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American Academy of Neurology (AAN): Annual Meeting 2017
New guideline: Address GTCS frequency to reduce SUDEP risk
BOSTON – Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.
Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.
Specifically, the guideline recommends that , she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”
Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”
The recommendations are based on moderate (Level B) evidence.
The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.
“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.
While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.
“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.
The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).
Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.
BOSTON – Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.
Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.
Specifically, the guideline recommends that , she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”
Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”
The recommendations are based on moderate (Level B) evidence.
The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.
“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.
While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.
“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.
The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).
Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.
BOSTON – Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.
Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.
Specifically, the guideline recommends that , she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”
Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”
The recommendations are based on moderate (Level B) evidence.
The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.
“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.
While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.
“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.
The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).
Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.
AT AAN 2017
Apomorphine Reduces Off Time for People with Advanced Parkinson’s Disease
BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.
“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”
Open-Label Data Suggest Efficacy
Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.
The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.
Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.
Comparing Apomorphine With Placebo
In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.
Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.
Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.
Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.
“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.
The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.
BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.
“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”
Open-Label Data Suggest Efficacy
Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.
The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.
Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.
Comparing Apomorphine With Placebo
In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.
Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.
Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.
Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.
“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.
The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.
BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.
“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”
Open-Label Data Suggest Efficacy
Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.
The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.
Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.
Comparing Apomorphine With Placebo
In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.
Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.
Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.
Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.
“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.
The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.
Parkinson’s patients’ quality of life improves after deep brain stimulation
BOSTON– Early results from an industry-funded registry of Parkinson’s disease patients who underwent deep brain stimulation (DBS) reveal that their quality-of-life scores grew by 22% at 6 months.
Patients, their caregivers, and their clinicians all overwhelmingly reported improvement.
The study, which examined two types of technology that are not approved in the United States, aims to fill a gap in DBS research: How do patients fare in normal conditions – “real life” – outside of clinical trials?
While DBS has been widely used for many years, “much of the available information is from controlled trials that usually select the best possible patients – relatively young and in the condition to go through a clinical trial. There’s less information about people who may not be in the best possible shape,” said Michele Tagliati, MD, professor and director of movement disorders at Cedars-Sinai Medical Center in Los Angeles. He did not take part in the study but is familiar with its findings.
The study’s lead author, Günther Deuschl, MD, PhD, of University Hospital Schleswig-Holstein, released preliminary findings at the annual meeting of the American Academy of Neurology.
The researchers have enrolled 203 patients who were treated with Boston Scientific’s Vercise DBS system, which is not approved for use in the United States. The researchers plan to track patients for 3 years.
“This is the first such industry-sponsored registry, which addresses a need in the field to track DBS practice and outcomes across multiple centers,” said Mark Richardson, MD, PhD, associate professor and director of epilepsy and movement disorders surgery at the University of Pittsburgh Medical Center. He did not take part in the study but is familiar with its findings.
The average age of participants is 59 years, which Dr. Deuschl said is a bit younger than many other studies, and 69% are male. Eighty-five serious adverse events were reported in 52 patients; 57 were not linked to the procedure. One patient died of a surgery-related hematoma.
At 6 months, patients reported a 22% improvement (P less than .0001) on the Parkinson’s Disease Questionnaire (PDQ-39), Dr. Deuschl said, and that level was sustained at 1 year.
That level of improvement is significant, Dr. Richardson said in an interview.
“Previous randomized, controlled trials have shown that patients who are candidates for DBS but who continue medical management alone are most likely to have no improvement at all on any quality of life measures,” he said. In addition, “6 months is fairly short, and some patients likely have not reached stable stimulation programming and effectiveness.”
The researchers also reported that more than 90% of patients, their clinicians, and their caregivers reported improvement.
This kind of study is valuable in light of skepticism about DBS, which is used to treat patients who do not respond to medication, Dr. Tagliati noted.
“Despite 15 years of [Food and Drug Administration] approval, there is still some form of resistance out there in referring patients with Parkinson’s at the right time when they can still fully benefit from the procedure,” he said. “Registries have this potential great benefit in terms of awareness and reassuring people.”
As for the high rating of improvement, Dr. Tagliati said it reflects what he sees in the clinic.
“Barring complications, we have very substantial satisfaction in our patients, definitely in the short term after DBS,” he said. “Over the long term, the picture is more difficult to read.”
Dr. Deuschl said researchers would like to add hundreds more patients to the study, and they hope to gain data about the differences between the results from standard and directional-lead DBS systems.
Boston Scientific funded the study, and some of the authors work for the company.
Dr. Richardson reported receiving research grant funding from Medtronic. Dr. Tagliati reported receiving funding from Abbott, Boston Scientific, Medtronic, and all DBS manufacturers, and his clinic is an investigational center for the Vercise DBS system. Dr. Deuschl reported receiving consulting fees from Boston Scientific, grant funding from Medtronic, lecture fees from Almirall and Novartis, and royalties from Thieme Publishers.
BOSTON– Early results from an industry-funded registry of Parkinson’s disease patients who underwent deep brain stimulation (DBS) reveal that their quality-of-life scores grew by 22% at 6 months.
Patients, their caregivers, and their clinicians all overwhelmingly reported improvement.
The study, which examined two types of technology that are not approved in the United States, aims to fill a gap in DBS research: How do patients fare in normal conditions – “real life” – outside of clinical trials?
While DBS has been widely used for many years, “much of the available information is from controlled trials that usually select the best possible patients – relatively young and in the condition to go through a clinical trial. There’s less information about people who may not be in the best possible shape,” said Michele Tagliati, MD, professor and director of movement disorders at Cedars-Sinai Medical Center in Los Angeles. He did not take part in the study but is familiar with its findings.
The study’s lead author, Günther Deuschl, MD, PhD, of University Hospital Schleswig-Holstein, released preliminary findings at the annual meeting of the American Academy of Neurology.
The researchers have enrolled 203 patients who were treated with Boston Scientific’s Vercise DBS system, which is not approved for use in the United States. The researchers plan to track patients for 3 years.
“This is the first such industry-sponsored registry, which addresses a need in the field to track DBS practice and outcomes across multiple centers,” said Mark Richardson, MD, PhD, associate professor and director of epilepsy and movement disorders surgery at the University of Pittsburgh Medical Center. He did not take part in the study but is familiar with its findings.
The average age of participants is 59 years, which Dr. Deuschl said is a bit younger than many other studies, and 69% are male. Eighty-five serious adverse events were reported in 52 patients; 57 were not linked to the procedure. One patient died of a surgery-related hematoma.
