MDedge Neurology

A first stroke in older adults is associated with substantial immediate and accelerated long-term cognitive decline, suggested a new study that underscores the need for continuous cognitive monitoring in this patient population.

Results from the study, which included 14 international cohorts of older adults, showed that stroke was associated with a significant acute decline in global cognition and a small, but significant, acceleration in the rate of cognitive decline over time.

Cognitive assessments in primary care are “crucial, especially since cognitive impairment is frequently missed or undiagnosed in hospitals,” lead author Jessica Lo, MSc, biostatistician and research associate with the Center for Healthy Brain Aging, University of New South Wales, Sydney, Australia, told this news organization.

She suggested clinicians incorporate long-term cognitive assessments into care plans, using more sensitive neuropsychological tests in primary care to detect early signs of cognitive impairment. “Early detection would enable timely interventions to improve outcomes,” Lo said.

She also noted that poststroke care typically includes physical rehabilitation but not cognitive rehabilitation, which many rehabilitation centers aren’t equipped to provide.

The study was published online in JAMA Network Open.
 

Mapping Cognitive Decline Trajectory

Cognitive impairment after stroke is common, but the trajectory of cognitive decline following a first stroke, relative to prestroke cognitive function, remains unclear.

The investigators leveraged data from 14 population-based cohort studies of 20,860 adults (mean age, 73 years; 59% women) to map the trajectory of cognitive function before and after a first stroke.

The primary outcome was global cognition, defined as the standardized average of four cognitive domains (language, memory, processing speed, and executive function).

During a mean follow-up of 7.5 years, 1041 (5%) adults (mean age, 79 years) experienced a first stroke, a mean of 4.5 years after study entry.

In adjusted analyses, stroke was associated with a significant acute decline of 0.25 SD in global cognition and a “small but significant” acceleration in the rate of decline of −0.038 SD per year, the authors reported.

Stroke was also associated with acute decline in all individual cognitive domains except for memory, with effect sizes ranging from −0.17 to −0.22 SD. Poststroke declines in Mini-Mental State Examination scores (−0.36 SD) were also noted.

In terms of cognitive trajectory, the rate of decline before stroke in survivors was similar to that seen in peers who didn’t have a stroke (−0.048 and −0.049 SD per year in global cognition, respectively).

The researchers did not identify any vascular risk factors moderating cognitive decline following a stroke, consistent with prior research. However, cognitive decline was significantly more rapid in individuals without stroke, regardless of any future stroke, who had a history of diabetes, hypertension, high cholesterol, cardiovascular disease, depression, smoking, or were APOE4 carriers.

“Targeting modifiable vascular risk factors at an early stage may reduce the risk of stroke but also subsequent risk of stroke-related cognitive decline and cognitive impairment,” the researchers noted.
 

A ‘Major Step’ in the Right Direction

As previously reported by this news organization, in 2023 the American Heart Association (AHA) issued a statement noting that screening for cognitive impairment should be part of multidisciplinary care for stroke survivors.

Commenting for this news organization, Mitchell Elkind, MD, MS, AHA chief clinical science officer, said these new data are consistent with current AHA guidelines and statements that “support screening for cognitive and functional decline in patients both acutely and over the long term after stroke.”

Elkind noted that the 2022 guideline for intracerebral hemorrhage states that cognitive screening should occur “across the continuum of inpatient care and at intervals in the outpatient setting” and provides recommendations for cognitive therapy.

“Our 2021 scientific statement on the primary care of patients after stroke also recommends screening for both depression and cognitive impairment over both the short- and long-term,” said Elkind, professor of neurology and epidemiology at Columbia University Irving Medical Center in New York City.

“These documents recognize the fact that function and cognition can continue to decline years after stroke and that patients’ rehabilitation and support needs may therefore change over time after stroke,” Elkind added.

The authors of an accompanying commentary called it a “major step” in the right direction for the future of long-term stroke outcome assessment.

“As we develop new devices, indications, and time windows for stroke treatment, it may perhaps be wise to ensure trials steer away from simpler outcomes to more complex, granular ones,” wrote Yasmin Sadigh, MSc, and Victor Volovici, MD, PhD, with Erasmus University Medical Center, Rotterdam, the Netherlands.

The study had no commercial funding. The authors and commentary writers and Elkind have declared no conflicts of interest.

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

A first stroke in older adults is associated with substantial immediate and accelerated long-term cognitive decline, suggested a new study that underscores the need for continuous cognitive monitoring in this patient population.

Results from the study, which included 14 international cohorts of older adults, showed that stroke was associated with a significant acute decline in global cognition and a small, but significant, acceleration in the rate of cognitive decline over time.

Cognitive assessments in primary care are “crucial, especially since cognitive impairment is frequently missed or undiagnosed in hospitals,” lead author Jessica Lo, MSc, biostatistician and research associate with the Center for Healthy Brain Aging, University of New South Wales, Sydney, Australia, told this news organization.

She suggested clinicians incorporate long-term cognitive assessments into care plans, using more sensitive neuropsychological tests in primary care to detect early signs of cognitive impairment. “Early detection would enable timely interventions to improve outcomes,” Lo said.

She also noted that poststroke care typically includes physical rehabilitation but not cognitive rehabilitation, which many rehabilitation centers aren’t equipped to provide.

The study was published online in JAMA Network Open.
 

Mapping Cognitive Decline Trajectory

Cognitive impairment after stroke is common, but the trajectory of cognitive decline following a first stroke, relative to prestroke cognitive function, remains unclear.

The investigators leveraged data from 14 population-based cohort studies of 20,860 adults (mean age, 73 years; 59% women) to map the trajectory of cognitive function before and after a first stroke.

The primary outcome was global cognition, defined as the standardized average of four cognitive domains (language, memory, processing speed, and executive function).

During a mean follow-up of 7.5 years, 1041 (5%) adults (mean age, 79 years) experienced a first stroke, a mean of 4.5 years after study entry.

In adjusted analyses, stroke was associated with a significant acute decline of 0.25 SD in global cognition and a “small but significant” acceleration in the rate of decline of −0.038 SD per year, the authors reported.

Stroke was also associated with acute decline in all individual cognitive domains except for memory, with effect sizes ranging from −0.17 to −0.22 SD. Poststroke declines in Mini-Mental State Examination scores (−0.36 SD) were also noted.

In terms of cognitive trajectory, the rate of decline before stroke in survivors was similar to that seen in peers who didn’t have a stroke (−0.048 and −0.049 SD per year in global cognition, respectively).

The researchers did not identify any vascular risk factors moderating cognitive decline following a stroke, consistent with prior research. However, cognitive decline was significantly more rapid in individuals without stroke, regardless of any future stroke, who had a history of diabetes, hypertension, high cholesterol, cardiovascular disease, depression, smoking, or were APOE4 carriers.

“Targeting modifiable vascular risk factors at an early stage may reduce the risk of stroke but also subsequent risk of stroke-related cognitive decline and cognitive impairment,” the researchers noted.
 

A ‘Major Step’ in the Right Direction

As previously reported by this news organization, in 2023 the American Heart Association (AHA) issued a statement noting that screening for cognitive impairment should be part of multidisciplinary care for stroke survivors.

Commenting for this news organization, Mitchell Elkind, MD, MS, AHA chief clinical science officer, said these new data are consistent with current AHA guidelines and statements that “support screening for cognitive and functional decline in patients both acutely and over the long term after stroke.”

Elkind noted that the 2022 guideline for intracerebral hemorrhage states that cognitive screening should occur “across the continuum of inpatient care and at intervals in the outpatient setting” and provides recommendations for cognitive therapy.

“Our 2021 scientific statement on the primary care of patients after stroke also recommends screening for both depression and cognitive impairment over both the short- and long-term,” said Elkind, professor of neurology and epidemiology at Columbia University Irving Medical Center in New York City.

“These documents recognize the fact that function and cognition can continue to decline years after stroke and that patients’ rehabilitation and support needs may therefore change over time after stroke,” Elkind added.

The authors of an accompanying commentary called it a “major step” in the right direction for the future of long-term stroke outcome assessment.

“As we develop new devices, indications, and time windows for stroke treatment, it may perhaps be wise to ensure trials steer away from simpler outcomes to more complex, granular ones,” wrote Yasmin Sadigh, MSc, and Victor Volovici, MD, PhD, with Erasmus University Medical Center, Rotterdam, the Netherlands.

The study had no commercial funding. The authors and commentary writers and Elkind have declared no conflicts of interest.

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

A first stroke in older adults is associated with substantial immediate and accelerated long-term cognitive decline, suggested a new study that underscores the need for continuous cognitive monitoring in this patient population.

Results from the study, which included 14 international cohorts of older adults, showed that stroke was associated with a significant acute decline in global cognition and a small, but significant, acceleration in the rate of cognitive decline over time.

Cognitive assessments in primary care are “crucial, especially since cognitive impairment is frequently missed or undiagnosed in hospitals,” lead author Jessica Lo, MSc, biostatistician and research associate with the Center for Healthy Brain Aging, University of New South Wales, Sydney, Australia, told this news organization.

She suggested clinicians incorporate long-term cognitive assessments into care plans, using more sensitive neuropsychological tests in primary care to detect early signs of cognitive impairment. “Early detection would enable timely interventions to improve outcomes,” Lo said.

She also noted that poststroke care typically includes physical rehabilitation but not cognitive rehabilitation, which many rehabilitation centers aren’t equipped to provide.

The study was published online in JAMA Network Open.
 

Mapping Cognitive Decline Trajectory

Cognitive impairment after stroke is common, but the trajectory of cognitive decline following a first stroke, relative to prestroke cognitive function, remains unclear.

The investigators leveraged data from 14 population-based cohort studies of 20,860 adults (mean age, 73 years; 59% women) to map the trajectory of cognitive function before and after a first stroke.

The primary outcome was global cognition, defined as the standardized average of four cognitive domains (language, memory, processing speed, and executive function).

During a mean follow-up of 7.5 years, 1041 (5%) adults (mean age, 79 years) experienced a first stroke, a mean of 4.5 years after study entry.

In adjusted analyses, stroke was associated with a significant acute decline of 0.25 SD in global cognition and a “small but significant” acceleration in the rate of decline of −0.038 SD per year, the authors reported.

Stroke was also associated with acute decline in all individual cognitive domains except for memory, with effect sizes ranging from −0.17 to −0.22 SD. Poststroke declines in Mini-Mental State Examination scores (−0.36 SD) were also noted.

In terms of cognitive trajectory, the rate of decline before stroke in survivors was similar to that seen in peers who didn’t have a stroke (−0.048 and −0.049 SD per year in global cognition, respectively).

The researchers did not identify any vascular risk factors moderating cognitive decline following a stroke, consistent with prior research. However, cognitive decline was significantly more rapid in individuals without stroke, regardless of any future stroke, who had a history of diabetes, hypertension, high cholesterol, cardiovascular disease, depression, smoking, or were APOE4 carriers.

“Targeting modifiable vascular risk factors at an early stage may reduce the risk of stroke but also subsequent risk of stroke-related cognitive decline and cognitive impairment,” the researchers noted.
 

A ‘Major Step’ in the Right Direction

As previously reported by this news organization, in 2023 the American Heart Association (AHA) issued a statement noting that screening for cognitive impairment should be part of multidisciplinary care for stroke survivors.

Commenting for this news organization, Mitchell Elkind, MD, MS, AHA chief clinical science officer, said these new data are consistent with current AHA guidelines and statements that “support screening for cognitive and functional decline in patients both acutely and over the long term after stroke.”

Elkind noted that the 2022 guideline for intracerebral hemorrhage states that cognitive screening should occur “across the continuum of inpatient care and at intervals in the outpatient setting” and provides recommendations for cognitive therapy.

“Our 2021 scientific statement on the primary care of patients after stroke also recommends screening for both depression and cognitive impairment over both the short- and long-term,” said Elkind, professor of neurology and epidemiology at Columbia University Irving Medical Center in New York City.

“These documents recognize the fact that function and cognition can continue to decline years after stroke and that patients’ rehabilitation and support needs may therefore change over time after stroke,” Elkind added.

The authors of an accompanying commentary called it a “major step” in the right direction for the future of long-term stroke outcome assessment.

“As we develop new devices, indications, and time windows for stroke treatment, it may perhaps be wise to ensure trials steer away from simpler outcomes to more complex, granular ones,” wrote Yasmin Sadigh, MSc, and Victor Volovici, MD, PhD, with Erasmus University Medical Center, Rotterdam, the Netherlands.

The study had no commercial funding. The authors and commentary writers and Elkind have declared no conflicts of interest.

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

Early recognition of neurodevelopmental concerns and timely access to services have been shown to result in better outcomes for young children. But not all instruments are of equal value, and new research has sought to identify the most useful among them.

For their research, published online in Developmental Medicine & Child Neurology, Andrea Burgess, PhD, of the University of Queensland in Brisbane, Australia, and her colleagues looked at two decades’ worth of systematic reviews of screening, assessment, and diagnostic tools used in children younger than 6 years.

Eighty-six clinical reviews and six practice guidelines, all published between 2000 and 2023, were included in the scoping review, which covered nearly 250 different multi-domain and domain- and disorder-specific tools.

The diagnostic instruments were those used to diagnose the most common early childhood disorders, including intellectual disability, global developmental delay, communication disorders, autism spectrum disorder, attention-deficit/hyperactivity disorder, cerebral palsy, movement disorders, and fetal alcohol spectrum disorder. Burgess and her colleagues sought to determine which tools had the strongest evidence behind them, noting that comparisons were inherently limited by differences in the tested populations, cutoff values, and other factors.

Burgess and her colleagues identified 67 instruments — about a third of those analyzed in the study — “with good discriminative or predictive validity for the screening and assessment of developmental concerns or disability.” Recommended tools were classified by tool type and by patient age groups.

The reason a tool might not be recommended, Burgess said in an email, was for lack of psychometric testing or published evidence, or because the tool was very narrow in scope (eg, covering only a single aspect of a domain), had a small time window for use, or was too new to have been captured in published systematic reviews.
 

Top Recommendations

Among multi-domain assessment tools, the Bayley Scales of Infant and Toddler Development, the Battelle Developmental Inventory, and the Mullen Scales of Early Learning all emerged as highly recommended. The top diagnostic screening tool for autism was the revised version of Social Attention and Communication Surveillance. For cerebral palsy, the top-rated diagnostic assessment tools were Prechtl’s Qualitative Assessment of General Movements and the Hammersmith Infant Neurological Examination.

Ratifying findings by other groups, the researchers determined the Ages & Stages Questionnaires, Third Edition (ASQ-3) to be the best overall multi-domain screening instrument for early childhood development, thanks to its simplicity and ease of use by a wide range of practitioner types. Burgess and her colleagues noted, however, that the ASQ-3 “will not identify all children with developmental concerns and may incorrectly identify others,” and that it may be more accurate in children 2 years or older.
 

Patient Care Setting and Cultural, Socioeconomic Factors Are Key

This news organization spoke to two clinicians working with these and similar tools in the United States. Both said that the care setting can also influence the utility of tools, with cultural and socioeconomic factors playing important roles.

Liz Schwandt, PsyD, an early intervention specialist in Los Angeles, said in an interview that children living in high-risk communities in the United States have a larger burden of developmental delays. But for many families in these communities, accessing care can be complex, which is why well-designed, efficient screening tools like ASQ-3 are especially valuable in practice.

“The reality is you have 10 minutes with a lot of families, and if it’s an emergency, you need to know,” she said. “The ASQ-3 has a very broad age range for this type of instrument and can be used by different practitioner types. The reason it’s successful lies in its parent-centric approach and inherent ease of use. It’s quick, and you can score it using pencil and paper while chatting with the parent, and you can use it for multiple siblings in the space of one appointment.”

