Aravindhan Veerapandiyan1, Katy Eichinger2, Debra Guntrum2, Jennifer Kwon3, Lindsay Baker2, Erin Collins2, Emma Ciafaloni2. 1. Department of Pediatrics, Division of Neurology, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas. 2. Department of Neurology, Division of Neuromuscular Medicine, University of Rochester Medical Center, Rochester, New York. 3. Department of Neurology, Division of Pediatric Neurology, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin.
Abstract
INTRODUCTION: Clinical trials data concerning use of nusinersen in older spinal muscular atrophy (SMA) patients is lacking. We describe our center's experience in using intrathecal nusinersen for older patients in the clinical setting. METHODS: Retrospective study. RESULTS: Twelve patients (12-52 years old) were treated with nusinersen. Mean follow-up duration was 17.4 months (range, 4-26 months). All patients had scoliosis; 10 had spinal fusion/instrumentation. All procedures (30 cervical and 57 lumbar punctures) were technically successful. The only side effects were postprocedural headache (9%) and site pain (5.7%). Functional assessments showed stability in 6/9 patients and improvement in 3/9 patients. Subjective improvements in endurance, hand strength, and bulbar functioning critical for activities of daily living were reported in 8/12 patients. None of the patients has discontinued treatment so far. DISCUSSION: Intrathecal nusinersen can be safely delivered in older SMA patients. Available functional outcome measures are not adequate to capture meaningful subjective improvements.
INTRODUCTION: Clinical trials data concerning use of nusinersen in older spinal muscular atrophy (SMA) patients is lacking. We describe our center's experience in using intrathecal nusinersen for older patients in the clinical setting. METHODS: Retrospective study. RESULTS: Twelve patients (12-52 years old) were treated with nusinersen. Mean follow-up duration was 17.4 months (range, 4-26 months). All patients had scoliosis; 10 had spinal fusion/instrumentation. All procedures (30 cervical and 57 lumbar punctures) were technically successful. The only side effects were postprocedural headache (9%) and site pain (5.7%). Functional assessments showed stability in 6/9 patients and improvement in 3/9 patients. Subjective improvements in endurance, hand strength, and bulbar functioning critical for activities of daily living were reported in 8/12 patients. None of the patients has discontinued treatment so far. DISCUSSION: Intrathecal nusinersen can be safely delivered in older SMApatients. Available functional outcome measures are not adequate to capture meaningful subjective improvements.
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