete response was 42 days,” Dr. Dickinson reported. Of the 38 patients with CR at the data cutoff point, 33 remained in complete remission (87%) based on IRC criteria. Complete response rates were consistent across prespecified subgroups, notably 42% of patients with no prior chimeric antigen receptor T-cell therapy and 70% and 34%, of those who were relapsed or refractory, respectively, to their last prior treatments.

The median duration of overall response was 18.4 months, and the median duration of complete response had not yet been estimated, Dr. Dickinson said. The median progression-free survival and overall survival rates were 4.9 months and 11.5 months, respectively, and the estimated 12-month overall survival was 49.8%.

“These are highly clinically significant results for this difficult to treat population,” Dr. Dickinson said in his presentation.

The most common adverse event was cytokine release syndrome (CRS), which occurred in 63% of patients. Of these, 3.9% were grade 3 or 4. Patients received corticosteroids (27.8%) or tocilizumab for management of CRS.

“As we have shown before, this is a first-course phenomenon, becoming far less frequent once step up dosing is complete,” Dr. Dickinson said in the presentation. “The median time to CRS is predictable, occurring around 10 hours after the IV infusion,” he said. Overall, 3.2% of the patients discontinued because of an adverse event.

A total of eight deaths occurred during the study; five of these were related to COVID-19 and the remaining three were in patients with manifest progression of disease.

Infections are to be expected in such a heavily treated population, and 14.9% of patients developed infections of grade 3 or higher, said Dr. Dickinson. Neutropenia of any grade occurred in 37.7%, febrile neutropenia in 2.6%. Neurological events occurred in 38.3% of patients; 3.2% were grade 3 or higher.

The study did not prospectively record immune effector cell–associated neurotoxicity syndrome, Dr. Dickinson said, but an estimate suggests a rate of 2.6%, and none of the events were considered to be related to glofitamab, he noted.

The researchers also looked at a supporting cohort of 35 patients with a median follow up of more than 2 years. In this group, the complete remission rate was 35% and the median duration of remission was 34.2 months. “Our six longest patients have been in remission for longer than 3 years,” Dr. Dickinson said.

The latest glofitamab data “reflect routine practice and an area of need for this disease,” said Dr. Dickinson.

“I think these results will prove to be very meaningful for our patients with large cell lymphoma, and this drug will prove to be an important treatment option,” he said.
 

 

 

More follow-up needed, but findings show promise

A number of CD20/CD3-bispecific antibodies are in development for patients with relapsed/refractory B-cell lymphomas, said study discussant Kerry J. Savage, MD, of the University of British Columbia, Vancouver, who served as the discussant for the session.

Glofitamab differs from other treatments in that it is bivalent for CD20 and monovalent for CD3, “which imparts greater potency,” she noted. Glofitamab also has a silent Fc region that is designed to extend half-life and reduce toxicity.

Patients in the current study had at least two prior regimens, and importantly, “CR rates were similar, regardless of prior therapy,” said Dr. Savage. The longer follow-up cohort provides “a hint that the response may be durable.”

Looking ahead, “the important thing will be response durability” with longer follow-up, she added. “We don’t know the curative potential yet, but the results are encouraging so far.”

In the meantime, “the best use of bispecific antibodies is through clinical trials,” Dr. Savage said. “Keep an eye out for bispecific antibody combination trials as well.”

The study was funded by F. Hoffmann–La Roche. Dr. Dickinson disclosed honoraria from or serving as a consultant to companies including Amgen, Bristol Myers-Squibb, Gilead Sciences, Janssen, MSD, Novartis, and Roche. Dr. Savage disclosed relationships, funding, and support from multiple companies including Bristol Myers-Squibb, Janssen Oncology, Kyowa, Merck, Novartis Canada Pharmaceuticals, Seattle Genetics and Roche.

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Motor abnormalities drive decreased function in schizophrenia

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Four common motor abnormalities in schizophrenia patients were associated with at least one poor functional outcome, based on data from 156 individuals.

