Members of the Food and Drug Administration’s Oncologic Drugs Advisory Committee voted 12-4 in support of approval of neratinib for single-agent extended adjuvant treatment of adults with early-stage HER2-positive breast cancer who received prior adjuvant trastuzumab-based therapy.

Members voting during the May 24 meeting expressed concern about the broadness of the indication proposed by the drug’s maker, Puma Biotechnology, but the majority said the risk-benefit profile of the drug is sufficient to support approval, particularly given the unmet need for such an agent. Those voting against approval argued that more data are needed to identify subpopulations of patients who would be most likely to benefit from treatment in order to narrow the indication.

[email protected]

Members of the Food and Drug Administration’s Oncologic Drugs Advisory Committee voted 12-4 in support of approval of neratinib for single-agent extended adjuvant treatment of adults with early-stage HER2-positive breast cancer who received prior adjuvant trastuzumab-based therapy.

Members voting during the May 24 meeting expressed concern about the broadness of the indication proposed by the drug’s maker, Puma Biotechnology, but the majority said the risk-benefit profile of the drug is sufficient to support approval, particularly given the unmet need for such an agent. Those voting against approval argued that more data are needed to identify subpopulations of patients who would be most likely to benefit from treatment in order to narrow the indication.

[email protected]

Members of the Food and Drug Administration’s Oncologic Drugs Advisory Committee voted 12-4 in support of approval of neratinib for single-agent extended adjuvant treatment of adults with early-stage HER2-positive breast cancer who received prior adjuvant trastuzumab-based therapy.

Members voting during the May 24 meeting expressed concern about the broadness of the indication proposed by the drug’s maker, Puma Biotechnology, but the majority said the risk-benefit profile of the drug is sufficient to support approval, particularly given the unmet need for such an agent. Those voting against approval argued that more data are needed to identify subpopulations of patients who would be most likely to benefit from treatment in order to narrow the indication.

[email protected]

The surgeons and society leaders involved in this summit want continued collaboration to become a cultural expectation. It is expected that future summits will be held in alternate venues: at the SVS Annual Meeting this year, at the STS Annual Meeting the next time.

“That shows it’s a team effort, that yes, we’re going to care for these complex patients together as a multidisciplinary aortic team, and collaborate nationally to exchange ideas,”according to Dr. Szeto and Dr. Lee.

The surgeons and society leaders involved in this summit want continued collaboration to become a cultural expectation. It is expected that future summits will be held in alternate venues: at the SVS Annual Meeting this year, at the STS Annual Meeting the next time.

“That shows it’s a team effort, that yes, we’re going to care for these complex patients together as a multidisciplinary aortic team, and collaborate nationally to exchange ideas,”according to Dr. Szeto and Dr. Lee.

The surgeons and society leaders involved in this summit want continued collaboration to become a cultural expectation. It is expected that future summits will be held in alternate venues: at the SVS Annual Meeting this year, at the STS Annual Meeting the next time.

“That shows it’s a team effort, that yes, we’re going to care for these complex patients together as a multidisciplinary aortic team, and collaborate nationally to exchange ideas,”according to Dr. Szeto and Dr. Lee.

BOSTON – Last December, the Food and Drug Administration approved nusinersen (Spinraza) as the only treatment for spinal muscular atrophy (SMA) based in part on startling interim results from a study in infants that ended early so all participants could get access to the drug.

Now the final study results are in, and they’re even more impressive: Patients who took the drug were less than half as likely to die as were those in a sham control group, and motor function improved in more than half of infants who took the medication, compared with none in the sham group.

The findings are “incredibly exciting,” said Charlotte J. Sumner, MD, in a discussant presentation at the annual meeting of the American Academy of Neurology. “The data teaches us that this drug is efficacious.”

However, Dr. Sumner, professor of neurology and neuroscience at Johns Hopkins University, Baltimore, cautioned that the drug is tremendously expensive, challenging to administer, and there are several questions about its use that remain unanswered.

SMA is most often caused by mutations in the SMN1 gene, leading to a loss of the protein called survival motor neuron (SMN), which is essential for the survival of motor neurons, except for a small amount produced from the SMN2 gene. The loss of motor neurons in the spinal cord and brainstem leads to weakness and atrophy of muscles used for crawling, walking, sitting up, controlling head movement, and in severe cases, breathing and swallowing. It affects an estimated 1 in 6,000 newborns and is the most common genetic cause of death in infants. Nusinersen is a antisense oligonucleotide that promotes transcription of the full-length SMN protein from the SMN2 gene.

At the AAN meeting, investigators presented the final results of two nusinersen phase III studies as well as interim data from a separate phase II trial.

The ENDEAR trial, a randomized, double-blind, sham-procedure controlled study, assigned 80 patients to 12-mg scaled equivalent doses of nusinersen delivered intrathecally (four doses over 2 months, then one every 4 months) and 41 to a sham procedure (a needle prick in the lower back). The subjects, aged 30-252 days at first dose, underwent 13 months of treatment and follow-up.

Researchers presented an end-of-study analysis of 110 subjects. They found that 51% of those who received the drug were “motor milestone responders” – meaning they had more modified Hammersmith Infant Neurological Examination Section 2 categories with improvement than worsening – compared with 41% at the interim analysis (P less than .0001). None of the sham group patients improved.

The patients treated with nusinersen lived longer: 39% of those in the sham procedure group died, compared with 16% of those in the nusinersen group (hazard ratio, 0.372; P = .0041). Event-free survival (survival without permanent ventilation) was also better in those who received the drugs: 61% in those who took nusinersen, compared with 32% in those who did not (HR, 0.53; P = .0046)

Also, 71% of those treated with the drug were considered responders at day 183 onward based on an improvement of 4 or more points on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale, whereas the response rate was only 3% in the sham group (P less than .0001).

The study authors report that nusinersen was well tolerated with adverse events that “were consistent with those expected in the general population of infants with SMA.” No adverse events were linked to the nusinersen treatment.

Researchers also presented the end-of-study results of the phase III CHERISH trial of 126 patients aged 2-12 years with later-onset SMA who could sit up but not walk. All participants were believed to have a life expectancy of at least 2 years.

The trial is a multicenter, randomized, double-blind, sham-procedure controlled study. The 84 subjects who received the drug improved an average of 3.9 points by the end of study (15 months) on the Hammersmith Functional Motor Scale Expanded score, a measurement of motor function in children with SMA (P = .0000001). The 42 children who received the sham treatment (a small needle prick on the lower back) declined by an average of 1.0 point.

Researchers report that children taking the drug had fewer adverse events, and most were not related to the drug itself.

All surviving participants who completed nusinersen investigational studies are being enrolled in the SHINE open-label extension trial.

Also at the AAN meeting, researchers released interim phase II results from the NURTURE trial, an open-label study of nusinersen in patients with diagnosed but presymptomatic SMA. The study, which is expected to be completed in 2022, recruited participants up to the age of 6 weeks.

The 20 enrolled infants were all alive at a median study period of 317.5 days, and none required assistance with respiration.

In terms of motor function and growth, most were making gains “generally consistent with normal development such as head control, independent sitting, standing and walking independently, as measured by validated scales,” according to a statement released by Biogen.

Three patients developed adverse events that may have been drug related, but the events were resolved and they remained in the trial.

In Dr. Sumner’s discussant presentation on the ENDEAR study, she cautioned that it’s still not known how much older patients with SMA will benefit from the drug treatment. It’s possible that younger patients may gain motor function while older patients will maintain what they have, she said.

In addition, it seems that “certain body segments may respond more robustly than some others. It’s possible that an infant may improve limb strength quite robustly but not lumbar strength.”

She pointed out other questions: Can the drug, which is difficult to administer, be stopped at some point? “We do think of SMA as a disease of development,” she said. “Maybe after development is finished we can withdraw the drug.”

Then there’s the issue of cost and whether insurers are willing to cover the drug. As Dr. Sumner pointed out, the drug is $125,000 per dose and $750,000 over the first year, then $325,000 a year.

It is clear, however, that “the early and efficient diagnosis of SMA is really important, particularly for infantile SMA,” she said. “Time is motor function.”

The studies were funded by Ionis Pharmaceuticals and Biogen. Dr. Sumner reports serving as a consultant for Biogen, Ionis, Avexis, and Roche. She has served on scientific advisory boards for the Cure SMA, SMA Foundation, and Muscular Dystrophy Association nonprofit foundations.

[email protected]

BOSTON – Last December, the Food and Drug Administration approved nusinersen (Spinraza) as the only treatment for spinal muscular atrophy (SMA) based in part on startling interim results from a study in infants that ended early so all participants could get access to the drug.

Now the final study results are in, and they’re even more impressive: Patients who took the drug were less than half as likely to die as were those in a sham control group, and motor function improved in more than half of infants who took the medication, compared with none in the sham group.

The findings are “incredibly exciting,” said Charlotte J. Sumner, MD, in a discussant presentation at the annual meeting of the American Academy of Neurology. “The data teaches us that this drug is efficacious.”