At 6 months, patients reported a 22% improvement (P less than .0001) on the Parkinson’s Disease Questionnaire (PDQ-39), Dr. Deuschl said, and that level was sustained at 1 year.
That level of improvement is significant, Dr. Richardson said in an interview.
“Previous randomized, controlled trials have shown that patients who are candidates for DBS but who continue medical management alone are most likely to have no improvement at all on any quality of life measures,” he said. In addition, “6 months is fairly short, and some patients likely have not reached stable stimulation programming and effectiveness.”
The researchers also reported that more than 90% of patients, their clinicians, and their caregivers reported improvement.
This kind of study is valuable in light of skepticism about DBS, which is used to treat patients who do not respond to medication, Dr. Tagliati noted.
“Despite 15 years of [Food and Drug Administration] approval, there is still some form of resistance out there in referring patients with Parkinson’s at the right time when they can still fully benefit from the procedure,” he said. “Registries have this potential great benefit in terms of awareness and reassuring people.”
As for the high rating of improvement, Dr. Tagliati said it reflects what he sees in the clinic.
“Barring complications, we have very substantial satisfaction in our patients, definitely in the short term after DBS,” he said. “Over the long term, the picture is more difficult to read.”
Dr. Deuschl said researchers would like to add hundreds more patients to the study, and they hope to gain data about the differences between the results from standard and directional-lead DBS systems.
Boston Scientific funded the study, and some of the authors work for the company.
Dr. Richardson reported receiving research grant funding from Medtronic. Dr. Tagliati reported receiving funding from Abbott, Boston Scientific, Medtronic, and all DBS manufacturers, and his clinic is an investigational center for the Vercise DBS system. Dr. Deuschl reported receiving consulting fees from Boston Scientific, grant funding from Medtronic, lecture fees from Almirall and Novartis, and royalties from Thieme Publishers.
BOSTON– Early results from an industry-funded registry of Parkinson’s disease patients who underwent deep brain stimulation (DBS) reveal that their quality-of-life scores grew by 22% at 6 months.
Patients, their caregivers, and their clinicians all overwhelmingly reported improvement.
The study, which examined two types of technology that are not approved in the United States, aims to fill a gap in DBS research: How do patients fare in normal conditions – “real life” – outside of clinical trials?
While DBS has been widely used for many years, “much of the available information is from controlled trials that usually select the best possible patients – relatively young and in the condition to go through a clinical trial. There’s less information about people who may not be in the best possible shape,” said Michele Tagliati, MD, professor and director of movement disorders at Cedars-Sinai Medical Center in Los Angeles. He did not take part in the study but is familiar with its findings.
The study’s lead author, Günther Deuschl, MD, PhD, of University Hospital Schleswig-Holstein, released preliminary findings at the annual meeting of the American Academy of Neurology.
The researchers have enrolled 203 patients who were treated with Boston Scientific’s Vercise DBS system, which is not approved for use in the United States. The researchers plan to track patients for 3 years.
“This is the first such industry-sponsored registry, which addresses a need in the field to track DBS practice and outcomes across multiple centers,” said Mark Richardson, MD, PhD, associate professor and director of epilepsy and movement disorders surgery at the University of Pittsburgh Medical Center. He did not take part in the study but is familiar with its findings.
The average age of participants is 59 years, which Dr. Deuschl said is a bit younger than many other studies, and 69% are male. Eighty-five serious adverse events were reported in 52 patients; 57 were not linked to the procedure. One patient died of a surgery-related hematoma.
At 6 months, patients reported a 22% improvement (P less than .0001) on the Parkinson’s Disease Questionnaire (PDQ-39), Dr. Deuschl said, and that level was sustained at 1 year.
That level of improvement is significant, Dr. Richardson said in an interview.
“Previous randomized, controlled trials have shown that patients who are candidates for DBS but who continue medical management alone are most likely to have no improvement at all on any quality of life measures,” he said. In addition, “6 months is fairly short, and some patients likely have not reached stable stimulation programming and effectiveness.”
The researchers also reported that more than 90% of patients, their clinicians, and their caregivers reported improvement.
This kind of study is valuable in light of skepticism about DBS, which is used to treat patients who do not respond to medication, Dr. Tagliati noted.
“Despite 15 years of [Food and Drug Administration] approval, there is still some form of resistance out there in referring patients with Parkinson’s at the right time when they can still fully benefit from the procedure,” he said. “Registries have this potential great benefit in terms of awareness and reassuring people.”
As for the high rating of improvement, Dr. Tagliati said it reflects what he sees in the clinic.
“Barring complications, we have very substantial satisfaction in our patients, definitely in the short term after DBS,” he said. “Over the long term, the picture is more difficult to read.”
Dr. Deuschl said researchers would like to add hundreds more patients to the study, and they hope to gain data about the differences between the results from standard and directional-lead DBS systems.
Boston Scientific funded the study, and some of the authors work for the company.
Dr. Richardson reported receiving research grant funding from Medtronic. Dr. Tagliati reported receiving funding from Abbott, Boston Scientific, Medtronic, and all DBS manufacturers, and his clinic is an investigational center for the Vercise DBS system. Dr. Deuschl reported receiving consulting fees from Boston Scientific, grant funding from Medtronic, lecture fees from Almirall and Novartis, and royalties from Thieme Publishers.
Prenotification, unequivocal stroke promote ultra-fast door-to-needle time
BOSTON – Ultra-fast door-to-needle times of 10 minutes or less for intravenous acute ischemic stroke thrombolysis can be safely achieved in carefully selected cases, according to a review of cases at an Austrian teaching hospital.
Raffi Topakian, MD, and his colleagues at the Academic Teaching Hospital Wels-Grieskirchen in Wels, Austria, followed a multidisciplinary intervention to reinforce key components of the well-known Helsinki model of acute stroke care to improve the intravenous thrombolysis rate and the median door-to-needle time (DNT) at the teaching hospital and analyzed data from 361 patients who underwent intravenous thrombolysis (IVT) for stroke there between July 2014 and September 2016. The IVT rate increased from 19% to about 27% after intervention, and the DNT during the study period was 60 minutes or less in 316 patients (87.5%), 30 minutes or less in 181 patients (50.1%), and 10 minutes or less in 63 patients (17.5%).
“Over the study period, we reduced the DNT time from 49 minutes to 25 minutes. This was significant, and the door-to-needle times were astonishingly similar for the in-hours service and the out-of-hour service,” he said at the annual meeting of the American Academy of Neurology.