Approximately half of adults with schizophrenia suffer from motor abnormalities that may impair their ability to work and decrease their quality of life, wrote Niluja Nadesalingam, MD, of the University of Bern, Switzerland, and colleagues. “Although previous reports show strong associations between single movement abnormalities and global as well as social functioning, we still struggle to understand the contribution of various motor domains,” they said.

Dr. Niluja Nadesalingam

The impact of these abnormalities on social and global functioning and on functional capacity remains unclear, but the researchers proposed that motor abnormalities would be associated with worse functional outcomes in schizophrenia patients.

In a study published in Comprehensive Psychiatry, the researchers identified patients with diagnosed schizophrenia spectrum disorders who were treated on an inpatient or outpatient basis at a single center. They collected data on five motor abnormalities: parkinsonism, catatonia, dyskinesia, neurological soft signs (NSS), and psychomotor slowing (PS). They assessed functional outcomes using the Global Assessment of Functioning (GAF), the Social and Occupational Functioning Assessment Scale (SOFAS), and the UCSD Performance-Based Skills Assessment (UPSA-B). The average age of the participants was 37.9 years and 88 of the 156 were male. The average duration of illness was 12.5 years.

Overall, patients with catatonia and parkinsonism scored significantly lower on GAF and SOFAS scale compared to those without catatonia and parkinsonism (P < .035 and P < .027, respectively).

No significant differences in functional outcomes appeared between patients with and without dyskinesia.

However, significant negative correlations were identified for parkinsonism and PS with GAF, SOFAS, and UPSA-B (P < .036 for all). “Our study further found that parkinsonism and psychomotor slowing also impair the functional capacity of patients,” which may be influenced by factors including deficits in social interaction and cognitive impairment, the researchers said.

Overall, the study findings demonstrate that motor abnormalities in patients with schizophrenia are strongly associated with poor functional outcomes, and the stronger the motor impairment, the worse the global and social functioning, the researchers said.

As for potential pathways, “motor abnormalities are readily observable signs, allowing laypersons to perceive subjects with schizophrenia as somebody with severe mental illness. Thus, motor abnormalities might lead to stigmatization of patients suffering from schizophrenia,” they wrote in their discussion.

The researchers emphasized the need to explore alternative treatment options that might improve motor abnormalities, such as transcranial magnetic stimulation, given the potential of antipsychotic medications to introduce additional motor abnormalities.

The study findings were limited by several factors including the potential for missed confounding variables, the small number of patients with dyskinesia, and the inability to deduce the course of illness because most of the patients were in psychotic episodes, the researchers noted.

However, the results suggest that specific motor abnormalities are associated with poor global and social functioning, and with reduced functional capacity, in adults with schizophrenia, the researchers said. “Future studies need to test whether amelioration of motor abnormalities may improve community functioning,” they concluded.

The study was supported by the Swiss National Science Foundation, the Bangerter Rhyner Foundation, and the Adrian and Simone Frutiger Foundation. Lead author Dr. Nadesalingam had no financial conflicts to disclose.

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Four common motor abnormalities in schizophrenia patients were associated with at least one poor functional outcome, based on data from 156 individuals.

Approximately half of adults with schizophrenia suffer from motor abnormalities that may impair their ability to work and decrease their quality of life, wrote Niluja Nadesalingam, MD, of the University of Bern, Switzerland, and colleagues. “Although previous reports show strong associations between single movement abnormalities and global as well as social functioning, we still struggle to understand the contribution of various motor domains,” they said.

Dr. Niluja Nadesalingam

The impact of these abnormalities on social and global functioning and on functional capacity remains unclear, but the researchers proposed that motor abnormalities would be associated with worse functional outcomes in schizophrenia patients.

In a study published in Comprehensive Psychiatry, the researchers identified patients with diagnosed schizophrenia spectrum disorders who were treated on an inpatient or outpatient basis at a single center. They collected data on five motor abnormalities: parkinsonism, catatonia, dyskinesia, neurological soft signs (NSS), and psychomotor slowing (PS). They assessed functional outcomes using the Global Assessment of Functioning (GAF), the Social and Occupational Functioning Assessment Scale (SOFAS), and the UCSD Performance-Based Skills Assessment (UPSA-B). The average age of the participants was 37.9 years and 88 of the 156 were male. The average duration of illness was 12.5 years.