However, Dr. Sumner, professor of neurology and neuroscience at Johns Hopkins University, Baltimore, cautioned that the drug is tremendously expensive, challenging to administer, and there are several questions about its use that remain unanswered.

SMA is most often caused by mutations in the SMN1 gene, leading to a loss of the protein called survival motor neuron (SMN), which is essential for the survival of motor neurons, except for a small amount produced from the SMN2 gene. The loss of motor neurons in the spinal cord and brainstem leads to weakness and atrophy of muscles used for crawling, walking, sitting up, controlling head movement, and in severe cases, breathing and swallowing. It affects an estimated 1 in 6,000 newborns and is the most common genetic cause of death in infants. Nusinersen is a antisense oligonucleotide that promotes transcription of the full-length SMN protein from the SMN2 gene.

At the AAN meeting, investigators presented the final results of two nusinersen phase III studies as well as interim data from a separate phase II trial.

The ENDEAR trial, a randomized, double-blind, sham-procedure controlled study, assigned 80 patients to 12-mg scaled equivalent doses of nusinersen delivered intrathecally (four doses over 2 months, then one every 4 months) and 41 to a sham procedure (a needle prick in the lower back). The subjects, aged 30-252 days at first dose, underwent 13 months of treatment and follow-up.

Researchers presented an end-of-study analysis of 110 subjects. They found that 51% of those who received the drug were “motor milestone responders” – meaning they had more modified Hammersmith Infant Neurological Examination Section 2 categories with improvement than worsening – compared with 41% at the interim analysis (P less than .0001). None of the sham group patients improved.

The patients treated with nusinersen lived longer: 39% of those in the sham procedure group died, compared with 16% of those in the nusinersen group (hazard ratio, 0.372; P = .0041). Event-free survival (survival without permanent ventilation) was also better in those who received the drugs: 61% in those who took nusinersen, compared with 32% in those who did not (HR, 0.53; P = .0046)

Also, 71% of those treated with the drug were considered responders at day 183 onward based on an improvement of 4 or more points on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale, whereas the response rate was only 3% in the sham group (P less than .0001).

The study authors report that nusinersen was well tolerated with adverse events that “were consistent with those expected in the general population of infants with SMA.” No adverse events were linked to the nusinersen treatment.

Researchers also presented the end-of-study results of the phase III CHERISH trial of 126 patients aged 2-12 years with later-onset SMA who could sit up but not walk. All participants were believed to have a life expectancy of at least 2 years.

The trial is a multicenter, randomized, double-blind, sham-procedure controlled study. The 84 subjects who received the drug improved an average of 3.9 points by the end of study (15 months) on the Hammersmith Functional Motor Scale Expanded score, a measurement of motor function in children with SMA (P = .0000001). The 42 children who received the sham treatment (a small needle prick on the lower back) declined by an average of 1.0 point.

Researchers report that children taking the drug had fewer adverse events, and most were not related to the drug itself.

All surviving participants who completed nusinersen investigational studies are being enrolled in the SHINE open-label extension trial.

Also at the AAN meeting, researchers released interim phase II results from the NURTURE trial, an open-label study of nusinersen in patients with diagnosed but presymptomatic SMA. The study, which is expected to be completed in 2022, recruited participants up to the age of 6 weeks.

The 20 enrolled infants were all alive at a median study period of 317.5 days, and none required assistance with respiration.

In terms of motor function and growth, most were making gains “generally consistent with normal development such as head control, independent sitting, standing and walking independently, as measured by validated scales,” according to a statement released by Biogen.

Three patients developed adverse events that may have been drug related, but the events were resolved and they remained in the trial.

In Dr. Sumner’s discussant presentation on the ENDEAR study, she cautioned that it’s still not known how much older patients with SMA will benefit from the drug treatment. It’s possible that younger patients may gain motor function while older patients will maintain what they have, she said.

In addition, it seems that “certain body segments may respond more robustly than some others. It’s possible that an infant may improve limb strength quite robustly but not lumbar strength.”

She pointed out other questions: Can the drug, which is difficult to administer, be stopped at some point? “We do think of SMA as a disease of development,” she said. “Maybe after development is finished we can withdraw the drug.”

Then there’s the issue of cost and whether insurers are willing to cover the drug. As Dr. Sumner pointed out, the drug is $125,000 per dose and $750,000 over the first year, then $325,000 a year.

It is clear, however, that “the early and efficient diagnosis of SMA is really important, particularly for infantile SMA,” she said. “Time is motor function.”

The studies were funded by Ionis Pharmaceuticals and Biogen. Dr. Sumner reports serving as a consultant for Biogen, Ionis, Avexis, and Roche. She has served on scientific advisory boards for the Cure SMA, SMA Foundation, and Muscular Dystrophy Association nonprofit foundations.

[email protected]

BOSTON – Last December, the Food and Drug Administration approved nusinersen (Spinraza) as the only treatment for spinal muscular atrophy (SMA) based in part on startling interim results from a study in infants that ended early so all participants could get access to the drug.

Now the final study results are in, and they’re even more impressive: Patients who took the drug were less than half as likely to die as were those in a sham control group, and motor function improved in more than half of infants who took the medication, compared with none in the sham group.

The findings are “incredibly exciting,” said Charlotte J. Sumner, MD, in a discussant presentation at the annual meeting of the American Academy of Neurology. “The data teaches us that this drug is efficacious.”

However, Dr. Sumner, professor of neurology and neuroscience at Johns Hopkins University, Baltimore, cautioned that the drug is tremendously expensive, challenging to administer, and there are several questions about its use that remain unanswered.

SMA is most often caused by mutations in the SMN1 gene, leading to a loss of the protein called survival motor neuron (SMN), which is essential for the survival of motor neurons, except for a small amount produced from the SMN2 gene. The loss of motor neurons in the spinal cord and brainstem leads to weakness and atrophy of muscles used for crawling, walking, sitting up, controlling head movement, and in severe cases, breathing and swallowing. It affects an estimated 1 in 6,000 newborns and is the most common genetic cause of death in infants. Nusinersen is a antisense oligonucleotide that promotes transcription of the full-length SMN protein from the SMN2 gene.

At the AAN meeting, investigators presented the final results of two nusinersen phase III studies as well as interim data from a separate phase II trial.

The ENDEAR trial, a randomized, double-blind, sham-procedure controlled study, assigned 80 patients to 12-mg scaled equivalent doses of nusinersen delivered intrathecally (four doses over 2 months, then one every 4 months) and 41 to a sham procedure (a needle prick in the lower back). The subjects, aged 30-252 days at first dose, underwent 13 months of treatment and follow-up.

Researchers presented an end-of-study analysis of 110 subjects. They found that 51% of those who received the drug were “motor milestone responders” – meaning they had more modified Hammersmith Infant Neurological Examination Section 2 categories with improvement than worsening – compared with 41% at the interim analysis (P less than .0001). None of the sham group patients improved.

The patients treated with nusinersen lived longer: 39% of those in the sham procedure group died, compared with 16% of those in the nusinersen group (hazard ratio, 0.372; P = .0041). Event-free survival (survival without permanent ventilation) was also better in those who received the drugs: 61% in those who took nusinersen, compared with 32% in those who did not (HR, 0.53; P = .0046)

Also, 71% of those treated with the drug were considered responders at day 183 onward based on an improvement of 4 or more points on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale, whereas the response rate was only 3% in the sham group (P less than .0001).

The study authors report that nusinersen was well tolerated with adverse events that “were consistent with those expected in the general population of infants with SMA.” No adverse events were linked to the nusinersen treatment.

Researchers also presented the end-of-study results of the phase III CHERISH trial of 126 patients aged 2-12 years with later-onset SMA who could sit up but not walk. All participants were believed to have a life expectancy of at least 2 years.

The trial is a multicenter, randomized, double-blind, sham-procedure controlled study. The 84 subjects who received the drug improved an average of 3.9 points by the end of study (15 months) on the Hammersmith Functional Motor Scale Expanded score, a measurement of motor function in children with SMA (P = .0000001). The 42 children who received the sham treatment (a small needle prick on the lower back) declined by an average of 1.0 point.

Researchers report that children taking the drug had fewer adverse events, and most were not related to the drug itself.

All surviving participants who completed nusinersen investigational studies are being enrolled in the SHINE open-label extension trial.

Also at the AAN meeting, researchers released interim phase II results from the NURTURE trial, an open-label study of nusinersen in patients with diagnosed but presymptomatic SMA. The study, which is expected to be completed in 2022, recruited participants up to the age of 6 weeks.

The 20 enrolled infants were all alive at a median study period of 317.5 days, and none required assistance with respiration.

In terms of motor function and growth, most were making gains “generally consistent with normal development such as head control, independent sitting, standing and walking independently, as measured by validated scales,” according to a statement released by Biogen.

Three patients developed adverse events that may have been drug related, but the events were resolved and they remained in the trial.