Further, the rate of prenotifications from emergency medical services rose from about 30% to 63% during the study period.
Patients with ultra-fast DNT vs. those with slower DNT were older, had more chronic heart failure, had more severe stroke (National Institutes of Health Stroke Scale score of 10 vs. 5), had more anterior circulation stroke and cardioembolic stroke, and had clear onset of stroke. Independent predictors of ultra-fast DNT included prenotification by EMS, anterior circulation syndrome, chronic heart failure, and having a stroke neurologist on duty, Dr. Topakian said.
“Ultra short DNTs can be achieved safely. The key is that we are prenotified by the EMS, that we can get all the relevant history details during transport, that there is a dedicated multidisciplinary stroke team and EMS staff, and that we have a seemingly unequivocal clinical scenario,” he said. “Out-of-hours DNT matched in-hours DNT, but the caveat is we’re talking about highly selected candidates; safety must not be sacrificed for the sake of speed, in all of our patients.”
Dr. Topakian has received personal compensation for activities with Novartis and Shire-Baxalta as an advisory board member; from Novartis, Pfizer, AbbVie, and Bayer for conference support; and from Pfizer as a speaker.
BOSTON – Ultra-fast door-to-needle times of 10 minutes or less for intravenous acute ischemic stroke thrombolysis can be safely achieved in carefully selected cases, according to a review of cases at an Austrian teaching hospital.
Raffi Topakian, MD, and his colleagues at the Academic Teaching Hospital Wels-Grieskirchen in Wels, Austria, followed a multidisciplinary intervention to reinforce key components of the well-known Helsinki model of acute stroke care to improve the intravenous thrombolysis rate and the median door-to-needle time (DNT) at the teaching hospital and analyzed data from 361 patients who underwent intravenous thrombolysis (IVT) for stroke there between July 2014 and September 2016. The IVT rate increased from 19% to about 27% after intervention, and the DNT during the study period was 60 minutes or less in 316 patients (87.5%), 30 minutes or less in 181 patients (50.1%), and 10 minutes or less in 63 patients (17.5%).
“Over the study period, we reduced the DNT time from 49 minutes to 25 minutes. This was significant, and the door-to-needle times were astonishingly similar for the in-hours service and the out-of-hour service,” he said at the annual meeting of the American Academy of Neurology.
Further, the rate of prenotifications from emergency medical services rose from about 30% to 63% during the study period.
Patients with ultra-fast DNT vs. those with slower DNT were older, had more chronic heart failure, had more severe stroke (National Institutes of Health Stroke Scale score of 10 vs. 5), had more anterior circulation stroke and cardioembolic stroke, and had clear onset of stroke. Independent predictors of ultra-fast DNT included prenotification by EMS, anterior circulation syndrome, chronic heart failure, and having a stroke neurologist on duty, Dr. Topakian said.
“Ultra short DNTs can be achieved safely. The key is that we are prenotified by the EMS, that we can get all the relevant history details during transport, that there is a dedicated multidisciplinary stroke team and EMS staff, and that we have a seemingly unequivocal clinical scenario,” he said. “Out-of-hours DNT matched in-hours DNT, but the caveat is we’re talking about highly selected candidates; safety must not be sacrificed for the sake of speed, in all of our patients.”
Dr. Topakian has received personal compensation for activities with Novartis and Shire-Baxalta as an advisory board member; from Novartis, Pfizer, AbbVie, and Bayer for conference support; and from Pfizer as a speaker.
BOSTON – Ultra-fast door-to-needle times of 10 minutes or less for intravenous acute ischemic stroke thrombolysis can be safely achieved in carefully selected cases, according to a review of cases at an Austrian teaching hospital.
Raffi Topakian, MD, and his colleagues at the Academic Teaching Hospital Wels-Grieskirchen in Wels, Austria, followed a multidisciplinary intervention to reinforce key components of the well-known Helsinki model of acute stroke care to improve the intravenous thrombolysis rate and the median door-to-needle time (DNT) at the teaching hospital and analyzed data from 361 patients who underwent intravenous thrombolysis (IVT) for stroke there between July 2014 and September 2016. The IVT rate increased from 19% to about 27% after intervention, and the DNT during the study period was 60 minutes or less in 316 patients (87.5%), 30 minutes or less in 181 patients (50.1%), and 10 minutes or less in 63 patients (17.5%).
“Over the study period, we reduced the DNT time from 49 minutes to 25 minutes. This was significant, and the door-to-needle times were astonishingly similar for the in-hours service and the out-of-hour service,” he said at the annual meeting of the American Academy of Neurology.
Further, the rate of prenotifications from emergency medical services rose from about 30% to 63% during the study period.
Patients with ultra-fast DNT vs. those with slower DNT were older, had more chronic heart failure, had more severe stroke (National Institutes of Health Stroke Scale score of 10 vs. 5), had more anterior circulation stroke and cardioembolic stroke, and had clear onset of stroke. Independent predictors of ultra-fast DNT included prenotification by EMS, anterior circulation syndrome, chronic heart failure, and having a stroke neurologist on duty, Dr. Topakian said.
“Ultra short DNTs can be achieved safely. The key is that we are prenotified by the EMS, that we can get all the relevant history details during transport, that there is a dedicated multidisciplinary stroke team and EMS staff, and that we have a seemingly unequivocal clinical scenario,” he said. “Out-of-hours DNT matched in-hours DNT, but the caveat is we’re talking about highly selected candidates; safety must not be sacrificed for the sake of speed, in all of our patients.”
Dr. Topakian has received personal compensation for activities with Novartis and Shire-Baxalta as an advisory board member; from Novartis, Pfizer, AbbVie, and Bayer for conference support; and from Pfizer as a speaker.
Key clinical point:
Major finding: Door-to-needle time of 10 minutes or less was achieved in 63 patients (17.5%).
Data source: A retrospective review of prospectively collected data from 361 patients.
Disclosures: Dr. Topakian has received personal compensation for activities with Novartis and Shire-Baxalta as an advisory board member; from Novartis, Pfizer, AbbVie, and Bayer for conference support; and from Pfizer as a speaker.
Sepsis survivors may have high risk for seizures
BOSTON – Survivors of sepsis face a significantly increased risk of seizures following an index hospitalization, regardless of any previous history of seizures or seizures occurring during hospitalization, according to findings from a retrospective, population-based cohort study.