With very young children, in whom neurodevelopmental concerns often overlap domains, Schwandt said it can be more important to flag a potential problem early and initiate a nonspecific developmental intervention than wait for results from more precise assessments using more specialized tools. These often require multiple, multi-hour appointments, which can be difficult to attain in lower-resource settings in the United States and can delay care, she said.

Liza Mackintosh, MD, a pediatrician at a federally funded healthcare center in Los Angeles that serves mostly publicly insured families, called validated first-line screening tools “incredibly important.” While rates of developmental screenings in pediatric clinics are increasing, there is still room for improvement, she said.

Mackintosh’s institution does not currently use the ASQ-3 but a different screening tool, called the Survey of Well-Being of Young Children (SWYC), that is embedded into the electronic health record. (The SWYC was not among the tools highlighted in Burgess and colleagues’ review.) Like the ASQ-3, it is short and efficient, she said, and it is used in all children in the recommended age ranges.

“Our visits are on average only 20 minutes,” Mackintosh said. “There’s not enough time for an in-depth developmental assessment. We will flag things such as a speech delay, gross motor delay, or fine motor delay” and refer to early intervention centers for more in-depth developmental assessments as needed, she said.

“The biggest job of pediatricians working in communities that are under-resourced is advocating for those early intervention services,” Mackintosh added. “We really see our job as doing the recommended screening, putting that together with what we’re seeing clinically and on history, and then advocating for the right next step or early intervention. Because sometimes the diagnosis is — I don’t want to say irrelevant, but your treatment plan is still going to be the same. So while I don’t have a formal diagnosis yet, the child definitely needs therapies and we’re still going to get those therapies.”

Burgess and her colleagues stressed in their paper the importance of selecting tools that are culturally appropriate for Indigenous communities in Australia, noting that “inappropriate tools may lead to over- or under-recognition of children with developmental concerns.”

Schwandt and Mackintosh said that the same applies in US settings.

“We’ve done a good job translating screening tools into Chinese, Spanish, Vietnamese, and Russian,” Schwandt said. “But some of them assume a way of taking care of children that is not always shared across cultures. The expectations of how children should play and interact with adults can be very different, and there needs to be an understanding of that. Just putting something in Vietnamese doesn’t mean that there are obvious analogues to understanding what the questionnaire is asking.”

Mackintosh concurred. “A lot of times our patients will not do well on screening, even though they’re fine, because they don’t have the exposure to that activity that’s being asked about. So — is the child scribbling with crayons? Is she climbing up a ladder at a playground? In order to be able to do that, you need to have an environment that you are doing it in. The screeners have to really be appropriate for what the child is exposed to. And sometimes our patients just don’t have that exposure.”

Burgess and colleagues’ study was funded by the Australian government and the Merchant Charitable Foundation. The authors disclosed no financial conflicts of interest. Schwandt and Mackintosh disclosed no conflicts of interest related to their comments.
 

A version of this article appeared on Medscape.com.

Early recognition of neurodevelopmental concerns and timely access to services have been shown to result in better outcomes for young children. But not all instruments are of equal value, and new research has sought to identify the most useful among them.

For their research, published online in Developmental Medicine & Child Neurology, Andrea Burgess, PhD, of the University of Queensland in Brisbane, Australia, and her colleagues looked at two decades’ worth of systematic reviews of screening, assessment, and diagnostic tools used in children younger than 6 years.

Eighty-six clinical reviews and six practice guidelines, all published between 2000 and 2023, were included in the scoping review, which covered nearly 250 different multi-domain and domain- and disorder-specific tools.

The diagnostic instruments were those used to diagnose the most common early childhood disorders, including intellectual disability, global developmental delay, communication disorders, autism spectrum disorder, attention-deficit/hyperactivity disorder, cerebral palsy, movement disorders, and fetal alcohol spectrum disorder. Burgess and her colleagues sought to determine which tools had the strongest evidence behind them, noting that comparisons were inherently limited by differences in the tested populations, cutoff values, and other factors.

Burgess and her colleagues identified 67 instruments — about a third of those analyzed in the study — “with good discriminative or predictive validity for the screening and assessment of developmental concerns or disability.” Recommended tools were classified by tool type and by patient age groups.

The reason a tool might not be recommended, Burgess said in an email, was for lack of psychometric testing or published evidence, or because the tool was very narrow in scope (eg, covering only a single aspect of a domain), had a small time window for use, or was too new to have been captured in published systematic reviews.
 

Top Recommendations

Among multi-domain assessment tools, the Bayley Scales of Infant and Toddler Development, the Battelle Developmental Inventory, and the Mullen Scales of Early Learning all emerged as highly recommended. The top diagnostic screening tool for autism was the revised version of Social Attention and Communication Surveillance. For cerebral palsy, the top-rated diagnostic assessment tools were Prechtl’s Qualitative Assessment of General Movements and the Hammersmith Infant Neurological Examination.

Ratifying findings by other groups, the researchers determined the Ages & Stages Questionnaires, Third Edition (ASQ-3) to be the best overall multi-domain screening instrument for early childhood development, thanks to its simplicity and ease of use by a wide range of practitioner types. Burgess and her colleagues noted, however, that the ASQ-3 “will not identify all children with developmental concerns and may incorrectly identify others,” and that it may be more accurate in children 2 years or older.
 

Patient Care Setting and Cultural, Socioeconomic Factors Are Key

This news organization spoke to two clinicians working with these and similar tools in the United States. Both said that the care setting can also influence the utility of tools, with cultural and socioeconomic factors playing important roles.

Liz Schwandt, PsyD, an early intervention specialist in Los Angeles, said in an interview that children living in high-risk communities in the United States have a larger burden of developmental delays. But for many families in these communities, accessing care can be complex, which is why well-designed, efficient screening tools like ASQ-3 are especially valuable in practice.

“The reality is you have 10 minutes with a lot of families, and if it’s an emergency, you need to know,” she said. “The ASQ-3 has a very broad age range for this type of instrument and can be used by different practitioner types. The reason it’s successful lies in its parent-centric approach and inherent ease of use. It’s quick, and you can score it using pencil and paper while chatting with the parent, and you can use it for multiple siblings in the space of one appointment.”

With very young children, in whom neurodevelopmental concerns often overlap domains, Schwandt said it can be more important to flag a potential problem early and initiate a nonspecific developmental intervention than wait for results from more precise assessments using more specialized tools. These often require multiple, multi-hour appointments, which can be difficult to attain in lower-resource settings in the United States and can delay care, she said.

Liza Mackintosh, MD, a pediatrician at a federally funded healthcare center in Los Angeles that serves mostly publicly insured families, called validated first-line screening tools “incredibly important.” While rates of developmental screenings in pediatric clinics are increasing, there is still room for improvement, she said.

Mackintosh’s institution does not currently use the ASQ-3 but a different screening tool, called the Survey of Well-Being of Young Children (SWYC), that is embedded into the electronic health record. (The SWYC was not among the tools highlighted in Burgess and colleagues’ review.) Like the ASQ-3, it is short and efficient, she said, and it is used in all children in the recommended age ranges.

“Our visits are on average only 20 minutes,” Mackintosh said. “There’s not enough time for an in-depth developmental assessment. We will flag things such as a speech delay, gross motor delay, or fine motor delay” and refer to early intervention centers for more in-depth developmental assessments as needed, she said.

“The biggest job of pediatricians working in communities that are under-resourced is advocating for those early intervention services,” Mackintosh added. “We really see our job as doing the recommended screening, putting that together with what we’re seeing clinically and on history, and then advocating for the right next step or early intervention. Because sometimes the diagnosis is — I don’t want to say irrelevant, but your treatment plan is still going to be the same. So while I don’t have a formal diagnosis yet, the child definitely needs therapies and we’re still going to get those therapies.”

Burgess and her colleagues stressed in their paper the importance of selecting tools that are culturally appropriate for Indigenous communities in Australia, noting that “inappropriate tools may lead to over- or under-recognition of children with developmental concerns.”

Schwandt and Mackintosh said that the same applies in US settings.

“We’ve done a good job translating screening tools into Chinese, Spanish, Vietnamese, and Russian,” Schwandt said. “But some of them assume a way of taking care of children that is not always shared across cultures. The expectations of how children should play and interact with adults can be very different, and there needs to be an understanding of that. Just putting something in Vietnamese doesn’t mean that there are obvious analogues to understanding what the questionnaire is asking.”

Mackintosh concurred. “A lot of times our patients will not do well on screening, even though they’re fine, because they don’t have the exposure to that activity that’s being asked about. So — is the child scribbling with crayons? Is she climbing up a ladder at a playground? In order to be able to do that, you need to have an environment that you are doing it in. The screeners have to really be appropriate for what the child is exposed to. And sometimes our patients just don’t have that exposure.”

Burgess and colleagues’ study was funded by the Australian government and the Merchant Charitable Foundation. The authors disclosed no financial conflicts of interest. Schwandt and Mackintosh disclosed no conflicts of interest related to their comments.
 

A version of this article appeared on Medscape.com.

Early recognition of neurodevelopmental concerns and timely access to services have been shown to result in better outcomes for young children. But not all instruments are of equal value, and new research has sought to identify the most useful among them.

For their research, published online in Developmental Medicine & Child Neurology, Andrea Burgess, PhD, of the University of Queensland in Brisbane, Australia, and her colleagues looked at two decades’ worth of systematic reviews of screening, assessment, and diagnostic tools used in children younger than 6 years.

Eighty-six clinical reviews and six practice guidelines, all published between 2000 and 2023, were included in the scoping review, which covered nearly 250 different multi-domain and domain- and disorder-specific tools.

The diagnostic instruments were those used to diagnose the most common early childhood disorders, including intellectual disability, global developmental delay, communication disorders, autism spectrum disorder, attention-deficit/hyperactivity disorder, cerebral palsy, movement disorders, and fetal alcohol spectrum disorder. Burgess and her colleagues sought to determine which tools had the strongest evidence behind them, noting that comparisons were inherently limited by differences in the tested populations, cutoff values, and other factors.

Burgess and her colleagues identified 67 instruments — about a third of those analyzed in the study — “with good discriminative or predictive validity for the screening and assessment of developmental concerns or disability.” Recommended tools were classified by tool type and by patient age groups.

The reason a tool might not be recommended, Burgess said in an email, was for lack of psychometric testing or published evidence, or because the tool was very narrow in scope (eg, covering only a single aspect of a domain), had a small time window for use, or was too new to have been captured in published systematic reviews.
 

Top Recommendations

Among multi-domain assessment tools, the Bayley Scales of Infant and Toddler Development, the Battelle Developmental Inventory, and the Mullen Scales of Early Learning all emerged as highly recommended. The top diagnostic screening tool for autism was the revised version of Social Attention and Communication Surveillance. For cerebral palsy, the top-rated diagnostic assessment tools were Prechtl’s Qualitative Assessment of General Movements and the Hammersmith Infant Neurological Examination.

Ratifying findings by other groups, the researchers determined the Ages & Stages Questionnaires, Third Edition (ASQ-3) to be the best overall multi-domain screening instrument for early childhood development, thanks to its simplicity and ease of use by a wide range of practitioner types. Burgess and her colleagues noted, however, that the ASQ-3 “will not identify all children with developmental concerns and may incorrectly identify others,” and that it may be more accurate in children 2 years or older.
 

Patient Care Setting and Cultural, Socioeconomic Factors Are Key

This news organization spoke to two clinicians working with these and similar tools in the United States. Both said that the care setting can also influence the utility of tools, with cultural and socioeconomic factors playing important roles.

Liz Schwandt, PsyD, an early intervention specialist in Los Angeles, said in an interview that children living in high-risk communities in the United States have a larger burden of developmental delays. But for many families in these communities, accessing care can be complex, which is why well-designed, efficient screening tools like ASQ-3 are especially valuable in practice.

“The reality is you have 10 minutes with a lot of families, and if it’s an emergency, you need to know,” she said. “The ASQ-3 has a very broad age range for this type of instrument and can be used by different practitioner types. The reason it’s successful lies in its parent-centric approach and inherent ease of use. It’s quick, and you can score it using pencil and paper while chatting with the parent, and you can use it for multiple siblings in the space of one appointment.”

With very young children, in whom neurodevelopmental concerns often overlap domains, Schwandt said it can be more important to flag a potential problem early and initiate a nonspecific developmental intervention than wait for results from more precise assessments using more specialized tools. These often require multiple, multi-hour appointments, which can be difficult to attain in lower-resource settings in the United States and can delay care, she said.

Liza Mackintosh, MD, a pediatrician at a federally funded healthcare center in Los Angeles that serves mostly publicly insured families, called validated first-line screening tools “incredibly important.” While rates of developmental screenings in pediatric clinics are increasing, there is still room for improvement, she said.

Mackintosh’s institution does not currently use the ASQ-3 but a different screening tool, called the Survey of Well-Being of Young Children (SWYC), that is embedded into the electronic health record. (The SWYC was not among the tools highlighted in Burgess and colleagues’ review.) Like the ASQ-3, it is short and efficient, she said, and it is used in all children in the recommended age ranges.

“Our visits are on average only 20 minutes,” Mackintosh said. “There’s not enough time for an in-depth developmental assessment. We will flag things such as a speech delay, gross motor delay, or fine motor delay” and refer to early intervention centers for more in-depth developmental assessments as needed, she said.

“The biggest job of pediatricians working in communities that are under-resourced is advocating for those early intervention services,” Mackintosh added. “We really see our job as doing the recommended screening, putting that together with what we’re seeing clinically and on history, and then advocating for the right next step or early intervention. Because sometimes the diagnosis is — I don’t want to say irrelevant, but your treatment plan is still going to be the same. So while I don’t have a formal diagnosis yet, the child definitely needs therapies and we’re still going to get those therapies.”

Burgess and her colleagues stressed in their paper the importance of selecting tools that are culturally appropriate for Indigenous communities in Australia, noting that “inappropriate tools may lead to over- or under-recognition of children with developmental concerns.”

Schwandt and Mackintosh said that the same applies in US settings.

“We’ve done a good job translating screening tools into Chinese, Spanish, Vietnamese, and Russian,” Schwandt said. “But some of them assume a way of taking care of children that is not always shared across cultures. The expectations of how children should play and interact with adults can be very different, and there needs to be an understanding of that. Just putting something in Vietnamese doesn’t mean that there are obvious analogues to understanding what the questionnaire is asking.”

Mackintosh concurred. “A lot of times our patients will not do well on screening, even though they’re fine, because they don’t have the exposure to that activity that’s being asked about. So — is the child scribbling with crayons? Is she climbing up a ladder at a playground? In order to be able to do that, you need to have an environment that you are doing it in. The screeners have to really be appropriate for what the child is exposed to. And sometimes our patients just don’t have that exposure.”

Burgess and colleagues’ study was funded by the Australian government and the Merchant Charitable Foundation. The authors disclosed no financial conflicts of interest. Schwandt and Mackintosh disclosed no conflicts of interest related to their comments.
 

A version of this article appeared on Medscape.com.

While Kellie Lease Stecher, MD, was working as an ob.gyn. in Minneapolis, Minnesota, a patient confided in her a sexual assault allegation about one of Stecher’s male colleagues. Stecher shared the allegation with her supervisor, who told Stecher not to file a report and chose not to address the issue with the patient. Stecher weighed how to do the right thing: Should she speak up? What were the ethical and legal implications of speaking up vs staying silent?

After seeking advice from her mentors, Stecher felt it was her moral and legal duty to report the allegation to the Minnesota Medical Board. Once she did, her supervisor chastised her repeatedly for reporting the allegation. Stecher soon found herself in a hostile work environment where she was regularly singled out and silenced by her supervisor and colleagues.