In Dr. Sumner’s discussant presentation on the ENDEAR study, she cautioned that it’s still not known how much older patients with SMA will benefit from the drug treatment. It’s possible that younger patients may gain motor function while older patients will maintain what they have, she said.

In addition, it seems that “certain body segments may respond more robustly than some others. It’s possible that an infant may improve limb strength quite robustly but not lumbar strength.”

She pointed out other questions: Can the drug, which is difficult to administer, be stopped at some point? “We do think of SMA as a disease of development,” she said. “Maybe after development is finished we can withdraw the drug.”

Then there’s the issue of cost and whether insurers are willing to cover the drug. As Dr. Sumner pointed out, the drug is $125,000 per dose and $750,000 over the first year, then $325,000 a year.

It is clear, however, that “the early and efficient diagnosis of SMA is really important, particularly for infantile SMA,” she said. “Time is motor function.”

The studies were funded by Ionis Pharmaceuticals and Biogen. Dr. Sumner reports serving as a consultant for Biogen, Ionis, Avexis, and Roche. She has served on scientific advisory boards for the Cure SMA, SMA Foundation, and Muscular Dystrophy Association nonprofit foundations.

[email protected]

The Food and Drug Administration has approved the first generic versions of Strattera (atomoxetine) for the treatment of attention-deficit/hyperactivity disorder, the agency announced May 30.

Apotex, Teva Pharmaceuticals USA, Aurobindo Pharma, and Glenmark Pharmaceuticals all gained approval to market generic atomoxetine at various strengths. All versions must be sold with a patient medication guide describing the uses and risks of atomoxetine and must also include a boxed warning detailing the potential for increased risk of suicidal ideation in children and adolescents.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

[email protected]

The Food and Drug Administration has approved the first generic versions of Strattera (atomoxetine) for the treatment of attention-deficit/hyperactivity disorder, the agency announced May 30.

Apotex, Teva Pharmaceuticals USA, Aurobindo Pharma, and Glenmark Pharmaceuticals all gained approval to market generic atomoxetine at various strengths. All versions must be sold with a patient medication guide describing the uses and risks of atomoxetine and must also include a boxed warning detailing the potential for increased risk of suicidal ideation in children and adolescents.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

[email protected]

The Food and Drug Administration has approved the first generic versions of Strattera (atomoxetine) for the treatment of attention-deficit/hyperactivity disorder, the agency announced May 30.

Apotex, Teva Pharmaceuticals USA, Aurobindo Pharma, and Glenmark Pharmaceuticals all gained approval to market generic atomoxetine at various strengths. All versions must be sold with a patient medication guide describing the uses and risks of atomoxetine and must also include a boxed warning detailing the potential for increased risk of suicidal ideation in children and adolescents.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

[email protected]

NEW ORLEANS—Most patients with relapsing-remitting multiple sclerosis (MS) with highly active disease remain free of new MRI activity for six years after receiving alemtuzumab, according to research presented at the 31st Annual Meeting of the Consortium of MS Centers. The durable effects may result from the distinct pattern of lymphocyte repopulation following treatment with alemtuzumab, which may lead to a rebalancing of the immune system, said Anthony Traboulsee, MD, Associate Professor of Neurology at the University of British Columbia in Vancouver. Additional mechanistic studies are needed to establish this hypothesis, the authors said.

To evaluate six-year MRI and brain volume loss outcomes in a subset of patient with highly active disease at baseline who were treated with alemtuzumab, Dr. Traboulsee and colleagues analyzed data from the CARE-MS I trial. In that trial, researchers randomized patients with active relapsing-remitting MS who were drug-naïve at baseline to alemtuzumab or interferon beta-1a. After two years, patients who received alemtuzumab had improved clinical and MRI outcomes, including brain loss volume, compared with patients who received interferon beta-1a. In addition, significantly more of these patients had no evidence of disease activity, compared with patients treated with interferon beta1-a.

In an extension study, patients could receive additional treatment with alemtuzumab (12 mg/day on three consecutive days one year or more after the most recent course) as needed for relapse or radiologic activity. During the extension, patients also could receive treatment with other licensed disease-modifying therapies at an investigator’s discretion. Retreatment criteria included one or more relapse, two or more new or enlarging T2 hyperintense lesions, or new gadolinium-enhancing T1 brain or spinal cord lesions on MRI. Researchers defined highly active disease as two or more relapses a year prior to randomization and one or more gadolinium-enhancing T1 lesion at core study baseline.

The researchers obtained MRI scans at baseline and yearly thereafter. Experts masked to patients’ original treatment group assignment analyzed the MRI scans. Investigators assessed the proportion of patients free of MRI disease activity (ie, no new or enlarging T2 hyperintense lesions or new gadolinium-enhancing T1 lesions) and the proportion of patients free of new non-enhancing T1 lesions. Brain volume loss was derived by brain parenchymal fraction change.

Of 376 patients who received alemtuzumab in the CARE-MS I trial, 105 met the criteria for highly active disease at core study baseline. Of these 105 patients, 99 remained in the study through Year 6. Through six years, 63% of patients did not receive additional treatment with alemtuzumab or another disease-modifying therapy. In each year through Year 6, a majority of patients were free of MRI disease activity (61 % at Year 6), free of new gadolinium-enhancing T1 lesions (83% at Year 6), and free of new or enlarging T2 hyperintense lesions (62% at Year 6). The investigators also observed that annually in Years 2 through 6, high proportions of patients had no new non-enhancing T1 hypointense lesions.

“These findings, along with long-term clinical efficacy, suggest that alemtuzumab may provide a unique treatment approach for relapsing-remitting MS patients with highly active disease, offering durable efficacy in the absence of continuous treatment,” said Dr. Traboulsee and colleagues.

NEW ORLEANS—Most patients with relapsing-remitting multiple sclerosis (MS) with highly active disease remain free of new MRI activity for six years after receiving alemtuzumab, according to research presented at the 31st Annual Meeting of the Consortium of MS Centers. The durable effects may result from the distinct pattern of lymphocyte repopulation following treatment with alemtuzumab, which may lead to a rebalancing of the immune system, said Anthony Traboulsee, MD, Associate Professor of Neurology at the University of British Columbia in Vancouver. Additional mechanistic studies are needed to establish this hypothesis, the authors said.

To evaluate six-year MRI and brain volume loss outcomes in a subset of patient with highly active disease at baseline who were treated with alemtuzumab, Dr. Traboulsee and colleagues analyzed data from the CARE-MS I trial. In that trial, researchers randomized patients with active relapsing-remitting MS who were drug-naïve at baseline to alemtuzumab or interferon beta-1a. After two years, patients who received alemtuzumab had improved clinical and MRI outcomes, including brain loss volume, compared with patients who received interferon beta-1a. In addition, significantly more of these patients had no evidence of disease activity, compared with patients treated with interferon beta1-a.

In an extension study, patients could receive additional treatment with alemtuzumab (12 mg/day on three consecutive days one year or more after the most recent course) as needed for relapse or radiologic activity. During the extension, patients also could receive treatment with other licensed disease-modifying therapies at an investigator’s discretion. Retreatment criteria included one or more relapse, two or more new or enlarging T2 hyperintense lesions, or new gadolinium-enhancing T1 brain or spinal cord lesions on MRI. Researchers defined highly active disease as two or more relapses a year prior to randomization and one or more gadolinium-enhancing T1 lesion at core study baseline.

The researchers obtained MRI scans at baseline and yearly thereafter. Experts masked to patients’ original treatment group assignment analyzed the MRI scans. Investigators assessed the proportion of patients free of MRI disease activity (ie, no new or enlarging T2 hyperintense lesions or new gadolinium-enhancing T1 lesions) and the proportion of patients free of new non-enhancing T1 lesions. Brain volume loss was derived by brain parenchymal fraction change.

Of 376 patients who received alemtuzumab in the CARE-MS I trial, 105 met the criteria for highly active disease at core study baseline. Of these 105 patients, 99 remained in the study through Year 6. Through six years, 63% of patients did not receive additional treatment with alemtuzumab or another disease-modifying therapy. In each year through Year 6, a majority of patients were free of MRI disease activity (61 % at Year 6), free of new gadolinium-enhancing T1 lesions (83% at Year 6), and free of new or enlarging T2 hyperintense lesions (62% at Year 6). The investigators also observed that annually in Years 2 through 6, high proportions of patients had no new non-enhancing T1 hypointense lesions.

“These findings, along with long-term clinical efficacy, suggest that alemtuzumab may provide a unique treatment approach for relapsing-remitting MS patients with highly active disease, offering durable efficacy in the absence of continuous treatment,” said Dr. Traboulsee and colleagues.