The risk for having subsequent seizures was highest for patients younger than 65 years but was still elevated above the general population for those aged 65 years or older, Michael Reznik, MD, reported at the annual meeting of the American Academy of Neurology.
Seizures are already a well-known complication of sepsis, and they also can occur alongside sepsis-associated encephalopathy, stroke, and neuromuscular disease. The frequency of sepsis-associated encephalopathy also has led to the recognition of postsepsis cognitive dysfunction, said Dr. Reznik, a neurocritical care fellow in the department of neurology at Weill Cornell Medicine and Columbia University Medical Center in New York.
It is unclear, however, how much of the risk for cognitive impairment after sepsis is due to pre-existing cognitive impairment, frailty, or lingering sedation effects, he said.
It’s possible, he noted, that “seizures may be more specific for structural brain injury, and I think our findings may support the hypothesis that sepsis could be associated with pathways leading to long-lasting brain injury that’s independent of other primary injuries that we have controlled for.”
Dr. Reznik and his coinvestigators used administrative claims data from all discharges from nonfederal emergency departments and acute care hospitals in California, New York, and Florida during 2005-2013 that had been collected as part of the federal Healthcare Cost and Utilization Project (HCUP). The HCUP assigns each patient a unique number that can be used to follow them anonymously through all subsequent hospitalizations. At each encounter, HCUP also tracks up to 25 discharge diagnoses that were present before hospital admission or developed during hospitalization, based on ICD-9-CM codes.
The investigators excluded patients with an ICD-9-CM diagnosis of seizures either before or during the index hospitalization for sepsis.
Overall, the 842,735 adult sepsis survivors in the study had a 6.67% cumulative rate of seizures over the 8-year period, compared with 1.27% in the general population. This translated to an incidence of about 1,288 per 100,000 patient-years in sepsis survivors, compared with 159 per 100,000 patient-years in the general population. The overall incidence rate ratio (IRR) for seizures among sepsis survivors was about 5, but was higher for those who also had neurologic dysfunction (such as encephalopathy, delirium, coma, or stupor) during their index hospitalization than in those without it (7.52 vs. 4.53). Sepsis survivors also had an elevated IRR of 5.42 for status epilepticus.
Sepsis survivors also had an elevated IRR of 4.35 for seizures when compared against control patients who were hospitalized for diagnoses other than sepsis and matched for age, sex, race, insurance, length of stay, discharge location, year of hospitalization, state, and the presence of codes for organ dysfunction.
The investigators confirmed the findings from the state-based HCUP analysis through inpatient and outpatient Medicare claims during 2008-1014 in a nationally representative sample of 5% of Medicare beneficiaries. These patients had an IRR for seizures of 2.72, and the IRR remained elevated (2.18) relative to patients who were hospitalized with diagnoses other than sepsis even when they excluded patients with ICD-9-CM codes for conditions that confer risk for seizures, including stroke, traumatic brain injury, CNS infection, or brain neoplasm. The seizure outcome in this analysis was defined as one or more inpatient claims for epilepsy or two or more outpatient claims within 3 months of each other.
Since the state-based HCUP data gave a much stronger association between sepsis and subsequent seizures than did the Medicare claims data, the investigators performed a post hoc stratified analysis according to age. Age proved to have a significant effect on the relationship between sepsis and subsequent seizures: Patients aged 65 years or older had an IRR of 2.83, compared with an IRR of 10.33 for those younger than 65.
In an interview, Dr. Reznik said that he sees the results as hypothesis generating and suggested they could serve as a “red flag” for neurologists that’s worth further investigation, given that studies suggest systemic infections and sepsis overall have long-term neurologic implications.
“I think there’s a possibility that, down the line, [sepsis] might be seen as a seizure risk factor, but unfortunately there are limitations from being based on an administrative data set,” he said.
The study was supported by a grant from the National Institute for Neurological Disorders and Stroke to one of the investigators and also by the Michael Goldberg Research Fund. Dr. Reznik had no disclosures to report.
BOSTON – Survivors of sepsis face a significantly increased risk of seizures following an index hospitalization, regardless of any previous history of seizures or seizures occurring during hospitalization, according to findings from a retrospective, population-based cohort study.
The risk for having subsequent seizures was highest for patients younger than 65 years but was still elevated above the general population for those aged 65 years or older, Michael Reznik, MD, reported at the annual meeting of the American Academy of Neurology.
Seizures are already a well-known complication of sepsis, and they also can occur alongside sepsis-associated encephalopathy, stroke, and neuromuscular disease. The frequency of sepsis-associated encephalopathy also has led to the recognition of postsepsis cognitive dysfunction, said Dr. Reznik, a neurocritical care fellow in the department of neurology at Weill Cornell Medicine and Columbia University Medical Center in New York.
It is unclear, however, how much of the risk for cognitive impairment after sepsis is due to pre-existing cognitive impairment, frailty, or lingering sedation effects, he said.
It’s possible, he noted, that “seizures may be more specific for structural brain injury, and I think our findings may support the hypothesis that sepsis could be associated with pathways leading to long-lasting brain injury that’s independent of other primary injuries that we have controlled for.”
Dr. Reznik and his coinvestigators used administrative claims data from all discharges from nonfederal emergency departments and acute care hospitals in California, New York, and Florida during 2005-2013 that had been collected as part of the federal Healthcare Cost and Utilization Project (HCUP). The HCUP assigns each patient a unique number that can be used to follow them anonymously through all subsequent hospitalizations. At each encounter, HCUP also tracks up to 25 discharge diagnoses that were present before hospital admission or developed during hospitalization, based on ICD-9-CM codes.
The investigators excluded patients with an ICD-9-CM diagnosis of seizures either before or during the index hospitalization for sepsis.
Overall, the 842,735 adult sepsis survivors in the study had a 6.67% cumulative rate of seizures over the 8-year period, compared with 1.27% in the general population. This translated to an incidence of about 1,288 per 100,000 patient-years in sepsis survivors, compared with 159 per 100,000 patient-years in the general population. The overall incidence rate ratio (IRR) for seizures among sepsis survivors was about 5, but was higher for those who also had neurologic dysfunction (such as encephalopathy, delirium, coma, or stupor) during their index hospitalization than in those without it (7.52 vs. 4.53). Sepsis survivors also had an elevated IRR of 5.42 for status epilepticus.
Sepsis survivors also had an elevated IRR of 4.35 for seizures when compared against control patients who were hospitalized for diagnoses other than sepsis and matched for age, sex, race, insurance, length of stay, discharge location, year of hospitalization, state, and the presence of codes for organ dysfunction.
