Avaliação da eficácia do nusinersena (Spinraza®) no tratamento de atrofia muscular espinhal: uma revisão sistemática

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Universidade Federal de Goiás


Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease characterized by mutations or deletions of the survival motor neuron 1 (SMN1) gene, which leads to degeneration of motor neurons of the spinal cord. Nusinersen, commercially known as Spinraza®, has been approved and incorporated into the Brazilian health system as the first pharmacological treatment for SMA, however, a number of ethical issues have been raised, indicating the need for further studies to confirm the drug’s efficacy. In view of this, the objectives of this study were to assess the efficacy of nusinersen (Spinraza®) in SMA’s treatment through a systematic review of randomized controlled trials (RCTs) and cohort studies, and to evaluate the methodological quality of the included studies. The identification and selection of the studies were conducted by two reviewers, blinded and independent in following databases: Cochrane Library, Embase, Lilacs (via Virtual Health Library) and Medline (via Pubmed). An additional search in references of the included articles and in the international database Clinical Trials was conducted to capture as many publications as possible. As inclusion criteria, were selected the full published full articles of human studies whose objective(s) was to evaluate the efficacy of nusinersena in SMA’s treatment. For quality assessment and bias identification, included RCTs were assessed according to the CONSORT (Consolidated Standards of Reporting Trials) Recommendation and cohort studies in accordance with STROBE (Strengthening the Reporting of Observational Studies in Epidemiology) Recommendation. The search in the databases returned 427 publications, of which 112 were removed by duplication and 309 excluded because they did not meet any of the inclusion criteria, totalizing six publications included in this review, of which two RCTs and four cohort studies. The total population of the analyzed articles yielded 461 both sexes individuals and diagnosed SMA Types I, II or III. The most used motor function improvement rating scales among the studies were HINE-2 and CHOP INTEND. The efficacy of nusinersen (Spinraza®) was compared between studies taking into account the research design, population included, and outcomes identified based on the progress of motor scale scores. The validity of the outcomes was discussed according to the quality of each publication and the risks of biases identified under the CONSORT and STROBE Recommendations. Financial support from the drug-producing industry was identified as the main bias in some of the studies. Scientific evidence has shown a significant improvement in motor function in patients treated with nusinersena, although the combination of the outcomes discussed in this review are not considered sufficient to confirm an increase in patient survival. It was identified that an improvement in the patterns of conduct and reporting of studies included in the review are important attitudes to be adopted.



BORGES, S. S. Avaliação da eficácia do nusinersena (Spinraza®) no tratamento de atrofia muscular espinhal: uma revisão sistemática. 2019. 113 f. Dissertação (Mestrado em Assistência e Avaliação em Saúde) - Universidade Federal de Goiás, Goiânia, 2019.