NEW ORLEANS—Most patients with relapsing-remitting multiple sclerosis (MS) with highly active disease remain free of new MRI activity for six years after receiving alemtuzumab, according to research presented at the 31st Annual Meeting of the Consortium of MS Centers. The durable effects may result from the distinct pattern of lymphocyte repopulation following treatment with alemtuzumab, which may lead to a rebalancing of the immune system, said Anthony Traboulsee, MD, Associate Professor of Neurology at the University of British Columbia in Vancouver. Additional mechanistic studies are needed to establish this hypothesis, the authors said.

To evaluate six-year MRI and brain volume loss outcomes in a subset of patient with highly active disease at baseline who were treated with alemtuzumab, Dr. Traboulsee and colleagues analyzed data from the CARE-MS I trial. In that trial, researchers randomized patients with active relapsing-remitting MS who were drug-naïve at baseline to alemtuzumab or interferon beta-1a. After two years, patients who received alemtuzumab had improved clinical and MRI outcomes, including brain loss volume, compared with patients who received interferon beta-1a. In addition, significantly more of these patients had no evidence of disease activity, compared with patients treated with interferon beta1-a.

In an extension study, patients could receive additional treatment with alemtuzumab (12 mg/day on three consecutive days one year or more after the most recent course) as needed for relapse or radiologic activity. During the extension, patients also could receive treatment with other licensed disease-modifying therapies at an investigator’s discretion. Retreatment criteria included one or more relapse, two or more new or enlarging T2 hyperintense lesions, or new gadolinium-enhancing T1 brain or spinal cord lesions on MRI. Researchers defined highly active disease as two or more relapses a year prior to randomization and one or more gadolinium-enhancing T1 lesion at core study baseline.

The researchers obtained MRI scans at baseline and yearly thereafter. Experts masked to patients’ original treatment group assignment analyzed the MRI scans. Investigators assessed the proportion of patients free of MRI disease activity (ie, no new or enlarging T2 hyperintense lesions or new gadolinium-enhancing T1 lesions) and the proportion of patients free of new non-enhancing T1 lesions. Brain volume loss was derived by brain parenchymal fraction change.

Of 376 patients who received alemtuzumab in the CARE-MS I trial, 105 met the criteria for highly active disease at core study baseline. Of these 105 patients, 99 remained in the study through Year 6. Through six years, 63% of patients did not receive additional treatment with alemtuzumab or another disease-modifying therapy. In each year through Year 6, a majority of patients were free of MRI disease activity (61 % at Year 6), free of new gadolinium-enhancing T1 lesions (83% at Year 6), and free of new or enlarging T2 hyperintense lesions (62% at Year 6). The investigators also observed that annually in Years 2 through 6, high proportions of patients had no new non-enhancing T1 hypointense lesions.

“These findings, along with long-term clinical efficacy, suggest that alemtuzumab may provide a unique treatment approach for relapsing-remitting MS patients with highly active disease, offering durable efficacy in the absence of continuous treatment,” said Dr. Traboulsee and colleagues.

BOSTON―Stress may be a modifiable risk factor for Parkinson’s disease progression, according to research presented at the 69th Annual Meeting of the American Academy of Neurology. In a study of more than 4,000 patients, a stress proxy score predicted mortality and was associated with worsening mobility. The findings suggest that stress reduction may be an effective intervention in Parkinson’s disease, said Amie Hiller, MD, Assistant Professor of Neurology at the Oregon Health and Science University in Portland.

“Potentially, stress reduction is something we could think about to slow Parkinson’s disease progression,” said Dr. Hiller. “Our goal is to not only treat symptoms of Parkinson’s disease, but to slow progression of the disease.”

Research suggests that stressful life events may increase the risk of Parkinson’s disease. In addition, animal studies indicate that stress damages dopamine cells, resulting in more severe parkinsonian symptoms.  In humans, acute stress can worsen motor symptoms, including bradykinesia, freezing, and tremor.

To examine the relationship between psychological stress and Parkinson’s disease progression, Dr. Hiller and colleagues analyzed data from the National Parkinson’s Foundation Quality Improvement Initiative. All 4,155 participants in the study were able to walk unassisted at baseline.

For each patient, investigators calculated a stress proxy score derived from the 39-item Parkinson’s Disease Questionnaire (PDQ-39) and the Multidimensional Caregiver Strain Index. They also calculated a mobility proxy score derived from the PDQ-39 and the Instrumented Timed Up and Go, and an overall health score derived from the PDQ-39, falls score, hospital admissions, and cognitive score.

Researchers also calculated patients’ levels of excess stress (ie, emotional stress above typical stress resulting from disease severity and overall health).

The stress proxy score predicted mortality, but the excess stress score did not. High baseline stress proxy scores and high levels of excess stress predicted worsening mobility.

“We need better data, as these were not data that we collected specifically to look at stress,” Dr. Hiller said. In addition, researchers need to conduct intervention studies to see if stress reduction benefits patients with Parkinson’s disease, she said.

—Erica Tricarico

BOSTON―Stress may be a modifiable risk factor for Parkinson’s disease progression, according to research presented at the 69th Annual Meeting of the American Academy of Neurology. In a study of more than 4,000 patients, a stress proxy score predicted mortality and was associated with worsening mobility. The findings suggest that stress reduction may be an effective intervention in Parkinson’s disease, said Amie Hiller, MD, Assistant Professor of Neurology at the Oregon Health and Science University in Portland.

“Potentially, stress reduction is something we could think about to slow Parkinson’s disease progression,” said Dr. Hiller. “Our goal is to not only treat symptoms of Parkinson’s disease, but to slow progression of the disease.”

Research suggests that stressful life events may increase the risk of Parkinson’s disease. In addition, animal studies indicate that stress damages dopamine cells, resulting in more severe parkinsonian symptoms.  In humans, acute stress can worsen motor symptoms, including bradykinesia, freezing, and tremor.

To examine the relationship between psychological stress and Parkinson’s disease progression, Dr. Hiller and colleagues analyzed data from the National Parkinson’s Foundation Quality Improvement Initiative. All 4,155 participants in the study were able to walk unassisted at baseline.

For each patient, investigators calculated a stress proxy score derived from the 39-item Parkinson’s Disease Questionnaire (PDQ-39) and the Multidimensional Caregiver Strain Index. They also calculated a mobility proxy score derived from the PDQ-39 and the Instrumented Timed Up and Go, and an overall health score derived from the PDQ-39, falls score, hospital admissions, and cognitive score.

Researchers also calculated patients’ levels of excess stress (ie, emotional stress above typical stress resulting from disease severity and overall health).

The stress proxy score predicted mortality, but the excess stress score did not. High baseline stress proxy scores and high levels of excess stress predicted worsening mobility.

“We need better data, as these were not data that we collected specifically to look at stress,” Dr. Hiller said. In addition, researchers need to conduct intervention studies to see if stress reduction benefits patients with Parkinson’s disease, she said.

—Erica Tricarico

BOSTON―Stress may be a modifiable risk factor for Parkinson’s disease progression, according to research presented at the 69th Annual Meeting of the American Academy of Neurology. In a study of more than 4,000 patients, a stress proxy score predicted mortality and was associated with worsening mobility. The findings suggest that stress reduction may be an effective intervention in Parkinson’s disease, said Amie Hiller, MD, Assistant Professor of Neurology at the Oregon Health and Science University in Portland.

“Potentially, stress reduction is something we could think about to slow Parkinson’s disease progression,” said Dr. Hiller. “Our goal is to not only treat symptoms of Parkinson’s disease, but to slow progression of the disease.”

Research suggests that stressful life events may increase the risk of Parkinson’s disease. In addition, animal studies indicate that stress damages dopamine cells, resulting in more severe parkinsonian symptoms.  In humans, acute stress can worsen motor symptoms, including bradykinesia, freezing, and tremor.

To examine the relationship between psychological stress and Parkinson’s disease progression, Dr. Hiller and colleagues analyzed data from the National Parkinson’s Foundation Quality Improvement Initiative. All 4,155 participants in the study were able to walk unassisted at baseline.

For each patient, investigators calculated a stress proxy score derived from the 39-item Parkinson’s Disease Questionnaire (PDQ-39) and the Multidimensional Caregiver Strain Index. They also calculated a mobility proxy score derived from the PDQ-39 and the Instrumented Timed Up and Go, and an overall health score derived from the PDQ-39, falls score, hospital admissions, and cognitive score.

Researchers also calculated patients’ levels of excess stress (ie, emotional stress above typical stress resulting from disease severity and overall health).

The stress proxy score predicted mortality, but the excess stress score did not. High baseline stress proxy scores and high levels of excess stress predicted worsening mobility.

“We need better data, as these were not data that we collected specifically to look at stress,” Dr. Hiller said. In addition, researchers need to conduct intervention studies to see if stress reduction benefits patients with Parkinson’s disease, she said.

—Erica Tricarico

BOSTON—Patients with epilepsy not only want to know about sudden unexpected death in epilepsy (SUDEP), they feel it is their right to be informed, according to survey results presented at the 69th Annual Meeting of the American Academy of Neurology. “The most astonishing finding of our study was that 100% of participants felt it was their right to be informed about SUDEP,” said Lucretia Long, MS, RN, CNP, Clinical Assistant Professor of Neurology and an epilepsy nurse practitioner at Ohio State University in Columbus.