The investigators confirmed the findings from the state-based HCUP analysis through inpatient and outpatient Medicare claims during 2008-1014 in a nationally representative sample of 5% of Medicare beneficiaries. These patients had an IRR for seizures of 2.72, and the IRR remained elevated (2.18) relative to patients who were hospitalized with diagnoses other than sepsis even when they excluded patients with ICD-9-CM codes for conditions that confer risk for seizures, including stroke, traumatic brain injury, CNS infection, or brain neoplasm. The seizure outcome in this analysis was defined as one or more inpatient claims for epilepsy or two or more outpatient claims within 3 months of each other.
Since the state-based HCUP data gave a much stronger association between sepsis and subsequent seizures than did the Medicare claims data, the investigators performed a post hoc stratified analysis according to age. Age proved to have a significant effect on the relationship between sepsis and subsequent seizures: Patients aged 65 years or older had an IRR of 2.83, compared with an IRR of 10.33 for those younger than 65.
In an interview, Dr. Reznik said that he sees the results as hypothesis generating and suggested they could serve as a “red flag” for neurologists that’s worth further investigation, given that studies suggest systemic infections and sepsis overall have long-term neurologic implications.
“I think there’s a possibility that, down the line, [sepsis] might be seen as a seizure risk factor, but unfortunately there are limitations from being based on an administrative data set,” he said.
The study was supported by a grant from the National Institute for Neurological Disorders and Stroke to one of the investigators and also by the Michael Goldberg Research Fund. Dr. Reznik had no disclosures to report.
BOSTON – Survivors of sepsis face a significantly increased risk of seizures following an index hospitalization, regardless of any previous history of seizures or seizures occurring during hospitalization, according to findings from a retrospective, population-based cohort study.
The risk for having subsequent seizures was highest for patients younger than 65 years but was still elevated above the general population for those aged 65 years or older, Michael Reznik, MD, reported at the annual meeting of the American Academy of Neurology.
Seizures are already a well-known complication of sepsis, and they also can occur alongside sepsis-associated encephalopathy, stroke, and neuromuscular disease. The frequency of sepsis-associated encephalopathy also has led to the recognition of postsepsis cognitive dysfunction, said Dr. Reznik, a neurocritical care fellow in the department of neurology at Weill Cornell Medicine and Columbia University Medical Center in New York.
It is unclear, however, how much of the risk for cognitive impairment after sepsis is due to pre-existing cognitive impairment, frailty, or lingering sedation effects, he said.
It’s possible, he noted, that “seizures may be more specific for structural brain injury, and I think our findings may support the hypothesis that sepsis could be associated with pathways leading to long-lasting brain injury that’s independent of other primary injuries that we have controlled for.”
Dr. Reznik and his coinvestigators used administrative claims data from all discharges from nonfederal emergency departments and acute care hospitals in California, New York, and Florida during 2005-2013 that had been collected as part of the federal Healthcare Cost and Utilization Project (HCUP). The HCUP assigns each patient a unique number that can be used to follow them anonymously through all subsequent hospitalizations. At each encounter, HCUP also tracks up to 25 discharge diagnoses that were present before hospital admission or developed during hospitalization, based on ICD-9-CM codes.
The investigators excluded patients with an ICD-9-CM diagnosis of seizures either before or during the index hospitalization for sepsis.
Overall, the 842,735 adult sepsis survivors in the study had a 6.67% cumulative rate of seizures over the 8-year period, compared with 1.27% in the general population. This translated to an incidence of about 1,288 per 100,000 patient-years in sepsis survivors, compared with 159 per 100,000 patient-years in the general population. The overall incidence rate ratio (IRR) for seizures among sepsis survivors was about 5, but was higher for those who also had neurologic dysfunction (such as encephalopathy, delirium, coma, or stupor) during their index hospitalization than in those without it (7.52 vs. 4.53). Sepsis survivors also had an elevated IRR of 5.42 for status epilepticus.
Sepsis survivors also had an elevated IRR of 4.35 for seizures when compared against control patients who were hospitalized for diagnoses other than sepsis and matched for age, sex, race, insurance, length of stay, discharge location, year of hospitalization, state, and the presence of codes for organ dysfunction.
The investigators confirmed the findings from the state-based HCUP analysis through inpatient and outpatient Medicare claims during 2008-1014 in a nationally representative sample of 5% of Medicare beneficiaries. These patients had an IRR for seizures of 2.72, and the IRR remained elevated (2.18) relative to patients who were hospitalized with diagnoses other than sepsis even when they excluded patients with ICD-9-CM codes for conditions that confer risk for seizures, including stroke, traumatic brain injury, CNS infection, or brain neoplasm. The seizure outcome in this analysis was defined as one or more inpatient claims for epilepsy or two or more outpatient claims within 3 months of each other.
Since the state-based HCUP data gave a much stronger association between sepsis and subsequent seizures than did the Medicare claims data, the investigators performed a post hoc stratified analysis according to age. Age proved to have a significant effect on the relationship between sepsis and subsequent seizures: Patients aged 65 years or older had an IRR of 2.83, compared with an IRR of 10.33 for those younger than 65.
In an interview, Dr. Reznik said that he sees the results as hypothesis generating and suggested they could serve as a “red flag” for neurologists that’s worth further investigation, given that studies suggest systemic infections and sepsis overall have long-term neurologic implications.
“I think there’s a possibility that, down the line, [sepsis] might be seen as a seizure risk factor, but unfortunately there are limitations from being based on an administrative data set,” he said.
The study was supported by a grant from the National Institute for Neurological Disorders and Stroke to one of the investigators and also by the Michael Goldberg Research Fund. Dr. Reznik had no disclosures to report.
Key clinical point:
Major finding: The overall incidence rate ratio for seizures among sepsis survivors was about 5, compared with the general population.
Data source: A retrospective, population-based cohort study of 842,735 sepsis survivors from three states during 2005-2013.
Disclosures: The study was supported by a grant from the National Institute for Neurological Disorders and Stroke to one of the investigators and also the Michael Goldberg Research Fund. Dr. Reznik had no disclosures to report.
Neurologists weigh in on rising drug prices
Neurologists will tackle the issues surrounding rising prices for neurological drug treatments in a set of presentations during the annual meeting of the American Academy of Neurology in Boston.
During the Contemporary Clinical Issues Plenary Session on April 24, Dennis N. Bourdette, MD, of Oregon Health and Science University, Portland, will give his presentation, “High Drug Prices: The Elephant in the Clinic.”
