Fatigue in adults with spinal muscular atrophy under treatment with nusinersen
| Autor: | O. von Velsen, Benjamin Stolte, Kathrin Kizina, Christoph Kleinschnitz, Andreas Totzeck, Claudia Ose, Saskia Bolz, Tim Hagenacker, Melina Schlag |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
Adult
Male Neuromuscular disease Oligonucleotides Medizin lcsh:Medicine Walk Test Article Muscular Atrophy Spinal Young Adult 03 medical and health sciences 0302 clinical medicine Quality of life Humans Medicine Motor neuron disease Young adult lcsh:Science Fatigue Multidisciplinary business.industry lcsh:R Progressive muscle weakness Spinal muscular atrophy Middle Aged Prognosis medicine.disease Survival of Motor Neuron 1 Protein Neurology Walk test 030220 oncology & carcinogenesis Anesthesia Ambulatory lcsh:Q Female Nusinersen business Neurological disorders 030217 neurology & neurosurgery Follow-Up Studies |
| Zdroj: | Scientific Reports Scientific Reports, Vol 10, Iss 1, Pp 1-11 (2020) |
| ISSN: | 2045-2322 |
| DOI: | 10.1038/s41598-020-68051-w |
| Popis: | 5q-Associated spinal muscular atrophy is a hereditary neuromuscular disease leading to progressive muscle weakness in which fatigue occurs and affects quality of life. Treatment with the antisense oligonucleotide nusinersen has been shown to improve motor function. Fatigue can be measured within the Fatigue Severity Scale (FSS). FSS is a self-reported questionnaire consisting of nine items to quantify fatigue severity within the last week. Higher values indicating a higher severity. Using the FSS, fatigue was measured in 28 adult patients, subdivided into ambulatory and non-ambulatory, suffering from a genetically confirmed 5q-SMA under treatment with nusinersen in accordance with the label. Correlations were performed among FSS and motor scales, 6-minute walk test (6MWT) and Hammersmiths Functional Motor Scale Expanded (HFMSE). Evaluation was performed prior to treatment initiation and after 6 and 10 months. The mean FSS score for all 28 patients at baseline was 4.61 ± 1.44. After 6 months mean FSS score significantly reduced to 3.92 ± 1.35. After 10 months mean FSS score had not differed from baseline, 3.84 ± 1.25. A moderate negative correlation of the difference of FSS and 6MWT after 6 months compared to baseline conditions was measured. Nusinersen reduces fatigue as measured by the FSS in adult patients with 5q-SMA transiently after initiation of treatment. There was no reduction of FSS 10 months after the beginning of treatment when compared to baseline. |
| Databáze: | OpenAIRE |
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