Screening for cognitive impairment should be part of multidisciplinary care for stroke survivors, the American Heart Association says in a new scientific statement.

“Cognitive impairment after stroke is very common, is associated with other post-stroke outcomes, and often has significant impact on the quality of life,” Nada El Husseini, MD, MHSc, chair of the scientific statement writing group, told this news organization.

“It is important to screen stroke survivors for cognitive impairment as well as for associated comorbidities such as mood and sleep disorders,” said Dr. El Husseini, associate professor of neurology at Duke University Medical Center in Durham, N.C.

The scientific statement was published online in Stroke. It’s the first to specifically focus on the cognitive impairment resulting from an overt stroke (ischemic or hemorrhagic).
 

‘Actionable’ considerations for care

The writing group performed a “scoping” review of the literature on the prevalence, diagnosis, and management of poststroke cognitive impairment (PSCI) to provide a framework for “actionable considerations” for clinical practice as well as to highlight gaps needing additional studies, Dr. El Husseini explained.

PSCI, ranging from mild to severe, occurs in up to 60% of stroke survivors in the first year after stroke; yet, it is often underreported and underdiagnosed, the writing group notes.

Up to 20% of stroke survivors who experience mild cognitive impairment fully recover cognitive function, and cognitive recovery is most likely within the first 6 months after a stroke.

However, improvement in cognitive impairment without return to prestroke levels is more frequent than is complete recovery. As many as one in three stroke survivors may develop dementia within 5 years of stroke.

The writing group also notes that PSCI is often associated with other conditions, including physical disability, sleep disorders, behavioral and personality changes, depression, and other neuropsychological changes – each of which may contribute to lower quality of life.

Currently, there is no “gold standard” for cognitive screening following stroke, but several brief cognitive screening tests, including the Mini–Mental State Examination and the Montreal Cognitive Assessment, are widely used to identify cognitive impairment after stroke.

The statement also highlights the importance of assessing cognitive changes over time after stroke. Stroke survivors who experience unexplained difficulties with cognitive-related activities of daily living, following care instructions, or providing a reliable health history may be candidates for additional cognitive screening.
 

Manage risk factors to prevent repeat stroke

“Anticipatory guidance regarding home and driving safety and, return to work (if applicable) along with interdisciplinary collaboration among different medical and ancillary specialists in the diagnosis and management of cognitive impairment is key for the holistic care of stroke survivors,” Dr. El Husseini told this news organization.

The multidisciplinary poststroke health care team could include neurologists, occupational therapists, speech therapists, nurses, neuropsychologists, gerontologists, and primary care providers.

“Because recurrent stroke is strongly associated with the development of cognitive impairment and dementia, prevention of recurrent strokes should be sought to decrease that risk,” Dr. El Husseini said. This includes addressing stroke risk factors, including high blood pressure, high cholesterol, type 2 diabetes, and atrial fibrillation.

The writing group says research is needed in the future to determine how cognitive impairment develops after stroke and the impact of nonbrain factors, including infection, frailty, and social factors.

Further research is also needed to determine best practices for cognitive screening after stroke, including the development and use of screening instruments that consider demographic, cultural, and linguistic factors in determining “normal” function.

“Perhaps the most pressing need, however, is the development of effective and culturally relevant treatments for poststroke cognitive impairment,” Dr. El Husseini said in a news release.

“We hope to see big enough clinical trials that assess various techniques, medications, and lifestyle changes in diverse groups of patients that may help improve cognitive function,” she added.

This scientific statement was prepared by the volunteer writing group on behalf of the AHA Stroke Council, the Council on Cardiovascular Radiology and Intervention, the Council on Hypertension, and the Council on Lifestyle and Cardiometabolic Health.
 

Screening for cognitive impairment should be part of multidisciplinary care for stroke survivors, the American Heart Association says in a new scientific statement.

“Cognitive impairment after stroke is very common, is associated with other post-stroke outcomes, and often has significant impact on the quality of life,” Nada El Husseini, MD, MHSc, chair of the scientific statement writing group, told this news organization.

“It is important to screen stroke survivors for cognitive impairment as well as for associated comorbidities such as mood and sleep disorders,” said Dr. El Husseini, associate professor of neurology at Duke University Medical Center in Durham, N.C.

The scientific statement was published online in Stroke. It’s the first to specifically focus on the cognitive impairment resulting from an overt stroke (ischemic or hemorrhagic).
 

‘Actionable’ considerations for care

The writing group performed a “scoping” review of the literature on the prevalence, diagnosis, and management of poststroke cognitive impairment (PSCI) to provide a framework for “actionable considerations” for clinical practice as well as to highlight gaps needing additional studies, Dr. El Husseini explained.

PSCI, ranging from mild to severe, occurs in up to 60% of stroke survivors in the first year after stroke; yet, it is often underreported and underdiagnosed, the writing group notes.

Up to 20% of stroke survivors who experience mild cognitive impairment fully recover cognitive function, and cognitive recovery is most likely within the first 6 months after a stroke.

However, improvement in cognitive impairment without return to prestroke levels is more frequent than is complete recovery. As many as one in three stroke survivors may develop dementia within 5 years of stroke.

The writing group also notes that PSCI is often associated with other conditions, including physical disability, sleep disorders, behavioral and personality changes, depression, and other neuropsychological changes – each of which may contribute to lower quality of life.

Currently, there is no “gold standard” for cognitive screening following stroke, but several brief cognitive screening tests, including the Mini–Mental State Examination and the Montreal Cognitive Assessment, are widely used to identify cognitive impairment after stroke.

The statement also highlights the importance of assessing cognitive changes over time after stroke. Stroke survivors who experience unexplained difficulties with cognitive-related activities of daily living, following care instructions, or providing a reliable health history may be candidates for additional cognitive screening.
 

Manage risk factors to prevent repeat stroke

“Anticipatory guidance regarding home and driving safety and, return to work (if applicable) along with interdisciplinary collaboration among different medical and ancillary specialists in the diagnosis and management of cognitive impairment is key for the holistic care of stroke survivors,” Dr. El Husseini told this news organization.

The multidisciplinary poststroke health care team could include neurologists, occupational therapists, speech therapists, nurses, neuropsychologists, gerontologists, and primary care providers.

“Because recurrent stroke is strongly associated with the development of cognitive impairment and dementia, prevention of recurrent strokes should be sought to decrease that risk,” Dr. El Husseini said. This includes addressing stroke risk factors, including high blood pressure, high cholesterol, type 2 diabetes, and atrial fibrillation.

The writing group says research is needed in the future to determine how cognitive impairment develops after stroke and the impact of nonbrain factors, including infection, frailty, and social factors.

Further research is also needed to determine best practices for cognitive screening after stroke, including the development and use of screening instruments that consider demographic, cultural, and linguistic factors in determining “normal” function.

“Perhaps the most pressing need, however, is the development of effective and culturally relevant treatments for poststroke cognitive impairment,” Dr. El Husseini said in a news release.

“We hope to see big enough clinical trials that assess various techniques, medications, and lifestyle changes in diverse groups of patients that may help improve cognitive function,” she added.

This scientific statement was prepared by the volunteer writing group on behalf of the AHA Stroke Council, the Council on Cardiovascular Radiology and Intervention, the Council on Hypertension, and the Council on Lifestyle and Cardiometabolic Health.
 

Screening for cognitive impairment should be part of multidisciplinary care for stroke survivors, the American Heart Association says in a new scientific statement.

“Cognitive impairment after stroke is very common, is associated with other post-stroke outcomes, and often has significant impact on the quality of life,” Nada El Husseini, MD, MHSc, chair of the scientific statement writing group, told this news organization.

“It is important to screen stroke survivors for cognitive impairment as well as for associated comorbidities such as mood and sleep disorders,” said Dr. El Husseini, associate professor of neurology at Duke University Medical Center in Durham, N.C.

The scientific statement was published online in Stroke. It’s the first to specifically focus on the cognitive impairment resulting from an overt stroke (ischemic or hemorrhagic).
 

‘Actionable’ considerations for care

The writing group performed a “scoping” review of the literature on the prevalence, diagnosis, and management of poststroke cognitive impairment (PSCI) to provide a framework for “actionable considerations” for clinical practice as well as to highlight gaps needing additional studies, Dr. El Husseini explained.

PSCI, ranging from mild to severe, occurs in up to 60% of stroke survivors in the first year after stroke; yet, it is often underreported and underdiagnosed, the writing group notes.

Up to 20% of stroke survivors who experience mild cognitive impairment fully recover cognitive function, and cognitive recovery is most likely within the first 6 months after a stroke.

However, improvement in cognitive impairment without return to prestroke levels is more frequent than is complete recovery. As many as one in three stroke survivors may develop dementia within 5 years of stroke.

The writing group also notes that PSCI is often associated with other conditions, including physical disability, sleep disorders, behavioral and personality changes, depression, and other neuropsychological changes – each of which may contribute to lower quality of life.

Currently, there is no “gold standard” for cognitive screening following stroke, but several brief cognitive screening tests, including the Mini–Mental State Examination and the Montreal Cognitive Assessment, are widely used to identify cognitive impairment after stroke.

The statement also highlights the importance of assessing cognitive changes over time after stroke. Stroke survivors who experience unexplained difficulties with cognitive-related activities of daily living, following care instructions, or providing a reliable health history may be candidates for additional cognitive screening.
 

Manage risk factors to prevent repeat stroke

“Anticipatory guidance regarding home and driving safety and, return to work (if applicable) along with interdisciplinary collaboration among different medical and ancillary specialists in the diagnosis and management of cognitive impairment is key for the holistic care of stroke survivors,” Dr. El Husseini told this news organization.

The multidisciplinary poststroke health care team could include neurologists, occupational therapists, speech therapists, nurses, neuropsychologists, gerontologists, and primary care providers.

“Because recurrent stroke is strongly associated with the development of cognitive impairment and dementia, prevention of recurrent strokes should be sought to decrease that risk,” Dr. El Husseini said. This includes addressing stroke risk factors, including high blood pressure, high cholesterol, type 2 diabetes, and atrial fibrillation.

The writing group says research is needed in the future to determine how cognitive impairment develops after stroke and the impact of nonbrain factors, including infection, frailty, and social factors.

Further research is also needed to determine best practices for cognitive screening after stroke, including the development and use of screening instruments that consider demographic, cultural, and linguistic factors in determining “normal” function.

“Perhaps the most pressing need, however, is the development of effective and culturally relevant treatments for poststroke cognitive impairment,” Dr. El Husseini said in a news release.

“We hope to see big enough clinical trials that assess various techniques, medications, and lifestyle changes in diverse groups of patients that may help improve cognitive function,” she added.

This scientific statement was prepared by the volunteer writing group on behalf of the AHA Stroke Council, the Council on Cardiovascular Radiology and Intervention, the Council on Hypertension, and the Council on Lifestyle and Cardiometabolic Health.
 

A brief scalable suicide risk assessment tool accurately predicts suicide risk in patients with serious mental illness (SMI), a new population-based study shows.

The 17-question Oxford Mental Illness and Suicide Tool (OxMIS) assessment is designed to predict 12-month suicide risk in people with schizophrenia spectrum disorders and bipolar disorder based on risk factors such as familial traits, antisocial traits, and information about self-harm.

“We have demonstrated the clinical utility of OxMIS in two separate studies and countries. As with any clinical risk prediction tool, it will not improve outcomes unless coupled with effective interventions,” lead investigator Amir Sariaslan, PhD, a senior research fellow in psychiatric epidemiology at the University of Oxford, England, told this news organization.

The findings were published online in Translational Psychiatry.
 

Twice validated

Dr. Sariaslan and his team originally developed and validated the OxMIS in a cohort of 75,000 people with SMI in Sweden. Recognizing the lack of externally validated prognostic models in the mental health field, the team wanted to validate the instrument in a new, population-based sample in Finland.

The investigators accessed information about patient diagnosis and treatment from the Finnish Care Register for Health Care, which contains de-identified information for all individuals between ages 15 and 65 years diagnosed with an SMI between Jan. 1, 1996, and Dec. 31, 2017.

They included 137,000 patients with somatic symptom disorder or bipolar disorder for a total of more than 5 million episodes of inpatient or outpatient treatment. Investigators linked the cohort to the Causes of Death Register to identify those who had died by suicide within 12 months of an index treatment episode, which investigators randomly selected for each person.

The investigators found that 1,475 individuals in the sample died by suicide within 1 year of their index episode (1.1%).

Each patient was assigned a clinical suicide risk score based on their clinical information, familial traits, prescription information, and comorbid conditions. Using OxMIS, the investigators found that the instrument accurately predicted suicide with an area under the curve of 0.70.

In other words, in 70% of the instances where the investigators randomly selected two people from the sample, one of whom died by suicide and the other of whom did not, the individual who died by suicide had a higher OxMIS risk score.

The investigators note the model overestimated the risk for patients who were at extremely high risk for suicide (those with a predicted suicide risk of > 5%). “In our complementary sensitivity analysis, we observed improved calibration in these patients when we assigned them a suicide risk prediction of no more than 5%,” they write.

Dr. Sariaslan said that the findings highlight the importance of safety planning interventions. “It is also essential to remember that OxMIS is not intended to replace clinical decision-making, but rather to support it,” he said.