“I got to a point where I felt like I couldn’t say anything at any meetings without somehow being targeted after the meeting. There was an individual who was even allowed to fat-shame me with no consequences,” Stecher said. “[Being bullied at work is] a struggle because you have no voice, you have no opportunities, and there’s someone who is intentionally making your life uncomfortable.”

Stecher’s experience is not unusual. Mistreatment is a common issue among healthcare workers, ranging from rudeness to bullying and harassment and permeating every level and specialty of the medical profession. A 2019 research review estimated that 26.3% of healthcare workers had experienced bullying and found bullying in healthcare to be associated with mental health problems such as burnout and depression, physical health problems such as insomnia and headaches, and physicians taking more sick leave.

The Medscape Physician Workplace Culture Report 2024 found similarly bleak results:

• 38% said workplace culture is declining.
• 70% don’t see a big commitment from employers for positive culture.
• 48% said staff isn’t committed to positive culture.

Toxicity’s ripple effects contribute to several issues in healthcare, including staffing shortages, physician attrition, inadequate leadership, and even suicide rates.

The irony, of course, is that most physicians enter the field to care for people. As individuals go from medical school to residency and on with the rest of their careers, they often experience a rude awakening.
 

It’s Everywhere

Noticing the prevalence of workplace bullying in the medical field, endocrinologist Farah Khan, MD, at UW Medicine in Seattle, Washington, decided to conduct a survey on the issue.

Khan collected 122 responses from colleagues, friends, and acquaintances in the field. When asked if they had ever been bullied in medicine, 68% of respondents said yes. But here’s the fascinating part: She tried to pinpoint one particular area or source of toxicity in the progression of a physician’s career — and couldn’t because it existed at all levels.

More than one third of respondents said their worst bullying experiences occurred in residency, while 30% said mistreatment was worst in medical school, and 24% indicated their worst experience had occurred once they became an attending.

The litany of experiences included being belittled, excluded, yelled at, criticized, shamed, unfairly blamed, threatened, sexually harassed, subjected to bigotry and slurs, and humiliated.

“What surprised me the most was how widespread this problem is and the many different layers of healthcare it permeates through, from operating room staff to medical students to hospital HR to residents and attendings,” Khan said of her findings.
 

Who Cares for the Caregivers?

When hematologist Mikkael Sekeres, MD, was in medical school, he seriously considered a career as a surgeon. Following success in his surgical rotations, he scrubbed in with a cardiothoracic surgeon who was well known for both his status as a surgeon and his fiery temper. Sekeres witnessed the surgeon yelling at whoever was nearby: Medical students, fellows, residents, operating room nurses.

“At the end of that experience, any passing thoughts I had of going into cardiothoracic surgery were gone,” Sekeres said. “Some of the people I met in surgery were truly wonderful. Some were unhappy people.”

He has clear ideas why. Mental health struggles that are all too common among physicians can be caused or exacerbated by mistreatment and can also lead a physician to mistreat others.

“People bully when they themselves are hurting,” Sekeres said. “It begs the question, why are people hurting? What’s driving them to be bullies? I think part of the reason is that they’re working really hard and they’re tired, and nobody’s caring for them. It’s hard to care for others when you feel as if you’re hurting more than they are.”

Gail Gazelle, MD, experienced something like this. In her case, the pressure to please and to be a perfect professional and mother affected how she interacted with those around her. While working as a hospice medical director and an academician and clinician at Harvard Medical School, Boston, Massachusetts, she found herself feeling exhausted and burnt out but simultaneously guilty for not doing enough at work or at home.

Guess what happened? She became irritable, lashing out at her son and not putting her best foot forward with coworkers or patients.

After trying traditional therapy and self-help through books and podcasts, Gazelle found her solution in life coaching. “I realized just how harsh I was being on myself and found ways to reverse that pattern,” she said. “I learned ways of regulating myself emotionally that I definitely didn’t learn in my training.”

Today, Gazelle works as a life coach herself, guiding physicians through common challenges of the profession — particularly bullying, which she sees often. She remembers one client, an oncologist, who was being targeted by a nurse practitioner she was training. The nurse practitioner began talking back to the oncologist, as well as gossiping and bad-mouthing her to the nurses in the practice. The nurses then began excluding the oncologist from their cafeteria table at lunchtime, which felt blatant in such a small practice.

A core component of Gazelle’s coaching strategy was helping the client reclaim her self-esteem by focusing on her strengths. She instructed the client to write down what went well that day each night rather than lying in bed ruminating. Such self-care strategies can not only help bullied physicians but also prevent some of the challenges that might cause a physician to bully or lash out at another in the first place.

Such strategies, along with the recent influx of wellness programs available in healthcare facilities, can help physicians cope with the mental health impacts of bullying and the job in general. But even life coaches like Gazelle acknowledge that they are often band-aids on the system’s deeper wounds. Bullying in healthcare is not an individual issue; at its core, it’s an institutional one.
 

Negative Hierarchies in Healthcare

When Stecher’s contract expired, she was fired by the supervisor who had been bullying her. Stecher has since filed a lawsuit, claiming sexual discrimination, defamation, and wrongful termination.

The medical field has a long history of hierarchy, and while this rigidity has softened over time, negative hierarchical dynamics are often perpetuated by leaders. Phenomena like cronyism and cliques and behaviors like petty gossip, lunchroom exclusion (which in the worst cases can mimic high school dynamics), and targeting can be at play in the healthcare workplace.

The classic examples, Stecher said, can usually be spotted: “If you threaten the status quo or offer different ideas, you are seen as a threat. Cronyism ... strict hierarchies ... people who elevate individuals in their social arena into leadership positions. Physicians don’t get the leadership training that they really need; they are often just dumped into roles with no previous experience because they’re someone’s golfing buddy.”

The question is how to get workplace culture momentum moving in a positive direction. When Gazelle’s clients are hesitant to voice concerns, she emphasizes doing so can and should benefit leadership, as well as patients and the wider healthcare system.

“The win-win is that you have a healthy culture of respect and dignity and civility rather than the opposite,” she said. “The leader will actually have more staff retention, which everybody’s concerned about, given the shortage of healthcare workers.”

And that’s a key incentive that may not be discussed as much: Talent drain from toxicity. The Medscape Workplace Culture Report asked about culture as it applies to physicians looking to join up. Notably, 93% of doctors say culture is important when mulling a job offer, 70% said culture is equal to money, and 18% ranked it as more important than money, and 46% say a positive atmosphere is the top priority.

Ultimately, it comes down to who is willing to step in and stand up. Respondents to Khan’s survey counted anonymous reporting systems, more supportive administration teams, and zero-tolerance policies as potential remedies. Gazelle, Sekeres, and Stecher all emphasize the need for zero-tolerance policies for bullying and mistreatment.

“We can’t afford to have things going on like this that just destroy the fabric of the healthcare endeavor,” Gazelle said. “They come out sideways eventually. They come out in terms of poor patient care because there are greater errors. There’s a lack of respect for patients. There’s anger and irritability and so much spillover. We have to have zero-tolerance policies from the top down.”

A version of this article appeared on Medscape.com.

While Kellie Lease Stecher, MD, was working as an ob.gyn. in Minneapolis, Minnesota, a patient confided in her a sexual assault allegation about one of Stecher’s male colleagues. Stecher shared the allegation with her supervisor, who told Stecher not to file a report and chose not to address the issue with the patient. Stecher weighed how to do the right thing: Should she speak up? What were the ethical and legal implications of speaking up vs staying silent?

After seeking advice from her mentors, Stecher felt it was her moral and legal duty to report the allegation to the Minnesota Medical Board. Once she did, her supervisor chastised her repeatedly for reporting the allegation. Stecher soon found herself in a hostile work environment where she was regularly singled out and silenced by her supervisor and colleagues.

“I got to a point where I felt like I couldn’t say anything at any meetings without somehow being targeted after the meeting. There was an individual who was even allowed to fat-shame me with no consequences,” Stecher said. “[Being bullied at work is] a struggle because you have no voice, you have no opportunities, and there’s someone who is intentionally making your life uncomfortable.”

Stecher’s experience is not unusual. Mistreatment is a common issue among healthcare workers, ranging from rudeness to bullying and harassment and permeating every level and specialty of the medical profession. A 2019 research review estimated that 26.3% of healthcare workers had experienced bullying and found bullying in healthcare to be associated with mental health problems such as burnout and depression, physical health problems such as insomnia and headaches, and physicians taking more sick leave.

The Medscape Physician Workplace Culture Report 2024 found similarly bleak results:

• 38% said workplace culture is declining.
• 70% don’t see a big commitment from employers for positive culture.
• 48% said staff isn’t committed to positive culture.

Toxicity’s ripple effects contribute to several issues in healthcare, including staffing shortages, physician attrition, inadequate leadership, and even suicide rates.

The irony, of course, is that most physicians enter the field to care for people. As individuals go from medical school to residency and on with the rest of their careers, they often experience a rude awakening.
 

It’s Everywhere

Noticing the prevalence of workplace bullying in the medical field, endocrinologist Farah Khan, MD, at UW Medicine in Seattle, Washington, decided to conduct a survey on the issue.

Khan collected 122 responses from colleagues, friends, and acquaintances in the field. When asked if they had ever been bullied in medicine, 68% of respondents said yes. But here’s the fascinating part: She tried to pinpoint one particular area or source of toxicity in the progression of a physician’s career — and couldn’t because it existed at all levels.

More than one third of respondents said their worst bullying experiences occurred in residency, while 30% said mistreatment was worst in medical school, and 24% indicated their worst experience had occurred once they became an attending.

The litany of experiences included being belittled, excluded, yelled at, criticized, shamed, unfairly blamed, threatened, sexually harassed, subjected to bigotry and slurs, and humiliated.

“What surprised me the most was how widespread this problem is and the many different layers of healthcare it permeates through, from operating room staff to medical students to hospital HR to residents and attendings,” Khan said of her findings.
 

Who Cares for the Caregivers?

When hematologist Mikkael Sekeres, MD, was in medical school, he seriously considered a career as a surgeon. Following success in his surgical rotations, he scrubbed in with a cardiothoracic surgeon who was well known for both his status as a surgeon and his fiery temper. Sekeres witnessed the surgeon yelling at whoever was nearby: Medical students, fellows, residents, operating room nurses.

“At the end of that experience, any passing thoughts I had of going into cardiothoracic surgery were gone,” Sekeres said. “Some of the people I met in surgery were truly wonderful. Some were unhappy people.”

He has clear ideas why. Mental health struggles that are all too common among physicians can be caused or exacerbated by mistreatment and can also lead a physician to mistreat others.

“People bully when they themselves are hurting,” Sekeres said. “It begs the question, why are people hurting? What’s driving them to be bullies? I think part of the reason is that they’re working really hard and they’re tired, and nobody’s caring for them. It’s hard to care for others when you feel as if you’re hurting more than they are.”

Gail Gazelle, MD, experienced something like this. In her case, the pressure to please and to be a perfect professional and mother affected how she interacted with those around her. While working as a hospice medical director and an academician and clinician at Harvard Medical School, Boston, Massachusetts, she found herself feeling exhausted and burnt out but simultaneously guilty for not doing enough at work or at home.

Guess what happened? She became irritable, lashing out at her son and not putting her best foot forward with coworkers or patients.

After trying traditional therapy and self-help through books and podcasts, Gazelle found her solution in life coaching. “I realized just how harsh I was being on myself and found ways to reverse that pattern,” she said. “I learned ways of regulating myself emotionally that I definitely didn’t learn in my training.”

Today, Gazelle works as a life coach herself, guiding physicians through common challenges of the profession — particularly bullying, which she sees often. She remembers one client, an oncologist, who was being targeted by a nurse practitioner she was training. The nurse practitioner began talking back to the oncologist, as well as gossiping and bad-mouthing her to the nurses in the practice. The nurses then began excluding the oncologist from their cafeteria table at lunchtime, which felt blatant in such a small practice.

A core component of Gazelle’s coaching strategy was helping the client reclaim her self-esteem by focusing on her strengths. She instructed the client to write down what went well that day each night rather than lying in bed ruminating. Such self-care strategies can not only help bullied physicians but also prevent some of the challenges that might cause a physician to bully or lash out at another in the first place.

Such strategies, along with the recent influx of wellness programs available in healthcare facilities, can help physicians cope with the mental health impacts of bullying and the job in general. But even life coaches like Gazelle acknowledge that they are often band-aids on the system’s deeper wounds. Bullying in healthcare is not an individual issue; at its core, it’s an institutional one.
 

Negative Hierarchies in Healthcare

When Stecher’s contract expired, she was fired by the supervisor who had been bullying her. Stecher has since filed a lawsuit, claiming sexual discrimination, defamation, and wrongful termination.

The medical field has a long history of hierarchy, and while this rigidity has softened over time, negative hierarchical dynamics are often perpetuated by leaders. Phenomena like cronyism and cliques and behaviors like petty gossip, lunchroom exclusion (which in the worst cases can mimic high school dynamics), and targeting can be at play in the healthcare workplace.

The classic examples, Stecher said, can usually be spotted: “If you threaten the status quo or offer different ideas, you are seen as a threat. Cronyism ... strict hierarchies ... people who elevate individuals in their social arena into leadership positions. Physicians don’t get the leadership training that they really need; they are often just dumped into roles with no previous experience because they’re someone’s golfing buddy.”

The question is how to get workplace culture momentum moving in a positive direction. When Gazelle’s clients are hesitant to voice concerns, she emphasizes doing so can and should benefit leadership, as well as patients and the wider healthcare system.

“The win-win is that you have a healthy culture of respect and dignity and civility rather than the opposite,” she said. “The leader will actually have more staff retention, which everybody’s concerned about, given the shortage of healthcare workers.”

And that’s a key incentive that may not be discussed as much: Talent drain from toxicity. The Medscape Workplace Culture Report asked about culture as it applies to physicians looking to join up. Notably, 93% of doctors say culture is important when mulling a job offer, 70% said culture is equal to money, and 18% ranked it as more important than money, and 46% say a positive atmosphere is the top priority.

Ultimately, it comes down to who is willing to step in and stand up. Respondents to Khan’s survey counted anonymous reporting systems, more supportive administration teams, and zero-tolerance policies as potential remedies. Gazelle, Sekeres, and Stecher all emphasize the need for zero-tolerance policies for bullying and mistreatment.

“We can’t afford to have things going on like this that just destroy the fabric of the healthcare endeavor,” Gazelle said. “They come out sideways eventually. They come out in terms of poor patient care because there are greater errors. There’s a lack of respect for patients. There’s anger and irritability and so much spillover. We have to have zero-tolerance policies from the top down.”

A version of this article appeared on Medscape.com.

While Kellie Lease Stecher, MD, was working as an ob.gyn. in Minneapolis, Minnesota, a patient confided in her a sexual assault allegation about one of Stecher’s male colleagues. Stecher shared the allegation with her supervisor, who told Stecher not to file a report and chose not to address the issue with the patient. Stecher weighed how to do the right thing: Should she speak up? What were the ethical and legal implications of speaking up vs staying silent?

After seeking advice from her mentors, Stecher felt it was her moral and legal duty to report the allegation to the Minnesota Medical Board. Once she did, her supervisor chastised her repeatedly for reporting the allegation. Stecher soon found herself in a hostile work environment where she was regularly singled out and silenced by her supervisor and colleagues.

“I got to a point where I felt like I couldn’t say anything at any meetings without somehow being targeted after the meeting. There was an individual who was even allowed to fat-shame me with no consequences,” Stecher said. “[Being bullied at work is] a struggle because you have no voice, you have no opportunities, and there’s someone who is intentionally making your life uncomfortable.”