Overall, patients with catatonia and parkinsonism scored significantly lower on GAF and SOFAS scale compared to those without catatonia and parkinsonism (P < .035 and P < .027, respectively).

No significant differences in functional outcomes appeared between patients with and without dyskinesia.

However, significant negative correlations were identified for parkinsonism and PS with GAF, SOFAS, and UPSA-B (P < .036 for all). “Our study further found that parkinsonism and psychomotor slowing also impair the functional capacity of patients,” which may be influenced by factors including deficits in social interaction and cognitive impairment, the researchers said.

Overall, the study findings demonstrate that motor abnormalities in patients with schizophrenia are strongly associated with poor functional outcomes, and the stronger the motor impairment, the worse the global and social functioning, the researchers said.

As for potential pathways, “motor abnormalities are readily observable signs, allowing laypersons to perceive subjects with schizophrenia as somebody with severe mental illness. Thus, motor abnormalities might lead to stigmatization of patients suffering from schizophrenia,” they wrote in their discussion.

The researchers emphasized the need to explore alternative treatment options that might improve motor abnormalities, such as transcranial magnetic stimulation, given the potential of antipsychotic medications to introduce additional motor abnormalities.

The study findings were limited by several factors including the potential for missed confounding variables, the small number of patients with dyskinesia, and the inability to deduce the course of illness because most of the patients were in psychotic episodes, the researchers noted.

However, the results suggest that specific motor abnormalities are associated with poor global and social functioning, and with reduced functional capacity, in adults with schizophrenia, the researchers said. “Future studies need to test whether amelioration of motor abnormalities may improve community functioning,” they concluded.

The study was supported by the Swiss National Science Foundation, the Bangerter Rhyner Foundation, and the Adrian and Simone Frutiger Foundation. Lead author Dr. Nadesalingam had no financial conflicts to disclose.

Four common motor abnormalities in schizophrenia patients were associated with at least one poor functional outcome, based on data from 156 individuals.

Approximately half of adults with schizophrenia suffer from motor abnormalities that may impair their ability to work and decrease their quality of life, wrote Niluja Nadesalingam, MD, of the University of Bern, Switzerland, and colleagues. “Although previous reports show strong associations between single movement abnormalities and global as well as social functioning, we still struggle to understand the contribution of various motor domains,” they said.

Dr. Niluja Nadesalingam

The impact of these abnormalities on social and global functioning and on functional capacity remains unclear, but the researchers proposed that motor abnormalities would be associated with worse functional outcomes in schizophrenia patients.

In a study published in Comprehensive Psychiatry, the researchers identified patients with diagnosed schizophrenia spectrum disorders who were treated on an inpatient or outpatient basis at a single center. They collected data on five motor abnormalities: parkinsonism, catatonia, dyskinesia, neurological soft signs (NSS), and psychomotor slowing (PS). They assessed functional outcomes using the Global Assessment of Functioning (GAF), the Social and Occupational Functioning Assessment Scale (SOFAS), and the UCSD Performance-Based Skills Assessment (UPSA-B). The average age of the participants was 37.9 years and 88 of the 156 were male. The average duration of illness was 12.5 years.

Overall, patients with catatonia and parkinsonism scored significantly lower on GAF and SOFAS scale compared to those without catatonia and parkinsonism (P < .035 and P < .027, respectively).

No significant differences in functional outcomes appeared between patients with and without dyskinesia.

However, significant negative correlations were identified for parkinsonism and PS with GAF, SOFAS, and UPSA-B (P < .036 for all). “Our study further found that parkinsonism and psychomotor slowing also impair the functional capacity of patients,” which may be influenced by factors including deficits in social interaction and cognitive impairment, the researchers said.

Overall, the study findings demonstrate that motor abnormalities in patients with schizophrenia are strongly associated with poor functional outcomes, and the stronger the motor impairment, the worse the global and social functioning, the researchers said.