As to whether the tool could be used in other populations, such as in the United States, Dr. Sariaslan said, “there is no good evidence that the contribution of risk factors to suicide in this population is different in the U.S. than in northern Europe, so there is no a priori reason to have to do multiple external validations before it can be used for research or clinical purposes.”
 

One size does not fit all

Commenting on the study, Ronald Kessler, PhD, McNeil Family Professor, department of health care policy at Harvard Medical School, Boston, said that he’d be “surprised” if OxMIS was adopted in the United States because there is already an existing tool that is “slightly more accurate,” which he helped develop.

Harvard Medical School

“In addition, when we start thinking about uses for such scales, it becomes clear that different scales should be used for different segments of the population, depending on intervention options,” Dr. Kessler said.

“So, for example, a different scale would probably be optimal in deciding how to manage psychiatric inpatients in the transition back to the community after hospital discharge than [it would be], say, in deciding how to respond to suicidality among patients presenting at an emergency department. No one scale will fit for all the scenarios in which prediction is desired,” he added.

The study was funded by the Academy of Finland. Dr. Kessler receives funding from the National Institute of Mental Health, Department of Defense, and Veterans Administration to develop suicide prediction models. Dr. Sariaslan has no disclosures to report.

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

A brief scalable suicide risk assessment tool accurately predicts suicide risk in patients with serious mental illness (SMI), a new population-based study shows.

The 17-question Oxford Mental Illness and Suicide Tool (OxMIS) assessment is designed to predict 12-month suicide risk in people with schizophrenia spectrum disorders and bipolar disorder based on risk factors such as familial traits, antisocial traits, and information about self-harm.

“We have demonstrated the clinical utility of OxMIS in two separate studies and countries. As with any clinical risk prediction tool, it will not improve outcomes unless coupled with effective interventions,” lead investigator Amir Sariaslan, PhD, a senior research fellow in psychiatric epidemiology at the University of Oxford, England, told this news organization.

The findings were published online in Translational Psychiatry.
 

Twice validated

Dr. Sariaslan and his team originally developed and validated the OxMIS in a cohort of 75,000 people with SMI in Sweden. Recognizing the lack of externally validated prognostic models in the mental health field, the team wanted to validate the instrument in a new, population-based sample in Finland.

The investigators accessed information about patient diagnosis and treatment from the Finnish Care Register for Health Care, which contains de-identified information for all individuals between ages 15 and 65 years diagnosed with an SMI between Jan. 1, 1996, and Dec. 31, 2017.

They included 137,000 patients with somatic symptom disorder or bipolar disorder for a total of more than 5 million episodes of inpatient or outpatient treatment. Investigators linked the cohort to the Causes of Death Register to identify those who had died by suicide within 12 months of an index treatment episode, which investigators randomly selected for each person.

The investigators found that 1,475 individuals in the sample died by suicide within 1 year of their index episode (1.1%).

Each patient was assigned a clinical suicide risk score based on their clinical information, familial traits, prescription information, and comorbid conditions. Using OxMIS, the investigators found that the instrument accurately predicted suicide with an area under the curve of 0.70.

In other words, in 70% of the instances where the investigators randomly selected two people from the sample, one of whom died by suicide and the other of whom did not, the individual who died by suicide had a higher OxMIS risk score.

The investigators note the model overestimated the risk for patients who were at extremely high risk for suicide (those with a predicted suicide risk of > 5%). “In our complementary sensitivity analysis, we observed improved calibration in these patients when we assigned them a suicide risk prediction of no more than 5%,” they write.

Dr. Sariaslan said that the findings highlight the importance of safety planning interventions. “It is also essential to remember that OxMIS is not intended to replace clinical decision-making, but rather to support it,” he said.

As to whether the tool could be used in other populations, such as in the United States, Dr. Sariaslan said, “there is no good evidence that the contribution of risk factors to suicide in this population is different in the U.S. than in northern Europe, so there is no a priori reason to have to do multiple external validations before it can be used for research or clinical purposes.”
 

One size does not fit all

Commenting on the study, Ronald Kessler, PhD, McNeil Family Professor, department of health care policy at Harvard Medical School, Boston, said that he’d be “surprised” if OxMIS was adopted in the United States because there is already an existing tool that is “slightly more accurate,” which he helped develop.

Harvard Medical School

“In addition, when we start thinking about uses for such scales, it becomes clear that different scales should be used for different segments of the population, depending on intervention options,” Dr. Kessler said.

“So, for example, a different scale would probably be optimal in deciding how to manage psychiatric inpatients in the transition back to the community after hospital discharge than [it would be], say, in deciding how to respond to suicidality among patients presenting at an emergency department. No one scale will fit for all the scenarios in which prediction is desired,” he added.

The study was funded by the Academy of Finland. Dr. Kessler receives funding from the National Institute of Mental Health, Department of Defense, and Veterans Administration to develop suicide prediction models. Dr. Sariaslan has no disclosures to report.

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

A brief scalable suicide risk assessment tool accurately predicts suicide risk in patients with serious mental illness (SMI), a new population-based study shows.

The 17-question Oxford Mental Illness and Suicide Tool (OxMIS) assessment is designed to predict 12-month suicide risk in people with schizophrenia spectrum disorders and bipolar disorder based on risk factors such as familial traits, antisocial traits, and information about self-harm.

“We have demonstrated the clinical utility of OxMIS in two separate studies and countries. As with any clinical risk prediction tool, it will not improve outcomes unless coupled with effective interventions,” lead investigator Amir Sariaslan, PhD, a senior research fellow in psychiatric epidemiology at the University of Oxford, England, told this news organization.

The findings were published online in Translational Psychiatry.
 

Twice validated

Dr. Sariaslan and his team originally developed and validated the OxMIS in a cohort of 75,000 people with SMI in Sweden. Recognizing the lack of externally validated prognostic models in the mental health field, the team wanted to validate the instrument in a new, population-based sample in Finland.

The investigators accessed information about patient diagnosis and treatment from the Finnish Care Register for Health Care, which contains de-identified information for all individuals between ages 15 and 65 years diagnosed with an SMI between Jan. 1, 1996, and Dec. 31, 2017.

They included 137,000 patients with somatic symptom disorder or bipolar disorder for a total of more than 5 million episodes of inpatient or outpatient treatment. Investigators linked the cohort to the Causes of Death Register to identify those who had died by suicide within 12 months of an index treatment episode, which investigators randomly selected for each person.

The investigators found that 1,475 individuals in the sample died by suicide within 1 year of their index episode (1.1%).

Each patient was assigned a clinical suicide risk score based on their clinical information, familial traits, prescription information, and comorbid conditions. Using OxMIS, the investigators found that the instrument accurately predicted suicide with an area under the curve of 0.70.

In other words, in 70% of the instances where the investigators randomly selected two people from the sample, one of whom died by suicide and the other of whom did not, the individual who died by suicide had a higher OxMIS risk score.

The investigators note the model overestimated the risk for patients who were at extremely high risk for suicide (those with a predicted suicide risk of > 5%). “In our complementary sensitivity analysis, we observed improved calibration in these patients when we assigned them a suicide risk prediction of no more than 5%,” they write.

Dr. Sariaslan said that the findings highlight the importance of safety planning interventions. “It is also essential to remember that OxMIS is not intended to replace clinical decision-making, but rather to support it,” he said.

As to whether the tool could be used in other populations, such as in the United States, Dr. Sariaslan said, “there is no good evidence that the contribution of risk factors to suicide in this population is different in the U.S. than in northern Europe, so there is no a priori reason to have to do multiple external validations before it can be used for research or clinical purposes.”
 

One size does not fit all

Commenting on the study, Ronald Kessler, PhD, McNeil Family Professor, department of health care policy at Harvard Medical School, Boston, said that he’d be “surprised” if OxMIS was adopted in the United States because there is already an existing tool that is “slightly more accurate,” which he helped develop.

Harvard Medical School

“In addition, when we start thinking about uses for such scales, it becomes clear that different scales should be used for different segments of the population, depending on intervention options,” Dr. Kessler said.

“So, for example, a different scale would probably be optimal in deciding how to manage psychiatric inpatients in the transition back to the community after hospital discharge than [it would be], say, in deciding how to respond to suicidality among patients presenting at an emergency department. No one scale will fit for all the scenarios in which prediction is desired,” he added.

The study was funded by the Academy of Finland. Dr. Kessler receives funding from the National Institute of Mental Health, Department of Defense, and Veterans Administration to develop suicide prediction models. Dr. Sariaslan has no disclosures to report.

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

NEW YORK – In a blood cancer conference session – “Will autologous stem cell transplant (ASCT) continue to be the standard of care (SoC) in frontline therapy for multiple myeloma (MM)?” – two experts weighed in on the merits of their approaches to treating MM at the Great Debates and Updates Hematologic Malignancies conference.

Hematologist-oncologists whose top priority is ensuring that patients have the best chance of progression-free survival (PFS) will continue to choose ASCT as a best practice, argued Amrita Krishnan, MD, hematologist at the Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope Comprehensive Cancer Center, Duarte, Calif.

credit Dr. Landgren

A differing perspective was presented by C. Ola Landgren, MD, PhD, hematologist at the Sylvester Comprehensive Cancer Center at the University of Miami. Dr. Landgren cited evidence that, for newly diagnosed MM patients treated successfully with modern combination therapies, ASCT is not a mandatory treatment step before starting maintenance therapy. 

Making a case for ASCT as the SoC, Dr. Krishnan noted, “based on the DETERMINATION trial [DT], there is far superior rate of PFS with patients who get ASCT up front, compared patients who got only conventional chemotherapy with lenalidomide, bortezomib, and dexamethasone [RVd]. PFS is the endpoint we look for in our treatment regimens.

“If you don’t use ASCT up front, you may lose the opportunity at later relapse. This is not to say that transplant is the only tool at our disposal. It is just an indispensable one. The GRIFFIN trial [GT] has shown us that robust combinations of drugs [both RVd and dexamethasone +RVd] can improve patient outcomes both before and after ASCT,” Dr. Krishnan concluded.

In his presentation, Dr. Landgren stated that, in the DT, while PFS is prolonged by the addition of ASCT to RVd, adding ASCT did not significantly increase overall survival (OS) rates. He added that treatment-related AEs of grade 3+ occurred in only 78.2% of patients on RVd versus 94.2% of RVd + ASCT patients.

“ASCT should not be the SoC frontline treatment in MM because it does not prolong OS. The IFM trial and the DT both show that there is no difference in OS between drug combination therapy followed by transplant and maintenance versus combination therapy alone, followed by transplant and maintenance. Furthermore, patients who get ASCT have higher risk of developing secondary malignancies, worse quality of life, and higher long-term morbidity with other conditions,” Dr. Landgren said.

He cited the MAIA trial administered daratumumab and lenalidomide plus dexamethasone (DRd) to patients who were too old or too frail to qualify for ASCT. Over half of patients in the DRd arm of MAIA had an estimated progression-free survival rate at 60 months.

“Furthermore, GT and the MANHATTAN clinical trials showed that we can safely add CD38-targeted monoclonal antibodies to standard combination therapies [lenalidomide, bortezomib, and dexamethasone (KRd)], resulting in higher rates of minimal-residual-disease (MRD) negativity. That means modern four-drug combination therapies [DR-RVd and DR-KRd] will allow more [and more newly diagnosed] MM patients to achieve MRD negativity in the absence of ASCT,” Dr. Landgren concluded.

courtesy of Dr. Richter

Asked to comment on the two viewpoints, Joshua Richter, MD, director of myeloma treatment at the Blavatnik Family Center at Chelsea Mount Sinai, New York, said: “With some patients, we can get similar outcomes, whether or not we do a transplant. Doctors need to be better at choosing who really needs ASCT. Older people with standard-risk disease or people who achieve MRD-negative status after pharmacological treatment might not need to receive a transplant as much as those who have bulk disease or high-risk cytogenetics.

“Although ASCT might not be the best frontline option for everyone, collecting cells from most patients and storing them has many advantages. It allows us to do have the option of ASCT in later lines of therapy. In some patients with low blood counts, we can use stored cells to reboot their marrow and make them eligible for trials of promising new drugs,” Dr. Richter said.

Dr. Krishnan disclosed relationships with Takeda, Amgen, GlaxoSmithKline, Bristol-Myers Squibb, Sanofi, Pfizer, Adaptive, Regeneron, Janssen, AstraZeneca, Artiva, and Sutro. Dr. Landgren reported ties with Amgen, BMS, Celgene, Janssen, Takedam Glenmark, Juno, Pfizer, Merck, and others. Dr. Richter disclosed relationships with Janssen, BMS, and Takeda.

NEW YORK – In a blood cancer conference session – “Will autologous stem cell transplant (ASCT) continue to be the standard of care (SoC) in frontline therapy for multiple myeloma (MM)?” – two experts weighed in on the merits of their approaches to treating MM at the Great Debates and Updates Hematologic Malignancies conference.

Hematologist-oncologists whose top priority is ensuring that patients have the best chance of progression-free survival (PFS) will continue to choose ASCT as a best practice, argued Amrita Krishnan, MD, hematologist at the Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope Comprehensive Cancer Center, Duarte, Calif.

credit Dr. Landgren

A differing perspective was presented by C. Ola Landgren, MD, PhD, hematologist at the Sylvester Comprehensive Cancer Center at the University of Miami. Dr. Landgren cited evidence that, for newly diagnosed MM patients treated successfully with modern combination therapies, ASCT is not a mandatory treatment step before starting maintenance therapy. 

