Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4
Verify – Wed, 16 Sep 2020 06:00:00 -0400
Bram De Wel,Veerle Goosens,Atka Sobota,Elke Van Camp,Ellen Geukens,Griet Van Kerschaver,Marlène Jagut,Kathleen Claes,Kristl G Claeys
Journal of neurology
CONCLUSIONS: We conclude that hand grip strength and hand motor function, as well as MRC sum scores improved significantly in nusinersen-treated adult patients with SMA types 3 and 4.
J Neurol. 2020 Sep 15. doi: 10.1007/s00415-020-10223-9. Online ahead of print.
BACKGROUND: Nusinersen recently became available as the first treatment for Spinal Muscular Atrophy (SMA) and data on its effectiveness and safety in adult SMA patients are still scarce.
METHODS: We evaluated the effectiveness and safety of nusinersen treatment during 14 months in 16 adult patients with SMA types 3 and 4 in a prospective study, and retrospectively detailed the natural history of 48 adult SMA patients types 2, 3 and 4.
RESULTS: Hand grip strength (p = 0.03), hand motor function (p = 0.04) as assessed by a sub-score of the Revised Upper Limb Module (RULM) and the Medical Research Council (MRC) sum score (p = 0.04) improved significantly at month 14. Importantly, the MRC sum score had declined significantly (p < 0.01) prior to start of treatment in these patients. A minimal clinically important difference (MCID) in the Hammersmith Functional Motor Scale Expanded (HFMSE) and RULM scores was achieved in 31% and 50% of the patients, respectively, but the mean changes from baseline failed to reach significance. Forced Vital Capacity (FVC) transiently increased at month 6 (p = 0.01), whereas the Peak Expiratory Flow (PEF) did not. The Activity Limitations scale declined significantly prior to start of treatment (p < 0.01) and showed an improvement with nusinersen which was not significant. The safety evaluation did not reveal serious adverse events and no signs of nephrotoxicity or antisense oligonucleotide (ASO)-mediated inflammation. CONCLUSIONS: We conclude that hand grip strength and hand motor function, as well as MRC sum scores improved significantly in nusinersen-treated adult patients with SMA types 3 and 4. PMID:32935160 | DOI:10.1007/s00415-020-10223-9 PUBMED ID: pubmed:32935160 OTHER ID: pmid:32935160,doi:10.1007/s00415-020-10223-9 PUBLICATION DATE: Wed, 16 Sep 2020 06:00:00 -0400 2020-09-16 RETRIEVAL DATE : 09/16/20 06:44AM LINK - PUBMED: https://pubmed.ncbi.nlm.nih.gov/32935160/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1l7lJQBQXfTBJkL86rnvYKzafMiKbgcUrlv_X8_D_H5EuRmkjR&fc=20200708141943&ff=20200916064412&v=2.11.5 LINK - DOI: https://doi.org/10.1007/s00415-020-10223-9 LINK - FULL TEXT: Pending NOTES: None