Dr. Bourdette will also address the problem on April 26 in a session called “Section Topic Controversies,” in which he and Dennis W. Choi, MD, PhD, of the State University of New York at Stony Brook will offer their opinions on how “Neurologists Should Take a Position Regarding the Cost of Neurological Treatments.” Dr. Bourdette is set to illustrate how “Neurologists Should More Vocally Protest Some of the Marked Price Increases Involving Neurological Treatments,” while Dr. Choi will provide rationale for his argument on why “Neurologists Should Influence Policies that Allow Companies to Commit More Resources to R&D for Neurological Diseases While Not Allowing for Rapacious Pricing Practices.” Following the discussion, Dr. Bourdette, Dr. Choi, and session moderators will have a panel discussion on the ways in which AAN members can work most productively for the benefit of their patients.
Sure to be discussed in the series of talks is a recent AAN position paper on prescription drug prices released in March that identified three distinct drug pricing challenges:
- Massive increase in the pricing of previously low-cost generic drugs used to treat common disorders without obvious increases in cost of production or distribution.
- Massive increase in the pricing for high-priced generic and brand name drugs used to treat serious disorders that are not protected by the Orphan Drug Act.
- The high cost of new medications used to treat rare disorders as defined by the Orphan Drug Act.
Neurologists will tackle the issues surrounding rising prices for neurological drug treatments in a set of presentations during the annual meeting of the American Academy of Neurology in Boston.
During the Contemporary Clinical Issues Plenary Session on April 24, Dennis N. Bourdette, MD, of Oregon Health and Science University, Portland, will give his presentation, “High Drug Prices: The Elephant in the Clinic.”
Dr. Bourdette will also address the problem on April 26 in a session called “Section Topic Controversies,” in which he and Dennis W. Choi, MD, PhD, of the State University of New York at Stony Brook will offer their opinions on how “Neurologists Should Take a Position Regarding the Cost of Neurological Treatments.” Dr. Bourdette is set to illustrate how “Neurologists Should More Vocally Protest Some of the Marked Price Increases Involving Neurological Treatments,” while Dr. Choi will provide rationale for his argument on why “Neurologists Should Influence Policies that Allow Companies to Commit More Resources to R&D for Neurological Diseases While Not Allowing for Rapacious Pricing Practices.” Following the discussion, Dr. Bourdette, Dr. Choi, and session moderators will have a panel discussion on the ways in which AAN members can work most productively for the benefit of their patients.
Sure to be discussed in the series of talks is a recent AAN position paper on prescription drug prices released in March that identified three distinct drug pricing challenges:
- Massive increase in the pricing of previously low-cost generic drugs used to treat common disorders without obvious increases in cost of production or distribution.
- Massive increase in the pricing for high-priced generic and brand name drugs used to treat serious disorders that are not protected by the Orphan Drug Act.
- The high cost of new medications used to treat rare disorders as defined by the Orphan Drug Act.
Neurologists will tackle the issues surrounding rising prices for neurological drug treatments in a set of presentations during the annual meeting of the American Academy of Neurology in Boston.
During the Contemporary Clinical Issues Plenary Session on April 24, Dennis N. Bourdette, MD, of Oregon Health and Science University, Portland, will give his presentation, “High Drug Prices: The Elephant in the Clinic.”
Dr. Bourdette will also address the problem on April 26 in a session called “Section Topic Controversies,” in which he and Dennis W. Choi, MD, PhD, of the State University of New York at Stony Brook will offer their opinions on how “Neurologists Should Take a Position Regarding the Cost of Neurological Treatments.” Dr. Bourdette is set to illustrate how “Neurologists Should More Vocally Protest Some of the Marked Price Increases Involving Neurological Treatments,” while Dr. Choi will provide rationale for his argument on why “Neurologists Should Influence Policies that Allow Companies to Commit More Resources to R&D for Neurological Diseases While Not Allowing for Rapacious Pricing Practices.” Following the discussion, Dr. Bourdette, Dr. Choi, and session moderators will have a panel discussion on the ways in which AAN members can work most productively for the benefit of their patients.
Sure to be discussed in the series of talks is a recent AAN position paper on prescription drug prices released in March that identified three distinct drug pricing challenges:
- Massive increase in the pricing of previously low-cost generic drugs used to treat common disorders without obvious increases in cost of production or distribution.
- Massive increase in the pricing for high-priced generic and brand name drugs used to treat serious disorders that are not protected by the Orphan Drug Act.
- The high cost of new medications used to treat rare disorders as defined by the Orphan Drug Act.
Bevy of monoclonal antibody migraine prevention trials awaits attendees
Calcitonin gene–related peptide (CGRP) monoclonal antibodies for migraine prevention will take center stage at the upcoming annual meeting of the American Academy of Neurology in Boston in a variety of talks aimed at describing the latest clinical trial findings.
Amaal J. Starling, MD, of the Mayo Clinic, Phoenix, Ariz., will lead off on the topic in the Contemporary Clinical Issues Plenary Session on April 24 by discussing in her presentation, “The Era of Targeted Preventive Treatment for Migraine: CGRP Monoclonal Antibodies,” the impact that CGRP monoclonal antibodies may have on migraine treatment
In the first of two presentations on the primary results of two phase III migraine prevention trials for the CGRP monoclonal antibody erenumab, also known as AMG 334, Peter Goadsby, MD, PhD, of the University of California, San Francisco, will report on the STRIVE trial in the Clinical Trials Plenary Session on April 25.
STRIVE is investigating two doses of erenumab against placebo in a 24-week trial examining the primary outcome of a change from baseline in mean monthly migraine days among 955 patients with a 1-year history of episodic migraine who are currently, have previously, or have never received migraine prophylactic medication. Patients will then be randomized after this initial double-blind treatment phase to 28 weeks of open-label active treatment with either dose of erenumab.
Later on April 25 in the Emerging Science Platform Session, David W. Dodick, MD, of the Mayo Clinic in Phoenix is set to describe the results of the phase III ARISE trial. Investigators in ARISE tested 12 weeks of one dosing regimen of erenumab versus placebo on the change from baseline in mean monthly migraine days in 577 patients with episodic migraine, followed by a 28-week open-label treatment phase.