Making a case for ASCT as the SoC, Dr. Krishnan noted, “based on the DETERMINATION trial [DT], there is far superior rate of PFS with patients who get ASCT up front, compared patients who got only conventional chemotherapy with lenalidomide, bortezomib, and dexamethasone [RVd]. PFS is the endpoint we look for in our treatment regimens.

“If you don’t use ASCT up front, you may lose the opportunity at later relapse. This is not to say that transplant is the only tool at our disposal. It is just an indispensable one. The GRIFFIN trial [GT] has shown us that robust combinations of drugs [both RVd and dexamethasone +RVd] can improve patient outcomes both before and after ASCT,” Dr. Krishnan concluded.

In his presentation, Dr. Landgren stated that, in the DT, while PFS is prolonged by the addition of ASCT to RVd, adding ASCT did not significantly increase overall survival (OS) rates. He added that treatment-related AEs of grade 3+ occurred in only 78.2% of patients on RVd versus 94.2% of RVd + ASCT patients.

“ASCT should not be the SoC frontline treatment in MM because it does not prolong OS. The IFM trial and the DT both show that there is no difference in OS between drug combination therapy followed by transplant and maintenance versus combination therapy alone, followed by transplant and maintenance. Furthermore, patients who get ASCT have higher risk of developing secondary malignancies, worse quality of life, and higher long-term morbidity with other conditions,” Dr. Landgren said.

He cited the MAIA trial administered daratumumab and lenalidomide plus dexamethasone (DRd) to patients who were too old or too frail to qualify for ASCT. Over half of patients in the DRd arm of MAIA had an estimated progression-free survival rate at 60 months.

“Furthermore, GT and the MANHATTAN clinical trials showed that we can safely add CD38-targeted monoclonal antibodies to standard combination therapies [lenalidomide, bortezomib, and dexamethasone (KRd)], resulting in higher rates of minimal-residual-disease (MRD) negativity. That means modern four-drug combination therapies [DR-RVd and DR-KRd] will allow more [and more newly diagnosed] MM patients to achieve MRD negativity in the absence of ASCT,” Dr. Landgren concluded.

courtesy of Dr. Richter

Asked to comment on the two viewpoints, Joshua Richter, MD, director of myeloma treatment at the Blavatnik Family Center at Chelsea Mount Sinai, New York, said: “With some patients, we can get similar outcomes, whether or not we do a transplant. Doctors need to be better at choosing who really needs ASCT. Older people with standard-risk disease or people who achieve MRD-negative status after pharmacological treatment might not need to receive a transplant as much as those who have bulk disease or high-risk cytogenetics.

“Although ASCT might not be the best frontline option for everyone, collecting cells from most patients and storing them has many advantages. It allows us to do have the option of ASCT in later lines of therapy. In some patients with low blood counts, we can use stored cells to reboot their marrow and make them eligible for trials of promising new drugs,” Dr. Richter said.

Dr. Krishnan disclosed relationships with Takeda, Amgen, GlaxoSmithKline, Bristol-Myers Squibb, Sanofi, Pfizer, Adaptive, Regeneron, Janssen, AstraZeneca, Artiva, and Sutro. Dr. Landgren reported ties with Amgen, BMS, Celgene, Janssen, Takedam Glenmark, Juno, Pfizer, Merck, and others. Dr. Richter disclosed relationships with Janssen, BMS, and Takeda.

NEW YORK – In a blood cancer conference session – “Will autologous stem cell transplant (ASCT) continue to be the standard of care (SoC) in frontline therapy for multiple myeloma (MM)?” – two experts weighed in on the merits of their approaches to treating MM at the Great Debates and Updates Hematologic Malignancies conference.

Hematologist-oncologists whose top priority is ensuring that patients have the best chance of progression-free survival (PFS) will continue to choose ASCT as a best practice, argued Amrita Krishnan, MD, hematologist at the Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope Comprehensive Cancer Center, Duarte, Calif.

credit Dr. Landgren

A differing perspective was presented by C. Ola Landgren, MD, PhD, hematologist at the Sylvester Comprehensive Cancer Center at the University of Miami. Dr. Landgren cited evidence that, for newly diagnosed MM patients treated successfully with modern combination therapies, ASCT is not a mandatory treatment step before starting maintenance therapy. 

Making a case for ASCT as the SoC, Dr. Krishnan noted, “based on the DETERMINATION trial [DT], there is far superior rate of PFS with patients who get ASCT up front, compared patients who got only conventional chemotherapy with lenalidomide, bortezomib, and dexamethasone [RVd]. PFS is the endpoint we look for in our treatment regimens.

“If you don’t use ASCT up front, you may lose the opportunity at later relapse. This is not to say that transplant is the only tool at our disposal. It is just an indispensable one. The GRIFFIN trial [GT] has shown us that robust combinations of drugs [both RVd and dexamethasone +RVd] can improve patient outcomes both before and after ASCT,” Dr. Krishnan concluded.

In his presentation, Dr. Landgren stated that, in the DT, while PFS is prolonged by the addition of ASCT to RVd, adding ASCT did not significantly increase overall survival (OS) rates. He added that treatment-related AEs of grade 3+ occurred in only 78.2% of patients on RVd versus 94.2% of RVd + ASCT patients.

“ASCT should not be the SoC frontline treatment in MM because it does not prolong OS. The IFM trial and the DT both show that there is no difference in OS between drug combination therapy followed by transplant and maintenance versus combination therapy alone, followed by transplant and maintenance. Furthermore, patients who get ASCT have higher risk of developing secondary malignancies, worse quality of life, and higher long-term morbidity with other conditions,” Dr. Landgren said.

He cited the MAIA trial administered daratumumab and lenalidomide plus dexamethasone (DRd) to patients who were too old or too frail to qualify for ASCT. Over half of patients in the DRd arm of MAIA had an estimated progression-free survival rate at 60 months.

“Furthermore, GT and the MANHATTAN clinical trials showed that we can safely add CD38-targeted monoclonal antibodies to standard combination therapies [lenalidomide, bortezomib, and dexamethasone (KRd)], resulting in higher rates of minimal-residual-disease (MRD) negativity. That means modern four-drug combination therapies [DR-RVd and DR-KRd] will allow more [and more newly diagnosed] MM patients to achieve MRD negativity in the absence of ASCT,” Dr. Landgren concluded.

courtesy of Dr. Richter

Asked to comment on the two viewpoints, Joshua Richter, MD, director of myeloma treatment at the Blavatnik Family Center at Chelsea Mount Sinai, New York, said: “With some patients, we can get similar outcomes, whether or not we do a transplant. Doctors need to be better at choosing who really needs ASCT. Older people with standard-risk disease or people who achieve MRD-negative status after pharmacological treatment might not need to receive a transplant as much as those who have bulk disease or high-risk cytogenetics.

“Although ASCT might not be the best frontline option for everyone, collecting cells from most patients and storing them has many advantages. It allows us to do have the option of ASCT in later lines of therapy. In some patients with low blood counts, we can use stored cells to reboot their marrow and make them eligible for trials of promising new drugs,” Dr. Richter said.

Dr. Krishnan disclosed relationships with Takeda, Amgen, GlaxoSmithKline, Bristol-Myers Squibb, Sanofi, Pfizer, Adaptive, Regeneron, Janssen, AstraZeneca, Artiva, and Sutro. Dr. Landgren reported ties with Amgen, BMS, Celgene, Janssen, Takedam Glenmark, Juno, Pfizer, Merck, and others. Dr. Richter disclosed relationships with Janssen, BMS, and Takeda.

The U.S. government plans to no longer require federal workers or international air travelers to be vaccinated for COVID-19, the Biden administration has announced. 

The move means vaccines will no longer be required for workers who are federal employees, federal contractors, Head Start early education employees, workers at Medicare-certified health care facilities, and those who work at U.S. borders. International air travelers will no longer be required to prove their vaccination status. The requirement will be lifted at the end of the day on May 11, which is also when the federal public health emergency declaration ends.

“While vaccination remains one of the most important tools in advancing the health and safety of employees and promoting the efficiency of workplaces, we are now in a different phase of our response when these measures are no longer necessary,” an announcement from the White House stated.

White House officials credited vaccine requirements with saving millions of lives, noting that the rules ensured “the safety of workers in critical workforces including those in the healthcare and education sectors, protecting themselves and the populations they serve, and strengthening their ability to provide services without disruptions to operations.”

More than 100 million people were subject to the vaccine requirement, The Associated Press reported. All but 2% of those covered by the mandate had received at least one dose or had a pending or approved exception on file by January 2022, the Biden administration said, noting that COVID deaths have dropped 95% since January 2021 and hospitalizations are down nearly 91%.

In January, vaccine requirements were lifted for U.S. military members.

On the government-run website Safer Federal Workforce, which helped affected organizations put federal COVID rules into place, agencies were told to “take no action to implement or enforce the COVID-19 vaccination requirement” at this time.

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

The U.S. government plans to no longer require federal workers or international air travelers to be vaccinated for COVID-19, the Biden administration has announced. 

The move means vaccines will no longer be required for workers who are federal employees, federal contractors, Head Start early education employees, workers at Medicare-certified health care facilities, and those who work at U.S. borders. International air travelers will no longer be required to prove their vaccination status. The requirement will be lifted at the end of the day on May 11, which is also when the federal public health emergency declaration ends.

“While vaccination remains one of the most important tools in advancing the health and safety of employees and promoting the efficiency of workplaces, we are now in a different phase of our response when these measures are no longer necessary,” an announcement from the White House stated.

White House officials credited vaccine requirements with saving millions of lives, noting that the rules ensured “the safety of workers in critical workforces including those in the healthcare and education sectors, protecting themselves and the populations they serve, and strengthening their ability to provide services without disruptions to operations.”

More than 100 million people were subject to the vaccine requirement, The Associated Press reported. All but 2% of those covered by the mandate had received at least one dose or had a pending or approved exception on file by January 2022, the Biden administration said, noting that COVID deaths have dropped 95% since January 2021 and hospitalizations are down nearly 91%.

In January, vaccine requirements were lifted for U.S. military members.

On the government-run website Safer Federal Workforce, which helped affected organizations put federal COVID rules into place, agencies were told to “take no action to implement or enforce the COVID-19 vaccination requirement” at this time.

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

The U.S. government plans to no longer require federal workers or international air travelers to be vaccinated for COVID-19, the Biden administration has announced. 

The move means vaccines will no longer be required for workers who are federal employees, federal contractors, Head Start early education employees, workers at Medicare-certified health care facilities, and those who work at U.S. borders. International air travelers will no longer be required to prove their vaccination status. The requirement will be lifted at the end of the day on May 11, which is also when the federal public health emergency declaration ends.

“While vaccination remains one of the most important tools in advancing the health and safety of employees and promoting the efficiency of workplaces, we are now in a different phase of our response when these measures are no longer necessary,” an announcement from the White House stated.

White House officials credited vaccine requirements with saving millions of lives, noting that the rules ensured “the safety of workers in critical workforces including those in the healthcare and education sectors, protecting themselves and the populations they serve, and strengthening their ability to provide services without disruptions to operations.”

More than 100 million people were subject to the vaccine requirement, The Associated Press reported. All but 2% of those covered by the mandate had received at least one dose or had a pending or approved exception on file by January 2022, the Biden administration said, noting that COVID deaths have dropped 95% since January 2021 and hospitalizations are down nearly 91%.

In January, vaccine requirements were lifted for U.S. military members.

On the government-run website Safer Federal Workforce, which helped affected organizations put federal COVID rules into place, agencies were told to “take no action to implement or enforce the COVID-19 vaccination requirement” at this time.

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

There has been an increase in the incidence and prevalence of myasthenia gravis in the United States, an analysis of new claims data shows. Investigators speculate the rise of this rare disorder may be due to “increased diagnosis and more awareness of the disease over time, which has been shown in several studies,” study investigator Ema Rodrigues, DSc, MPH, with Alexion Pharmaceuticals, Boston.

Dr. Rodrigues presented her research at the 2023 annual meeting of the American Academy of Neurology.

Myasthenia gravis is a rare neuromuscular disease characterized by muscle weakness and fatigue caused by the binding of autoantibodies at the neuromuscular junction. It affects the voluntary muscles of the body, especially those that control the eyes, mouth, throat, and limbs.

In Europe, the incidence and prevalence of myasthenia gravis has increased for the past several decades. In the United States, increasing prevalence has also been observed, but recent estimates are lacking, making it tough to gauge the true burden of disease, Dr. Rodrigues explained. 
 

Claims-based analysis

To investigate, Dr. Rodrigues and colleagues analyzed claims data (commercial, Medicare, and Medicaid) and electronic health records representing over 300 million patients in the United States from 2011 to present.

They calculated sex- and age-specific incidence and prevalence of myasthenia gravis for the year 2021 using U.S. Census data.

Prevalent patients were identified as having one or more myasthenia gravis records in 2021 and two or more myasthenia gravis records, at least 30 days apart, from 2016 to 2021. This cohort had 78,225 patients.

