Details

Autor(en) / Beteiligte

• Vázquez‐Costa, Juan F.
• Povedano, Mónica
• Nascimiento‐Osorio, Andrés E.
• Moreno Escribano, Antonio
• Kapetanovic Garcia, Solange
• Dominguez, Raul
• Exposito, Jessica M.
• González, Laura
• Marco, Carla
• Medina Castillo, Julita
• Muelas, Nuria
• Natera de Benito, Daniel
• Ñungo Garzón, Nancy Carolina
• Pitarch Castellano, Inmaculada
• Sevilla, Teresa
• Hervás, David

Titel

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

Ist Teil von

• European journal of neurology, 2022-11, Vol.29 (11), p.3337-3346

Ort / Verlag

England: John Wiley & Sons, Inc

Erscheinungsjahr

2022

Quelle

Wiley-Blackwell Journals

Beschreibungen/Notizen

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