The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials

Medicina

Basel Abdelazeem¹  Mahmoud Shaqfeh²  Nahla Ahmed El-Shahat³  Mennatullah Mohamed Eltaras³  Kirellos Said Abbas⁴  James Robert Brašić⁵ 

[1] Department of Medicine, McLaren Health Care, Flint, MI 48532, USA;Department of Neurology, McLaren Health Care, Flint, MI 48532, USA;Faculty of Medicine for Girls, Al-Azhar University, Cairo 11754, Egypt;Faculty of Medicine, Alexandria University, Alexandria 22621, Egypt;Section of High Resolution Brain Positron Emission Tomography Imaging, The Russell H. Morgan Department of Radiology and Radiological Science, Division of Nuclear Medicine and Molecular Imaging, The Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA;

关键词: adverse and beneficial effects;    alpha motor neuron;    bias;    brainstem;    evidence;    neurodegenerative;   

DOI  :  10.3390/medicina58020213

来源: DOAJ

【 摘 要 】

Background and objectives: Spinal muscular atrophy (SMA) is a neurodegenerative disease that leads to progressive proximal muscle weakness and muscle atrophy. To assess the beneficial and adverse effects of nusinersen, a promising intervention for SMA, we conducted a systematic search and meta-analysis of the published randomized control trials (RCTs) of nusinersen for SMA. Materials and methods: Utilizing the Preferred Reporting for Systematic Review and Meta-Analysis (PRISMA), we searched PubMed, Scopus, Web of Science, Cochrane Central, and Clinicaltrials.gov from inception to 22 July 2021. Results: Three RCTs satisfying the inclusion and exclusion criteria covered 274 patients: 178 patients in the nusinersen group. Our results show a significant risk difference (RD) in the motor milestone response (RD: 0.51; 95% CI: 0.39, 0.62; p < 0.00001) and improvement in the HINE-2 score (RD: 0.26; 95% CI: 0.12, 0.40; p < 0.0003) in the nusinersen group compared to the control group. Moreover, a significant decrease in the risk ratio (RR) for severe adverse events (RR: 0.72; 95% CI: 0.57, 0.92; p = 0.007) and any adverse event leading to treatment discontinuation (RR: 0.40; 95% CI: 0.22, 0.74; p = 0.004) was observed. An insignificant result was found for any adverse effects (RR: 0.93; 95% CI: 0.97, 1.01; p = 0.14) and for serious adverse effects (RR: 0.81; 95% CI: 0.60, 1.07; p = 0.14). Conclusions: This review provides evidence that nusinersen treatment was effective in treatment for infants with SMA and was associated with fewer severe adverse events; however, more RCTs are needed to establish evidence.

【 授权许可】

Unknown