Stecher’s experience is not unusual. Mistreatment is a common issue among healthcare workers, ranging from rudeness to bullying and harassment and permeating every level and specialty of the medical profession. A 2019 research review estimated that 26.3% of healthcare workers had experienced bullying and found bullying in healthcare to be associated with mental health problems such as burnout and depression, physical health problems such as insomnia and headaches, and physicians taking more sick leave.

The Medscape Physician Workplace Culture Report 2024 found similarly bleak results:

• 38% said workplace culture is declining.
• 70% don’t see a big commitment from employers for positive culture.
• 48% said staff isn’t committed to positive culture.

Toxicity’s ripple effects contribute to several issues in healthcare, including staffing shortages, physician attrition, inadequate leadership, and even suicide rates.

The irony, of course, is that most physicians enter the field to care for people. As individuals go from medical school to residency and on with the rest of their careers, they often experience a rude awakening.
 

It’s Everywhere

Noticing the prevalence of workplace bullying in the medical field, endocrinologist Farah Khan, MD, at UW Medicine in Seattle, Washington, decided to conduct a survey on the issue.

Khan collected 122 responses from colleagues, friends, and acquaintances in the field. When asked if they had ever been bullied in medicine, 68% of respondents said yes. But here’s the fascinating part: She tried to pinpoint one particular area or source of toxicity in the progression of a physician’s career — and couldn’t because it existed at all levels.

More than one third of respondents said their worst bullying experiences occurred in residency, while 30% said mistreatment was worst in medical school, and 24% indicated their worst experience had occurred once they became an attending.

The litany of experiences included being belittled, excluded, yelled at, criticized, shamed, unfairly blamed, threatened, sexually harassed, subjected to bigotry and slurs, and humiliated.

“What surprised me the most was how widespread this problem is and the many different layers of healthcare it permeates through, from operating room staff to medical students to hospital HR to residents and attendings,” Khan said of her findings.
 

Who Cares for the Caregivers?

When hematologist Mikkael Sekeres, MD, was in medical school, he seriously considered a career as a surgeon. Following success in his surgical rotations, he scrubbed in with a cardiothoracic surgeon who was well known for both his status as a surgeon and his fiery temper. Sekeres witnessed the surgeon yelling at whoever was nearby: Medical students, fellows, residents, operating room nurses.

“At the end of that experience, any passing thoughts I had of going into cardiothoracic surgery were gone,” Sekeres said. “Some of the people I met in surgery were truly wonderful. Some were unhappy people.”

He has clear ideas why. Mental health struggles that are all too common among physicians can be caused or exacerbated by mistreatment and can also lead a physician to mistreat others.

“People bully when they themselves are hurting,” Sekeres said. “It begs the question, why are people hurting? What’s driving them to be bullies? I think part of the reason is that they’re working really hard and they’re tired, and nobody’s caring for them. It’s hard to care for others when you feel as if you’re hurting more than they are.”

Gail Gazelle, MD, experienced something like this. In her case, the pressure to please and to be a perfect professional and mother affected how she interacted with those around her. While working as a hospice medical director and an academician and clinician at Harvard Medical School, Boston, Massachusetts, she found herself feeling exhausted and burnt out but simultaneously guilty for not doing enough at work or at home.

Guess what happened? She became irritable, lashing out at her son and not putting her best foot forward with coworkers or patients.

After trying traditional therapy and self-help through books and podcasts, Gazelle found her solution in life coaching. “I realized just how harsh I was being on myself and found ways to reverse that pattern,” she said. “I learned ways of regulating myself emotionally that I definitely didn’t learn in my training.”

Today, Gazelle works as a life coach herself, guiding physicians through common challenges of the profession — particularly bullying, which she sees often. She remembers one client, an oncologist, who was being targeted by a nurse practitioner she was training. The nurse practitioner began talking back to the oncologist, as well as gossiping and bad-mouthing her to the nurses in the practice. The nurses then began excluding the oncologist from their cafeteria table at lunchtime, which felt blatant in such a small practice.

A core component of Gazelle’s coaching strategy was helping the client reclaim her self-esteem by focusing on her strengths. She instructed the client to write down what went well that day each night rather than lying in bed ruminating. Such self-care strategies can not only help bullied physicians but also prevent some of the challenges that might cause a physician to bully or lash out at another in the first place.

Such strategies, along with the recent influx of wellness programs available in healthcare facilities, can help physicians cope with the mental health impacts of bullying and the job in general. But even life coaches like Gazelle acknowledge that they are often band-aids on the system’s deeper wounds. Bullying in healthcare is not an individual issue; at its core, it’s an institutional one.
 

Negative Hierarchies in Healthcare

When Stecher’s contract expired, she was fired by the supervisor who had been bullying her. Stecher has since filed a lawsuit, claiming sexual discrimination, defamation, and wrongful termination.

The medical field has a long history of hierarchy, and while this rigidity has softened over time, negative hierarchical dynamics are often perpetuated by leaders. Phenomena like cronyism and cliques and behaviors like petty gossip, lunchroom exclusion (which in the worst cases can mimic high school dynamics), and targeting can be at play in the healthcare workplace.

The classic examples, Stecher said, can usually be spotted: “If you threaten the status quo or offer different ideas, you are seen as a threat. Cronyism ... strict hierarchies ... people who elevate individuals in their social arena into leadership positions. Physicians don’t get the leadership training that they really need; they are often just dumped into roles with no previous experience because they’re someone’s golfing buddy.”

The question is how to get workplace culture momentum moving in a positive direction. When Gazelle’s clients are hesitant to voice concerns, she emphasizes doing so can and should benefit leadership, as well as patients and the wider healthcare system.

“The win-win is that you have a healthy culture of respect and dignity and civility rather than the opposite,” she said. “The leader will actually have more staff retention, which everybody’s concerned about, given the shortage of healthcare workers.”

And that’s a key incentive that may not be discussed as much: Talent drain from toxicity. The Medscape Workplace Culture Report asked about culture as it applies to physicians looking to join up. Notably, 93% of doctors say culture is important when mulling a job offer, 70% said culture is equal to money, and 18% ranked it as more important than money, and 46% say a positive atmosphere is the top priority.

Ultimately, it comes down to who is willing to step in and stand up. Respondents to Khan’s survey counted anonymous reporting systems, more supportive administration teams, and zero-tolerance policies as potential remedies. Gazelle, Sekeres, and Stecher all emphasize the need for zero-tolerance policies for bullying and mistreatment.

“We can’t afford to have things going on like this that just destroy the fabric of the healthcare endeavor,” Gazelle said. “They come out sideways eventually. They come out in terms of poor patient care because there are greater errors. There’s a lack of respect for patients. There’s anger and irritability and so much spillover. We have to have zero-tolerance policies from the top down.”

A version of this article appeared on Medscape.com.

A group of nurse practitioners (NPs) employed by the state of New York has sued the state, alleging that their employer has them doing the work of physicians but underpays them.

The New York State Civil Service Commission understates the job function of NPs, overstates their dependence on physicians, and inadequately pays them for their work, according to the complaint filed in the US District Court for the Northern District of New York.

The nurses claim the mistreatment is a consequence of the fact that “at least 80% of the state’s employed NPs are women.”

Michael H. Sussman, a Goshen, New York–based attorney for the nurses, said in an interview that New York NPs are increasingly being used essentially as doctors at state-run facilities, including prisons, yet the state has failed to adequately pay them.

The lawsuit comes after a decade-long attempt by NPs to attain equitable pay and the ability to advance their civil service careers, he said.

“New York state has not addressed the heart of the issue, which is that the classification of this position is much lower than other positions in the state which are not so female-dominated and which engage in very similar activities,” Sussman said.

The lawsuit claims that “the work of NPs is complex, equaling that of a medical specialist, psychiatrist, or clinical physician.”

A spokesman for the New York State Civil Service Commission declined comment, saying the department does not comment on pending litigation.
 

Novel Gender Discrimination Argument

Gender discrimination is a relatively new argument avenue in the larger equal work, equal pay debate, said Joanne Spetz, PhD, director of the Institute for Health Policy Studies at the University of California, San Francisco.

“This is the first time I’ve heard of [such] a case being really gender discrimination focused,” she said in an interview. “On one level, I think it’s groundbreaking as a legal approach, but it’s also limited because it’s focused on public, state employees.”

Spetz noted that New York has significantly expanded NPs’ scope of practice, enacting in 2022 legislation that granted NPs full practice authority. The law means NPs can evaluate, order, diagnose, manage treatments, and prescribe medications for patients without physician supervision.

“They are in a role where they are stepping back and saying, ‘Wait, why are [we] not receiving equal pay for equal work?’ ” Spetz said. “It’s a totally fair area for debate, especially because they are now authorized to do essentially equal work with a high degree of autonomy.”
 

Debate Over Pay Grade

The nurses’ complaint centers on the New York State Civil Service Commission’s classification for NPs, which hasn’t changed since 2006. NPs are classified at grade 24, and they have no possibility of internal advancement associated with their title, according to the legal complaint filed on September 17.

To comply with a state legislative directive, the commission in 2018 conducted a study of the NP classification but recommended against reclassification or implementing a career ladder. The study noted the subordinate role of NPs to physicians and the substantial difference between physician classification (entry at grade 34) and that of NPs, psychologists (grade 25), and pharmacists (grade 25).

The study concluded that higher classified positions have higher levels of educational attainment and licensure requirements and no supervision or collaboration requirements, according to the complaint.

At the time, groups such as the Nurse Practitioner Association and the Public Employees Federation (PEF) criticized the findings, but the commission stuck to its classification.

Following the NP Modernization Act that allowed NPs to practice independently, PEF sought an increase for NPs to grade 28 with a progression to grade 34 depending on experience.

“But to this date, despite altering the starting salaries of NPs, defendants have failed and refused to alter the compensation offered to the substantial majority of NPs, and each plaintiff remains cabined in a grade 24 with a discriminatorily low salary when compared with males in other job classifications doing highly similar functions,” the lawsuit contended.

Six plaintiffs are named in the lawsuit, all of whom are women and work for state agencies. Plaintiff Rachel Burns, for instance, works as a psychiatric mental health NP in West Seneca and is responsible for performing psychiatric evaluations for patients, diagnosis, prescribing medication, ordering labs, and determining risks. The evaluations are identical for a psychiatrist and require her to complete the same forms, according to the suit.

Another plaintiff, Amber Hawthorne Lashway, works at a correctional facility in Altona, where for many years she was the sole medical provider, according to the lawsuit. Lashway’s duties, which include diagnoses and treatment of inmates’ medical conditions, mirror those performed by clinical physicians, the suit stated.

The plaintiffs are requesting the court accept jurisdiction of the matter and certify the class they seek to represent. They are also demanding prospective pay equity and compensatory damages for the distress caused by “the long-standing discriminatory” treatment by the state.

The Civil Service Commission and state of New York have not yet responded to the complaint. Their responses are due on November 12.
 

Attorney: Case Impact Limited

Benjamin McMichael, PhD, JD, said the New York case is not surprising as more states across the country are granting nurses more practice autonomy. The current landscape tends to favor the nurses, he said, with about half of states now allowing NPs full practice authority.

“I think the [New York] NPs are correct that they are underpaid,” said McMichael, an associate professor of law and director of the Interdisciplinary Legal Studies Initiative at The University of Alabama in Tuscaloosa. “With that said, the nature of the case does not clearly lend itself to national change.”

The fact that the NP plaintiffs are employed by the state means they are using a specific set of laws to advance their cause, he said. Other NPs in other employment situations may not have access to the same laws.
 

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

A group of nurse practitioners (NPs) employed by the state of New York has sued the state, alleging that their employer has them doing the work of physicians but underpays them.

The New York State Civil Service Commission understates the job function of NPs, overstates their dependence on physicians, and inadequately pays them for their work, according to the complaint filed in the US District Court for the Northern District of New York.

The nurses claim the mistreatment is a consequence of the fact that “at least 80% of the state’s employed NPs are women.”

Michael H. Sussman, a Goshen, New York–based attorney for the nurses, said in an interview that New York NPs are increasingly being used essentially as doctors at state-run facilities, including prisons, yet the state has failed to adequately pay them.

The lawsuit comes after a decade-long attempt by NPs to attain equitable pay and the ability to advance their civil service careers, he said.

“New York state has not addressed the heart of the issue, which is that the classification of this position is much lower than other positions in the state which are not so female-dominated and which engage in very similar activities,” Sussman said.

The lawsuit claims that “the work of NPs is complex, equaling that of a medical specialist, psychiatrist, or clinical physician.”

A spokesman for the New York State Civil Service Commission declined comment, saying the department does not comment on pending litigation.
 

Novel Gender Discrimination Argument

Gender discrimination is a relatively new argument avenue in the larger equal work, equal pay debate, said Joanne Spetz, PhD, director of the Institute for Health Policy Studies at the University of California, San Francisco.

“This is the first time I’ve heard of [such] a case being really gender discrimination focused,” she said in an interview. “On one level, I think it’s groundbreaking as a legal approach, but it’s also limited because it’s focused on public, state employees.”

Spetz noted that New York has significantly expanded NPs’ scope of practice, enacting in 2022 legislation that granted NPs full practice authority. The law means NPs can evaluate, order, diagnose, manage treatments, and prescribe medications for patients without physician supervision.

“They are in a role where they are stepping back and saying, ‘Wait, why are [we] not receiving equal pay for equal work?’ ” Spetz said. “It’s a totally fair area for debate, especially because they are now authorized to do essentially equal work with a high degree of autonomy.”
 

Debate Over Pay Grade

The nurses’ complaint centers on the New York State Civil Service Commission’s classification for NPs, which hasn’t changed since 2006. NPs are classified at grade 24, and they have no possibility of internal advancement associated with their title, according to the legal complaint filed on September 17.

To comply with a state legislative directive, the commission in 2018 conducted a study of the NP classification but recommended against reclassification or implementing a career ladder. The study noted the subordinate role of NPs to physicians and the substantial difference between physician classification (entry at grade 34) and that of NPs, psychologists (grade 25), and pharmacists (grade 25).

The study concluded that higher classified positions have higher levels of educational attainment and licensure requirements and no supervision or collaboration requirements, according to the complaint.

At the time, groups such as the Nurse Practitioner Association and the Public Employees Federation (PEF) criticized the findings, but the commission stuck to its classification.

Following the NP Modernization Act that allowed NPs to practice independently, PEF sought an increase for NPs to grade 28 with a progression to grade 34 depending on experience.

“But to this date, despite altering the starting salaries of NPs, defendants have failed and refused to alter the compensation offered to the substantial majority of NPs, and each plaintiff remains cabined in a grade 24 with a discriminatorily low salary when compared with males in other job classifications doing highly similar functions,” the lawsuit contended.

Six plaintiffs are named in the lawsuit, all of whom are women and work for state agencies. Plaintiff Rachel Burns, for instance, works as a psychiatric mental health NP in West Seneca and is responsible for performing psychiatric evaluations for patients, diagnosis, prescribing medication, ordering labs, and determining risks. The evaluations are identical for a psychiatrist and require her to complete the same forms, according to the suit.

Another plaintiff, Amber Hawthorne Lashway, works at a correctional facility in Altona, where for many years she was the sole medical provider, according to the lawsuit. Lashway’s duties, which include diagnoses and treatment of inmates’ medical conditions, mirror those performed by clinical physicians, the suit stated.

The plaintiffs are requesting the court accept jurisdiction of the matter and certify the class they seek to represent. They are also demanding prospective pay equity and compensatory damages for the distress caused by “the long-standing discriminatory” treatment by the state.

