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Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters
Journal article   Open access   Peer reviewed

Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters

Benjamin Stolte, Michael Nonnemacher, Kathrin Kizina, Saskia Bolz, Andreas Totzeck, Andreas Thimm, Bernd Wagner, Cornelius Deuschl, Christoph Kleinschnitz and Tim Hagenacker
Journal of neurology, v 268(12), pp 4667-4679
Dec 2021
DOI: https://doi.org/10.1007/s00415-021-10569-8
PMID: 33899154
Featured in Collection :   UN Sustainable Development Goals @ Drexel
url
https://link.springer.com/content/pdf/10.1007/s00415-021-10569-8.pdfView
Published, Version of Record (VoR) Open
url
https://doi.org/10.1007/s00415-021-10569-8View
Published, Version of Record (VoR) Open

Abstract

Adult Humans Laboratories Muscular Atrophy, Spinal - drug therapy Oligonucleotides Retrospective Studies Spinal Muscular Atrophies of Childhood
Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) that improves motor function in patients with spinal muscular atrophy (SMA). In addition to efficacy, the safety of a therapy is the decisive factor for the success of the treatment. For some ASOs, various organ toxicities have been described, such as thrombocytopenia, renal and liver impairment, or coagulation abnormalities. However, systematic data on laboratory parameters under treatment with nusinersen are mainly available from studies in infants and children. Therefore, our aim was to assess the safety of nusinersen therapy in adult SMA patients. Laboratory data from 404 nusinersen injections performed in 50 adult patients with SMA type 2 and type 3 were retrospectively analyzed. The total observation period was 76.9 patient-years, and patients received up to 12 injections. Our data provides no new safety concerns. In cerebrospinal fluid (CSF), the mean white blood cell count and lactate remained stable over time. Total CSF protein increased by 2.9 mg/dL. No change in mean platelet count was observed under therapy. Only one patient showed sporadic mild thrombocytopenia. Coagulation parameters and inflammatory markers were stable. The mean creatinine level decreased by 0.09 mg/dL. Analysis of mean liver enzyme levels revealed no relevant changes during treatment. Our data demonstrate a favorable safety profile of nusinersen therapy in adult SMA patients under longer-term "real-world" conditions. In particular, we found no evidence of clinically relevant platelet declines, coagulopathies, or renal or hepatic organ toxicities, which are common concerns with the use of ASOs.

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#3 Good Health and Well-Being

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Web of Science research areas
Clinical Neurology
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