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Safety, Tolerability, and Effect of Nusinersen in Non-ambulatory Adults With Spinal Muscular Atrophy. | LitMetric

AI Article Synopsis

  • The study examined the safety and effectiveness of nusinersen treatment in non-ambulatory adults with spinal muscular atrophy (SMA), focusing on participants aged 18 and older with genetic confirmation.
  • Outcomes were evaluated at 2, 6, 10, and 14 months post-treatment using various measures, but most showed stability rather than improvement, with adverse events including hospitalizations for pneumonia and other infections.
  • Overall, nusinersen was deemed safe and well-tolerated, leading to stable functional outcomes; however, there were indications of increased motor unit size and stability in CMAP without significant clinical improvements.

Article Abstract

Investigation of the safety, tolerability, and treatment effect of nusinersen treatment in non-ambulatory adults with spinal muscular atrophy (SMA). Non-ambulatory individuals, aged 18 years or older with genetically confirmed 5q SMA were enrolled. In participants with spinal fusion, fluoroscopy guided cervical C1-C2 lateral approach was used. Outcomes at 2, 6, 10, and 14 months post-treatment were compared with baseline assessment. Forced vital capacity (FVC) was the primary outcome, and RULM, HFMSE, the modified SMA-FRS, and ulnar nerve electrophysiology [compound muscle action potential (CMAP), single motor unit size, and motor unit number] were secondary. Adverse and serious adverse events and clinically significant vital sign or lab abnormalities were recorded. Results from 12 women and 7 men (mean age: 39.7 ± 13.9, range: 21-64 years) were analyzed. No clinically significant changes of vital signs or laboratory parameters were observed. Five participants were hospitalized for pneumonia. Other adverse events included headache, back pain, cervical injection site pain, and upper respiratory and urinary tract infections. High baseline protein/creatinine ratio without significant change on treatment noted in 4 participants. FVC was feasible in all participants. HFMSE and RULM were not feasible in the majority of participants. FVC and functional outcomes were stable without improvement. CMAP and single motor unit potential sizes showed enlargement while motor unit numbers were stable. Nusinersen, including C1/C2 delivery, was safe overall and well-tolerated. Several outcome measures were limited by floor effect. Overall, treatment resulted in stability of motor outcomes, but motor unit and CMAP size were increased.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8085528PMC
http://dx.doi.org/10.3389/fneur.2021.650532DOI Listing

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