Phase II study results of erenumab in the prevention of chronic migraine can be viewed April 28 in the “Headache: Clinical Trials and Disease Burden” platform session. The trial tested two doses of erenumab against placebo to detect differences in the change in monthly migraine days from baseline in the last 4 weeks of the trial’s 12-week double-blind treatment phase. The phase II trial results of a different CGRP monoclonal antibody known as eptinezumab or ALD403 in the prevention of chronic migraine will also be reported in the same platform session. The study evaluated a variety of doses of the drug for superiority against placebo in reducing the number of migraine days by 75% over 12 weeks.
Calcitonin gene–related peptide (CGRP) monoclonal antibodies for migraine prevention will take center stage at the upcoming annual meeting of the American Academy of Neurology in Boston in a variety of talks aimed at describing the latest clinical trial findings.
Amaal J. Starling, MD, of the Mayo Clinic, Phoenix, Ariz., will lead off on the topic in the Contemporary Clinical Issues Plenary Session on April 24 by discussing in her presentation, “The Era of Targeted Preventive Treatment for Migraine: CGRP Monoclonal Antibodies,” the impact that CGRP monoclonal antibodies may have on migraine treatment
In the first of two presentations on the primary results of two phase III migraine prevention trials for the CGRP monoclonal antibody erenumab, also known as AMG 334, Peter Goadsby, MD, PhD, of the University of California, San Francisco, will report on the STRIVE trial in the Clinical Trials Plenary Session on April 25.
STRIVE is investigating two doses of erenumab against placebo in a 24-week trial examining the primary outcome of a change from baseline in mean monthly migraine days among 955 patients with a 1-year history of episodic migraine who are currently, have previously, or have never received migraine prophylactic medication. Patients will then be randomized after this initial double-blind treatment phase to 28 weeks of open-label active treatment with either dose of erenumab.
Later on April 25 in the Emerging Science Platform Session, David W. Dodick, MD, of the Mayo Clinic in Phoenix is set to describe the results of the phase III ARISE trial. Investigators in ARISE tested 12 weeks of one dosing regimen of erenumab versus placebo on the change from baseline in mean monthly migraine days in 577 patients with episodic migraine, followed by a 28-week open-label treatment phase.
Phase II study results of erenumab in the prevention of chronic migraine can be viewed April 28 in the “Headache: Clinical Trials and Disease Burden” platform session. The trial tested two doses of erenumab against placebo to detect differences in the change in monthly migraine days from baseline in the last 4 weeks of the trial’s 12-week double-blind treatment phase. The phase II trial results of a different CGRP monoclonal antibody known as eptinezumab or ALD403 in the prevention of chronic migraine will also be reported in the same platform session. The study evaluated a variety of doses of the drug for superiority against placebo in reducing the number of migraine days by 75% over 12 weeks.
Calcitonin gene–related peptide (CGRP) monoclonal antibodies for migraine prevention will take center stage at the upcoming annual meeting of the American Academy of Neurology in Boston in a variety of talks aimed at describing the latest clinical trial findings.
Amaal J. Starling, MD, of the Mayo Clinic, Phoenix, Ariz., will lead off on the topic in the Contemporary Clinical Issues Plenary Session on April 24 by discussing in her presentation, “The Era of Targeted Preventive Treatment for Migraine: CGRP Monoclonal Antibodies,” the impact that CGRP monoclonal antibodies may have on migraine treatment
In the first of two presentations on the primary results of two phase III migraine prevention trials for the CGRP monoclonal antibody erenumab, also known as AMG 334, Peter Goadsby, MD, PhD, of the University of California, San Francisco, will report on the STRIVE trial in the Clinical Trials Plenary Session on April 25.
STRIVE is investigating two doses of erenumab against placebo in a 24-week trial examining the primary outcome of a change from baseline in mean monthly migraine days among 955 patients with a 1-year history of episodic migraine who are currently, have previously, or have never received migraine prophylactic medication. Patients will then be randomized after this initial double-blind treatment phase to 28 weeks of open-label active treatment with either dose of erenumab.
Later on April 25 in the Emerging Science Platform Session, David W. Dodick, MD, of the Mayo Clinic in Phoenix is set to describe the results of the phase III ARISE trial. Investigators in ARISE tested 12 weeks of one dosing regimen of erenumab versus placebo on the change from baseline in mean monthly migraine days in 577 patients with episodic migraine, followed by a 28-week open-label treatment phase.
Phase II study results of erenumab in the prevention of chronic migraine can be viewed April 28 in the “Headache: Clinical Trials and Disease Burden” platform session. The trial tested two doses of erenumab against placebo to detect differences in the change in monthly migraine days from baseline in the last 4 weeks of the trial’s 12-week double-blind treatment phase. The phase II trial results of a different CGRP monoclonal antibody known as eptinezumab or ALD403 in the prevention of chronic migraine will also be reported in the same platform session. The study evaluated a variety of doses of the drug for superiority against placebo in reducing the number of migraine days by 75% over 12 weeks.
AAN spotlights spinal muscular atrophy clinical research
A variety of plenary and emerging science sessions at this year’s annual meeting of the American Academy of Neurology in Boston will highlight clinical research efforts to treat children with spinal muscular atrophy.
At the Hot Topics Plenary Session on April 22, Claudia A. Chiriboga, MD, of Columbia University, New York, will discuss the results of clinical trials involving antisense oligonucleotide treatments for spinal muscular atrophy (SMA), including the recently approved nusinersen (Spinraza), which promotes transcription of the full-length survival motor neuron (SMN) protein from the SMN2 gene.
In the first of two reports on new clinical research about nusinersen, Nancy L. Kuntz, MD, of Ann & Robert H. Lurie Children’s Hospital of Chicago will present the initial interim efficacy and safety findings from the phase III international ENDEAR study on April 24 at the Contemporary Clinical Issues Plenary Session. The study of 122 infants with SMA is comparing intrathecal administration of nusinersen against a sham procedure of a small needle prick on the lower back to look for differences at day 402 in the primary outcome of the percentage of patients who attain motor milestones as assessed by section 2 of the Hammersmith Infant Neurological Examination or the time to death or need for respiratory intervention. Charlotte J. Sumner, MD, of Johns Hopkins University, Baltimore, will discuss the study following Dr. Kuntz’s presentation.
The second nusinersen trial to be reported at the meeting will describe interim results of the drug’s efficacy and safety in children with later-onset SMA in the phase III CHERISH study. At the Emerging Science Platform Session on April 25, Richard S. Finkel, MD, of Nemours Children’s Hospital in Orlando, Fla., will discuss how the primary outcome of the Hammersmith Functional Motor Scale–Expanded score changed from baseline to 15 months following intrathecal injection or a sham procedure in children aged 2-12 years.