As for potential pathways, “motor abnormalities are readily observable signs, allowing laypersons to perceive subjects with schizophrenia as somebody with severe mental illness. Thus, motor abnormalities might lead to stigmatization of patients suffering from schizophrenia,” they wrote in their discussion.

The researchers emphasized the need to explore alternative treatment options that might improve motor abnormalities, such as transcranial magnetic stimulation, given the potential of antipsychotic medications to introduce additional motor abnormalities.

The study findings were limited by several factors including the potential for missed confounding variables, the small number of patients with dyskinesia, and the inability to deduce the course of illness because most of the patients were in psychotic episodes, the researchers noted.

However, the results suggest that specific motor abnormalities are associated with poor global and social functioning, and with reduced functional capacity, in adults with schizophrenia, the researchers said. “Future studies need to test whether amelioration of motor abnormalities may improve community functioning,” they concluded.

The study was supported by the Swiss National Science Foundation, the Bangerter Rhyner Foundation, and the Adrian and Simone Frutiger Foundation. Lead author Dr. Nadesalingam had no financial conflicts to disclose.

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Cochlear implants benefit deaf children with developmental delays

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Tue, 06/07/2022 - 16:14

Deaf babies and toddlers with developmental delays may benefit significantly from receiving cochlear implants over hearing aids.

A new study, published in the journal Pediatrics, pushes against the notion that children with low nonverbal cognition and adaptive functioning skills won’t improve if given cochlear implants. Some insurers cover hearing aids for children with developmental disorders but not the implants, which can cost between $60,000 and $100,000 per ear.

“We were surprised [by] the large magnitude of the improvements, not only in quality of life, but also in cognition, ability to function in daily living situations, and speech and language,” lead author John S. Oghalai, MD, of the University of Southern California, Los Angeles, told this news organization. “Remember, these are children with substantial developmental delays. Any improvements are incredibly important and meaningful.”

All children with severe hearing loss should be referred for cochlear implant evaluation, “regardless of the presence of other disabilities,” Dr. Oghalai said. “The younger this referral happens, the better the outcomes will be.”

Dr. Oghalai and his colleagues reviewed data from 204 children approximately 1-3 years old with hearing aids receiving treatment in Texas and California. Of these, 138 received a cochlear implant and had normal cognitive skills and social competence (referred to as adaptive behavior). Another 37 received a cochlear implant and also met criteria for early developmental impairment (EDI), defined by measures of nonverbal cognitive scores and adaptive functioning.

A third group of 29 children with EDI continued with hearing aids without a cochlear implant.

The children were evaluated annually for 1-5 years, with the average follow-up of 2 years. At baseline, no significant differences were noted between the children with EDI who received implants and those who did not on cognition, language, auditory skills, or measures of parental or child stress.

Overall, children who received implants scored higher on cognitive and social measures than those who continued using hearing aids.

Compared with children with EDI who received implants, children without EDI who received implants had significantly higher developmental scores by the study’s end (P ≤ .001), whereas children with EDI who did not receive implants had significantly lower scores (P ≤ .04).

Children who received implants, and their parents, also experienced less stress than those who did not receive the devices, according to the researchers.

Dr. Oghalai and colleagues also measured developmental trajectories for each cohort. Children without delays who received implants had the best outcomes, but those with EDI who received implants had better outcomes than those with EDI and hearing aids.
 

Findings ‘overdue’

“This study is overdue,” Howard Francis, MD, chair of the department of head and neck surgery & communication sciences at Duke University, Durham, N.C., told this news organization.

Dr. Francis called the new research “reasonably powered and designed,” and said it “documents benefits in the cognitive, language, and patient-child relationship domains” in children who received cochlear implants “compared to children with similar levels of developmental delay whose hearing loss was treated using hearing aids.”  

However, “larger studies will be needed to account for potential effects of older age at intervention in the hearing aid group,” he said. Socioeconomic effects are a topic for future research as well, Dr. Francis added.

The researchers initially wanted to perform a controlled clinical trial. However, by the time they secured funding, health insurance policy had changed to cover cochlear implants for children without EDI because of demonstrated benefits shown in studies.