Patients with epilepsy are 24 times more likely to die suddenly, compared with age-matched controls. SUDEP is responsible for 2% to 18% of epilepsy-related deaths and is the most common epilepsy-related cause of death, particularly in patients with intractable seizures. SUDEP takes more lives annually in the US than sudden infant death syndrome (SIDS).

The decision to discuss SUDEP with patients has created much debate over the years. “Many physicians are hesitant to discuss this, due to their perception of imposing unnecessary fear, while others argue that SUDEP disclosure could aid in improving patient compliance and also encourage patients to modify factors that can lower seizure thresholds,” Ms. Long explained.

The Patient's Perspective

SUDEP etiology has been studied, as have physician perspectives on the matter, but limited data exist regarding patients’ feelings about SUDEP disclosure, Ms. Long said.

She and colleagues assessed via questionnaire adult patient feelings and viewpoints associated with SUDEP. For their study, every third patient at the Comprehensive Epilepsy Center at the Wexner Medical Center at Ohio State University was given a one-page SUDEP information sheet. The information sheet is available on www.epilepsy.com and was developed by the National Epilepsy Foundation of America. Patients who consented were then asked to complete an eight-item questionnaire assessing their perception of SUDEP disclosure.

To be eligible for the study, patients had to have a confirmed diagnosis of epilepsy and be able to read and write English. Those with psychogenic nonepileptic seizures or other psychiatric conditions were excluded. However, patients with comorbid depression were eligible to participate in the study.

A total of 67 patients received the handout; 97% of those patients completed the questionnaire. Fifty-eight percent were female, the overwhelming majority of patients had a high school diploma or associate degree, and most patients resided with family members or caregivers. The majority of patients had a long-standing history of epilepsy; 76% had been diagnosed with epilepsy for 10 years or longer, and almost 46% of patients had a history of generalized tonic-clonic seizures. “A history of tonic-clonic seizures is important, particularly as we look at risk factor reduction,” Ms. Long said. “We know, for example, that patients with tonic-clonic seizures, especially nocturnal intractable tonic-clonic seizures, are more likely to die from SUDEP.”

A Right to Know

Survey results revealed that 100% of respondents felt it was their right to be informed about SUDEP. “This was regardless of participant education, sex, and age,” Ms. Long reported. “Both male and female, young and old, and those with higher versus lower education levels all felt that it was their right to be informed about SUDEP.” About 84% of patients also felt that SUDEP disclosure would encourage them to be more adherent to their antiepileptic drug regimen, and 87% felt that SUDEP disclosure would encourage them to avoid factors that can lower seizure threshold.

Thirty percent of patients surveyed admitted that SUDEP disclosure made them more frightened. “This is particularly interesting because most physicians are a little hesitant to discuss this due to fear,” Ms. Long said. “However, 100% of the 30% still felt it was their right to be informed.”

When asked when patients should be informed, 90% of patients felt they should be informed shortly after diagnosis, and 50% felt that they should be informed about SUDEP at first diagnosis.

“Patients not only want to be informed about SUDEP, they actually feel it is their right to be informed,” Ms. Long said. “In our study, patients felt that physicians should be responsible for disclosing SUDEP and that SUDEP awareness motivated them to be more compliant and also to better manage those factors that lower seizure threshold.”

In summation, Ms. Long cautioned against generalizing her study findings. “These were patients with a long-standing history of epilepsy—10 years or longer. These were not patients who were newly diagnosed. But a very interesting finding is that 50% felt they should be informed at first
diagnosis.”

—Glenn S. Williams

Suggested Reading

Keddie S, Angus-Leppan H, Parker T, et al. Discussing sudden unexpected death in epilepsy: are we empowering our patients? A questionnaire survey. JRSM Open. 2016;7(9):2054270416654358.

Xu Z, Ayyappan S, Seneviratne U. Sudden unexpected death in epilepsy (SUDEP): what do patients think? Epilepsy Behav. 2015;42:29-34.

BOSTON—Patients with epilepsy not only want to know about sudden unexpected death in epilepsy (SUDEP), they feel it is their right to be informed, according to survey results presented at the 69th Annual Meeting of the American Academy of Neurology. “The most astonishing finding of our study was that 100% of participants felt it was their right to be informed about SUDEP,” said Lucretia Long, MS, RN, CNP, Clinical Assistant Professor of Neurology and an epilepsy nurse practitioner at Ohio State University in Columbus.

Patients with epilepsy are 24 times more likely to die suddenly, compared with age-matched controls. SUDEP is responsible for 2% to 18% of epilepsy-related deaths and is the most common epilepsy-related cause of death, particularly in patients with intractable seizures. SUDEP takes more lives annually in the US than sudden infant death syndrome (SIDS).

The decision to discuss SUDEP with patients has created much debate over the years. “Many physicians are hesitant to discuss this, due to their perception of imposing unnecessary fear, while others argue that SUDEP disclosure could aid in improving patient compliance and also encourage patients to modify factors that can lower seizure thresholds,” Ms. Long explained.

The Patient's Perspective

SUDEP etiology has been studied, as have physician perspectives on the matter, but limited data exist regarding patients’ feelings about SUDEP disclosure, Ms. Long said.

She and colleagues assessed via questionnaire adult patient feelings and viewpoints associated with SUDEP. For their study, every third patient at the Comprehensive Epilepsy Center at the Wexner Medical Center at Ohio State University was given a one-page SUDEP information sheet. The information sheet is available on www.epilepsy.com and was developed by the National Epilepsy Foundation of America. Patients who consented were then asked to complete an eight-item questionnaire assessing their perception of SUDEP disclosure.

To be eligible for the study, patients had to have a confirmed diagnosis of epilepsy and be able to read and write English. Those with psychogenic nonepileptic seizures or other psychiatric conditions were excluded. However, patients with comorbid depression were eligible to participate in the study.

A total of 67 patients received the handout; 97% of those patients completed the questionnaire. Fifty-eight percent were female, the overwhelming majority of patients had a high school diploma or associate degree, and most patients resided with family members or caregivers. The majority of patients had a long-standing history of epilepsy; 76% had been diagnosed with epilepsy for 10 years or longer, and almost 46% of patients had a history of generalized tonic-clonic seizures. “A history of tonic-clonic seizures is important, particularly as we look at risk factor reduction,” Ms. Long said. “We know, for example, that patients with tonic-clonic seizures, especially nocturnal intractable tonic-clonic seizures, are more likely to die from SUDEP.”

A Right to Know

Survey results revealed that 100% of respondents felt it was their right to be informed about SUDEP. “This was regardless of participant education, sex, and age,” Ms. Long reported. “Both male and female, young and old, and those with higher versus lower education levels all felt that it was their right to be informed about SUDEP.” About 84% of patients also felt that SUDEP disclosure would encourage them to be more adherent to their antiepileptic drug regimen, and 87% felt that SUDEP disclosure would encourage them to avoid factors that can lower seizure threshold.

Thirty percent of patients surveyed admitted that SUDEP disclosure made them more frightened. “This is particularly interesting because most physicians are a little hesitant to discuss this due to fear,” Ms. Long said. “However, 100% of the 30% still felt it was their right to be informed.”

When asked when patients should be informed, 90% of patients felt they should be informed shortly after diagnosis, and 50% felt that they should be informed about SUDEP at first diagnosis.

“Patients not only want to be informed about SUDEP, they actually feel it is their right to be informed,” Ms. Long said. “In our study, patients felt that physicians should be responsible for disclosing SUDEP and that SUDEP awareness motivated them to be more compliant and also to better manage those factors that lower seizure threshold.”

In summation, Ms. Long cautioned against generalizing her study findings. “These were patients with a long-standing history of epilepsy—10 years or longer. These were not patients who were newly diagnosed. But a very interesting finding is that 50% felt they should be informed at first
diagnosis.”

—Glenn S. Williams

Suggested Reading

Keddie S, Angus-Leppan H, Parker T, et al. Discussing sudden unexpected death in epilepsy: are we empowering our patients? A questionnaire survey. JRSM Open. 2016;7(9):2054270416654358.

Xu Z, Ayyappan S, Seneviratne U. Sudden unexpected death in epilepsy (SUDEP): what do patients think? Epilepsy Behav. 2015;42:29-34.

BOSTON—Patients with epilepsy not only want to know about sudden unexpected death in epilepsy (SUDEP), they feel it is their right to be informed, according to survey results presented at the 69th Annual Meeting of the American Academy of Neurology. “The most astonishing finding of our study was that 100% of participants felt it was their right to be informed about SUDEP,” said Lucretia Long, MS, RN, CNP, Clinical Assistant Professor of Neurology and an epilepsy nurse practitioner at Ohio State University in Columbus.