Incident patients were identified as those with a myasthenia gravis record in 2021 and no previous myasthenia gravis record from 2019 to 2020. This cohort had 4,214 patients.

For both the prevalent and incident cohort, the distribution of male and female patients was roughly 50/50, with a slightly higher proportion of females in the incident cohort, Dr. Rodrigues reported.

When looking at age groups, there were “very few pediatric patients,” she noted, with less than 1% of the patients under the age of 12. The highest proportion of patients were 65 years or older. The mean age was 67 in the prevalent cohort and 68 in the incident cohort.

In 2021, the overall incidence of myasthenia gravis was 3.2 per 100,000 with similar estimates for males and females (3.2 vs. 3.1 per 100,000, respectively).

Total prevalence was estimated to be 37.0 per 100,000 with sex-specific estimates being comparable at 37.3 and 36.7 per 100,000 for males and females, respectively.

The incidence and prevalence of myasthenia gravis increased with age, ranging from 0.3 and 0.4 per 100,000, respectively, in children younger than age 2 years, to 10.2 and 116.8 per 100,000, respectively, in people 65 and older.

These estimates are “significantly higher” than those from a prior U.S. analysis from 2003, Dr. Rodrigues told attendees, but they are quite similar to the estimates that were reported in Sweden in 2020.

A limitation of the analysis is that patients who do not seek care regularly may have not been identified due to inclusion criteria, potentially leading to underestimates. Also, no information was available on the myasthenia gravis subtype (ocular vs. generalized).
 

Underestimated burden

Reached for comment, Richard J. Nowak, MD, MS, director of the Yale Myasthenia Gravis Clinic, Yale School of Medicine, New Haven, Conn., noted that the new report, “albeit limited as a claims-based analysis, presents modern data on incidence and prevalence of myasthenia gravis in the United States.”

“It suggests that the current estimates of myasthenia gravis in the United States are too low and that the true impact/burden of myasthenia gravis is greater. While we are unable to verify the accuracy of the diagnosis, the total myasthenia gravis population is likely to be about 100,000, which is higher than prior estimates.”

“This, in fact, might be driven by greater disease awareness and increased diagnosis along with decreased mortality and longer life expectancy,” Dr. Nowak said.

“Anecdotally, we are most certainly seeing patients with new-onset myasthenia gravis in their 70s, 80s, and even 90s in recent years. The EXPLORE-MG registry published data from a tertiary center on age of onset breakdown showing myasthenia gravis can present at any age,” Dr. Nowak added. 

Funding for the study was provided by Alexion, AstraZeneca Rare Disease. Dr. Rodrigues receives compensation and owns stock as an employee of Alexion, AstraZeneca Rare Diseases. Dr. Nowak has no relevant disclosures.

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

There has been an increase in the incidence and prevalence of myasthenia gravis in the United States, an analysis of new claims data shows. Investigators speculate the rise of this rare disorder may be due to “increased diagnosis and more awareness of the disease over time, which has been shown in several studies,” study investigator Ema Rodrigues, DSc, MPH, with Alexion Pharmaceuticals, Boston.

Dr. Rodrigues presented her research at the 2023 annual meeting of the American Academy of Neurology.

Myasthenia gravis is a rare neuromuscular disease characterized by muscle weakness and fatigue caused by the binding of autoantibodies at the neuromuscular junction. It affects the voluntary muscles of the body, especially those that control the eyes, mouth, throat, and limbs.

In Europe, the incidence and prevalence of myasthenia gravis has increased for the past several decades. In the United States, increasing prevalence has also been observed, but recent estimates are lacking, making it tough to gauge the true burden of disease, Dr. Rodrigues explained. 
 

Claims-based analysis

To investigate, Dr. Rodrigues and colleagues analyzed claims data (commercial, Medicare, and Medicaid) and electronic health records representing over 300 million patients in the United States from 2011 to present.

They calculated sex- and age-specific incidence and prevalence of myasthenia gravis for the year 2021 using U.S. Census data.

Prevalent patients were identified as having one or more myasthenia gravis records in 2021 and two or more myasthenia gravis records, at least 30 days apart, from 2016 to 2021. This cohort had 78,225 patients.

Incident patients were identified as those with a myasthenia gravis record in 2021 and no previous myasthenia gravis record from 2019 to 2020. This cohort had 4,214 patients.

For both the prevalent and incident cohort, the distribution of male and female patients was roughly 50/50, with a slightly higher proportion of females in the incident cohort, Dr. Rodrigues reported.

When looking at age groups, there were “very few pediatric patients,” she noted, with less than 1% of the patients under the age of 12. The highest proportion of patients were 65 years or older. The mean age was 67 in the prevalent cohort and 68 in the incident cohort.

In 2021, the overall incidence of myasthenia gravis was 3.2 per 100,000 with similar estimates for males and females (3.2 vs. 3.1 per 100,000, respectively).

Total prevalence was estimated to be 37.0 per 100,000 with sex-specific estimates being comparable at 37.3 and 36.7 per 100,000 for males and females, respectively.

The incidence and prevalence of myasthenia gravis increased with age, ranging from 0.3 and 0.4 per 100,000, respectively, in children younger than age 2 years, to 10.2 and 116.8 per 100,000, respectively, in people 65 and older.

These estimates are “significantly higher” than those from a prior U.S. analysis from 2003, Dr. Rodrigues told attendees, but they are quite similar to the estimates that were reported in Sweden in 2020.

A limitation of the analysis is that patients who do not seek care regularly may have not been identified due to inclusion criteria, potentially leading to underestimates. Also, no information was available on the myasthenia gravis subtype (ocular vs. generalized).
 

Underestimated burden

Reached for comment, Richard J. Nowak, MD, MS, director of the Yale Myasthenia Gravis Clinic, Yale School of Medicine, New Haven, Conn., noted that the new report, “albeit limited as a claims-based analysis, presents modern data on incidence and prevalence of myasthenia gravis in the United States.”

“It suggests that the current estimates of myasthenia gravis in the United States are too low and that the true impact/burden of myasthenia gravis is greater. While we are unable to verify the accuracy of the diagnosis, the total myasthenia gravis population is likely to be about 100,000, which is higher than prior estimates.”

“This, in fact, might be driven by greater disease awareness and increased diagnosis along with decreased mortality and longer life expectancy,” Dr. Nowak said.

“Anecdotally, we are most certainly seeing patients with new-onset myasthenia gravis in their 70s, 80s, and even 90s in recent years. The EXPLORE-MG registry published data from a tertiary center on age of onset breakdown showing myasthenia gravis can present at any age,” Dr. Nowak added. 

Funding for the study was provided by Alexion, AstraZeneca Rare Disease. Dr. Rodrigues receives compensation and owns stock as an employee of Alexion, AstraZeneca Rare Diseases. Dr. Nowak has no relevant disclosures.

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

There has been an increase in the incidence and prevalence of myasthenia gravis in the United States, an analysis of new claims data shows. Investigators speculate the rise of this rare disorder may be due to “increased diagnosis and more awareness of the disease over time, which has been shown in several studies,” study investigator Ema Rodrigues, DSc, MPH, with Alexion Pharmaceuticals, Boston.

Dr. Rodrigues presented her research at the 2023 annual meeting of the American Academy of Neurology.

Myasthenia gravis is a rare neuromuscular disease characterized by muscle weakness and fatigue caused by the binding of autoantibodies at the neuromuscular junction. It affects the voluntary muscles of the body, especially those that control the eyes, mouth, throat, and limbs.

In Europe, the incidence and prevalence of myasthenia gravis has increased for the past several decades. In the United States, increasing prevalence has also been observed, but recent estimates are lacking, making it tough to gauge the true burden of disease, Dr. Rodrigues explained. 
 

Claims-based analysis

To investigate, Dr. Rodrigues and colleagues analyzed claims data (commercial, Medicare, and Medicaid) and electronic health records representing over 300 million patients in the United States from 2011 to present.

They calculated sex- and age-specific incidence and prevalence of myasthenia gravis for the year 2021 using U.S. Census data.

Prevalent patients were identified as having one or more myasthenia gravis records in 2021 and two or more myasthenia gravis records, at least 30 days apart, from 2016 to 2021. This cohort had 78,225 patients.

Incident patients were identified as those with a myasthenia gravis record in 2021 and no previous myasthenia gravis record from 2019 to 2020. This cohort had 4,214 patients.

For both the prevalent and incident cohort, the distribution of male and female patients was roughly 50/50, with a slightly higher proportion of females in the incident cohort, Dr. Rodrigues reported.

When looking at age groups, there were “very few pediatric patients,” she noted, with less than 1% of the patients under the age of 12. The highest proportion of patients were 65 years or older. The mean age was 67 in the prevalent cohort and 68 in the incident cohort.

In 2021, the overall incidence of myasthenia gravis was 3.2 per 100,000 with similar estimates for males and females (3.2 vs. 3.1 per 100,000, respectively).

Total prevalence was estimated to be 37.0 per 100,000 with sex-specific estimates being comparable at 37.3 and 36.7 per 100,000 for males and females, respectively.

The incidence and prevalence of myasthenia gravis increased with age, ranging from 0.3 and 0.4 per 100,000, respectively, in children younger than age 2 years, to 10.2 and 116.8 per 100,000, respectively, in people 65 and older.

These estimates are “significantly higher” than those from a prior U.S. analysis from 2003, Dr. Rodrigues told attendees, but they are quite similar to the estimates that were reported in Sweden in 2020.

A limitation of the analysis is that patients who do not seek care regularly may have not been identified due to inclusion criteria, potentially leading to underestimates. Also, no information was available on the myasthenia gravis subtype (ocular vs. generalized).
 

Underestimated burden

Reached for comment, Richard J. Nowak, MD, MS, director of the Yale Myasthenia Gravis Clinic, Yale School of Medicine, New Haven, Conn., noted that the new report, “albeit limited as a claims-based analysis, presents modern data on incidence and prevalence of myasthenia gravis in the United States.”

“It suggests that the current estimates of myasthenia gravis in the United States are too low and that the true impact/burden of myasthenia gravis is greater. While we are unable to verify the accuracy of the diagnosis, the total myasthenia gravis population is likely to be about 100,000, which is higher than prior estimates.”

“This, in fact, might be driven by greater disease awareness and increased diagnosis along with decreased mortality and longer life expectancy,” Dr. Nowak said.

“Anecdotally, we are most certainly seeing patients with new-onset myasthenia gravis in their 70s, 80s, and even 90s in recent years. The EXPLORE-MG registry published data from a tertiary center on age of onset breakdown showing myasthenia gravis can present at any age,” Dr. Nowak added. 

Funding for the study was provided by Alexion, AstraZeneca Rare Disease. Dr. Rodrigues receives compensation and owns stock as an employee of Alexion, AstraZeneca Rare Diseases. Dr. Nowak has no relevant disclosures.

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

Higher step counts and cardiorespiratory fitness in adolescents were associated with a lower heart rate at rest – confirming an association long identified in adults, according to a study presented at the Pediatric Academic Societies annual meeting.

The new findings provide a new means for pediatricians to measure physical fitness, the researchers said.

“It really changes the way we evaluate kids’ fitness and gives us a new method of judging physical fitness other than body mass index,” said Susan Gasparino, MD, an instructor in pediatrics at the University of Rochester (N.Y.) Medical Center, who led the study.

Using data from the 2005 to 2006 National Health and Nutrition Examination Survey, Dr. Gasparino and her colleagues examined the association between resting heart rate (RHR) and step count among 899 children and 1,640 adolescents aged 6-19 years.

In the adolescent group, the mean RHR was 74.9 among those who walked more than 10,000 steps per day (n = 414) and 79.3 for those whose step counts fell below that cutoff (n = 1,226) (P < .001). For each additional 1,000 steps per day, RHR decreased by an average of 0.7 beats per minute in this group (P < .001).

In the younger age group, mean RHR was 85.3 among children who took more than 10,000 steps per day (n = 447) and 86.3 among those who did not reach that threshold (n = 452) (P = .29). For each additional 1,000 steps per day, RHR decreased by an average of 0.3 bpm in this group (P = .02)

Dr. Gasparino said next steps in research could include controlling for confounders, such as baseline anxiety and medications that could blunt the heart rate.
 

Broader implications

If similar results bear out in future studies, monitoring RHR could be incorporated into fitness programs for children and adolescents. Doing so could obviate “the need for intensive treadmill assessments using VO_2max, time-consuming and emotionally fraught school-based physical fitness tests, and the fear and potential shame of the scale,” the researchers said.

Dr. Gasparino said measuring RHR during a 3-minute step test could help organizations and governments determine whether fitness programs are improving cardiovascular and overall health and could help them direct “funding and resources to the programs that are effective.” Such a test could also be incorporated into pediatrician wellness checks, she noted.

“It’s an exciting development, and [RHR measurement] holds a lot of promise as a clinical tool that can be applicable in a lot of settings,” said Nicholas M. Edwards, MD, MPH, a sports medicine pediatrician and an associate professor of orthopedics at the University of Minnesota in Minneapolis.

Dr. Edwards said that, because measurement of fitness in clinical settings is difficult, finding ways to “assess fitness in the office with the equipment already at hand would be a superb development.”

If use of RHR to measure fitness “is validated in a clinical setting,” Dr. Edwards said, “I think adoption would be a natural next step.”