The Civil Service Commission and state of New York have not yet responded to the complaint. Their responses are due on November 12.
 

Attorney: Case Impact Limited

Benjamin McMichael, PhD, JD, said the New York case is not surprising as more states across the country are granting nurses more practice autonomy. The current landscape tends to favor the nurses, he said, with about half of states now allowing NPs full practice authority.

“I think the [New York] NPs are correct that they are underpaid,” said McMichael, an associate professor of law and director of the Interdisciplinary Legal Studies Initiative at The University of Alabama in Tuscaloosa. “With that said, the nature of the case does not clearly lend itself to national change.”

The fact that the NP plaintiffs are employed by the state means they are using a specific set of laws to advance their cause, he said. Other NPs in other employment situations may not have access to the same laws.
 

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

A group of nurse practitioners (NPs) employed by the state of New York has sued the state, alleging that their employer has them doing the work of physicians but underpays them.

The New York State Civil Service Commission understates the job function of NPs, overstates their dependence on physicians, and inadequately pays them for their work, according to the complaint filed in the US District Court for the Northern District of New York.

The nurses claim the mistreatment is a consequence of the fact that “at least 80% of the state’s employed NPs are women.”

Michael H. Sussman, a Goshen, New York–based attorney for the nurses, said in an interview that New York NPs are increasingly being used essentially as doctors at state-run facilities, including prisons, yet the state has failed to adequately pay them.

The lawsuit comes after a decade-long attempt by NPs to attain equitable pay and the ability to advance their civil service careers, he said.

“New York state has not addressed the heart of the issue, which is that the classification of this position is much lower than other positions in the state which are not so female-dominated and which engage in very similar activities,” Sussman said.

The lawsuit claims that “the work of NPs is complex, equaling that of a medical specialist, psychiatrist, or clinical physician.”

A spokesman for the New York State Civil Service Commission declined comment, saying the department does not comment on pending litigation.
 

Novel Gender Discrimination Argument

Gender discrimination is a relatively new argument avenue in the larger equal work, equal pay debate, said Joanne Spetz, PhD, director of the Institute for Health Policy Studies at the University of California, San Francisco.

“This is the first time I’ve heard of [such] a case being really gender discrimination focused,” she said in an interview. “On one level, I think it’s groundbreaking as a legal approach, but it’s also limited because it’s focused on public, state employees.”

Spetz noted that New York has significantly expanded NPs’ scope of practice, enacting in 2022 legislation that granted NPs full practice authority. The law means NPs can evaluate, order, diagnose, manage treatments, and prescribe medications for patients without physician supervision.

“They are in a role where they are stepping back and saying, ‘Wait, why are [we] not receiving equal pay for equal work?’ ” Spetz said. “It’s a totally fair area for debate, especially because they are now authorized to do essentially equal work with a high degree of autonomy.”
 

Debate Over Pay Grade

The nurses’ complaint centers on the New York State Civil Service Commission’s classification for NPs, which hasn’t changed since 2006. NPs are classified at grade 24, and they have no possibility of internal advancement associated with their title, according to the legal complaint filed on September 17.

To comply with a state legislative directive, the commission in 2018 conducted a study of the NP classification but recommended against reclassification or implementing a career ladder. The study noted the subordinate role of NPs to physicians and the substantial difference between physician classification (entry at grade 34) and that of NPs, psychologists (grade 25), and pharmacists (grade 25).

The study concluded that higher classified positions have higher levels of educational attainment and licensure requirements and no supervision or collaboration requirements, according to the complaint.

At the time, groups such as the Nurse Practitioner Association and the Public Employees Federation (PEF) criticized the findings, but the commission stuck to its classification.

Following the NP Modernization Act that allowed NPs to practice independently, PEF sought an increase for NPs to grade 28 with a progression to grade 34 depending on experience.

“But to this date, despite altering the starting salaries of NPs, defendants have failed and refused to alter the compensation offered to the substantial majority of NPs, and each plaintiff remains cabined in a grade 24 with a discriminatorily low salary when compared with males in other job classifications doing highly similar functions,” the lawsuit contended.

Six plaintiffs are named in the lawsuit, all of whom are women and work for state agencies. Plaintiff Rachel Burns, for instance, works as a psychiatric mental health NP in West Seneca and is responsible for performing psychiatric evaluations for patients, diagnosis, prescribing medication, ordering labs, and determining risks. The evaluations are identical for a psychiatrist and require her to complete the same forms, according to the suit.

Another plaintiff, Amber Hawthorne Lashway, works at a correctional facility in Altona, where for many years she was the sole medical provider, according to the lawsuit. Lashway’s duties, which include diagnoses and treatment of inmates’ medical conditions, mirror those performed by clinical physicians, the suit stated.

The plaintiffs are requesting the court accept jurisdiction of the matter and certify the class they seek to represent. They are also demanding prospective pay equity and compensatory damages for the distress caused by “the long-standing discriminatory” treatment by the state.

The Civil Service Commission and state of New York have not yet responded to the complaint. Their responses are due on November 12.
 

Attorney: Case Impact Limited

Benjamin McMichael, PhD, JD, said the New York case is not surprising as more states across the country are granting nurses more practice autonomy. The current landscape tends to favor the nurses, he said, with about half of states now allowing NPs full practice authority.

“I think the [New York] NPs are correct that they are underpaid,” said McMichael, an associate professor of law and director of the Interdisciplinary Legal Studies Initiative at The University of Alabama in Tuscaloosa. “With that said, the nature of the case does not clearly lend itself to national change.”

The fact that the NP plaintiffs are employed by the state means they are using a specific set of laws to advance their cause, he said. Other NPs in other employment situations may not have access to the same laws.
 

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

Getting drugs to the brain is difficult. The very thing designed to protect the brain’s environment — the blood-brain barrier (BBB) — is one of the main reasons diseases like Alzheimer’s are so hard to treat.

And even if a drug can cross the BBB, it’s difficult to ensure it reaches specific areas of the brain like the hippocampus, which is located deep within the brain and notoriously difficult to target with conventional drugs.

However, new research shows that novel bioengineered proteins can target neurons in the hippocampus. Using a mouse model, the researchers found that these proteins could be delivered to the hippocampus intranasally — through the nose via a spray.

“This is an urgent topic because many potential therapeutic agents do not readily cross the blood-brain barrier or have limited effects even after intranasal delivery,” said Konrad Talbot, PhD, professor of neurosurgery and pathology at Loma Linda University, Loma Linda, California, who was not involved in the study.

This is the first time a protein drug, which is larger than many drug molecules, has been specifically delivered to the hippocampus, said Noriyasu Kamei, PhD, a professor of pharmaceutical science at Kobe Gakuin University in Kobe, Japan, and lead author of the study.
 

How Did They Do It?

“Smuggle” may be a flip term, but it’s not inaccurate.

Insulin has the ability to cross the BBB, so the team began with insulin as the vehicle. By attaching other molecules to an insulin fragment, researchers theorized they could create an insulin fusion protein that can be transported across the BBB and into the brain via a process called macropinocytosis.

They executed this technique in mice by fusing florescent proteins to insulin. To treat Alzheimer’s or other diseases, they would want to fuse therapeutic molecules to the insulin for brain delivery — a future step for their research.

Other groups are studying a similar approach using transferrin receptor instead of insulin to shuttle molecules across the BBB. However, the transferrin receptor doesn’t make it to the hippocampus, Kamei said.

A benefit of their system, Kamei pointed out, is that because the method just requires a small piece of insulin to work, it’s straightforward to produce in bacteria. Importantly, he said, the insulin fusion protein should not affect blood glucose levels.
 

Why Insulin?

Aside from its ability to cross the BBB, the team thought to use insulin as the basis of a fusion protein because of their previous work.

“I found that insulin has the unique characteristics to be accumulated specifically in the hippocampal neuronal layers,” Kamei explained. That potential for accumulation is key, as they can deliver more of a drug that way.

In their past work, Kamei and colleagues also found that it could be delivered from the nose to the brain, indicating that it may be possible to use a simple nasal spray.

“The potential for noninvasive delivery of proteins by intranasal administration to the hippocampal neurons is novel,” said John Varghese, PhD, professor of neurology at University of California Los Angeles (he was not involved in the study). He noted that it’s also possible that this method could be harnessed to treat other brain diseases.

There are other drugs that treat central nervous system diseases, such as desmopressin and buserelin, which are available as nasal sprays. However, these drugs are synthetic hormones, and though relatively small molecules, they do not cross the BBB.

There are also antibody treatments for Alzheimer’s, such as aducanumab (which will soon be discontinued), lecanemab, and donanemab; however, they aren’t always effective and they require an intravenous infusion, and while they cross the BBB to a degree, to bolster delivery to the brain, studies have proposed additional methods like focused ultrasound.

“Neuronal uptake of drugs potentially therapeutic for Alzheimer’s may be significantly enhanced by fusion of those drugs with insulin. This should be a research priority,” said Talbot.

While this is exciting and has potential, such drugs won’t be available anytime soon. Kamei would like to complete the research at a basic level in 5 years, including testing insulin fused with larger proteins such as therapeutic antibodies. If all goes well, they’ll move on to testing insulin fusion drugs in people.
 

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

Getting drugs to the brain is difficult. The very thing designed to protect the brain’s environment — the blood-brain barrier (BBB) — is one of the main reasons diseases like Alzheimer’s are so hard to treat.

And even if a drug can cross the BBB, it’s difficult to ensure it reaches specific areas of the brain like the hippocampus, which is located deep within the brain and notoriously difficult to target with conventional drugs.

However, new research shows that novel bioengineered proteins can target neurons in the hippocampus. Using a mouse model, the researchers found that these proteins could be delivered to the hippocampus intranasally — through the nose via a spray.

“This is an urgent topic because many potential therapeutic agents do not readily cross the blood-brain barrier or have limited effects even after intranasal delivery,” said Konrad Talbot, PhD, professor of neurosurgery and pathology at Loma Linda University, Loma Linda, California, who was not involved in the study.

This is the first time a protein drug, which is larger than many drug molecules, has been specifically delivered to the hippocampus, said Noriyasu Kamei, PhD, a professor of pharmaceutical science at Kobe Gakuin University in Kobe, Japan, and lead author of the study.
 

How Did They Do It?

“Smuggle” may be a flip term, but it’s not inaccurate.

Insulin has the ability to cross the BBB, so the team began with insulin as the vehicle. By attaching other molecules to an insulin fragment, researchers theorized they could create an insulin fusion protein that can be transported across the BBB and into the brain via a process called macropinocytosis.

They executed this technique in mice by fusing florescent proteins to insulin. To treat Alzheimer’s or other diseases, they would want to fuse therapeutic molecules to the insulin for brain delivery — a future step for their research.

Other groups are studying a similar approach using transferrin receptor instead of insulin to shuttle molecules across the BBB. However, the transferrin receptor doesn’t make it to the hippocampus, Kamei said.

A benefit of their system, Kamei pointed out, is that because the method just requires a small piece of insulin to work, it’s straightforward to produce in bacteria. Importantly, he said, the insulin fusion protein should not affect blood glucose levels.
 

Why Insulin?

Aside from its ability to cross the BBB, the team thought to use insulin as the basis of a fusion protein because of their previous work.

“I found that insulin has the unique characteristics to be accumulated specifically in the hippocampal neuronal layers,” Kamei explained. That potential for accumulation is key, as they can deliver more of a drug that way.

In their past work, Kamei and colleagues also found that it could be delivered from the nose to the brain, indicating that it may be possible to use a simple nasal spray.

“The potential for noninvasive delivery of proteins by intranasal administration to the hippocampal neurons is novel,” said John Varghese, PhD, professor of neurology at University of California Los Angeles (he was not involved in the study). He noted that it’s also possible that this method could be harnessed to treat other brain diseases.

There are other drugs that treat central nervous system diseases, such as desmopressin and buserelin, which are available as nasal sprays. However, these drugs are synthetic hormones, and though relatively small molecules, they do not cross the BBB.

There are also antibody treatments for Alzheimer’s, such as aducanumab (which will soon be discontinued), lecanemab, and donanemab; however, they aren’t always effective and they require an intravenous infusion, and while they cross the BBB to a degree, to bolster delivery to the brain, studies have proposed additional methods like focused ultrasound.

“Neuronal uptake of drugs potentially therapeutic for Alzheimer’s may be significantly enhanced by fusion of those drugs with insulin. This should be a research priority,” said Talbot.

While this is exciting and has potential, such drugs won’t be available anytime soon. Kamei would like to complete the research at a basic level in 5 years, including testing insulin fused with larger proteins such as therapeutic antibodies. If all goes well, they’ll move on to testing insulin fusion drugs in people.
 

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

Getting drugs to the brain is difficult. The very thing designed to protect the brain’s environment — the blood-brain barrier (BBB) — is one of the main reasons diseases like Alzheimer’s are so hard to treat.

And even if a drug can cross the BBB, it’s difficult to ensure it reaches specific areas of the brain like the hippocampus, which is located deep within the brain and notoriously difficult to target with conventional drugs.

However, new research shows that novel bioengineered proteins can target neurons in the hippocampus. Using a mouse model, the researchers found that these proteins could be delivered to the hippocampus intranasally — through the nose via a spray.

“This is an urgent topic because many potential therapeutic agents do not readily cross the blood-brain barrier or have limited effects even after intranasal delivery,” said Konrad Talbot, PhD, professor of neurosurgery and pathology at Loma Linda University, Loma Linda, California, who was not involved in the study.

This is the first time a protein drug, which is larger than many drug molecules, has been specifically delivered to the hippocampus, said Noriyasu Kamei, PhD, a professor of pharmaceutical science at Kobe Gakuin University in Kobe, Japan, and lead author of the study.
 

How Did They Do It?

“Smuggle” may be a flip term, but it’s not inaccurate.

Insulin has the ability to cross the BBB, so the team began with insulin as the vehicle. By attaching other molecules to an insulin fragment, researchers theorized they could create an insulin fusion protein that can be transported across the BBB and into the brain via a process called macropinocytosis.

They executed this technique in mice by fusing florescent proteins to insulin. To treat Alzheimer’s or other diseases, they would want to fuse therapeutic molecules to the insulin for brain delivery — a future step for their research.

Other groups are studying a similar approach using transferrin receptor instead of insulin to shuttle molecules across the BBB. However, the transferrin receptor doesn’t make it to the hippocampus, Kamei said.

A benefit of their system, Kamei pointed out, is that because the method just requires a small piece of insulin to work, it’s straightforward to produce in bacteria. Importantly, he said, the insulin fusion protein should not affect blood glucose levels.
 

Why Insulin?

Aside from its ability to cross the BBB, the team thought to use insulin as the basis of a fusion protein because of their previous work.

“I found that insulin has the unique characteristics to be accumulated specifically in the hippocampal neuronal layers,” Kamei explained. That potential for accumulation is key, as they can deliver more of a drug that way.

In their past work, Kamei and colleagues also found that it could be delivered from the nose to the brain, indicating that it may be possible to use a simple nasal spray.

“The potential for noninvasive delivery of proteins by intranasal administration to the hippocampal neurons is novel,” said John Varghese, PhD, professor of neurology at University of California Los Angeles (he was not involved in the study). He noted that it’s also possible that this method could be harnessed to treat other brain diseases.

There are other drugs that treat central nervous system diseases, such as desmopressin and buserelin, which are available as nasal sprays. However, these drugs are synthetic hormones, and though relatively small molecules, they do not cross the BBB.

