Seguridad y tolerabilidad del nusinersen y onasemnogene como tratamientos para la atrofia muscular espinal: revisión sistemática

Abstract

Introduction: Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by a deletion in exon 7 of the SMN1 gene on chromosome 5q13. This alteration leads to a reduction in SMN protein, which is essential for the function of motor neurons. The most affected age group is children, who present clinical features such as asthenia, atrophy, paralysis, and, in severe cases, respiratory failure. For this reason, it is essential to analyze the safety and tolerability of therapies such as nusinersen and onasemnogene in treating this disease. Objective: To compare the safety and tolerability between nusinersen and onasemnogene as treatments for spinal muscular atrophy. Methodology: A systematic review was conducted through searches in databases such as Scopus, PubMed, and ProQuest, using inclusion and exclusion criteria, keywords based on health descriptors, and Boolean operators such as "AND" and "OR." The results will be presented in tables following the PRISMA method, and the methodological quality will be analyzed using the Newcastle-Ottawa Scale. Results: A total of 105 publications were found, of which 18 in English were selected, including clinical trials, systematic reviews, and retrospective observational studies. Conclusions: According to the collected data, nusinersen is safer than onasemnogene, with side effects managed according to each patient's symptomatology. Specifically, tolerability with onasemnogene is greater in patients (infants) with SMA type 1 and in patients with SMA type 2 and 3 (preschoolers and adults) treated with nusinersen.