Dr. Edwards has disclosed no relevant financial relationships.

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

Higher step counts and cardiorespiratory fitness in adolescents were associated with a lower heart rate at rest – confirming an association long identified in adults, according to a study presented at the Pediatric Academic Societies annual meeting.

The new findings provide a new means for pediatricians to measure physical fitness, the researchers said.

“It really changes the way we evaluate kids’ fitness and gives us a new method of judging physical fitness other than body mass index,” said Susan Gasparino, MD, an instructor in pediatrics at the University of Rochester (N.Y.) Medical Center, who led the study.

Using data from the 2005 to 2006 National Health and Nutrition Examination Survey, Dr. Gasparino and her colleagues examined the association between resting heart rate (RHR) and step count among 899 children and 1,640 adolescents aged 6-19 years.

In the adolescent group, the mean RHR was 74.9 among those who walked more than 10,000 steps per day (n = 414) and 79.3 for those whose step counts fell below that cutoff (n = 1,226) (P < .001). For each additional 1,000 steps per day, RHR decreased by an average of 0.7 beats per minute in this group (P < .001).

In the younger age group, mean RHR was 85.3 among children who took more than 10,000 steps per day (n = 447) and 86.3 among those who did not reach that threshold (n = 452) (P = .29). For each additional 1,000 steps per day, RHR decreased by an average of 0.3 bpm in this group (P = .02)

Dr. Gasparino said next steps in research could include controlling for confounders, such as baseline anxiety and medications that could blunt the heart rate.
 

Broader implications

If similar results bear out in future studies, monitoring RHR could be incorporated into fitness programs for children and adolescents. Doing so could obviate “the need for intensive treadmill assessments using VO_2max, time-consuming and emotionally fraught school-based physical fitness tests, and the fear and potential shame of the scale,” the researchers said.

Dr. Gasparino said measuring RHR during a 3-minute step test could help organizations and governments determine whether fitness programs are improving cardiovascular and overall health and could help them direct “funding and resources to the programs that are effective.” Such a test could also be incorporated into pediatrician wellness checks, she noted.

“It’s an exciting development, and [RHR measurement] holds a lot of promise as a clinical tool that can be applicable in a lot of settings,” said Nicholas M. Edwards, MD, MPH, a sports medicine pediatrician and an associate professor of orthopedics at the University of Minnesota in Minneapolis.

Dr. Edwards said that, because measurement of fitness in clinical settings is difficult, finding ways to “assess fitness in the office with the equipment already at hand would be a superb development.”

If use of RHR to measure fitness “is validated in a clinical setting,” Dr. Edwards said, “I think adoption would be a natural next step.”

Dr. Edwards has disclosed no relevant financial relationships.

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

Higher step counts and cardiorespiratory fitness in adolescents were associated with a lower heart rate at rest – confirming an association long identified in adults, according to a study presented at the Pediatric Academic Societies annual meeting.

The new findings provide a new means for pediatricians to measure physical fitness, the researchers said.

“It really changes the way we evaluate kids’ fitness and gives us a new method of judging physical fitness other than body mass index,” said Susan Gasparino, MD, an instructor in pediatrics at the University of Rochester (N.Y.) Medical Center, who led the study.

Using data from the 2005 to 2006 National Health and Nutrition Examination Survey, Dr. Gasparino and her colleagues examined the association between resting heart rate (RHR) and step count among 899 children and 1,640 adolescents aged 6-19 years.

In the adolescent group, the mean RHR was 74.9 among those who walked more than 10,000 steps per day (n = 414) and 79.3 for those whose step counts fell below that cutoff (n = 1,226) (P < .001). For each additional 1,000 steps per day, RHR decreased by an average of 0.7 beats per minute in this group (P < .001).

In the younger age group, mean RHR was 85.3 among children who took more than 10,000 steps per day (n = 447) and 86.3 among those who did not reach that threshold (n = 452) (P = .29). For each additional 1,000 steps per day, RHR decreased by an average of 0.3 bpm in this group (P = .02)

Dr. Gasparino said next steps in research could include controlling for confounders, such as baseline anxiety and medications that could blunt the heart rate.
 

Broader implications

If similar results bear out in future studies, monitoring RHR could be incorporated into fitness programs for children and adolescents. Doing so could obviate “the need for intensive treadmill assessments using VO_2max, time-consuming and emotionally fraught school-based physical fitness tests, and the fear and potential shame of the scale,” the researchers said.

Dr. Gasparino said measuring RHR during a 3-minute step test could help organizations and governments determine whether fitness programs are improving cardiovascular and overall health and could help them direct “funding and resources to the programs that are effective.” Such a test could also be incorporated into pediatrician wellness checks, she noted.

“It’s an exciting development, and [RHR measurement] holds a lot of promise as a clinical tool that can be applicable in a lot of settings,” said Nicholas M. Edwards, MD, MPH, a sports medicine pediatrician and an associate professor of orthopedics at the University of Minnesota in Minneapolis.

Dr. Edwards said that, because measurement of fitness in clinical settings is difficult, finding ways to “assess fitness in the office with the equipment already at hand would be a superb development.”

If use of RHR to measure fitness “is validated in a clinical setting,” Dr. Edwards said, “I think adoption would be a natural next step.”

Dr. Edwards has disclosed no relevant financial relationships.

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

BOSTON – When it comes to relieving migraine, triptans, ergots, and antiemetics are the most effective classes of medications, a new real-world analysis of data on more than 3 million migraine attacks shows.

The findings “align with results of clinical trials and recommendations from clinical treatment guidelines” and provide insights to complement clinical practice, said study investigator Chia-Chun Chiang, MD, a neurologist with Mayo Clinic, Rochester, Minn.

The findings were presented at the 2023 annual meeting of the American Academy of Neurology.
 

The power of big data

Despite a wide variety of acute migraine medications that are available, large-scale, head-to-head comparisons of treatment effectiveness from real-world patient experience reports are lacking, Dr. Chiang explained.

“To the best of our knowledge, this is the first study that simultaneously compared multiple acute migraine medications using a Big Data analysis approach based on real-world patient-provided data,” she said.

The researchers extracted more than 10 million self-reported migraine attack records from a migraine smartphone app called Migraine Buddy, where users can document whether a treatment was helpful, somewhat helpful, unsure, or unhelpful.

They analyzed 25 acute medications among seven classes: acetaminophen, nonsteroidal anti-inflammatory drugs (NSAIDs), triptans, combination analgesics (acetaminophen/aspirin/caffeine), ergots, antiemetics, and opioids. The newer gepants and ditan medication classes of medications were not included because of the relatively lower numbers of usage when data was extracted (2014-2020).

The researchers employed a two-level nested logistic regression model to analyze the odds of treatment effectiveness of each medication by adjusting concurrent medications and the covariance within the same user.

The final analysis included more than 3.1 million migraine attacks among 278,000 users globally.

Using ibuprofen as the reference, triptans, ergots, and antiemetics had the highest efficacy with mean odds ratios of 4.8, 3.02, and 2.67, respectively, followed by opioids (OR, 2.49), NSAIDs (OR, 1.94), combination analgesics (OR, 1.69), others (OR, 1.49), and acetaminophen (OR, 0.83).

Individual medications with the highest patient-reported effectiveness were eletriptan (Relpax; OR, 6.1), zolmitriptan (Zomig; OR, 5.7) and sumatriptan (Zecuity; OR, 5.2).

This migraine medication comparative effectiveness analysis, based on patient-reported outcomes, “supports and complements the treatment recommendations from national headache societies based on randomized controlled trials and meta-analyses and strongly supports the use of triptans,” Dr. Chiang said.
 

End of trial-and-error?

Commenting on this research, Shaheen Lakhan, MD, PhD, a neurologist and researcher in Boston, said “This is a great study of Big Data in that it shows the power of the smartphone to collect real-world data and smart researchers like at Mayo Clinic to analyze them.”

“The study sheds light on how different therapeutics compare with each other. The next iteration of this line of research, I would hope, would be to determine if particular medications are effective for a particular migraine population, and even down to individuals with migraine,” said Dr. Lakhan, who wasn’t involved in the study.

“Once those models are appropriately built, long gone will be the era of trial-and-error medicine,” Dr. Lakhan added.

The study had no specific funding. Dr. Chiang has served as a consultant for Satsuma. Dr. Lakhan reports no relevant financial relationships.

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

BOSTON – When it comes to relieving migraine, triptans, ergots, and antiemetics are the most effective classes of medications, a new real-world analysis of data on more than 3 million migraine attacks shows.

The findings “align with results of clinical trials and recommendations from clinical treatment guidelines” and provide insights to complement clinical practice, said study investigator Chia-Chun Chiang, MD, a neurologist with Mayo Clinic, Rochester, Minn.

The findings were presented at the 2023 annual meeting of the American Academy of Neurology.
 

The power of big data

Despite a wide variety of acute migraine medications that are available, large-scale, head-to-head comparisons of treatment effectiveness from real-world patient experience reports are lacking, Dr. Chiang explained.

“To the best of our knowledge, this is the first study that simultaneously compared multiple acute migraine medications using a Big Data analysis approach based on real-world patient-provided data,” she said.

The researchers extracted more than 10 million self-reported migraine attack records from a migraine smartphone app called Migraine Buddy, where users can document whether a treatment was helpful, somewhat helpful, unsure, or unhelpful.

They analyzed 25 acute medications among seven classes: acetaminophen, nonsteroidal anti-inflammatory drugs (NSAIDs), triptans, combination analgesics (acetaminophen/aspirin/caffeine), ergots, antiemetics, and opioids. The newer gepants and ditan medication classes of medications were not included because of the relatively lower numbers of usage when data was extracted (2014-2020).

The researchers employed a two-level nested logistic regression model to analyze the odds of treatment effectiveness of each medication by adjusting concurrent medications and the covariance within the same user.

The final analysis included more than 3.1 million migraine attacks among 278,000 users globally.

Using ibuprofen as the reference, triptans, ergots, and antiemetics had the highest efficacy with mean odds ratios of 4.8, 3.02, and 2.67, respectively, followed by opioids (OR, 2.49), NSAIDs (OR, 1.94), combination analgesics (OR, 1.69), others (OR, 1.49), and acetaminophen (OR, 0.83).

Individual medications with the highest patient-reported effectiveness were eletriptan (Relpax; OR, 6.1), zolmitriptan (Zomig; OR, 5.7) and sumatriptan (Zecuity; OR, 5.2).

This migraine medication comparative effectiveness analysis, based on patient-reported outcomes, “supports and complements the treatment recommendations from national headache societies based on randomized controlled trials and meta-analyses and strongly supports the use of triptans,” Dr. Chiang said.
 

End of trial-and-error?

Commenting on this research, Shaheen Lakhan, MD, PhD, a neurologist and researcher in Boston, said “This is a great study of Big Data in that it shows the power of the smartphone to collect real-world data and smart researchers like at Mayo Clinic to analyze them.”

“The study sheds light on how different therapeutics compare with each other. The next iteration of this line of research, I would hope, would be to determine if particular medications are effective for a particular migraine population, and even down to individuals with migraine,” said Dr. Lakhan, who wasn’t involved in the study.

“Once those models are appropriately built, long gone will be the era of trial-and-error medicine,” Dr. Lakhan added.

The study had no specific funding. Dr. Chiang has served as a consultant for Satsuma. Dr. Lakhan reports no relevant financial relationships.

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

BOSTON – When it comes to relieving migraine, triptans, ergots, and antiemetics are the most effective classes of medications, a new real-world analysis of data on more than 3 million migraine attacks shows.

The findings “align with results of clinical trials and recommendations from clinical treatment guidelines” and provide insights to complement clinical practice, said study investigator Chia-Chun Chiang, MD, a neurologist with Mayo Clinic, Rochester, Minn.

The findings were presented at the 2023 annual meeting of the American Academy of Neurology.
 

The power of big data

Despite a wide variety of acute migraine medications that are available, large-scale, head-to-head comparisons of treatment effectiveness from real-world patient experience reports are lacking, Dr. Chiang explained.

“To the best of our knowledge, this is the first study that simultaneously compared multiple acute migraine medications using a Big Data analysis approach based on real-world patient-provided data,” she said.

The researchers extracted more than 10 million self-reported migraine attack records from a migraine smartphone app called Migraine Buddy, where users can document whether a treatment was helpful, somewhat helpful, unsure, or unhelpful.

They analyzed 25 acute medications among seven classes: acetaminophen, nonsteroidal anti-inflammatory drugs (NSAIDs), triptans, combination analgesics (acetaminophen/aspirin/caffeine), ergots, antiemetics, and opioids. The newer gepants and ditan medication classes of medications were not included because of the relatively lower numbers of usage when data was extracted (2014-2020).

The researchers employed a two-level nested logistic regression model to analyze the odds of treatment effectiveness of each medication by adjusting concurrent medications and the covariance within the same user.

The final analysis included more than 3.1 million migraine attacks among 278,000 users globally.

Using ibuprofen as the reference, triptans, ergots, and antiemetics had the highest efficacy with mean odds ratios of 4.8, 3.02, and 2.67, respectively, followed by opioids (OR, 2.49), NSAIDs (OR, 1.94), combination analgesics (OR, 1.69), others (OR, 1.49), and acetaminophen (OR, 0.83).

