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Abstract Details

Real-World Patterns of Nusinersen Use in Adult Patients with Spinal Muscular Atrophy (SMA) in the US: A Multi-Site Chart Review Study
Child Neurology and Developmental Neurology
Child Neurology and Developmental Neurology Posters (7:00 AM-5:00 PM)
077

To describe patterns of nusinersen use among adults with SMA treated with nusinersen in US clinics.

Nusinersen, an antisense oligonucleotide approved for the treatment of SMA, is administered intrathecally at doses of 12 mg with 3 loading doses at 14-day intervals and a 4th loading dose 30 days thereafter. Maintenance doses are administered every 4 months. Limited evidence exists on real-world adherence to nusinersen.

Data were curated from an ongoing multi-site retrospective chart review study of nusinersen-treated adults in the US. Eligible patients were age ≥18 years at treatment initiation between January 2017 and February 2019 with a genetically confirmed diagnosis of SMA. The percentage of patients who discontinued (defined as having 2 missed doses) and days between doses are reported. Doses were considered not on time using grace periods of ±7 days for loading doses and ±28 days for maintenance doses.   
Retrospective data were collected from electronic health records through August 2019 from 38 patients. In this interim analysis the median age at treatment initiation was 28.5 years (range 20-68), 57.9% were SMA Type III, and 86.8% were nonambulatory at baseline, suggesting loss of motor function. Patients had a median follow-up time of 15 months (range: 4-27) with a median of 6 doses received (range: 4-10). 86.8% patients continuously received nusinersen: 4 discontinued after the loading dose period and 1 discontinued after the 1st maintenance dose. 76.3% patients received all nusinersen doses on time. Of the 9 patients with at least one dose earlier or later than the expected date, all patients resumed nusinersen after that dose. The final study data from a larger sample will be presented.

Nusinersen-treated adult patients varied widely in age. The vast majority of patients remained on drug receiving treatment according to the dosing schedule.

Sponsored by: Biogen
Authors/Disclosures
Lauren B. Elman, MD
PRESENTER 		Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche/Genentech. Dr. Elman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Edgewise Therapeutics. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis Pharamaceuticals. Dr. Elman has received publishing royalties from a publication relating to health care.
No disclosure on file
Crystal M. Proud, MD (CSG Child & Adolescent Neurology) Dr. Proud has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avexis. Dr. Proud has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Proud has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sarepta. Dr. Proud has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Avexis. Dr. Proud has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Proud has received research support from Biogen. The institution of Dr. Proud has received research support from Sarepta. The institution of Dr. Proud has received research support from Avexis. The institution of Dr. Proud has received research support from PTC. The institution of Dr. Proud has received research support from CSL Behring. The institution of Dr. Proud has received research support from Scholar Rock. The institution of Dr. Proud has received research support from Catabasis.
Margaret R. Frey, DO Dr. Frey has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Biogen. Dr. Frey has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Frey has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Allergan. Dr. Frey has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen.
Senda Ajroud-Driss, MD, FÂ鶹´«Ã½Ó³»­ (Northwestern University) Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Astrazeneca. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Ajroud-Driss has received research support from NIH. The institution of Dr. Ajroud-Driss has received research support from University of Minesota/FDA. The institution of Dr. Ajroud-Driss has received research support from Edgewise Therapeutics. The institution of Dr. Ajroud-Driss has received research support from Biogen. The institution of Dr. Ajroud-Driss has received research support from Woolsey Pharmaceuticals. The institution of Dr. Ajroud-Driss has received research support from Uniqure Biopharma. The institution of Dr. Ajroud-Driss has received research support from Coya Therapeutics. The institution of Dr. Ajroud-Driss has received research support from Novartis. The institution of Dr. Ajroud-Driss has received research support from Alnylam pharmaceuticals. The institution of Dr. Ajroud-Driss has received research support from Healey and AMG center for ALS . The institution of Dr. Ajroud-Driss has received research support from les Turner ALS Foundation. The institution of Dr. Ajroud-Driss has received research support from Hopewell. The institution of Dr. Ajroud-Driss has received research support from Peripheral neuropathy foundation. Dr. Ajroud-Driss has received publishing royalties from a publication relating to health care.
M E. McCormick, DO Dr. McCormick has nothing to disclose.
David Michelson, MD (Loma Linda University Health Care) Dr. Michelson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech. Dr. Michelson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for OptumRx. The institution of Dr. Michelson has received research support from UCB SA. The institution of Dr. Michelson has received research support from Teva Pharmaceuticals USA. The institution of Dr. Michelson has received research support from PTC Therapeutics, Inc. The institution of Dr. Michelson has received research support from Novartis. The institution of Dr. Michelson has received research support from Biogen, Inc. Dr. Michelson has received publishing royalties from a publication relating to health care.
Michael Cartwright, MD (Wake Forest School of Medicine) Dr. Cartwright has received publishing royalties from a publication relating to health care.
Terry D. Heiman-Patterson, MD (Temple University Lewis Katz School of Medicine) Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Heiman-Patterson has received personal compensation in the range of $0-$499 for serving as a Consultant for novartis. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for biogen. The institution of Dr. Heiman-Patterson has received research support from MTPA. The institution of Dr. Heiman-Patterson has received research support from State of Pennsylvania . The institution of Dr. Heiman-Patterson has received research support from ALS Association. The institution of Dr. Heiman-Patterson has received research support from ALS United. The institution of Dr. Heiman-Patterson has received research support from ALS Hope Foundation.
Joseph Choi, MD (INOVA Health Systems) Dr. Choi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Choi has received personal compensation in the range of $500-$4,999 for serving as a Case reviewer with Megellan Rx Management.
No disclosure on file
No disclosure on file
No disclosure on file
Angela Paradis Angela Paradis has received personal compensation for serving as an employee of Biogen. An immediate family member of Angela Paradis has received personal compensation for serving as an employee of Biogen. Angela Paradis has received stock or an ownership interest from Biogen. An immediate family member of Angela Paradis has received stock or an ownership interest from Biogen.