Patients with epilepsy are 24 times more likely to die suddenly, compared with age-matched controls. SUDEP is responsible for 2% to 18% of epilepsy-related deaths and is the most common epilepsy-related cause of death, particularly in patients with intractable seizures. SUDEP takes more lives annually in the US than sudden infant death syndrome (SIDS).

The decision to discuss SUDEP with patients has created much debate over the years. “Many physicians are hesitant to discuss this, due to their perception of imposing unnecessary fear, while others argue that SUDEP disclosure could aid in improving patient compliance and also encourage patients to modify factors that can lower seizure thresholds,” Ms. Long explained.

The Patient's Perspective

SUDEP etiology has been studied, as have physician perspectives on the matter, but limited data exist regarding patients’ feelings about SUDEP disclosure, Ms. Long said.

She and colleagues assessed via questionnaire adult patient feelings and viewpoints associated with SUDEP. For their study, every third patient at the Comprehensive Epilepsy Center at the Wexner Medical Center at Ohio State University was given a one-page SUDEP information sheet. The information sheet is available on www.epilepsy.com and was developed by the National Epilepsy Foundation of America. Patients who consented were then asked to complete an eight-item questionnaire assessing their perception of SUDEP disclosure.

To be eligible for the study, patients had to have a confirmed diagnosis of epilepsy and be able to read and write English. Those with psychogenic nonepileptic seizures or other psychiatric conditions were excluded. However, patients with comorbid depression were eligible to participate in the study.

A total of 67 patients received the handout; 97% of those patients completed the questionnaire. Fifty-eight percent were female, the overwhelming majority of patients had a high school diploma or associate degree, and most patients resided with family members or caregivers. The majority of patients had a long-standing history of epilepsy; 76% had been diagnosed with epilepsy for 10 years or longer, and almost 46% of patients had a history of generalized tonic-clonic seizures. “A history of tonic-clonic seizures is important, particularly as we look at risk factor reduction,” Ms. Long said. “We know, for example, that patients with tonic-clonic seizures, especially nocturnal intractable tonic-clonic seizures, are more likely to die from SUDEP.”

A Right to Know

Survey results revealed that 100% of respondents felt it was their right to be informed about SUDEP. “This was regardless of participant education, sex, and age,” Ms. Long reported. “Both male and female, young and old, and those with higher versus lower education levels all felt that it was their right to be informed about SUDEP.” About 84% of patients also felt that SUDEP disclosure would encourage them to be more adherent to their antiepileptic drug regimen, and 87% felt that SUDEP disclosure would encourage them to avoid factors that can lower seizure threshold.

Thirty percent of patients surveyed admitted that SUDEP disclosure made them more frightened. “This is particularly interesting because most physicians are a little hesitant to discuss this due to fear,” Ms. Long said. “However, 100% of the 30% still felt it was their right to be informed.”

When asked when patients should be informed, 90% of patients felt they should be informed shortly after diagnosis, and 50% felt that they should be informed about SUDEP at first diagnosis.

“Patients not only want to be informed about SUDEP, they actually feel it is their right to be informed,” Ms. Long said. “In our study, patients felt that physicians should be responsible for disclosing SUDEP and that SUDEP awareness motivated them to be more compliant and also to better manage those factors that lower seizure threshold.”

In summation, Ms. Long cautioned against generalizing her study findings. “These were patients with a long-standing history of epilepsy—10 years or longer. These were not patients who were newly diagnosed. But a very interesting finding is that 50% felt they should be informed at first
diagnosis.”

—Glenn S. Williams

Suggested Reading

Keddie S, Angus-Leppan H, Parker T, et al. Discussing sudden unexpected death in epilepsy: are we empowering our patients? A questionnaire survey. JRSM Open. 2016;7(9):2054270416654358.

Xu Z, Ayyappan S, Seneviratne U. Sudden unexpected death in epilepsy (SUDEP): what do patients think? Epilepsy Behav. 2015;42:29-34.

BOSTON—Women athletes are 50% more likely to have a sports-related concussion than male athletes, according to research presented at the 69th Annual Meeting of the American Academy of Neurology.

“The findings from this study highlight the need for more research on the gender differences in concussion,” said James Noble, MD, Assistant Professor of Clinical Neurology at Columbia University in New York.

Little is known about the occurrence, severity, and recovery of sports-related concussion, especially among female athletes, since previous studies typically focused on male athletes. Gender-balanced studies have been limited by small sample size, incomplete or variable follow-up, or referral bias to tertiary concussion care centers. As a result, Dr. Noble and colleagues sought to determine gender differences in the incidence, symptomatology, neuropsychologic testing, and return-to-play length of sports-related concussion in collegiate varsity athletes.

For the study, Dr. Noble and colleagues followed 1,203 athletes at Columbia University from 2000 to 2014. In all, 822 of the participants were men, and 381 participants were women. All participants played sports such as soccer, basketball, and football.

Researchers assessed participants’ thinking skills and processing speed before and after a concussion. In addition, investigators tracked symptoms and when participants returned to play after a concussion.

A total of 228 athletes had at least one concussion, including 23% of the women (n = 88) and 17% of the men (n = 140). In addition, women who played soccer and basketball were more likely to have a concussion than their male counterparts. Finally, athletes who had had a previous concussion were three times more likely to have another concussion, compared with athletes who had never had a concussion.

The investigators also noted that women recovered from concussion about as quickly as men. Both men and women had a median return-to-play time of 10 days. Concussion symptoms were similar for men and women, although amnesia occurred more frequently in men (44% vs 31%), and insomnia occurred more frequently in women (42% vs 29%).

BOSTON—Women athletes are 50% more likely to have a sports-related concussion than male athletes, according to research presented at the 69th Annual Meeting of the American Academy of Neurology.

“The findings from this study highlight the need for more research on the gender differences in concussion,” said James Noble, MD, Assistant Professor of Clinical Neurology at Columbia University in New York.

Little is known about the occurrence, severity, and recovery of sports-related concussion, especially among female athletes, since previous studies typically focused on male athletes. Gender-balanced studies have been limited by small sample size, incomplete or variable follow-up, or referral bias to tertiary concussion care centers. As a result, Dr. Noble and colleagues sought to determine gender differences in the incidence, symptomatology, neuropsychologic testing, and return-to-play length of sports-related concussion in collegiate varsity athletes.

For the study, Dr. Noble and colleagues followed 1,203 athletes at Columbia University from 2000 to 2014. In all, 822 of the participants were men, and 381 participants were women. All participants played sports such as soccer, basketball, and football.

Researchers assessed participants’ thinking skills and processing speed before and after a concussion. In addition, investigators tracked symptoms and when participants returned to play after a concussion.

A total of 228 athletes had at least one concussion, including 23% of the women (n = 88) and 17% of the men (n = 140). In addition, women who played soccer and basketball were more likely to have a concussion than their male counterparts. Finally, athletes who had had a previous concussion were three times more likely to have another concussion, compared with athletes who had never had a concussion.

The investigators also noted that women recovered from concussion about as quickly as men. Both men and women had a median return-to-play time of 10 days. Concussion symptoms were similar for men and women, although amnesia occurred more frequently in men (44% vs 31%), and insomnia occurred more frequently in women (42% vs 29%).

BOSTON—Women athletes are 50% more likely to have a sports-related concussion than male athletes, according to research presented at the 69th Annual Meeting of the American Academy of Neurology.

“The findings from this study highlight the need for more research on the gender differences in concussion,” said James Noble, MD, Assistant Professor of Clinical Neurology at Columbia University in New York.

Little is known about the occurrence, severity, and recovery of sports-related concussion, especially among female athletes, since previous studies typically focused on male athletes. Gender-balanced studies have been limited by small sample size, incomplete or variable follow-up, or referral bias to tertiary concussion care centers. As a result, Dr. Noble and colleagues sought to determine gender differences in the incidence, symptomatology, neuropsychologic testing, and return-to-play length of sports-related concussion in collegiate varsity athletes.

For the study, Dr. Noble and colleagues followed 1,203 athletes at Columbia University from 2000 to 2014. In all, 822 of the participants were men, and 381 participants were women. All participants played sports such as soccer, basketball, and football.

Researchers assessed participants’ thinking skills and processing speed before and after a concussion. In addition, investigators tracked symptoms and when participants returned to play after a concussion.

A total of 228 athletes had at least one concussion, including 23% of the women (n = 88) and 17% of the men (n = 140). In addition, women who played soccer and basketball were more likely to have a concussion than their male counterparts. Finally, athletes who had had a previous concussion were three times more likely to have another concussion, compared with athletes who had never had a concussion.

The investigators also noted that women recovered from concussion about as quickly as men. Both men and women had a median return-to-play time of 10 days. Concussion symptoms were similar for men and women, although amnesia occurred more frequently in men (44% vs 31%), and insomnia occurred more frequently in women (42% vs 29%).