Individual medications with the highest patient-reported effectiveness were eletriptan (Relpax; OR, 6.1), zolmitriptan (Zomig; OR, 5.7) and sumatriptan (Zecuity; OR, 5.2).

This migraine medication comparative effectiveness analysis, based on patient-reported outcomes, “supports and complements the treatment recommendations from national headache societies based on randomized controlled trials and meta-analyses and strongly supports the use of triptans,” Dr. Chiang said.
 

End of trial-and-error?

Commenting on this research, Shaheen Lakhan, MD, PhD, a neurologist and researcher in Boston, said “This is a great study of Big Data in that it shows the power of the smartphone to collect real-world data and smart researchers like at Mayo Clinic to analyze them.”

“The study sheds light on how different therapeutics compare with each other. The next iteration of this line of research, I would hope, would be to determine if particular medications are effective for a particular migraine population, and even down to individuals with migraine,” said Dr. Lakhan, who wasn’t involved in the study.

“Once those models are appropriately built, long gone will be the era of trial-and-error medicine,” Dr. Lakhan added.

The study had no specific funding. Dr. Chiang has served as a consultant for Satsuma. Dr. Lakhan reports no relevant financial relationships.

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

BOSTON – Results of a phase 2 study demonstrate potential Alzheimer’s disease–modifying effects of an investigational oral antiamyloid agent, represented by positive changes in plasma and imaging biomarkers of Alzheimer’s disease pathology.

Use of the drug, ALZ-801 (Alzheon), led to a significant reduction of plasma phosphorylated–tau 181 (p-tau₁₈₁) , a marker of amyloid-induced neuronal injury in Alzheimer’s disease, as well as slowing of hippocampal atrophy and stabilization of cognition.

“The 12-month results of our phase 2 trial support the finding that ALZ-801 blocks misfolding of amyloid monomers and subsequent formation of neurotoxic amyloid oligomers, the key initial step in the amyloid aggregation cascade, which leads to a rapid and sustained reduction of brain neurodegeneration as measured by plasma p-tau₁₈₁,” John Hey, PhD, Alzheon’s chief scientific officer, said in a statement.

“The severalfold greater reduction on the p-tau₁₈₁ biomarker in plasma compared to plaque-clearing antiamyloid antibodies, combined with preservation of brain hippocampal volume and their positive correlations with cognitive benefits, further validate the disease-modifying effects of ALZ-801 in Alzheimer’s patients,” Dr. Hey added.

The results were presented at the 2023 annual meeting of the American Academy of Neurology.
 

ALZ-801 is an optimized prodrug of tramiprosate that has been shown to inhibit amyloid-beta 42 aggregation into toxic oligomers.

The ongoing phase 2 study is evaluating the effects of oral ALZ-801 (265 mg twice daily) on biomarkers of Alzheimer’s disease pathology for 84 adults with early Alzheimer’s disease who have either the APOE4/4 or APOE3/4 genotype. These genotypes represent the majority of patients with Alzheimer’s disease.

The mean age of the cohort was 69 years, and 51% are women; 70% had mild cognitive impairment, and 30% had mild Alzheimer’s disease. The mean Mini-Mental State Examination score for the cohort was 26.0. Roughly half were taking a cholinesterase inhibitor.

Significant plasma p-tau₁₈₁ reduction was observed at 13 weeks. Levels were reduced by 41% by 52 weeks (P = .016). There was also a significant 5% reduction in plasma amyloid-beta 42 and 40 at 52 weeks (P = .002 and P = .005, respectively), Dr. Hey reported.

After 12 months of treatment, hippocampal atrophy was reduced by about 23%, and expansion of ventricular volume was reduced by about 15%, both in comparison with matched controls from the Alzheimer’s Disease Neuroimaging Initiative.

Composite cognitive z-score improved significantly at 13 and 26 weeks and remained above baseline at 52 weeks in comparison with matched ADNI controls. “These are very promising data,” Dr. Hey told conference attendees.

He noted that the safety profile of ALZ-801 remains favorable and consistent with prior safety data. Common adverse events were mild nausea and SARS-CoV-2 infection. There were no drug-related serious events or amyloid-related imaging abnormalities–edema (ARIA-E).

The phase 3 APOLLOE4 study of ALZ-801 is underway. This double-blind, randomized study is comparing oral ALZ-801 with placebo over 78 weeks for roughly 300 adults with early Alzheimer’s disease who have the APOE4/4 genotype. APOLLOE4 is expected to be completed in mid 2024.

The APOLLOE4 study is supported by a $47 million grant from the National Institute on Aging. The U.S. Food and Drug Administration has granted ALZ-801 fast-track designation.
 

More accessible option?

Reached for comment, Percy Griffin, PhD, Alzheimer’s Association director of scientific engagement, noted that the “biggest difference between this drug and others is that it is taken orally, rather than delivered through an infusion. This is important and valuable for reducing patient and caregiver burden and increasing ease of use and access.”

It’s also noteworthy that ALZ-801 was not associated with ARIA-E, “which has been reported in other antiamyloid trials and can occasionally be serious,” Dr. Griffin said.

Overall, he said the results are “encouraging, but more work is needed. If studies results continue to be positive, this treatment may provide a more accessible option for people who are at higher risk of ARIA,” Dr. Griffin said.

The study was funded by Alzheon. Dr. Hey is an employee of Alzheon and holds stock in the company. Dr. Griffin has disclosed no relevant financial relationships.

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

BOSTON – Results of a phase 2 study demonstrate potential Alzheimer’s disease–modifying effects of an investigational oral antiamyloid agent, represented by positive changes in plasma and imaging biomarkers of Alzheimer’s disease pathology.

Use of the drug, ALZ-801 (Alzheon), led to a significant reduction of plasma phosphorylated–tau 181 (p-tau₁₈₁) , a marker of amyloid-induced neuronal injury in Alzheimer’s disease, as well as slowing of hippocampal atrophy and stabilization of cognition.

“The 12-month results of our phase 2 trial support the finding that ALZ-801 blocks misfolding of amyloid monomers and subsequent formation of neurotoxic amyloid oligomers, the key initial step in the amyloid aggregation cascade, which leads to a rapid and sustained reduction of brain neurodegeneration as measured by plasma p-tau₁₈₁,” John Hey, PhD, Alzheon’s chief scientific officer, said in a statement.

“The severalfold greater reduction on the p-tau₁₈₁ biomarker in plasma compared to plaque-clearing antiamyloid antibodies, combined with preservation of brain hippocampal volume and their positive correlations with cognitive benefits, further validate the disease-modifying effects of ALZ-801 in Alzheimer’s patients,” Dr. Hey added.

The results were presented at the 2023 annual meeting of the American Academy of Neurology.
 

ALZ-801 is an optimized prodrug of tramiprosate that has been shown to inhibit amyloid-beta 42 aggregation into toxic oligomers.

The ongoing phase 2 study is evaluating the effects of oral ALZ-801 (265 mg twice daily) on biomarkers of Alzheimer’s disease pathology for 84 adults with early Alzheimer’s disease who have either the APOE4/4 or APOE3/4 genotype. These genotypes represent the majority of patients with Alzheimer’s disease.

The mean age of the cohort was 69 years, and 51% are women; 70% had mild cognitive impairment, and 30% had mild Alzheimer’s disease. The mean Mini-Mental State Examination score for the cohort was 26.0. Roughly half were taking a cholinesterase inhibitor.

Significant plasma p-tau₁₈₁ reduction was observed at 13 weeks. Levels were reduced by 41% by 52 weeks (P = .016). There was also a significant 5% reduction in plasma amyloid-beta 42 and 40 at 52 weeks (P = .002 and P = .005, respectively), Dr. Hey reported.

After 12 months of treatment, hippocampal atrophy was reduced by about 23%, and expansion of ventricular volume was reduced by about 15%, both in comparison with matched controls from the Alzheimer’s Disease Neuroimaging Initiative.

Composite cognitive z-score improved significantly at 13 and 26 weeks and remained above baseline at 52 weeks in comparison with matched ADNI controls. “These are very promising data,” Dr. Hey told conference attendees.

He noted that the safety profile of ALZ-801 remains favorable and consistent with prior safety data. Common adverse events were mild nausea and SARS-CoV-2 infection. There were no drug-related serious events or amyloid-related imaging abnormalities–edema (ARIA-E).

The phase 3 APOLLOE4 study of ALZ-801 is underway. This double-blind, randomized study is comparing oral ALZ-801 with placebo over 78 weeks for roughly 300 adults with early Alzheimer’s disease who have the APOE4/4 genotype. APOLLOE4 is expected to be completed in mid 2024.

The APOLLOE4 study is supported by a $47 million grant from the National Institute on Aging. The U.S. Food and Drug Administration has granted ALZ-801 fast-track designation.
 

More accessible option?

Reached for comment, Percy Griffin, PhD, Alzheimer’s Association director of scientific engagement, noted that the “biggest difference between this drug and others is that it is taken orally, rather than delivered through an infusion. This is important and valuable for reducing patient and caregiver burden and increasing ease of use and access.”

It’s also noteworthy that ALZ-801 was not associated with ARIA-E, “which has been reported in other antiamyloid trials and can occasionally be serious,” Dr. Griffin said.

Overall, he said the results are “encouraging, but more work is needed. If studies results continue to be positive, this treatment may provide a more accessible option for people who are at higher risk of ARIA,” Dr. Griffin said.

The study was funded by Alzheon. Dr. Hey is an employee of Alzheon and holds stock in the company. Dr. Griffin has disclosed no relevant financial relationships.

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

BOSTON – Results of a phase 2 study demonstrate potential Alzheimer’s disease–modifying effects of an investigational oral antiamyloid agent, represented by positive changes in plasma and imaging biomarkers of Alzheimer’s disease pathology.

Use of the drug, ALZ-801 (Alzheon), led to a significant reduction of plasma phosphorylated–tau 181 (p-tau₁₈₁) , a marker of amyloid-induced neuronal injury in Alzheimer’s disease, as well as slowing of hippocampal atrophy and stabilization of cognition.

“The 12-month results of our phase 2 trial support the finding that ALZ-801 blocks misfolding of amyloid monomers and subsequent formation of neurotoxic amyloid oligomers, the key initial step in the amyloid aggregation cascade, which leads to a rapid and sustained reduction of brain neurodegeneration as measured by plasma p-tau₁₈₁,” John Hey, PhD, Alzheon’s chief scientific officer, said in a statement.

“The severalfold greater reduction on the p-tau₁₈₁ biomarker in plasma compared to plaque-clearing antiamyloid antibodies, combined with preservation of brain hippocampal volume and their positive correlations with cognitive benefits, further validate the disease-modifying effects of ALZ-801 in Alzheimer’s patients,” Dr. Hey added.

The results were presented at the 2023 annual meeting of the American Academy of Neurology.
 

ALZ-801 is an optimized prodrug of tramiprosate that has been shown to inhibit amyloid-beta 42 aggregation into toxic oligomers.

The ongoing phase 2 study is evaluating the effects of oral ALZ-801 (265 mg twice daily) on biomarkers of Alzheimer’s disease pathology for 84 adults with early Alzheimer’s disease who have either the APOE4/4 or APOE3/4 genotype. These genotypes represent the majority of patients with Alzheimer’s disease.

The mean age of the cohort was 69 years, and 51% are women; 70% had mild cognitive impairment, and 30% had mild Alzheimer’s disease. The mean Mini-Mental State Examination score for the cohort was 26.0. Roughly half were taking a cholinesterase inhibitor.

Significant plasma p-tau₁₈₁ reduction was observed at 13 weeks. Levels were reduced by 41% by 52 weeks (P = .016). There was also a significant 5% reduction in plasma amyloid-beta 42 and 40 at 52 weeks (P = .002 and P = .005, respectively), Dr. Hey reported.

After 12 months of treatment, hippocampal atrophy was reduced by about 23%, and expansion of ventricular volume was reduced by about 15%, both in comparison with matched controls from the Alzheimer’s Disease Neuroimaging Initiative.

Composite cognitive z-score improved significantly at 13 and 26 weeks and remained above baseline at 52 weeks in comparison with matched ADNI controls. “These are very promising data,” Dr. Hey told conference attendees.

He noted that the safety profile of ALZ-801 remains favorable and consistent with prior safety data. Common adverse events were mild nausea and SARS-CoV-2 infection. There were no drug-related serious events or amyloid-related imaging abnormalities–edema (ARIA-E).

The phase 3 APOLLOE4 study of ALZ-801 is underway. This double-blind, randomized study is comparing oral ALZ-801 with placebo over 78 weeks for roughly 300 adults with early Alzheimer’s disease who have the APOE4/4 genotype. APOLLOE4 is expected to be completed in mid 2024.

The APOLLOE4 study is supported by a $47 million grant from the National Institute on Aging. The U.S. Food and Drug Administration has granted ALZ-801 fast-track designation.
 

More accessible option?

Reached for comment, Percy Griffin, PhD, Alzheimer’s Association director of scientific engagement, noted that the “biggest difference between this drug and others is that it is taken orally, rather than delivered through an infusion. This is important and valuable for reducing patient and caregiver burden and increasing ease of use and access.”

