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Journal of Neurology
Published in: Journal of Neurology 12/2021

Open Access 01-12-2021 | Spinal Muscular Atrophy | Original Communication

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

Authors: Benjamin Stolte, Michael Nonnemacher, Kathrin Kizina, Saskia Bolz, Andreas Totzeck, Andreas Thimm, Bernd Wagner, Cornelius Deuschl, Christoph Kleinschnitz, Tim Hagenacker

Published in: Journal of Neurology | Issue 12/2021

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Abstract

Background

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.

Methods

Laboratory data from 404 nusinersen injections performed in 50 adult patients with SMA type 2 and type 3 were retrospectively analyzed.

Results

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.

Conclusion

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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Metadata
Title
Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters
Authors
Benjamin Stolte
Michael Nonnemacher
Kathrin Kizina
Saskia Bolz
Andreas Totzeck
Andreas Thimm
Bernd Wagner
Cornelius Deuschl
Christoph Kleinschnitz
Tim Hagenacker
Publication date
01-12-2021
Publisher
Springer Berlin Heidelberg
Published in
Journal of Neurology / Issue 12/2021
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI
https://doi.org/10.1007/s00415-021-10569-8

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