Nusinersen in adult patients with 5q spinal muscular atrophy: A multicenter observational cohorts' study

• DOI: 10.1111/ene.15501
• Publication Date: 2022-07-25T05:34:32Z
• Authors: Juan F. Vázquez‐C...; Mónica Povedano; Andrés E. Nascimi...; Antonio Moreno Es...; Solange Kapetanov...; Raul Dominguez; Jessica M. Exposito; Laura González; Carla Marco; Julita Medina Cas...; Nuria Muelas; Daniel Natera de ...; Nancy Carolina Ñu...; Inmaculada Pitarc...; Teresa Sevilla; David Hervás
• Keywords: Progressive muscular atrophy

The aim was to assess the safety and efficacy of nusinersen in adult 5q spinal muscular atrophy (SMA) patients.Patients older than 15 years followed for at least 6 months with one motor scale (Hammersmith Functional Motor Scale Expanded, HFMSE; Revised Upper Limb Module, RULM) five referral centers were included. clinical patients' global impression change (CGI-C PGI-C) recorded treated patients last visit. scales (Egen Klassification, EK2; Amyotrophic Lateral Sclerosis Rating Scale, ALSFRS-R) percentage predicted forced vital capacity collected when available.Seventy-nine SMA (39 nusinersen) Compared untreated patients, showed a significant improvement 2 points (±0.46) RULM (p < 0.001) after months. After mean follow-up 16 months, treatment associated HFMSE (odds ratio [OR] 1.15, p = 0.006), 6-min walk test (OR 1.07, EK2 0.81, 0.001). more experienced clinically meaningful improvements all scales, but these differences statistically only 0.033), ALSFRS-R 0.005) According CGI-C PGI-C, 64.1% 61.5% improved treatment. Being non-sitter less response treatment, whilst longer time better response. Most (77%) presented adverse event, mostly mild.Nusinersen is some patients. severely affected complex spines are probably those most unfavorable risk-benefit ratio.