It’s also noteworthy that ALZ-801 was not associated with ARIA-E, “which has been reported in other antiamyloid trials and can occasionally be serious,” Dr. Griffin said.

Overall, he said the results are “encouraging, but more work is needed. If studies results continue to be positive, this treatment may provide a more accessible option for people who are at higher risk of ARIA,” Dr. Griffin said.

The study was funded by Alzheon. Dr. Hey is an employee of Alzheon and holds stock in the company. Dr. Griffin has disclosed no relevant financial relationships.

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

What is the significance of the recent TRIANGLE study on mantle cell lymphoma (MCL)?

Dr. LaCasce: The TRIANGLE study is extremely important in previously untreated, transplant-eligible patients with MCL. The cutoff age for transplants varies by center and is between 60 and 75 years. In the absence of a TP53 mutation, we have typically used induction chemotherapy followed by autologous stem-cell transplant (ASCT), followed by 3 years of maintenance rituximab. Obviously, this is a lot of therapy.

The TRIANGLE study was a 3-arm study in which ibrutinib-containing therapy was compared with standard RCHOP/RDHAP followed by ASCT. Maintenance rituximab became standard of care midway through the trial and was added. In the first experimental arm, ibrutinib was combined with RCHOP and then given as maintenance for 2 years following ASCT. The second experimental arm included the same schedule of ibrutinib and omitted the ASCT.

The results are early, but what has been presented thus far, ibrutinib induction and maintenance with ASCT is clearly superior to the standard arm with ASCT. Although the data are not statistically mature, the failure-free survival of the 2 ibrutinib arms was similar, suggesting that transplant may not be necessary. Longer follow-up is necessary to confirm this conclusion and assess overall survival in all 3 arms.

If the results hold, ASCT could become a thing of the past or perhaps used in the second line. With the favorable activity of chimeric antigen receptor (CAR) T-cell therapy, however, it is unclear whether ASCT would be used in second line. Avoiding the sequential use of ASCT and CAR T-cell therapy is appealing given the stem-cell damage that can result. It is appealing to think about not using ASCT upfront, because ASCT increases the risk of myelodysplastic syndrome.

The TRIANGLE data are likely to change the frontline management of MCL. Although ibrutinib was the first Bruton tyrosine kinase (BTK) inhibitor approved in MCL and has obviously changed the field dramatically, it is significantly less well-tolerated than the next generation of drugs—acalabrutinib and zanubrutinib. I suspect these will be substituted for ibrutinib and we will see even more tolerable upfront regimens for patients with newly diagnosed MCL.

Have there been any disparities that you found in patients newly diagnosed with MCL regarding age, sex, or ethnicity?

Dr. LaCasce: MCL typically affects patients in their 60s. It is rare in young patients, and approximately 75% of the cases are male. If you look at the demographics, it is more common in White patients and less common in Hispanic and African American patients. In addition, there is an association with farming, which likely contribute to the demographics of patients with MCL.

What is your recommended approach to managing patients newly diagnosed with MCL in your day-to-day practice?

Dr. LaCasce: Management is a bit tricky right now because the TRIANGLE study is not part of any guidelines thus far. Therefore, most would argue the standard treatment continues to include ASCT upfront. There is an important, large randomized  study (NCT03267433) going on in the United States that is assessing the role of ASCT in patients who are in MRD-negative complete remission at the end of induction therapy. These patients are randomized to ASCT plus maintenance rituximab versus maintenance rituximab alone.

We are still enrolling patients to participate in this study, which is addressing a different question than TRIANGLE. I think we will learn a lot from this study. For patients who are not interested in participating in this study, we talk about the risks and benefits of ASCT.

One or 2 years ago, I would have strongly encouraged patients who were appropriate candidates to consider transplant in first remission. With the TRIANGLE data, however, and now that we have CAR T-cell therapy, I think it is more important to tailor the recommendation to the individual patient. If a patient is reluctant about ASCT and the associated risks, I do not push it.

If patients want the most aggressive approach associated with the longest remissions, at this moment, before TRIANGLE findings have been adopted into guidelines, I continue to recommend ASCT. For patients who have TP53 mutation, however, we treat with typically less aggressive therapy, as this patient population does not benefit from ASCT. We look forward to more data incorporating BTK inhibitors upfront, particularly for this group of patients, who tend to have a more adverse prognosis.

Do you feel MCL data and clinical trials are important areas of focus for your colleagues?

Dr. LaCasce: Yes. I think it is a rapidly evolving field, which is really exciting. We are seeing data now from the bispecific antibodies in the relapsed/refractory setting. We also need more data using pirtobrutinib for patients who have had BTK inhibitors and compare pirtobrutinib (a non-covalent BTK inhibitor) with the covalent BTK inhibitors.

I would strongly encourage patients to participate in clinical trials so that we can better answer these important questions. When patients go online and read about MCL, they often see a median survival of 3 to 4 years, which is completely outdated. The overall prognosis of MCL has changed dramatically since I have been in the field. Hopefully, survival will continue to improve, and therapies will become more tolerable, as well.

What is the significance of the recent TRIANGLE study on mantle cell lymphoma (MCL)?

Dr. LaCasce: The TRIANGLE study is extremely important in previously untreated, transplant-eligible patients with MCL. The cutoff age for transplants varies by center and is between 60 and 75 years. In the absence of a TP53 mutation, we have typically used induction chemotherapy followed by autologous stem-cell transplant (ASCT), followed by 3 years of maintenance rituximab. Obviously, this is a lot of therapy.

The TRIANGLE study was a 3-arm study in which ibrutinib-containing therapy was compared with standard RCHOP/RDHAP followed by ASCT. Maintenance rituximab became standard of care midway through the trial and was added. In the first experimental arm, ibrutinib was combined with RCHOP and then given as maintenance for 2 years following ASCT. The second experimental arm included the same schedule of ibrutinib and omitted the ASCT.

The results are early, but what has been presented thus far, ibrutinib induction and maintenance with ASCT is clearly superior to the standard arm with ASCT. Although the data are not statistically mature, the failure-free survival of the 2 ibrutinib arms was similar, suggesting that transplant may not be necessary. Longer follow-up is necessary to confirm this conclusion and assess overall survival in all 3 arms.

If the results hold, ASCT could become a thing of the past or perhaps used in the second line. With the favorable activity of chimeric antigen receptor (CAR) T-cell therapy, however, it is unclear whether ASCT would be used in second line. Avoiding the sequential use of ASCT and CAR T-cell therapy is appealing given the stem-cell damage that can result. It is appealing to think about not using ASCT upfront, because ASCT increases the risk of myelodysplastic syndrome.

The TRIANGLE data are likely to change the frontline management of MCL. Although ibrutinib was the first Bruton tyrosine kinase (BTK) inhibitor approved in MCL and has obviously changed the field dramatically, it is significantly less well-tolerated than the next generation of drugs—acalabrutinib and zanubrutinib. I suspect these will be substituted for ibrutinib and we will see even more tolerable upfront regimens for patients with newly diagnosed MCL.

Have there been any disparities that you found in patients newly diagnosed with MCL regarding age, sex, or ethnicity?

Dr. LaCasce: MCL typically affects patients in their 60s. It is rare in young patients, and approximately 75% of the cases are male. If you look at the demographics, it is more common in White patients and less common in Hispanic and African American patients. In addition, there is an association with farming, which likely contribute to the demographics of patients with MCL.

What is your recommended approach to managing patients newly diagnosed with MCL in your day-to-day practice?

Dr. LaCasce: Management is a bit tricky right now because the TRIANGLE study is not part of any guidelines thus far. Therefore, most would argue the standard treatment continues to include ASCT upfront. There is an important, large randomized  study (NCT03267433) going on in the United States that is assessing the role of ASCT in patients who are in MRD-negative complete remission at the end of induction therapy. These patients are randomized to ASCT plus maintenance rituximab versus maintenance rituximab alone.

We are still enrolling patients to participate in this study, which is addressing a different question than TRIANGLE. I think we will learn a lot from this study. For patients who are not interested in participating in this study, we talk about the risks and benefits of ASCT.

One or 2 years ago, I would have strongly encouraged patients who were appropriate candidates to consider transplant in first remission. With the TRIANGLE data, however, and now that we have CAR T-cell therapy, I think it is more important to tailor the recommendation to the individual patient. If a patient is reluctant about ASCT and the associated risks, I do not push it.

If patients want the most aggressive approach associated with the longest remissions, at this moment, before TRIANGLE findings have been adopted into guidelines, I continue to recommend ASCT. For patients who have TP53 mutation, however, we treat with typically less aggressive therapy, as this patient population does not benefit from ASCT. We look forward to more data incorporating BTK inhibitors upfront, particularly for this group of patients, who tend to have a more adverse prognosis.

Do you feel MCL data and clinical trials are important areas of focus for your colleagues?

Dr. LaCasce: Yes. I think it is a rapidly evolving field, which is really exciting. We are seeing data now from the bispecific antibodies in the relapsed/refractory setting. We also need more data using pirtobrutinib for patients who have had BTK inhibitors and compare pirtobrutinib (a non-covalent BTK inhibitor) with the covalent BTK inhibitors.

I would strongly encourage patients to participate in clinical trials so that we can better answer these important questions. When patients go online and read about MCL, they often see a median survival of 3 to 4 years, which is completely outdated. The overall prognosis of MCL has changed dramatically since I have been in the field. Hopefully, survival will continue to improve, and therapies will become more tolerable, as well.

What is the significance of the recent TRIANGLE study on mantle cell lymphoma (MCL)?

Dr. LaCasce: The TRIANGLE study is extremely important in previously untreated, transplant-eligible patients with MCL. The cutoff age for transplants varies by center and is between 60 and 75 years. In the absence of a TP53 mutation, we have typically used induction chemotherapy followed by autologous stem-cell transplant (ASCT), followed by 3 years of maintenance rituximab. Obviously, this is a lot of therapy.

The TRIANGLE study was a 3-arm study in which ibrutinib-containing therapy was compared with standard RCHOP/RDHAP followed by ASCT. Maintenance rituximab became standard of care midway through the trial and was added. In the first experimental arm, ibrutinib was combined with RCHOP and then given as maintenance for 2 years following ASCT. The second experimental arm included the same schedule of ibrutinib and omitted the ASCT.

The results are early, but what has been presented thus far, ibrutinib induction and maintenance with ASCT is clearly superior to the standard arm with ASCT. Although the data are not statistically mature, the failure-free survival of the 2 ibrutinib arms was similar, suggesting that transplant may not be necessary. Longer follow-up is necessary to confirm this conclusion and assess overall survival in all 3 arms.

If the results hold, ASCT could become a thing of the past or perhaps used in the second line. With the favorable activity of chimeric antigen receptor (CAR) T-cell therapy, however, it is unclear whether ASCT would be used in second line. Avoiding the sequential use of ASCT and CAR T-cell therapy is appealing given the stem-cell damage that can result. It is appealing to think about not using ASCT upfront, because ASCT increases the risk of myelodysplastic syndrome.

The TRIANGLE data are likely to change the frontline management of MCL. Although ibrutinib was the first Bruton tyrosine kinase (BTK) inhibitor approved in MCL and has obviously changed the field dramatically, it is significantly less well-tolerated than the next generation of drugs—acalabrutinib and zanubrutinib. I suspect these will be substituted for ibrutinib and we will see even more tolerable upfront regimens for patients with newly diagnosed MCL.

Have there been any disparities that you found in patients newly diagnosed with MCL regarding age, sex, or ethnicity?

Dr. LaCasce: MCL typically affects patients in their 60s. It is rare in young patients, and approximately 75% of the cases are male. If you look at the demographics, it is more common in White patients and less common in Hispanic and African American patients. In addition, there is an association with farming, which likely contribute to the demographics of patients with MCL.

What is your recommended approach to managing patients newly diagnosed with MCL in your day-to-day practice?

Dr. LaCasce: Management is a bit tricky right now because the TRIANGLE study is not part of any guidelines thus far. Therefore, most would argue the standard treatment continues to include ASCT upfront. There is an important, large randomized  study (NCT03267433) going on in the United States that is assessing the role of ASCT in patients who are in MRD-negative complete remission at the end of induction therapy. These patients are randomized to ASCT plus maintenance rituximab versus maintenance rituximab alone.

We are still enrolling patients to participate in this study, which is addressing a different question than TRIANGLE. I think we will learn a lot from this study. For patients who are not interested in participating in this study, we talk about the risks and benefits of ASCT.

One or 2 years ago, I would have strongly encouraged patients who were appropriate candidates to consider transplant in first remission. With the TRIANGLE data, however, and now that we have CAR T-cell therapy, I think it is more important to tailor the recommendation to the individual patient. If a patient is reluctant about ASCT and the associated risks, I do not push it.

If patients want the most aggressive approach associated with the longest remissions, at this moment, before TRIANGLE findings have been adopted into guidelines, I continue to recommend ASCT. For patients who have TP53 mutation, however, we treat with typically less aggressive therapy, as this patient population does not benefit from ASCT. We look forward to more data incorporating BTK inhibitors upfront, particularly for this group of patients, who tend to have a more adverse prognosis.

Do you feel MCL data and clinical trials are important areas of focus for your colleagues?