There are also antibody treatments for Alzheimer’s, such as aducanumab (which will soon be discontinued), lecanemab, and donanemab; however, they aren’t always effective and they require an intravenous infusion, and while they cross the BBB to a degree, to bolster delivery to the brain, studies have proposed additional methods like focused ultrasound.

“Neuronal uptake of drugs potentially therapeutic for Alzheimer’s may be significantly enhanced by fusion of those drugs with insulin. This should be a research priority,” said Talbot.

While this is exciting and has potential, such drugs won’t be available anytime soon. Kamei would like to complete the research at a basic level in 5 years, including testing insulin fused with larger proteins such as therapeutic antibodies. If all goes well, they’ll move on to testing insulin fusion drugs in people.
 

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

PHILADELPHIA — Pimavanserin (Nuplazid, Acadia) is noninferior to quetiapine in patients with Parkinson’s disease psychosis at 56 days, results from a phase 3 trial showed.

In the first prospective comparison of the two antipsychotics in this patient population, pimavanserin yielded significant improvement across all parameters of efficacy without worsening motor symptoms and was very well tolerated, said study investigator Amey Mane, MD, Sun Pharma Laboratories, Mumbai, India.

Psychosis occurs in about 50% patients with Parkinson’s disease and is a major risk factor for hospitalization, nursing home placement, and mortality.

Antipsychotics are used to treat Parkinson’s disease psychosis, but evidence for the efficacy of quetiapine is inconsistent and clozapine requires regular monitoring for agranulocytosis, said Dr. Mane. Cholinergic blockade by these drugs can also increase non-motor symptoms such as constipation, drooling, and cognitive impairment.

Pimavanserin is an oral 5-HT2A inverse agonist and antagonist and the only Food and Drug Administration–approved medication for Parkinson’s disease psychosis, he said. The drug was approved in 2016, and its label was updated in 2023 to clarify that it can be used to treat patients with Parkinson’s disease psychosis, who also have dementia.

“To the best of our understanding, this is the first completed prospective study of pimavanserin with an active comparator, quetiapine,” in Parkinson’s disease psychosis, he said.

The findings were presented in a late-breaking abstract session at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) 2024.
 

Primary Outcome at 56 Days

The assessor-blinded study enrolled 247 patients with Parkinson’s disease for at least 1 year, who were Hoehn and Yahr stage 3 or higher, with hallucinations and/or delusions on a stable dose of Parkinson’s disease medication for at least 4 weeks. The average duration of psychosis was 1.2 years.

Patients were randomly assigned to receive daily pimavanserin 34 mg or quetiapine 25-200 mg for 56 days and evaluated at baseline and days 14, 28, 42, and 56.

The mean change in Scale for the Assessment of Positive Symptoms–Parkinson’s disease (SAPS-PD) nine-item total scores improved from baseline in both groups at all visits (P < .0001) and was significantly greater at 42 days with pimavanserin than with quetiapine (−7.15 vs −6.33; P = .029).

The primary outcome of mean change in SAPS-PD total score at day 56 was −9.64 in the pimavanserin group and −8.37 in the quetiapine group (P = .008). The between-group difference was −1.27, and the upper bound of the 95% CI (−2.77 to 0.24) was lower than the prespecified margin of 0.9, demonstrating noninferiority, Dr. Mane said.
 

Secondary Endpoints and Safety

Pimavanserin was associated with significantly greater improvement than quetiapine for the following secondary outcomes:

• SAPS-Hallucinations and Delusions at day 42 (mean, −12.70 vs −11.40; P = .009) and day 56 (mean, −17.00 vs −15.60; P = .007)
• SAPS-Hallucinations at day 42 (mean, −5.61 vs −4.75; P = .01) and day 56 (mean, −7.33 vs −6.52; P = .02)
• Clinical Global Impression-Improvement score at day 56 (−1.90 vs −1.59; P = .01)
• Scales for Outcomes in Parkinson’s disease (SCOPA) scores for nighttime sleep at day 14 (−1.12 vs −0.85; P = .03) and SCOPA daytime wakefulness at day 28 (−2.42 vs −1.70; P = .01)

Treatment-emergent adverse events (TEAEs) were reported in 7.5% and 13.5% of the pimavanserin and quetiapine groups, respectively.

Five TEAEs, all of mild intensity, were reported as related to study drugs: Pyrexia (1), headache (1), and nasopharyngitis (2) with pimavanserin and headache (1) with quetiapine, Dr. Mane said. There was one unrelated fatal stroke in the quetiapine group. No drug discontinuations occurred because of TEAEs.
 

Delayed Onset of Action?

During a discussion of the results, Hubert Fernandez, MD, director, Center for Neurological Restoration, Cleveland Clinic in Ohio, asked whether the investigators observed a difference in onset between the two drugs.

“Our general impression in the United States is that pimavanserin has a slower uptake in efficacy as compared with quetiapine. If it [quetiapine] works, it works the next day or the day after, whereas with pimavanserin you have to wait for a week or 2. I was just wondering if that’s validated or just anecdotal experience,” he said.

Dr. Mane said the study showed no difference in efficacy at 14 days and greater improvement in efficacy between days 14 and 56.

Another attendee pointed out that quetiapine is particularly good at inducing sleep and asked whether some of the observed differences, especially early on, were due to the need to rapidly titrate quetiapine to induce sleep and get the sleep-wake cycle back on track.

“We did discuss this with most of our investigators, and they gave the same reason. It’s the titration with the quetiapine, and that’s why it’s seen in the early parts,” said Dr. Mane.

Reached for comment, Regina Katzenschlager, MD, Karl Landsteiner Institute for Neuroimmunological and Neurodegenerative Disorders, Klinik Donaustadt, Vienna, Austria, said the majority of drugs commonly used for other types of psychosis cannot be used in PD because of motor worsening.

“Quetiapine is one of the very, very few options we have to treat people with Parkinson’s psychosis because it leads to little, if any, worsening and is the best tolerated,” she said. “Everything else is almost absolutely contraindicated. So that’s why an additional drug — this one has a slightly different mechanism — is incredibly helpful in the clinic because not everyone responds to quetiapine.”

Dr. Katzenschlager pointed out that pimavanserin is not approved in Europe and that the present study was conducted for regulatory purposes in India.

Dr. Mane is an employee of Sun Pharma Laboratories. Dr. Katzenschlager reported having no relevant financial relationships.

A version of this article appeared on Medscape.com.

PHILADELPHIA — Pimavanserin (Nuplazid, Acadia) is noninferior to quetiapine in patients with Parkinson’s disease psychosis at 56 days, results from a phase 3 trial showed.

In the first prospective comparison of the two antipsychotics in this patient population, pimavanserin yielded significant improvement across all parameters of efficacy without worsening motor symptoms and was very well tolerated, said study investigator Amey Mane, MD, Sun Pharma Laboratories, Mumbai, India.

Psychosis occurs in about 50% patients with Parkinson’s disease and is a major risk factor for hospitalization, nursing home placement, and mortality.

Antipsychotics are used to treat Parkinson’s disease psychosis, but evidence for the efficacy of quetiapine is inconsistent and clozapine requires regular monitoring for agranulocytosis, said Dr. Mane. Cholinergic blockade by these drugs can also increase non-motor symptoms such as constipation, drooling, and cognitive impairment.

Pimavanserin is an oral 5-HT2A inverse agonist and antagonist and the only Food and Drug Administration–approved medication for Parkinson’s disease psychosis, he said. The drug was approved in 2016, and its label was updated in 2023 to clarify that it can be used to treat patients with Parkinson’s disease psychosis, who also have dementia.

“To the best of our understanding, this is the first completed prospective study of pimavanserin with an active comparator, quetiapine,” in Parkinson’s disease psychosis, he said.

The findings were presented in a late-breaking abstract session at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) 2024.
 

Primary Outcome at 56 Days

The assessor-blinded study enrolled 247 patients with Parkinson’s disease for at least 1 year, who were Hoehn and Yahr stage 3 or higher, with hallucinations and/or delusions on a stable dose of Parkinson’s disease medication for at least 4 weeks. The average duration of psychosis was 1.2 years.

Patients were randomly assigned to receive daily pimavanserin 34 mg or quetiapine 25-200 mg for 56 days and evaluated at baseline and days 14, 28, 42, and 56.

The mean change in Scale for the Assessment of Positive Symptoms–Parkinson’s disease (SAPS-PD) nine-item total scores improved from baseline in both groups at all visits (P < .0001) and was significantly greater at 42 days with pimavanserin than with quetiapine (−7.15 vs −6.33; P = .029).

The primary outcome of mean change in SAPS-PD total score at day 56 was −9.64 in the pimavanserin group and −8.37 in the quetiapine group (P = .008). The between-group difference was −1.27, and the upper bound of the 95% CI (−2.77 to 0.24) was lower than the prespecified margin of 0.9, demonstrating noninferiority, Dr. Mane said.
 

Secondary Endpoints and Safety

Pimavanserin was associated with significantly greater improvement than quetiapine for the following secondary outcomes:

• SAPS-Hallucinations and Delusions at day 42 (mean, −12.70 vs −11.40; P = .009) and day 56 (mean, −17.00 vs −15.60; P = .007)
• SAPS-Hallucinations at day 42 (mean, −5.61 vs −4.75; P = .01) and day 56 (mean, −7.33 vs −6.52; P = .02)
• Clinical Global Impression-Improvement score at day 56 (−1.90 vs −1.59; P = .01)
• Scales for Outcomes in Parkinson’s disease (SCOPA) scores for nighttime sleep at day 14 (−1.12 vs −0.85; P = .03) and SCOPA daytime wakefulness at day 28 (−2.42 vs −1.70; P = .01)

Treatment-emergent adverse events (TEAEs) were reported in 7.5% and 13.5% of the pimavanserin and quetiapine groups, respectively.

Five TEAEs, all of mild intensity, were reported as related to study drugs: Pyrexia (1), headache (1), and nasopharyngitis (2) with pimavanserin and headache (1) with quetiapine, Dr. Mane said. There was one unrelated fatal stroke in the quetiapine group. No drug discontinuations occurred because of TEAEs.
 

Delayed Onset of Action?

During a discussion of the results, Hubert Fernandez, MD, director, Center for Neurological Restoration, Cleveland Clinic in Ohio, asked whether the investigators observed a difference in onset between the two drugs.

“Our general impression in the United States is that pimavanserin has a slower uptake in efficacy as compared with quetiapine. If it [quetiapine] works, it works the next day or the day after, whereas with pimavanserin you have to wait for a week or 2. I was just wondering if that’s validated or just anecdotal experience,” he said.

Dr. Mane said the study showed no difference in efficacy at 14 days and greater improvement in efficacy between days 14 and 56.

Another attendee pointed out that quetiapine is particularly good at inducing sleep and asked whether some of the observed differences, especially early on, were due to the need to rapidly titrate quetiapine to induce sleep and get the sleep-wake cycle back on track.

“We did discuss this with most of our investigators, and they gave the same reason. It’s the titration with the quetiapine, and that’s why it’s seen in the early parts,” said Dr. Mane.

Reached for comment, Regina Katzenschlager, MD, Karl Landsteiner Institute for Neuroimmunological and Neurodegenerative Disorders, Klinik Donaustadt, Vienna, Austria, said the majority of drugs commonly used for other types of psychosis cannot be used in PD because of motor worsening.

“Quetiapine is one of the very, very few options we have to treat people with Parkinson’s psychosis because it leads to little, if any, worsening and is the best tolerated,” she said. “Everything else is almost absolutely contraindicated. So that’s why an additional drug — this one has a slightly different mechanism — is incredibly helpful in the clinic because not everyone responds to quetiapine.”

Dr. Katzenschlager pointed out that pimavanserin is not approved in Europe and that the present study was conducted for regulatory purposes in India.

Dr. Mane is an employee of Sun Pharma Laboratories. Dr. Katzenschlager reported having no relevant financial relationships.

A version of this article appeared on Medscape.com.

PHILADELPHIA — Pimavanserin (Nuplazid, Acadia) is noninferior to quetiapine in patients with Parkinson’s disease psychosis at 56 days, results from a phase 3 trial showed.

In the first prospective comparison of the two antipsychotics in this patient population, pimavanserin yielded significant improvement across all parameters of efficacy without worsening motor symptoms and was very well tolerated, said study investigator Amey Mane, MD, Sun Pharma Laboratories, Mumbai, India.

Psychosis occurs in about 50% patients with Parkinson’s disease and is a major risk factor for hospitalization, nursing home placement, and mortality.

Antipsychotics are used to treat Parkinson’s disease psychosis, but evidence for the efficacy of quetiapine is inconsistent and clozapine requires regular monitoring for agranulocytosis, said Dr. Mane. Cholinergic blockade by these drugs can also increase non-motor symptoms such as constipation, drooling, and cognitive impairment.

Pimavanserin is an oral 5-HT2A inverse agonist and antagonist and the only Food and Drug Administration–approved medication for Parkinson’s disease psychosis, he said. The drug was approved in 2016, and its label was updated in 2023 to clarify that it can be used to treat patients with Parkinson’s disease psychosis, who also have dementia.

“To the best of our understanding, this is the first completed prospective study of pimavanserin with an active comparator, quetiapine,” in Parkinson’s disease psychosis, he said.

The findings were presented in a late-breaking abstract session at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) 2024.
 

Primary Outcome at 56 Days

The assessor-blinded study enrolled 247 patients with Parkinson’s disease for at least 1 year, who were Hoehn and Yahr stage 3 or higher, with hallucinations and/or delusions on a stable dose of Parkinson’s disease medication for at least 4 weeks. The average duration of psychosis was 1.2 years.

Patients were randomly assigned to receive daily pimavanserin 34 mg or quetiapine 25-200 mg for 56 days and evaluated at baseline and days 14, 28, 42, and 56.

The mean change in Scale for the Assessment of Positive Symptoms–Parkinson’s disease (SAPS-PD) nine-item total scores improved from baseline in both groups at all visits (P < .0001) and was significantly greater at 42 days with pimavanserin than with quetiapine (−7.15 vs −6.33; P = .029).

The primary outcome of mean change in SAPS-PD total score at day 56 was −9.64 in the pimavanserin group and −8.37 in the quetiapine group (P = .008). The between-group difference was −1.27, and the upper bound of the 95% CI (−2.77 to 0.24) was lower than the prespecified margin of 0.9, demonstrating noninferiority, Dr. Mane said.
 

Secondary Endpoints and Safety

Pimavanserin was associated with significantly greater improvement than quetiapine for the following secondary outcomes:

• SAPS-Hallucinations and Delusions at day 42 (mean, −12.70 vs −11.40; P = .009) and day 56 (mean, −17.00 vs −15.60; P = .007)
• SAPS-Hallucinations at day 42 (mean, −5.61 vs −4.75; P = .01) and day 56 (mean, −7.33 vs −6.52; P = .02)
• Clinical Global Impression-Improvement score at day 56 (−1.90 vs −1.59; P = .01)
• Scales for Outcomes in Parkinson’s disease (SCOPA) scores for nighttime sleep at day 14 (−1.12 vs −0.85; P = .03) and SCOPA daytime wakefulness at day 28 (−2.42 vs −1.70; P = .01)

Treatment-emergent adverse events (TEAEs) were reported in 7.5% and 13.5% of the pimavanserin and quetiapine groups, respectively.

Five TEAEs, all of mild intensity, were reported as related to study drugs: Pyrexia (1), headache (1), and nasopharyngitis (2) with pimavanserin and headache (1) with quetiapine, Dr. Mane said. There was one unrelated fatal stroke in the quetiapine group. No drug discontinuations occurred because of TEAEs.
 

Delayed Onset of Action?

During a discussion of the results, Hubert Fernandez, MD, director, Center for Neurological Restoration, Cleveland Clinic in Ohio, asked whether the investigators observed a difference in onset between the two drugs.