BOSTON—Cannabidiol (CBD) as an add-on therapy may reduce drop seizures by 50% in some adults and children with Lennox-Gastaut syndrome (LGS), according to research presented at the 69th Annual Meeting of the American Academy of Neurology. “Our study found that CBD shows great promise, in that it may reduce seizures that are otherwise difficult to control,” said Anup Patel, MD, a pediatric neurologist at Nationwide Children’s Hospital in Columbus, Ohio.

Evaluating CBD in LGS

LGS is a severe form of epilepsy that starts in childhood and causes multiple kinds of seizures, including drop seizures and tonic-clonic seizures, which can lead to serious injuries. To evaluate the efficacy of add-on CBD for the treatment of drop seizures associated with LGS, Dr. Patel and colleagues conducted a randomized, double-blind, placebo-controlled trial.

Eligible participants were between ages 2 and 55 and had a clinical diagnosis of LGS, eight or more drop seizures during a four-week baseline, and documented failures on one or more antiepileptic drugs. Participants received 20 mg/kg/day of CBD, 10 mg/kg/day of CBD, or placebo for 14 weeks, in addition to their current medications. The primary efficacy end point was the percentage change from baseline in drop seizures per month over the course of the study.

Researchers randomized 225 patients; 76 patients received 20 mg/kg/day of CBD, 73 patients received 10 mg/kg/day of CBD, and 76 patients received placebo. At baseline, the participants had a median monthly drop seizure frequency of 85, and they had previously failed a median of six epilepsy drugs. Participants were taking a median of three epilepsy drugs, in addition to CBD or placebo, throughout the study.

CBD Versus Placebo

Investigators observed a significantly greater reduction in drop seizure frequency in patients who received 20 mg/kg/day of CBD (42%) or 10 mg/kg/day of CBD (37%) than in patients who received placebo (17%). In addition, about 40% of patients who received CBD had at least a 50% reduction in drop seizures, compared with 15% of patients who received placebo.

Ninety-four percent of patients who received the higher dose of CBD reported adverse events, compared with 84% of participants who received the lower dose of CBD and 72% of participants who received placebo. The two most commonly reported adverse events were somnolence and decreased appetite. Most adverse events were mild or moderate. Treatment-related serious adverse events occurred in five patients who received 20 mg/kg/day of CBD and in two patients who received 10 mg/kg/day of CBD. No serious adverse events were reported in the placebo group, and no one died in any of the treatment groups.

In addition, patients who received CBD were more likely to report that their overall condition had improved, compared with patients who received placebo. Sixty-six percent of patients who received CBD reported improvement, compared with 44% of patients who received placebo.

“Our results suggest that CBD may be effective for people with Lennox-Gastaut syndrome in treating drop seizures,” said Dr. Patel. “While there were more side effects for those taking CBD, they were mostly well tolerated. I believe that it may become an important new treatment option for these patients.”

This study was supported by GW Pharmaceuticals, the developer of the CBD formulation.

Suggested Reading

Hussain SA, Zhou R, Jacobson C, et al. Perceived efficacy of cannabidiol-enriched cannabis extracts for treatment of pediatric epilepsy: A potential role for infantile spasms and Lennox-Gastaut syndrome. Epilepsy Behav. 2015;47:138-141.

BOSTON—Cannabidiol (CBD) as an add-on therapy may reduce drop seizures by 50% in some adults and children with Lennox-Gastaut syndrome (LGS), according to research presented at the 69th Annual Meeting of the American Academy of Neurology. “Our study found that CBD shows great promise, in that it may reduce seizures that are otherwise difficult to control,” said Anup Patel, MD, a pediatric neurologist at Nationwide Children’s Hospital in Columbus, Ohio.

Evaluating CBD in LGS

LGS is a severe form of epilepsy that starts in childhood and causes multiple kinds of seizures, including drop seizures and tonic-clonic seizures, which can lead to serious injuries. To evaluate the efficacy of add-on CBD for the treatment of drop seizures associated with LGS, Dr. Patel and colleagues conducted a randomized, double-blind, placebo-controlled trial.

Eligible participants were between ages 2 and 55 and had a clinical diagnosis of LGS, eight or more drop seizures during a four-week baseline, and documented failures on one or more antiepileptic drugs. Participants received 20 mg/kg/day of CBD, 10 mg/kg/day of CBD, or placebo for 14 weeks, in addition to their current medications. The primary efficacy end point was the percentage change from baseline in drop seizures per month over the course of the study.

Researchers randomized 225 patients; 76 patients received 20 mg/kg/day of CBD, 73 patients received 10 mg/kg/day of CBD, and 76 patients received placebo. At baseline, the participants had a median monthly drop seizure frequency of 85, and they had previously failed a median of six epilepsy drugs. Participants were taking a median of three epilepsy drugs, in addition to CBD or placebo, throughout the study.

CBD Versus Placebo

Investigators observed a significantly greater reduction in drop seizure frequency in patients who received 20 mg/kg/day of CBD (42%) or 10 mg/kg/day of CBD (37%) than in patients who received placebo (17%). In addition, about 40% of patients who received CBD had at least a 50% reduction in drop seizures, compared with 15% of patients who received placebo.

Ninety-four percent of patients who received the higher dose of CBD reported adverse events, compared with 84% of participants who received the lower dose of CBD and 72% of participants who received placebo. The two most commonly reported adverse events were somnolence and decreased appetite. Most adverse events were mild or moderate. Treatment-related serious adverse events occurred in five patients who received 20 mg/kg/day of CBD and in two patients who received 10 mg/kg/day of CBD. No serious adverse events were reported in the placebo group, and no one died in any of the treatment groups.

In addition, patients who received CBD were more likely to report that their overall condition had improved, compared with patients who received placebo. Sixty-six percent of patients who received CBD reported improvement, compared with 44% of patients who received placebo.

“Our results suggest that CBD may be effective for people with Lennox-Gastaut syndrome in treating drop seizures,” said Dr. Patel. “While there were more side effects for those taking CBD, they were mostly well tolerated. I believe that it may become an important new treatment option for these patients.”

This study was supported by GW Pharmaceuticals, the developer of the CBD formulation.

Suggested Reading

Hussain SA, Zhou R, Jacobson C, et al. Perceived efficacy of cannabidiol-enriched cannabis extracts for treatment of pediatric epilepsy: A potential role for infantile spasms and Lennox-Gastaut syndrome. Epilepsy Behav. 2015;47:138-141.

BOSTON—Cannabidiol (CBD) as an add-on therapy may reduce drop seizures by 50% in some adults and children with Lennox-Gastaut syndrome (LGS), according to research presented at the 69th Annual Meeting of the American Academy of Neurology. “Our study found that CBD shows great promise, in that it may reduce seizures that are otherwise difficult to control,” said Anup Patel, MD, a pediatric neurologist at Nationwide Children’s Hospital in Columbus, Ohio.

Evaluating CBD in LGS

LGS is a severe form of epilepsy that starts in childhood and causes multiple kinds of seizures, including drop seizures and tonic-clonic seizures, which can lead to serious injuries. To evaluate the efficacy of add-on CBD for the treatment of drop seizures associated with LGS, Dr. Patel and colleagues conducted a randomized, double-blind, placebo-controlled trial.

Eligible participants were between ages 2 and 55 and had a clinical diagnosis of LGS, eight or more drop seizures during a four-week baseline, and documented failures on one or more antiepileptic drugs. Participants received 20 mg/kg/day of CBD, 10 mg/kg/day of CBD, or placebo for 14 weeks, in addition to their current medications. The primary efficacy end point was the percentage change from baseline in drop seizures per month over the course of the study.

Researchers randomized 225 patients; 76 patients received 20 mg/kg/day of CBD, 73 patients received 10 mg/kg/day of CBD, and 76 patients received placebo. At baseline, the participants had a median monthly drop seizure frequency of 85, and they had previously failed a median of six epilepsy drugs. Participants were taking a median of three epilepsy drugs, in addition to CBD or placebo, throughout the study.

CBD Versus Placebo

Investigators observed a significantly greater reduction in drop seizure frequency in patients who received 20 mg/kg/day of CBD (42%) or 10 mg/kg/day of CBD (37%) than in patients who received placebo (17%). In addition, about 40% of patients who received CBD had at least a 50% reduction in drop seizures, compared with 15% of patients who received placebo.

Ninety-four percent of patients who received the higher dose of CBD reported adverse events, compared with 84% of participants who received the lower dose of CBD and 72% of participants who received placebo. The two most commonly reported adverse events were somnolence and decreased appetite. Most adverse events were mild or moderate. Treatment-related serious adverse events occurred in five patients who received 20 mg/kg/day of CBD and in two patients who received 10 mg/kg/day of CBD. No serious adverse events were reported in the placebo group, and no one died in any of the treatment groups.

In addition, patients who received CBD were more likely to report that their overall condition had improved, compared with patients who received placebo. Sixty-six percent of patients who received CBD reported improvement, compared with 44% of patients who received placebo.

