The efficacy of Nusinersen treatment in children with spinal muscular atrophy: a systematic review

*Correspondence: [e-mail].

Abstract

Background: Spinal muscular atrophy (SMA) is a degenerative neuromuscular genetic disorder characterized by the progressive loss of spinal and brainstem motor neurons. Nusinersen has allowed an increase in life expectancy of SMA type 1 patients and provided the acquisition of milestones of motor development.

Objective: To analyze the efficacy of Nusinersen treatment in children with spinal muscular atrophy.

Methods: A literature review was carried out using the descriptors: Spinal; Muscular; Atrophy; Nusinersen; Treatment; Pediatric and the Boolean operator “AND”. The databases used were Pubmed, BVS and Scielo, adding the Free Full Text filter and restricting it to the period between 2010 and 2023, reaching 141 results in Pubmed. From these, 15 were selected by title. In the Virtual Health Library, 5 articles were selected by title, while in Scielo, 6 were selected by title. The final selection consisted of 26 articles, including randomized clinical trials, meta-analyses, systematic reviews and review, with publication data in the last 13 years.

Results: The main randomized clinical studies showed an improvement in motor results in children with SMA manifested by treatment with Nusinersen, in addition to an improvement in quality of life and nutritional support. Thus, after the institution of drug therapy, there was a greater differentiation in the age of infant death and the need for long-term ventilation. Nusinersen was also able to reduce mortality in children who weren’t in long-term ventilation, as well as increase the development of motor functions in SMA types 1 to 3. It was shown that, if the medication is instituted before the child develops symptoms, there is a better prognosis in relation to motor movements. The final results showed that 57% of the children who received Nusinersen had improvement in motricity compared to 26% of the control group, from the beginning of the treatment until the 15th month of follow-up. The incidence of adverse effects was similar in the control group and in the Nusinersen group.

Conclusion: Treatment with Nusinersen proved to be efficient in children with SMA, helping with motor activity and respiratory support, allowing a better quality of life.

 

MissingLinkText

Publikationsverlauf

Artikel online veröffentlicht:
12. Mai 2025