They also were unable to determine the reasons for families’ decisions to choose implants or hearing aids and were unable to assess the impact of insurance on the choice of implantation. But they did find that families with insurers who would cover implants often did choose the devices. Children were also followed for an average of 2 years, so long-term outcomes are unknown.  

Despite these limitations, the results support the value of cochlear implantation in children with disabilities and developmental delays, and it should be discussed with parents, the researchers concluded.

“Cochlear implants are just a tool; they do not provide speech and language,” Dr. Oghalai said. “Any child with severe hearing loss requires significant therapy and education via sign language, auditory-verbal therapy, or both. Making the decision about what type of therapy to do is personal, and it depends upon the family and the options that are available to them in their community.”

The study was funded by the National Institutes of Health. The researchers and Dr. Francis have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

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Deaf babies and toddlers with developmental delays may benefit significantly from receiving cochlear implants over hearing aids.

A new study, published in the journal Pediatrics, pushes against the notion that children with low nonverbal cognition and adaptive functioning skills won’t improve if given cochlear implants. Some insurers cover hearing aids for children with developmental disorders but not the implants, which can cost between $60,000 and $100,000 per ear.

“We were surprised [by] the large magnitude of the improvements, not only in quality of life, but also in cognition, ability to function in daily living situations, and speech and language,” lead author John S. Oghalai, MD, of the University of Southern California, Los Angeles, told this news organization. “Remember, these are children with substantial developmental delays. Any improvements are incredibly important and meaningful.”

All children with severe hearing loss should be referred for cochlear implant evaluation, “regardless of the presence of other disabilities,” Dr. Oghalai said. “The younger this referral happens, the better the outcomes will be.”

Dr. Oghalai and his colleagues reviewed data from 204 children approximately 1-3 years old with hearing aids receiving treatment in Texas and California. Of these, 138 received a cochlear implant and had normal cognitive skills and social competence (referred to as adaptive behavior). Another 37 received a cochlear implant and also met criteria for early developmental impairment (EDI), defined by measures of nonverbal cognitive scores and adaptive functioning.

A third group of 29 children with EDI continued with hearing aids without a cochlear implant.

The children were evaluated annually for 1-5 years, with the average follow-up of 2 years. At baseline, no significant differences were noted between the children with EDI who received implants and those who did not on cognition, language, auditory skills, or measures of parental or child stress.

Overall, children who received implants scored higher on cognitive and social measures than those who continued using hearing aids.

Compared with children with EDI who received implants, children without EDI who received implants had significantly higher developmental scores by the study’s end (P ≤ .001), whereas children with EDI who did not receive implants had significantly lower scores (P ≤ .04).

Children who received implants, and their parents, also experienced less stress than those who did not receive the devices, according to the researchers.

Dr. Oghalai and colleagues also measured developmental trajectories for each cohort. Children without delays who received implants had the best outcomes, but those with EDI who received implants had better outcomes than those with EDI and hearing aids.
 

Findings ‘overdue’

“This study is overdue,” Howard Francis, MD, chair of the department of head and neck surgery & communication sciences at Duke University, Durham, N.C., told this news organization.

Dr. Francis called the new research “reasonably powered and designed,” and said it “documents benefits in the cognitive, language, and patient-child relationship domains” in children who received cochlear implants “compared to children with similar levels of developmental delay whose hearing loss was treated using hearing aids.”  

However, “larger studies will be needed to account for potential effects of older age at intervention in the hearing aid group,” he said. Socioeconomic effects are a topic for future research as well, Dr. Francis added.

The researchers initially wanted to perform a controlled clinical trial. However, by the time they secured funding, health insurance policy had changed to cover cochlear implants for children without EDI because of demonstrated benefits shown in studies.

They also were unable to determine the reasons for families’ decisions to choose implants or hearing aids and were unable to assess the impact of insurance on the choice of implantation. But they did find that families with insurers who would cover implants often did choose the devices. Children were also followed for an average of 2 years, so long-term outcomes are unknown.  

Despite these limitations, the results support the value of cochlear implantation in children with disabilities and developmental delays, and it should be discussed with parents, the researchers concluded.