“Our general impression in the United States is that pimavanserin has a slower uptake in efficacy as compared with quetiapine. If it [quetiapine] works, it works the next day or the day after, whereas with pimavanserin you have to wait for a week or 2. I was just wondering if that’s validated or just anecdotal experience,” he said.

Dr. Mane said the study showed no difference in efficacy at 14 days and greater improvement in efficacy between days 14 and 56.

Another attendee pointed out that quetiapine is particularly good at inducing sleep and asked whether some of the observed differences, especially early on, were due to the need to rapidly titrate quetiapine to induce sleep and get the sleep-wake cycle back on track.

“We did discuss this with most of our investigators, and they gave the same reason. It’s the titration with the quetiapine, and that’s why it’s seen in the early parts,” said Dr. Mane.

Reached for comment, Regina Katzenschlager, MD, Karl Landsteiner Institute for Neuroimmunological and Neurodegenerative Disorders, Klinik Donaustadt, Vienna, Austria, said the majority of drugs commonly used for other types of psychosis cannot be used in PD because of motor worsening.

“Quetiapine is one of the very, very few options we have to treat people with Parkinson’s psychosis because it leads to little, if any, worsening and is the best tolerated,” she said. “Everything else is almost absolutely contraindicated. So that’s why an additional drug — this one has a slightly different mechanism — is incredibly helpful in the clinic because not everyone responds to quetiapine.”

Dr. Katzenschlager pointed out that pimavanserin is not approved in Europe and that the present study was conducted for regulatory purposes in India.

Dr. Mane is an employee of Sun Pharma Laboratories. Dr. Katzenschlager reported having no relevant financial relationships.

A version of this article appeared on Medscape.com.

The peer-review process, which is used by scientific journals to validate legitimate research, is now under legal scrutiny. The US District Court for the Southern District of New York will soon rule on whether scientific publishers have compromised this system for profit. In mid-September, University of California, Los Angeles neuroscientist Lucina Uddin filed a class action lawsuit against six leading academic publishers — Elsevier, Wolters Kluwer, Wiley, Sage Publications, Taylor & Francis, and Springer Nature — accusing them of violating antitrust laws and obstructing academic research.

The lawsuit targets several long-standing practices in scientific publishing, including the lack of compensation for peer reviewers, restrictions that require submitting to only one journal at a time, and bans on sharing manuscripts under review. Uddin’s complaint argues that these practices contribute to inefficiencies in the review process, thus delaying the publication of critical discoveries, which could hinder research, clinical advancements, and the development of new medical treatments.

The suit also noted that these publishers generated $10 billion in revenue in 2023 in peer-reviewed journals. However, the complaint seemingly overlooks the widespread practice of preprint repositories, where many manuscripts are shared while awaiting peer review.
 

Flawed Reviews

A growing number of studies have highlighted subpar or unethical behaviors among reviewers, who are supposed to adhere to the highest standards of methodological rigor, both in conducting research and reviewing work for journals. One recent study published in Scientometrics in August examined 263 reviews from 37 journals across various disciplines and found alarming patterns of duplication. Many of the reviews contained identical or highly similar language. Some reviewers were found to be suggesting that the authors expand their bibliographies to include the reviewers’ own work, thus inflating their citation counts.

As María Ángeles Oviedo-García from the University of Seville in Spain, pointed out: “The analysis of 263 review reports shows a pattern of vague, repetitive statements — often identical or very similar — along with coercive citations, ultimately resulting in misleading reviews.”

Experts in research integrity and ethics argue that while issues persist, the integrity of scientific research is improving. Increasing research and public disclosure reflect a heightened awareness of problems long overlooked.

“There is indeed a problem with research reliability, but it’s not as widespread or severe as some portray,” said Daniele Fanelli, a metascientist at the London School of Economics and Political Science in England. Speaking to this news organization, Fanelli, who has been studying scientific misconduct for about 20 years, noted that while his early work left him disillusioned, further research has replaced his cynicism with what he describes as healthy skepticism and a more optimistic outlook. Fanelli also collaborates with the Luxembourg Agency for Research Integrity and the Advisory Committee on Research Ethics and Bioethics at the Italian National Research Council (CNR), where he helped develop the first research integrity guidelines.
 

Lack of Awareness

A recurring challenge is the difficulty in distinguishing between honest mistakes and intentional misconduct. “This is why greater investment in education is essential,” said Daniel Pizzolato, European Network of Research Ethics Committees, Bonn, Germany, and the Centre for Biomedical Ethics and Law, KU Leuven in Belgium.

While Pizzolato acknowledged that institutions such as the CNR in Italy provide a positive example, awareness of research integrity is generally still lacking across much of Europe, and there are few offices dedicated to promoting research integrity. However, he pointed to promising developments in other countries. “In France and Denmark, researchers are required to be familiar with integrity norms because codes of conduct have legal standing. Some major international funding bodies like the European Molecular Biology Organization are making participation in research integrity courses a condition for receiving grants.”

Pizzolato remains optimistic. “There is a growing willingness to move past this impasse,” he said.

A recent study published in The Journal of Clinical Epidemiology reveals troubling gaps in how retracted biomedical articles are flagged and cited. Led by Caitlin Bakkera, Department of Epidemiology, Maastricht University, Maastricht, the Netherlands, the research sought to determine whether articles retracted because of errors or fraud were properly flagged across various databases.

The results were concerning: Less than 5% of retracted articles had consistent retraction notices across all databases that hosted them, and less than 50% of citations referenced the retraction. None of the 414 retraction notices analyzed met best-practice guidelines for completeness. Bakkera and colleagues warned that these shortcomings threaten the integrity of public health research.
 

Fanelli’s Perspective

Despite the concerns, Fanelli remains calm. “Science is based on debate and a perspective called organized skepticism, which helps reveal the truth,” he explained. “While there is often excessive skepticism today, the overall quality of clinical trials is improving.

“It’s important to remember that reliable results take time and shouldn’t depend on the outcome of a single study. It’s essential to consider the broader context, the history of the research field, and potential conflicts of interest, both financial and otherwise. Biomedical research requires constant updates,” he concluded.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The peer-review process, which is used by scientific journals to validate legitimate research, is now under legal scrutiny. The US District Court for the Southern District of New York will soon rule on whether scientific publishers have compromised this system for profit. In mid-September, University of California, Los Angeles neuroscientist Lucina Uddin filed a class action lawsuit against six leading academic publishers — Elsevier, Wolters Kluwer, Wiley, Sage Publications, Taylor & Francis, and Springer Nature — accusing them of violating antitrust laws and obstructing academic research.

The lawsuit targets several long-standing practices in scientific publishing, including the lack of compensation for peer reviewers, restrictions that require submitting to only one journal at a time, and bans on sharing manuscripts under review. Uddin’s complaint argues that these practices contribute to inefficiencies in the review process, thus delaying the publication of critical discoveries, which could hinder research, clinical advancements, and the development of new medical treatments.

The suit also noted that these publishers generated $10 billion in revenue in 2023 in peer-reviewed journals. However, the complaint seemingly overlooks the widespread practice of preprint repositories, where many manuscripts are shared while awaiting peer review.
 

Flawed Reviews

A growing number of studies have highlighted subpar or unethical behaviors among reviewers, who are supposed to adhere to the highest standards of methodological rigor, both in conducting research and reviewing work for journals. One recent study published in Scientometrics in August examined 263 reviews from 37 journals across various disciplines and found alarming patterns of duplication. Many of the reviews contained identical or highly similar language. Some reviewers were found to be suggesting that the authors expand their bibliographies to include the reviewers’ own work, thus inflating their citation counts.

As María Ángeles Oviedo-García from the University of Seville in Spain, pointed out: “The analysis of 263 review reports shows a pattern of vague, repetitive statements — often identical or very similar — along with coercive citations, ultimately resulting in misleading reviews.”

Experts in research integrity and ethics argue that while issues persist, the integrity of scientific research is improving. Increasing research and public disclosure reflect a heightened awareness of problems long overlooked.

“There is indeed a problem with research reliability, but it’s not as widespread or severe as some portray,” said Daniele Fanelli, a metascientist at the London School of Economics and Political Science in England. Speaking to this news organization, Fanelli, who has been studying scientific misconduct for about 20 years, noted that while his early work left him disillusioned, further research has replaced his cynicism with what he describes as healthy skepticism and a more optimistic outlook. Fanelli also collaborates with the Luxembourg Agency for Research Integrity and the Advisory Committee on Research Ethics and Bioethics at the Italian National Research Council (CNR), where he helped develop the first research integrity guidelines.
 

Lack of Awareness

A recurring challenge is the difficulty in distinguishing between honest mistakes and intentional misconduct. “This is why greater investment in education is essential,” said Daniel Pizzolato, European Network of Research Ethics Committees, Bonn, Germany, and the Centre for Biomedical Ethics and Law, KU Leuven in Belgium.

While Pizzolato acknowledged that institutions such as the CNR in Italy provide a positive example, awareness of research integrity is generally still lacking across much of Europe, and there are few offices dedicated to promoting research integrity. However, he pointed to promising developments in other countries. “In France and Denmark, researchers are required to be familiar with integrity norms because codes of conduct have legal standing. Some major international funding bodies like the European Molecular Biology Organization are making participation in research integrity courses a condition for receiving grants.”

Pizzolato remains optimistic. “There is a growing willingness to move past this impasse,” he said.

A recent study published in The Journal of Clinical Epidemiology reveals troubling gaps in how retracted biomedical articles are flagged and cited. Led by Caitlin Bakkera, Department of Epidemiology, Maastricht University, Maastricht, the Netherlands, the research sought to determine whether articles retracted because of errors or fraud were properly flagged across various databases.

The results were concerning: Less than 5% of retracted articles had consistent retraction notices across all databases that hosted them, and less than 50% of citations referenced the retraction. None of the 414 retraction notices analyzed met best-practice guidelines for completeness. Bakkera and colleagues warned that these shortcomings threaten the integrity of public health research.
 

Fanelli’s Perspective

Despite the concerns, Fanelli remains calm. “Science is based on debate and a perspective called organized skepticism, which helps reveal the truth,” he explained. “While there is often excessive skepticism today, the overall quality of clinical trials is improving.

“It’s important to remember that reliable results take time and shouldn’t depend on the outcome of a single study. It’s essential to consider the broader context, the history of the research field, and potential conflicts of interest, both financial and otherwise. Biomedical research requires constant updates,” he concluded.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The peer-review process, which is used by scientific journals to validate legitimate research, is now under legal scrutiny. The US District Court for the Southern District of New York will soon rule on whether scientific publishers have compromised this system for profit. In mid-September, University of California, Los Angeles neuroscientist Lucina Uddin filed a class action lawsuit against six leading academic publishers — Elsevier, Wolters Kluwer, Wiley, Sage Publications, Taylor & Francis, and Springer Nature — accusing them of violating antitrust laws and obstructing academic research.

The lawsuit targets several long-standing practices in scientific publishing, including the lack of compensation for peer reviewers, restrictions that require submitting to only one journal at a time, and bans on sharing manuscripts under review. Uddin’s complaint argues that these practices contribute to inefficiencies in the review process, thus delaying the publication of critical discoveries, which could hinder research, clinical advancements, and the development of new medical treatments.

The suit also noted that these publishers generated $10 billion in revenue in 2023 in peer-reviewed journals. However, the complaint seemingly overlooks the widespread practice of preprint repositories, where many manuscripts are shared while awaiting peer review.
 

Flawed Reviews

A growing number of studies have highlighted subpar or unethical behaviors among reviewers, who are supposed to adhere to the highest standards of methodological rigor, both in conducting research and reviewing work for journals. One recent study published in Scientometrics in August examined 263 reviews from 37 journals across various disciplines and found alarming patterns of duplication. Many of the reviews contained identical or highly similar language. Some reviewers were found to be suggesting that the authors expand their bibliographies to include the reviewers’ own work, thus inflating their citation counts.

As María Ángeles Oviedo-García from the University of Seville in Spain, pointed out: “The analysis of 263 review reports shows a pattern of vague, repetitive statements — often identical or very similar — along with coercive citations, ultimately resulting in misleading reviews.”

Experts in research integrity and ethics argue that while issues persist, the integrity of scientific research is improving. Increasing research and public disclosure reflect a heightened awareness of problems long overlooked.

“There is indeed a problem with research reliability, but it’s not as widespread or severe as some portray,” said Daniele Fanelli, a metascientist at the London School of Economics and Political Science in England. Speaking to this news organization, Fanelli, who has been studying scientific misconduct for about 20 years, noted that while his early work left him disillusioned, further research has replaced his cynicism with what he describes as healthy skepticism and a more optimistic outlook. Fanelli also collaborates with the Luxembourg Agency for Research Integrity and the Advisory Committee on Research Ethics and Bioethics at the Italian National Research Council (CNR), where he helped develop the first research integrity guidelines.
 

Lack of Awareness

A recurring challenge is the difficulty in distinguishing between honest mistakes and intentional misconduct. “This is why greater investment in education is essential,” said Daniel Pizzolato, European Network of Research Ethics Committees, Bonn, Germany, and the Centre for Biomedical Ethics and Law, KU Leuven in Belgium.

While Pizzolato acknowledged that institutions such as the CNR in Italy provide a positive example, awareness of research integrity is generally still lacking across much of Europe, and there are few offices dedicated to promoting research integrity. However, he pointed to promising developments in other countries. “In France and Denmark, researchers are required to be familiar with integrity norms because codes of conduct have legal standing. Some major international funding bodies like the European Molecular Biology Organization are making participation in research integrity courses a condition for receiving grants.”

Pizzolato remains optimistic. “There is a growing willingness to move past this impasse,” he said.

A recent study published in The Journal of Clinical Epidemiology reveals troubling gaps in how retracted biomedical articles are flagged and cited. Led by Caitlin Bakkera, Department of Epidemiology, Maastricht University, Maastricht, the Netherlands, the research sought to determine whether articles retracted because of errors or fraud were properly flagged across various databases.

The results were concerning: Less than 5% of retracted articles had consistent retraction notices across all databases that hosted them, and less than 50% of citations referenced the retraction. None of the 414 retraction notices analyzed met best-practice guidelines for completeness. Bakkera and colleagues warned that these shortcomings threaten the integrity of public health research.
 

Fanelli’s Perspective

Despite the concerns, Fanelli remains calm. “Science is based on debate and a perspective called organized skepticism, which helps reveal the truth,” he explained. “While there is often excessive skepticism today, the overall quality of clinical trials is improving.

“It’s important to remember that reliable results take time and shouldn’t depend on the outcome of a single study. It’s essential to consider the broader context, the history of the research field, and potential conflicts of interest, both financial and otherwise. Biomedical research requires constant updates,” he concluded.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The latest research on therapeutic management of patients with relapsing-remitting multiple sclerosis (MS) presented at the European Committee for Treatment and Research in Multiple Sclerosis 2024 Congress is reported by Dr Patricia Coyle from Stony Brook University Hospital, in Stony Brook, New York. 

Dr Coyle first discusses a registry study looking at initiation of monoclonal antibody therapy for patients with pediatric-onset MS. Results showed a significant reduction in disability at age 23 and beyond when therapy was initiated in childhood.   

Next, Dr Coyle discusses a trial examining the safety and efficacy of frexalimab, a second-generation anti-CD40L antibody. In an open-label extension trial through 72 weeks, frexalimab provided a sustained reduction of disease activity, as measured by MRI, and was well tolerated. 