“Our results suggest that CBD may be effective for people with Lennox-Gastaut syndrome in treating drop seizures,” said Dr. Patel. “While there were more side effects for those taking CBD, they were mostly well tolerated. I believe that it may become an important new treatment option for these patients.”

This study was supported by GW Pharmaceuticals, the developer of the CBD formulation.

Suggested Reading

Hussain SA, Zhou R, Jacobson C, et al. Perceived efficacy of cannabidiol-enriched cannabis extracts for treatment of pediatric epilepsy: A potential role for infantile spasms and Lennox-Gastaut syndrome. Epilepsy Behav. 2015;47:138-141.

NEW ORLEANS—Two studies presented at the 31st Annual Meeting of the Consortium of Multiple Sclerosis (MS) Centers examined the impact of MS and treatment among patients who are members of an online social network. Researchers found that, among patients who were taking oral disease-modifying treatments (DMTs), how the drug was taken was the most important factor in selecting the therapy (36% of patients) almost as often as the doctor’s recommendation (40% of patients), according to the results of a patient survey. In a separate study that analyzed comments made on the social network, researchers found that adverse events were the most commonly stated reason for stopping a treatment (56%).

In one study, Terrie Livingston, PharmD, an employee of Biogen in Weston, Massachusetts, and colleagues conducted a patient survey to assess the impact of MS on day-to-day life, self-reported symptoms, patient descriptions of disability progression, trade-offs when deciding which DMT to take, and what health care providers should know to better manage MS.

In November 2014, researchers sent an email invitation to the entire MyMSTeam community, a social network of approximately 25,000 people (currently more than 75,000 people) diagnosed with MS. In all, 1,107 members responded to the survey.

Overall, the most commonly reported symptoms were fatigue (82% of patients), problems with mobility (64%), and poor balance (64%). In addition, weakness, cognitive issues, and depression were common. The respondents defined disability progression as increased limitations on their ability to get around, having to sacrifice normal daily activities, and losing their ability to be independent. Patients measured the efficacy of DMTs by evaluating the treatments’ ability to slow progression, prevent further physical disability, prevent new lesions, and improve quality of life.

In a separate study, Dr. Livingston and colleagues conducted an analysis of organic discussions within MyMSTeam from January 2015 to April 2016. Researchers anonymized, coded, categorized, and analyzed 3,300 verbatim comments by key themes. The analysis focused on 12 DMTs. Discussions were overlaid with patient self-reported data on gender, age, date of diagnosis, MS type, current DMT, and effectiveness ratings.

Common discussion topics included side effects experienced; reasons for stopping treatment; perceived efficacy; questions, hopes, and concerns about starting a new DMT; rituals to mitigate side effects; and insurance or financial hurdles to getting on or staying on treatment.

Discussions about infusion therapies tended to focus on receiving treatment and issues related to progressive multifocal leukoencephalopathy.

When discussing Tysabri (natalizumab), patients most commonly discussed elevated energy levels post infusion around day 14. Patients also reported increased fatigue in the days prior to the next infusion and extreme fatigue on the day of treatment. In addition, patients shared their experiences with managing tolerability issues, most commonly flu-like symptoms with interferons and gastrointestinal effects with Tecfidera (dimethyl fumarate).

Dr. Livingston and colleagues said that their research “will better allow medical professionals to treat the individual holistically, and not just the disease itself. Additionally, helping patients understand MS progression will allow specialists to set realistic expectations for treating the disease and to help their patients better prepare for the future.”

NEW ORLEANS—Two studies presented at the 31st Annual Meeting of the Consortium of Multiple Sclerosis (MS) Centers examined the impact of MS and treatment among patients who are members of an online social network. Researchers found that, among patients who were taking oral disease-modifying treatments (DMTs), how the drug was taken was the most important factor in selecting the therapy (36% of patients) almost as often as the doctor’s recommendation (40% of patients), according to the results of a patient survey. In a separate study that analyzed comments made on the social network, researchers found that adverse events were the most commonly stated reason for stopping a treatment (56%).

In one study, Terrie Livingston, PharmD, an employee of Biogen in Weston, Massachusetts, and colleagues conducted a patient survey to assess the impact of MS on day-to-day life, self-reported symptoms, patient descriptions of disability progression, trade-offs when deciding which DMT to take, and what health care providers should know to better manage MS.

In November 2014, researchers sent an email invitation to the entire MyMSTeam community, a social network of approximately 25,000 people (currently more than 75,000 people) diagnosed with MS. In all, 1,107 members responded to the survey.

Overall, the most commonly reported symptoms were fatigue (82% of patients), problems with mobility (64%), and poor balance (64%). In addition, weakness, cognitive issues, and depression were common. The respondents defined disability progression as increased limitations on their ability to get around, having to sacrifice normal daily activities, and losing their ability to be independent. Patients measured the efficacy of DMTs by evaluating the treatments’ ability to slow progression, prevent further physical disability, prevent new lesions, and improve quality of life.

In a separate study, Dr. Livingston and colleagues conducted an analysis of organic discussions within MyMSTeam from January 2015 to April 2016. Researchers anonymized, coded, categorized, and analyzed 3,300 verbatim comments by key themes. The analysis focused on 12 DMTs. Discussions were overlaid with patient self-reported data on gender, age, date of diagnosis, MS type, current DMT, and effectiveness ratings.

Common discussion topics included side effects experienced; reasons for stopping treatment; perceived efficacy; questions, hopes, and concerns about starting a new DMT; rituals to mitigate side effects; and insurance or financial hurdles to getting on or staying on treatment.

Discussions about infusion therapies tended to focus on receiving treatment and issues related to progressive multifocal leukoencephalopathy.

When discussing Tysabri (natalizumab), patients most commonly discussed elevated energy levels post infusion around day 14. Patients also reported increased fatigue in the days prior to the next infusion and extreme fatigue on the day of treatment. In addition, patients shared their experiences with managing tolerability issues, most commonly flu-like symptoms with interferons and gastrointestinal effects with Tecfidera (dimethyl fumarate).

Dr. Livingston and colleagues said that their research “will better allow medical professionals to treat the individual holistically, and not just the disease itself. Additionally, helping patients understand MS progression will allow specialists to set realistic expectations for treating the disease and to help their patients better prepare for the future.”

NEW ORLEANS—Two studies presented at the 31st Annual Meeting of the Consortium of Multiple Sclerosis (MS) Centers examined the impact of MS and treatment among patients who are members of an online social network. Researchers found that, among patients who were taking oral disease-modifying treatments (DMTs), how the drug was taken was the most important factor in selecting the therapy (36% of patients) almost as often as the doctor’s recommendation (40% of patients), according to the results of a patient survey. In a separate study that analyzed comments made on the social network, researchers found that adverse events were the most commonly stated reason for stopping a treatment (56%).

In one study, Terrie Livingston, PharmD, an employee of Biogen in Weston, Massachusetts, and colleagues conducted a patient survey to assess the impact of MS on day-to-day life, self-reported symptoms, patient descriptions of disability progression, trade-offs when deciding which DMT to take, and what health care providers should know to better manage MS.

In November 2014, researchers sent an email invitation to the entire MyMSTeam community, a social network of approximately 25,000 people (currently more than 75,000 people) diagnosed with MS. In all, 1,107 members responded to the survey.

Overall, the most commonly reported symptoms were fatigue (82% of patients), problems with mobility (64%), and poor balance (64%). In addition, weakness, cognitive issues, and depression were common. The respondents defined disability progression as increased limitations on their ability to get around, having to sacrifice normal daily activities, and losing their ability to be independent. Patients measured the efficacy of DMTs by evaluating the treatments’ ability to slow progression, prevent further physical disability, prevent new lesions, and improve quality of life.

In a separate study, Dr. Livingston and colleagues conducted an analysis of organic discussions within MyMSTeam from January 2015 to April 2016. Researchers anonymized, coded, categorized, and analyzed 3,300 verbatim comments by key themes. The analysis focused on 12 DMTs. Discussions were overlaid with patient self-reported data on gender, age, date of diagnosis, MS type, current DMT, and effectiveness ratings.

Common discussion topics included side effects experienced; reasons for stopping treatment; perceived efficacy; questions, hopes, and concerns about starting a new DMT; rituals to mitigate side effects; and insurance or financial hurdles to getting on or staying on treatment.

Discussions about infusion therapies tended to focus on receiving treatment and issues related to progressive multifocal leukoencephalopathy.

When discussing Tysabri (natalizumab), patients most commonly discussed elevated energy levels post infusion around day 14. Patients also reported increased fatigue in the days prior to the next infusion and extreme fatigue on the day of treatment. In addition, patients shared their experiences with managing tolerability issues, most commonly flu-like symptoms with interferons and gastrointestinal effects with Tecfidera (dimethyl fumarate).

Dr. Livingston and colleagues said that their research “will better allow medical professionals to treat the individual holistically, and not just the disease itself. Additionally, helping patients understand MS progression will allow specialists to set realistic expectations for treating the disease and to help their patients better prepare for the future.”