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Muscle Nerve. 2019 Nov 26. doi: 10.1002/mus.26769. [Epub ahead of print]

Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience.

Author information

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.

KEYWORDS:

SMA; adolescents; adults; intrathecal; nusinersen; spinraza

PMID:
31773738
DOI:
10.1002/mus.26769

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