Journal article
Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters
Journal of neurology, Vol.268(12), pp.4667-4679
Dec 2021
PMID: 33899154
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
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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Details
- Title
- Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters
- Creators
- Benjamin Stolte - Essen University HospitalMichael Nonnemacher - Essen University HospitalKathrin Kizina - Essen University HospitalSaskia Bolz - Essen University HospitalAndreas Totzeck - Essen University HospitalAndreas Thimm - Essen University HospitalBernd Wagner - Essen University HospitalCornelius Deuschl - Essen University HospitalChristoph Kleinschnitz - Essen University HospitalTim Hagenacker - Essen University Hospital
- Publication Details
- Journal of neurology, Vol.268(12), pp.4667-4679
- Publisher
- Springer Nature
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Microbiology and Immunology
- Identifiers
- 991020100070204721
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- Clinical Neurology