Dr. LaCasce: Yes. I think it is a rapidly evolving field, which is really exciting. We are seeing data now from the bispecific antibodies in the relapsed/refractory setting. We also need more data using pirtobrutinib for patients who have had BTK inhibitors and compare pirtobrutinib (a non-covalent BTK inhibitor) with the covalent BTK inhibitors.

I would strongly encourage patients to participate in clinical trials so that we can better answer these important questions. When patients go online and read about MCL, they often see a median survival of 3 to 4 years, which is completely outdated. The overall prognosis of MCL has changed dramatically since I have been in the field. Hopefully, survival will continue to improve, and therapies will become more tolerable, as well.

MANCHESTER, England – Data from the COVAD-2 e-survey suggest that people with a rheumatic disease are twice as likely as are those without to experience long-term effects after contracting COVID-19.

The prevalence of post–COVID-19 condition (PCC), the term the World Health Organization advocates for describing the widely popularized term long COVID, was 10.8% among people with autoimmune rheumatic diseases (AIRDs) vs. 5.3% among those with no autoimmune condition (designated as “healthy controls”). The odds ratio was 2.1, with a 95% confidence interval of 1.4-3.2 and a P-value of .002.

The prevalence in people with nonrheumatic autoimmune diseases was also higher than it was in the control participants but still lower, at 7.3%, than in those with AIRDs.

Sara Freeman/MDedge News

“Our findings highlight the importance of close monitoring for PCC,” Arvind Nune, MBBCh, MSc, said in a virtual poster presentation at the annual meeting of the British Society for Rheumatology.

They also show the need for “appropriate referral for optimized multidisciplinary care for patients with autoimmune rheumatic diseases during the recovery period following COVID-19,” added Dr. Nune, who works for Southport (England) and Ormskirk Hospital NHS Trust.

In an interview, he noted that it was patients who had a severe COVID-19 course or had other coexisting conditions that appeared to experience more long-term effects than did their less-affected counterparts.

Commenting on the study, Jeffrey A. Sparks, MD, MMSc, told this news organization: “This is one of the first studies to find that the prevalence of long COVID is higher among people with systemic rheumatic diseases than those without.”

Dr. Sparks, who is based at Brigham and Women’s Hospital and Harvard Medical School in Boston, added: “Since the symptoms of long COVID and rheumatic diseases can overlap substantially, more work will need to be done to determine whether COVID may have induced flares, new symptoms, or whether the finding is due to the presence of the chronic rheumatic disease.”
 

The COVAD study

Using an electronic survey platform, the COVAD study has been set up to look at the long-term efficacy and safety of COVID-19 vaccinations in patients with AIRDs. It’s now a large international effort involving more than 150 collaborating clinics in 106 countries.

A huge amount of data has been collected. “We collected demographics, details of autoimmune disease, including treatment, comorbidity, COVID infection, vaccination history and outcomes, date on flares, and validated patient-reported outcomes, including pain, fatigue, physical function, and quality of life,” Dr. Nune said in his presentation.

A total of 12,358 people who were invited to participate responded to the e-survey. Of them, 2,640 were confirmed to have COVID-19. Because the analysis aimed to look at PCC, anyone who had completed the survey less than 3 months after infection was excluded. This left 1,677 eligible respondents, of whom, an overall 8.7% (n = 136) were identified as having PCC.

“The [WHO] definition for PCC was employed, which is persistent signs or symptoms beyond 3 months of COVID-19 infection lasting at least 2 months,” Dr. Nune told this news organization.

“Symptoms could be anything from fatigue to breathlessness to arthralgias,” he added. However, the focus of the present analysis was to look at how many people were experiencing the condition rather than specific symptoms.

A higher risk for PCC was seen in women than in men (OR, 2.9; 95% CI, 1.1-7.7; P = .037) in the entire cohort.

In addition, those with comorbidities were found to have a greater chance of long-term sequelae from COVID-19 than were those without comorbid disease (OR, 2.8; 95% CI, 1.4-5.7; P = .005).

Patients who experienced more severe acute COVID-19, such as those who needed intensive care treatment, oxygen therapy, or advanced treatment for COVID-19 with monoclonal antibodies, were significantly more likely to later have PCC than were those who did not (OR, 3.8; 95% CI, 1.1-13.6; P = .039).

Having PCC was also associated with poorer patient-reported outcomes for physical function, compared with not having PCC. “However, no association with disease flares of underlying rheumatic diseases or immunosuppressive drugs used were noted,” Dr. Nune said.

These new findings from the COVAD study should be published soon. Dr. Nune suggested that the findings might be used to help identify patients early so that they can be referred to the appropriate services in good time.

The COVAD study was independently supported. Dr. Nune reports no relevant financial relationships. Dr. Sparks is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Llura Gund Award for Rheumatoid Arthritis Research and Care. Dr. Sparks has received research support from Bristol-Myers Squibb and performed consultancy for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova Diagnostics, Janssen, Optum, and Pfizer.
 

MANCHESTER, England – Data from the COVAD-2 e-survey suggest that people with a rheumatic disease are twice as likely as are those without to experience long-term effects after contracting COVID-19.

The prevalence of post–COVID-19 condition (PCC), the term the World Health Organization advocates for describing the widely popularized term long COVID, was 10.8% among people with autoimmune rheumatic diseases (AIRDs) vs. 5.3% among those with no autoimmune condition (designated as “healthy controls”). The odds ratio was 2.1, with a 95% confidence interval of 1.4-3.2 and a P-value of .002.

The prevalence in people with nonrheumatic autoimmune diseases was also higher than it was in the control participants but still lower, at 7.3%, than in those with AIRDs.

Sara Freeman/MDedge News

“Our findings highlight the importance of close monitoring for PCC,” Arvind Nune, MBBCh, MSc, said in a virtual poster presentation at the annual meeting of the British Society for Rheumatology.

They also show the need for “appropriate referral for optimized multidisciplinary care for patients with autoimmune rheumatic diseases during the recovery period following COVID-19,” added Dr. Nune, who works for Southport (England) and Ormskirk Hospital NHS Trust.

In an interview, he noted that it was patients who had a severe COVID-19 course or had other coexisting conditions that appeared to experience more long-term effects than did their less-affected counterparts.

Commenting on the study, Jeffrey A. Sparks, MD, MMSc, told this news organization: “This is one of the first studies to find that the prevalence of long COVID is higher among people with systemic rheumatic diseases than those without.”

Dr. Sparks, who is based at Brigham and Women’s Hospital and Harvard Medical School in Boston, added: “Since the symptoms of long COVID and rheumatic diseases can overlap substantially, more work will need to be done to determine whether COVID may have induced flares, new symptoms, or whether the finding is due to the presence of the chronic rheumatic disease.”
 

The COVAD study

Using an electronic survey platform, the COVAD study has been set up to look at the long-term efficacy and safety of COVID-19 vaccinations in patients with AIRDs. It’s now a large international effort involving more than 150 collaborating clinics in 106 countries.

A huge amount of data has been collected. “We collected demographics, details of autoimmune disease, including treatment, comorbidity, COVID infection, vaccination history and outcomes, date on flares, and validated patient-reported outcomes, including pain, fatigue, physical function, and quality of life,” Dr. Nune said in his presentation.

A total of 12,358 people who were invited to participate responded to the e-survey. Of them, 2,640 were confirmed to have COVID-19. Because the analysis aimed to look at PCC, anyone who had completed the survey less than 3 months after infection was excluded. This left 1,677 eligible respondents, of whom, an overall 8.7% (n = 136) were identified as having PCC.

“The [WHO] definition for PCC was employed, which is persistent signs or symptoms beyond 3 months of COVID-19 infection lasting at least 2 months,” Dr. Nune told this news organization.

“Symptoms could be anything from fatigue to breathlessness to arthralgias,” he added. However, the focus of the present analysis was to look at how many people were experiencing the condition rather than specific symptoms.

A higher risk for PCC was seen in women than in men (OR, 2.9; 95% CI, 1.1-7.7; P = .037) in the entire cohort.

In addition, those with comorbidities were found to have a greater chance of long-term sequelae from COVID-19 than were those without comorbid disease (OR, 2.8; 95% CI, 1.4-5.7; P = .005).

Patients who experienced more severe acute COVID-19, such as those who needed intensive care treatment, oxygen therapy, or advanced treatment for COVID-19 with monoclonal antibodies, were significantly more likely to later have PCC than were those who did not (OR, 3.8; 95% CI, 1.1-13.6; P = .039).

Having PCC was also associated with poorer patient-reported outcomes for physical function, compared with not having PCC. “However, no association with disease flares of underlying rheumatic diseases or immunosuppressive drugs used were noted,” Dr. Nune said.

These new findings from the COVAD study should be published soon. Dr. Nune suggested that the findings might be used to help identify patients early so that they can be referred to the appropriate services in good time.

The COVAD study was independently supported. Dr. Nune reports no relevant financial relationships. Dr. Sparks is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Llura Gund Award for Rheumatoid Arthritis Research and Care. Dr. Sparks has received research support from Bristol-Myers Squibb and performed consultancy for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova Diagnostics, Janssen, Optum, and Pfizer.
 

MANCHESTER, England – Data from the COVAD-2 e-survey suggest that people with a rheumatic disease are twice as likely as are those without to experience long-term effects after contracting COVID-19.

The prevalence of post–COVID-19 condition (PCC), the term the World Health Organization advocates for describing the widely popularized term long COVID, was 10.8% among people with autoimmune rheumatic diseases (AIRDs) vs. 5.3% among those with no autoimmune condition (designated as “healthy controls”). The odds ratio was 2.1, with a 95% confidence interval of 1.4-3.2 and a P-value of .002.

The prevalence in people with nonrheumatic autoimmune diseases was also higher than it was in the control participants but still lower, at 7.3%, than in those with AIRDs.

Sara Freeman/MDedge News

“Our findings highlight the importance of close monitoring for PCC,” Arvind Nune, MBBCh, MSc, said in a virtual poster presentation at the annual meeting of the British Society for Rheumatology.

They also show the need for “appropriate referral for optimized multidisciplinary care for patients with autoimmune rheumatic diseases during the recovery period following COVID-19,” added Dr. Nune, who works for Southport (England) and Ormskirk Hospital NHS Trust.

In an interview, he noted that it was patients who had a severe COVID-19 course or had other coexisting conditions that appeared to experience more long-term effects than did their less-affected counterparts.

Commenting on the study, Jeffrey A. Sparks, MD, MMSc, told this news organization: “This is one of the first studies to find that the prevalence of long COVID is higher among people with systemic rheumatic diseases than those without.”

Dr. Sparks, who is based at Brigham and Women’s Hospital and Harvard Medical School in Boston, added: “Since the symptoms of long COVID and rheumatic diseases can overlap substantially, more work will need to be done to determine whether COVID may have induced flares, new symptoms, or whether the finding is due to the presence of the chronic rheumatic disease.”
 

The COVAD study

Using an electronic survey platform, the COVAD study has been set up to look at the long-term efficacy and safety of COVID-19 vaccinations in patients with AIRDs. It’s now a large international effort involving more than 150 collaborating clinics in 106 countries.

A huge amount of data has been collected. “We collected demographics, details of autoimmune disease, including treatment, comorbidity, COVID infection, vaccination history and outcomes, date on flares, and validated patient-reported outcomes, including pain, fatigue, physical function, and quality of life,” Dr. Nune said in his presentation.

A total of 12,358 people who were invited to participate responded to the e-survey. Of them, 2,640 were confirmed to have COVID-19. Because the analysis aimed to look at PCC, anyone who had completed the survey less than 3 months after infection was excluded. This left 1,677 eligible respondents, of whom, an overall 8.7% (n = 136) were identified as having PCC.

“The [WHO] definition for PCC was employed, which is persistent signs or symptoms beyond 3 months of COVID-19 infection lasting at least 2 months,” Dr. Nune told this news organization.

“Symptoms could be anything from fatigue to breathlessness to arthralgias,” he added. However, the focus of the present analysis was to look at how many people were experiencing the condition rather than specific symptoms.

A higher risk for PCC was seen in women than in men (OR, 2.9; 95% CI, 1.1-7.7; P = .037) in the entire cohort.

In addition, those with comorbidities were found to have a greater chance of long-term sequelae from COVID-19 than were those without comorbid disease (OR, 2.8; 95% CI, 1.4-5.7; P = .005).

Patients who experienced more severe acute COVID-19, such as those who needed intensive care treatment, oxygen therapy, or advanced treatment for COVID-19 with monoclonal antibodies, were significantly more likely to later have PCC than were those who did not (OR, 3.8; 95% CI, 1.1-13.6; P = .039).

Having PCC was also associated with poorer patient-reported outcomes for physical function, compared with not having PCC. “However, no association with disease flares of underlying rheumatic diseases or immunosuppressive drugs used were noted,” Dr. Nune said.

These new findings from the COVAD study should be published soon. Dr. Nune suggested that the findings might be used to help identify patients early so that they can be referred to the appropriate services in good time.

The COVAD study was independently supported. Dr. Nune reports no relevant financial relationships. Dr. Sparks is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the R. Bruce and Joan M. Mickey Research Scholar Fund, and the Llura Gund Award for Rheumatoid Arthritis Research and Care. Dr. Sparks has received research support from Bristol-Myers Squibb and performed consultancy for AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Gilead, Inova Diagnostics, Janssen, Optum, and Pfizer.