She then details a study looking at the effects of disease-modifying therapies (DMTs) on pregnancy outcomes in patients with MS. Using a German MS registry, researchers looked at 3722 pregnancies, 2885 with DMT exposure, and concluded that most pregnancy outcomes are unaffected by DMT exposure; however, the data showed the potential risk for reduced birth rates. 

Finally, Dr Coyle examines the efficacy of the Bruton tyrosine kinase (BTK) inhibitor tolebrutinib, as evidenced by the HERCULES trial and the two GEMINI trials. In HERCULES, the BTK inhibitor reduced 6-month disability progression by a significant 31% compared with placebo.  

--

Patricia K. Coyle, MD, Professor and Interim Chair, Department of Neurology; Director, MS Comprehensive Care Center, Stony Brook University Hospital, Stony Brook, New York

Patricia K. Coyle, MD, has disclosed the following relevant financial relationships: 
 
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Accordant; Amgen; Biogen; Bristol Myers Squibb; Eli Lilly & Company; EMD Serono; GSK; Genentech; Horizon; LabCorp; Mylan; Novartis; Sanofi Genzyme; Viatris 
Received research grant from: Celgene; CorEvitas LLC; Genentech/Roche; National Institute of Neurological Disorders and Stroke; Sanofi Genzyme

The latest research on therapeutic management of patients with relapsing-remitting multiple sclerosis (MS) presented at the European Committee for Treatment and Research in Multiple Sclerosis 2024 Congress is reported by Dr Patricia Coyle from Stony Brook University Hospital, in Stony Brook, New York. 

Dr Coyle first discusses a registry study looking at initiation of monoclonal antibody therapy for patients with pediatric-onset MS. Results showed a significant reduction in disability at age 23 and beyond when therapy was initiated in childhood.   

Next, Dr Coyle discusses a trial examining the safety and efficacy of frexalimab, a second-generation anti-CD40L antibody. In an open-label extension trial through 72 weeks, frexalimab provided a sustained reduction of disease activity, as measured by MRI, and was well tolerated. 

She then details a study looking at the effects of disease-modifying therapies (DMTs) on pregnancy outcomes in patients with MS. Using a German MS registry, researchers looked at 3722 pregnancies, 2885 with DMT exposure, and concluded that most pregnancy outcomes are unaffected by DMT exposure; however, the data showed the potential risk for reduced birth rates. 

Finally, Dr Coyle examines the efficacy of the Bruton tyrosine kinase (BTK) inhibitor tolebrutinib, as evidenced by the HERCULES trial and the two GEMINI trials. In HERCULES, the BTK inhibitor reduced 6-month disability progression by a significant 31% compared with placebo.  

--

Patricia K. Coyle, MD, Professor and Interim Chair, Department of Neurology; Director, MS Comprehensive Care Center, Stony Brook University Hospital, Stony Brook, New York

Patricia K. Coyle, MD, has disclosed the following relevant financial relationships: 
 
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Accordant; Amgen; Biogen; Bristol Myers Squibb; Eli Lilly & Company; EMD Serono; GSK; Genentech; Horizon; LabCorp; Mylan; Novartis; Sanofi Genzyme; Viatris 
Received research grant from: Celgene; CorEvitas LLC; Genentech/Roche; National Institute of Neurological Disorders and Stroke; Sanofi Genzyme

The latest research on therapeutic management of patients with relapsing-remitting multiple sclerosis (MS) presented at the European Committee for Treatment and Research in Multiple Sclerosis 2024 Congress is reported by Dr Patricia Coyle from Stony Brook University Hospital, in Stony Brook, New York. 

Dr Coyle first discusses a registry study looking at initiation of monoclonal antibody therapy for patients with pediatric-onset MS. Results showed a significant reduction in disability at age 23 and beyond when therapy was initiated in childhood.   

Next, Dr Coyle discusses a trial examining the safety and efficacy of frexalimab, a second-generation anti-CD40L antibody. In an open-label extension trial through 72 weeks, frexalimab provided a sustained reduction of disease activity, as measured by MRI, and was well tolerated. 

She then details a study looking at the effects of disease-modifying therapies (DMTs) on pregnancy outcomes in patients with MS. Using a German MS registry, researchers looked at 3722 pregnancies, 2885 with DMT exposure, and concluded that most pregnancy outcomes are unaffected by DMT exposure; however, the data showed the potential risk for reduced birth rates. 

Finally, Dr Coyle examines the efficacy of the Bruton tyrosine kinase (BTK) inhibitor tolebrutinib, as evidenced by the HERCULES trial and the two GEMINI trials. In HERCULES, the BTK inhibitor reduced 6-month disability progression by a significant 31% compared with placebo.  

--

Patricia K. Coyle, MD, Professor and Interim Chair, Department of Neurology; Director, MS Comprehensive Care Center, Stony Brook University Hospital, Stony Brook, New York

Patricia K. Coyle, MD, has disclosed the following relevant financial relationships: 
 
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Accordant; Amgen; Biogen; Bristol Myers Squibb; Eli Lilly & Company; EMD Serono; GSK; Genentech; Horizon; LabCorp; Mylan; Novartis; Sanofi Genzyme; Viatris 
Received research grant from: Celgene; CorEvitas LLC; Genentech/Roche; National Institute of Neurological Disorders and Stroke; Sanofi Genzyme

Summary:

In this segment, the speaker discusses how AI is revolutionizing the drug repurposing process. Previously, drug repurposing was limited by manual research on individual diseases and drugs. With AI, scientists can now analyze a vast array of drugs and diseases simultaneously, generating a ranking system based on the likelihood of success. The Center for Cytokine Storm Treatment and Laboratory, along with the platform Every Cure, uses AI to score 3000 drugs against 18,000 diseases. This platform dramatically reduces the time and resources required for drug repurposing, enabling predictions that can be tested in a fraction of the time.
 

Key Takeaways:

AI is accelerating the drug repurposing process, offering faster and more comprehensive analysis of possible drug-disease matches.

The AI-based platform assigns a likelihood score to each potential match, streamlining the process for testing and validation.

The ability to analyze global biomedical knowledge in 24 hours is unprecedented, reducing research costs and increasing the speed of drug discovery.
 

Our Editors Also Recommend: 

AI’s Drug Revolution, Part 1: Faster Trials and Approvals

From AI to Obesity Drugs to Soaring Costs: Medscape Hot Topics in the Medical Profession Report 2024

AI Voice Analysis for Diabetes Screening Shows Promise

To see the full event recording, click here.
 

A version of this article appeared on Medscape.com.

Summary:

In this segment, the speaker discusses how AI is revolutionizing the drug repurposing process. Previously, drug repurposing was limited by manual research on individual diseases and drugs. With AI, scientists can now analyze a vast array of drugs and diseases simultaneously, generating a ranking system based on the likelihood of success. The Center for Cytokine Storm Treatment and Laboratory, along with the platform Every Cure, uses AI to score 3000 drugs against 18,000 diseases. This platform dramatically reduces the time and resources required for drug repurposing, enabling predictions that can be tested in a fraction of the time.
 

Key Takeaways:

AI is accelerating the drug repurposing process, offering faster and more comprehensive analysis of possible drug-disease matches.

The AI-based platform assigns a likelihood score to each potential match, streamlining the process for testing and validation.

The ability to analyze global biomedical knowledge in 24 hours is unprecedented, reducing research costs and increasing the speed of drug discovery.
 

Our Editors Also Recommend: 

AI’s Drug Revolution, Part 1: Faster Trials and Approvals

From AI to Obesity Drugs to Soaring Costs: Medscape Hot Topics in the Medical Profession Report 2024

AI Voice Analysis for Diabetes Screening Shows Promise

To see the full event recording, click here.
 

A version of this article appeared on Medscape.com.

Summary:

In this segment, the speaker discusses how AI is revolutionizing the drug repurposing process. Previously, drug repurposing was limited by manual research on individual diseases and drugs. With AI, scientists can now analyze a vast array of drugs and diseases simultaneously, generating a ranking system based on the likelihood of success. The Center for Cytokine Storm Treatment and Laboratory, along with the platform Every Cure, uses AI to score 3000 drugs against 18,000 diseases. This platform dramatically reduces the time and resources required for drug repurposing, enabling predictions that can be tested in a fraction of the time.
 

Key Takeaways:

AI is accelerating the drug repurposing process, offering faster and more comprehensive analysis of possible drug-disease matches.

The AI-based platform assigns a likelihood score to each potential match, streamlining the process for testing and validation.

The ability to analyze global biomedical knowledge in 24 hours is unprecedented, reducing research costs and increasing the speed of drug discovery.
 

Our Editors Also Recommend: 

AI’s Drug Revolution, Part 1: Faster Trials and Approvals

From AI to Obesity Drugs to Soaring Costs: Medscape Hot Topics in the Medical Profession Report 2024

AI Voice Analysis for Diabetes Screening Shows Promise

To see the full event recording, click here.
 

A version of this article appeared on Medscape.com.

Editor’s Note
By Glenn S. Williams
In this year’s Rare Neurological Disease Special Report, we focus on rare neurological diseases that have new therapies that have been recently approved as well as conditions for which the treatment pipeline is robust.

A Note From NORD
By Pamela Gavin
Through NORD’s collaboration with Neurology Reviews, we share cutting-edge research and insights from leading medical experts, including specialists from the NORD Rare Disease Centers of Excellence network, about the latest advances in the treatment of rare neurological conditions.

Genetic Testing for ALS, Now a Standard, Creates a Path Toward Individualized Care
By Ted Bosworth
Overall, there is a sense of progress in ALS. The hope is that clinical research is reaching a tipping point where targeted treatments may offer hope to patients with ALS.

Myasthenia Gravis: Patient Choice, Cultural Change
By John Jesitus
Used appropriately, newer treatments for myasthenia gravis can provide dramatic results faster and more safely than broad immunosuppressants.

Promise for Disease-Modifying Therapies to Tame Huntington’s Disease
By Neil Osterweil
Much progress has been made in managing the symptoms of Huntington’s disease, but the real excitement lies in the development of disease-modifying drugs and genetic therapy.

Diagnosing and Managing Duchenne Muscular Dystrophy: Tips for Practicing Clinicians
By Batya Swift Yasgur, MA, LSW
Healthcare providers should be familiar enough with Duchenne muscular dystrophy to provide timely diagnosis and early intervention as well as practical and emotional support to the patient and family/caregivers.

Neuromyelitis Optica: Historically Misdiagnosed — Now Demands Prompt Treatment
By Kate Johnson
Rapid diagnosis and treatment of NMO “means potentially preventing future devastating neurologic injury.”

Untangling CIDP
By Jennie Smith
Though a preferred biomarker remains elusive, this difficult-to-diagnose neuropathy has seen important recent advances in diagnosis and treatment.

Newborn Screening Programs: What Do Clinicians Need to Know?
By Batya Swift Yasgur, MA, LSW
The goal of newborn screening is to identify babies with genetic disorders who otherwise have no obvious symptoms.

Balancing Act: Weighing the Pros and Cons of Genetic Testing in Rare Diseases
By Frieda Wiley
While genetic testing may offer great potential for providing answers to patients and clinicians seeking insight into a rare disorder, the technology holds some pros and cons that neurologists should be aware of.

Editor’s Note
By Glenn S. Williams
In this year’s Rare Neurological Disease Special Report, we focus on rare neurological diseases that have new therapies that have been recently approved as well as conditions for which the treatment pipeline is robust.

A Note From NORD
By Pamela Gavin
Through NORD’s collaboration with Neurology Reviews, we share cutting-edge research and insights from leading medical experts, including specialists from the NORD Rare Disease Centers of Excellence network, about the latest advances in the treatment of rare neurological conditions.

Genetic Testing for ALS, Now a Standard, Creates a Path Toward Individualized Care
By Ted Bosworth
Overall, there is a sense of progress in ALS. The hope is that clinical research is reaching a tipping point where targeted treatments may offer hope to patients with ALS.

Myasthenia Gravis: Patient Choice, Cultural Change
By John Jesitus
Used appropriately, newer treatments for myasthenia gravis can provide dramatic results faster and more safely than broad immunosuppressants.

Promise for Disease-Modifying Therapies to Tame Huntington’s Disease
By Neil Osterweil
Much progress has been made in managing the symptoms of Huntington’s disease, but the real excitement lies in the development of disease-modifying drugs and genetic therapy.

Diagnosing and Managing Duchenne Muscular Dystrophy: Tips for Practicing Clinicians
By Batya Swift Yasgur, MA, LSW
Healthcare providers should be familiar enough with Duchenne muscular dystrophy to provide timely diagnosis and early intervention as well as practical and emotional support to the patient and family/caregivers.

Neuromyelitis Optica: Historically Misdiagnosed — Now Demands Prompt Treatment
By Kate Johnson
Rapid diagnosis and treatment of NMO “means potentially preventing future devastating neurologic injury.”

Untangling CIDP
By Jennie Smith
Though a preferred biomarker remains elusive, this difficult-to-diagnose neuropathy has seen important recent advances in diagnosis and treatment.

Newborn Screening Programs: What Do Clinicians Need to Know?
By Batya Swift Yasgur, MA, LSW
The goal of newborn screening is to identify babies with genetic disorders who otherwise have no obvious symptoms.

Balancing Act: Weighing the Pros and Cons of Genetic Testing in Rare Diseases
By Frieda Wiley
While genetic testing may offer great potential for providing answers to patients and clinicians seeking insight into a rare disorder, the technology holds some pros and cons that neurologists should be aware of.

Editor’s Note
By Glenn S. Williams
In this year’s Rare Neurological Disease Special Report, we focus on rare neurological diseases that have new therapies that have been recently approved as well as conditions for which the treatment pipeline is robust.

A Note From NORD
By Pamela Gavin
Through NORD’s collaboration with Neurology Reviews, we share cutting-edge research and insights from leading medical experts, including specialists from the NORD Rare Disease Centers of Excellence network, about the latest advances in the treatment of rare neurological conditions.

Genetic Testing for ALS, Now a Standard, Creates a Path Toward Individualized Care
By Ted Bosworth
Overall, there is a sense of progress in ALS. The hope is that clinical research is reaching a tipping point where targeted treatments may offer hope to patients with ALS.

Myasthenia Gravis: Patient Choice, Cultural Change
By John Jesitus
Used appropriately, newer treatments for myasthenia gravis can provide dramatic results faster and more safely than broad immunosuppressants.

Promise for Disease-Modifying Therapies to Tame Huntington’s Disease
By Neil Osterweil
Much progress has been made in managing the symptoms of Huntington’s disease, but the real excitement lies in the development of disease-modifying drugs and genetic therapy.

Diagnosing and Managing Duchenne Muscular Dystrophy: Tips for Practicing Clinicians
By Batya Swift Yasgur, MA, LSW
Healthcare providers should be familiar enough with Duchenne muscular dystrophy to provide timely diagnosis and early intervention as well as practical and emotional support to the patient and family/caregivers.

Neuromyelitis Optica: Historically Misdiagnosed — Now Demands Prompt Treatment
By Kate Johnson
Rapid diagnosis and treatment of NMO “means potentially preventing future devastating neurologic injury.”

Untangling CIDP
By Jennie Smith
Though a preferred biomarker remains elusive, this difficult-to-diagnose neuropathy has seen important recent advances in diagnosis and treatment.

Newborn Screening Programs: What Do Clinicians Need to Know?
By Batya Swift Yasgur, MA, LSW
The goal of newborn screening is to identify babies with genetic disorders who otherwise have no obvious symptoms.

Balancing Act: Weighing the Pros and Cons of Genetic Testing in Rare Diseases
By Frieda Wiley
While genetic testing may offer great potential for providing answers to patients and clinicians seeking insight into a rare disorder, the technology holds some pros and cons that neurologists should be aware of.