“Cochlear implants are just a tool; they do not provide speech and language,” Dr. Oghalai said. “Any child with severe hearing loss requires significant therapy and education via sign language, auditory-verbal therapy, or both. Making the decision about what type of therapy to do is personal, and it depends upon the family and the options that are available to them in their community.”

The study was funded by the National Institutes of Health. The researchers and Dr. Francis have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Deaf babies and toddlers with developmental delays may benefit significantly from receiving cochlear implants over hearing aids.

A new study, published in the journal Pediatrics, pushes against the notion that children with low nonverbal cognition and adaptive functioning skills won’t improve if given cochlear implants. Some insurers cover hearing aids for children with developmental disorders but not the implants, which can cost between $60,000 and $100,000 per ear.

“We were surprised [by] the large magnitude of the improvements, not only in quality of life, but also in cognition, ability to function in daily living situations, and speech and language,” lead author John S. Oghalai, MD, of the University of Southern California, Los Angeles, told this news organization. “Remember, these are children with substantial developmental delays. Any improvements are incredibly important and meaningful.”

All children with severe hearing loss should be referred for cochlear implant evaluation, “regardless of the presence of other disabilities,” Dr. Oghalai said. “The younger this referral happens, the better the outcomes will be.”

Dr. Oghalai and his colleagues reviewed data from 204 children approximately 1-3 years old with hearing aids receiving treatment in Texas and California. Of these, 138 received a cochlear implant and had normal cognitive skills and social competence (referred to as adaptive behavior). Another 37 received a cochlear implant and also met criteria for early developmental impairment (EDI), defined by measures of nonverbal cognitive scores and adaptive functioning.

A third group of 29 children with EDI continued with hearing aids without a cochlear implant.

The children were evaluated annually for 1-5 years, with the average follow-up of 2 years. At baseline, no significant differences were noted between the children with EDI who received implants and those who did not on cognition, language, auditory skills, or measures of parental or child stress.

Overall, children who received implants scored higher on cognitive and social measures than those who continued using hearing aids.

Compared with children with EDI who received implants, children without EDI who received implants had significantly higher developmental scores by the study’s end (P ≤ .001), whereas children with EDI who did not receive implants had significantly lower scores (P ≤ .04).

Children who received implants, and their parents, also experienced less stress than those who did not receive the devices, according to the researchers.

Dr. Oghalai and colleagues also measured developmental trajectories for each cohort. Children without delays who received implants had the best outcomes, but those with EDI who received implants had better outcomes than those with EDI and hearing aids.
 

Findings ‘overdue’

“This study is overdue,” Howard Francis, MD, chair of the department of head and neck surgery & communication sciences at Duke University, Durham, N.C., told this news organization.

Dr. Francis called the new research “reasonably powered and designed,” and said it “documents benefits in the cognitive, language, and patient-child relationship domains” in children who received cochlear implants “compared to children with similar levels of developmental delay whose hearing loss was treated using hearing aids.”  

However, “larger studies will be needed to account for potential effects of older age at intervention in the hearing aid group,” he said. Socioeconomic effects are a topic for future research as well, Dr. Francis added.

The researchers initially wanted to perform a controlled clinical trial. However, by the time they secured funding, health insurance policy had changed to cover cochlear implants for children without EDI because of demonstrated benefits shown in studies.

They also were unable to determine the reasons for families’ decisions to choose implants or hearing aids and were unable to assess the impact of insurance on the choice of implantation. But they did find that families with insurers who would cover implants often did choose the devices. Children were also followed for an average of 2 years, so long-term outcomes are unknown.  

Despite these limitations, the results support the value of cochlear implantation in children with disabilities and developmental delays, and it should be discussed with parents, the researchers concluded.

“Cochlear implants are just a tool; they do not provide speech and language,” Dr. Oghalai said. “Any child with severe hearing loss requires significant therapy and education via sign language, auditory-verbal therapy, or both. Making the decision about what type of therapy to do is personal, and it depends upon the family and the options that are available to them in their community.”

The study was funded by the National Institutes of Health. The researchers and Dr. Francis have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

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