An investigational SMA type 1 treatment just beginning testing in clinical trials will also receive attention in a plenary session and a platform session. In the Clinical Trials Plenary Session on April 25, Jerry R. Mendell, MD, of Nationwide Children’s Hospital, Columbus, Ohio, will report on the first gene therapy trial for SMA type 1, a phase I trial of AVXS-101, which delivers the SMN gene in a AAV9 viral vector that is able to cross the blood-brain barrier. The primary objective of the trial is to assess safety of a single intravenous dose. The secondary objectives include survival (avoidance of death/permanent-ventilation) and the ability to sit unassisted. Other analyses of data from the phase I trial will be reported during the “Motor Neuron Diseases: Biomarkers, Outcome Measures, and Therapeutics,” platform session on April 24, including the evaluation of preexisting anti-AAV9 antibodies and the proportion of patients who achieve CHOP-INTEND scores of 50 and above and sit unassisted.
A variety of plenary and emerging science sessions at this year’s annual meeting of the American Academy of Neurology in Boston will highlight clinical research efforts to treat children with spinal muscular atrophy.
At the Hot Topics Plenary Session on April 22, Claudia A. Chiriboga, MD, of Columbia University, New York, will discuss the results of clinical trials involving antisense oligonucleotide treatments for spinal muscular atrophy (SMA), including the recently approved nusinersen (Spinraza), which promotes transcription of the full-length survival motor neuron (SMN) protein from the SMN2 gene.
In the first of two reports on new clinical research about nusinersen, Nancy L. Kuntz, MD, of Ann & Robert H. Lurie Children’s Hospital of Chicago will present the initial interim efficacy and safety findings from the phase III international ENDEAR study on April 24 at the Contemporary Clinical Issues Plenary Session. The study of 122 infants with SMA is comparing intrathecal administration of nusinersen against a sham procedure of a small needle prick on the lower back to look for differences at day 402 in the primary outcome of the percentage of patients who attain motor milestones as assessed by section 2 of the Hammersmith Infant Neurological Examination or the time to death or need for respiratory intervention. Charlotte J. Sumner, MD, of Johns Hopkins University, Baltimore, will discuss the study following Dr. Kuntz’s presentation.
The second nusinersen trial to be reported at the meeting will describe interim results of the drug’s efficacy and safety in children with later-onset SMA in the phase III CHERISH study. At the Emerging Science Platform Session on April 25, Richard S. Finkel, MD, of Nemours Children’s Hospital in Orlando, Fla., will discuss how the primary outcome of the Hammersmith Functional Motor Scale–Expanded score changed from baseline to 15 months following intrathecal injection or a sham procedure in children aged 2-12 years.
An investigational SMA type 1 treatment just beginning testing in clinical trials will also receive attention in a plenary session and a platform session. In the Clinical Trials Plenary Session on April 25, Jerry R. Mendell, MD, of Nationwide Children’s Hospital, Columbus, Ohio, will report on the first gene therapy trial for SMA type 1, a phase I trial of AVXS-101, which delivers the SMN gene in a AAV9 viral vector that is able to cross the blood-brain barrier. The primary objective of the trial is to assess safety of a single intravenous dose. The secondary objectives include survival (avoidance of death/permanent-ventilation) and the ability to sit unassisted. Other analyses of data from the phase I trial will be reported during the “Motor Neuron Diseases: Biomarkers, Outcome Measures, and Therapeutics,” platform session on April 24, including the evaluation of preexisting anti-AAV9 antibodies and the proportion of patients who achieve CHOP-INTEND scores of 50 and above and sit unassisted.
A variety of plenary and emerging science sessions at this year’s annual meeting of the American Academy of Neurology in Boston will highlight clinical research efforts to treat children with spinal muscular atrophy.
At the Hot Topics Plenary Session on April 22, Claudia A. Chiriboga, MD, of Columbia University, New York, will discuss the results of clinical trials involving antisense oligonucleotide treatments for spinal muscular atrophy (SMA), including the recently approved nusinersen (Spinraza), which promotes transcription of the full-length survival motor neuron (SMN) protein from the SMN2 gene.
In the first of two reports on new clinical research about nusinersen, Nancy L. Kuntz, MD, of Ann & Robert H. Lurie Children’s Hospital of Chicago will present the initial interim efficacy and safety findings from the phase III international ENDEAR study on April 24 at the Contemporary Clinical Issues Plenary Session. The study of 122 infants with SMA is comparing intrathecal administration of nusinersen against a sham procedure of a small needle prick on the lower back to look for differences at day 402 in the primary outcome of the percentage of patients who attain motor milestones as assessed by section 2 of the Hammersmith Infant Neurological Examination or the time to death or need for respiratory intervention. Charlotte J. Sumner, MD, of Johns Hopkins University, Baltimore, will discuss the study following Dr. Kuntz’s presentation.
The second nusinersen trial to be reported at the meeting will describe interim results of the drug’s efficacy and safety in children with later-onset SMA in the phase III CHERISH study. At the Emerging Science Platform Session on April 25, Richard S. Finkel, MD, of Nemours Children’s Hospital in Orlando, Fla., will discuss how the primary outcome of the Hammersmith Functional Motor Scale–Expanded score changed from baseline to 15 months following intrathecal injection or a sham procedure in children aged 2-12 years.
An investigational SMA type 1 treatment just beginning testing in clinical trials will also receive attention in a plenary session and a platform session. In the Clinical Trials Plenary Session on April 25, Jerry R. Mendell, MD, of Nationwide Children’s Hospital, Columbus, Ohio, will report on the first gene therapy trial for SMA type 1, a phase I trial of AVXS-101, which delivers the SMN gene in a AAV9 viral vector that is able to cross the blood-brain barrier. The primary objective of the trial is to assess safety of a single intravenous dose. The secondary objectives include survival (avoidance of death/permanent-ventilation) and the ability to sit unassisted. Other analyses of data from the phase I trial will be reported during the “Motor Neuron Diseases: Biomarkers, Outcome Measures, and Therapeutics,” platform session on April 24, including the evaluation of preexisting anti-AAV9 antibodies and the proportion of patients who achieve CHOP-INTEND scores of 50 and above and sit unassisted.
Coming Soon: Highlights from AAN's Annual Meeting
Check back for highlights from this year's meeting in Boston.
Check back for highlights from this year's meeting in Boston.
Check back for highlights from this year's meeting in Boston.