Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Meta-Analysis
. 2024 May 7;19(5):e0302860.
doi: 10.1371/journal.pone.0302860. eCollection 2024.

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Brígida Dias Fernandes  1   2 , Bárbara Corrêa Krug  1   3 , Fernanda D'Athayde Rodrigues  1   4 , Hérica Núbia Cardoso Cirilo  1   5 , Stéfani Sousa Borges  1 , Ida Vanessa D Schwartz  6 , Livia Fernandes Probst  1 , Ivan Zimmermann  1   7
Affiliations
Meta-Analysis

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Brígida Dias Fernandes et al. PLoS One. .

Abstract

Background: Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety.

Objective: Conduct a meta-analysis to assess the efficacy and safety of onasemnogene abeparvovec in patients diagnosed with SMA type 1, based on the available evidence.

Methods: This article results from searches conducted on databases up to November 2022. Outcomes of interest were global survival and event-free survival, improvement in motor function and treatment-related adverse events. Risk of bias assessment and certainty of evidence were performed for each outcome. Proportional meta-analysis models were performed when applicable.

Results: Four reports of three open-label, non-comparative clinical trials covering 67 patients were included. Meta-analyses of data available in a 12-month follow-up estimate a global survival of 97.56% (95%CI: 92.55 to 99.86, I2 = 0%, n = 67), an event-free survival of 96.5% (95%CI: 90.76 to 99.54, I2 = 32%, n = 66) and a CHOP-INTEND score ≥ 40 points proportion of 87.28% (95%CI: 69.81 to 97.83, I2 = 69%, n = 67). Proportion of 52.64% (95%CI: 27.11 to 77.45, I2 = 78%, n = 67) of treatment-related adverse events was estimated.

Conclusion: The results indicate a potential change in the natural history of type 1 SMA, but the methodological limitations of the studies make the real extent of the technology's long-term benefits uncertain.

PubMed Disclaimer

Conflict of interest statement

The authors have declared that no competing interests exist.

Figures

Fig 1
Fig 1. Flowchart of studies’ selection.
Adapted from: Page et al., 2021 [19]. Available at: http://www.prisma-statement.org/.
Fig 2
Fig 2
Risk of bias assessment, outcomes of global survival and mechanical-ventilation-free survival (A), improvement in motor function (B) and treatment-related adverse events (C).
Fig 3
Fig 3
Meta-analysis of a) overall survival, b) event-free survival and c) CHOP-INTEND ≥ 40 following administration of onasemnogene abeparvovec in SMA type 1 patients.
Fig 4
Fig 4
Meta-analysis of a) serious adverse events and b) drug-related adverse events following administration of onasemnogene abeparvovec in SMA type 1 patients.
See this image and copyright information in PMC

References

    1. Prior TW, Leach ME, Finanger E. Spinal Muscular Atrophy. 2000. Feb 24 [Updated 2020 Dec 3]. In: Adam MP, Mirzaa GM, Pagon RA, et al.., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2023. - PubMed
    1. Mercuri E, Bertini E, Iannaccone ST. Childhood spinal muscular atrophy: controversies and challenges. Lancet Neurol. 2012. May;11(5):443–52. doi: 10.1016/S1474-4422(12)70061-3 . - DOI - PubMed
    1. Verhaert IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al.. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy—a literature review. Orphanet J Rare Dis. 2017. Jul 4;12(1):124. doi: 10.1186/s13023-017-0671-8 - DOI - PMC - PubMed
    1. Kingsmore SF, Henderson A, Owen MJ, et al.. Measurement of genetic diseases as a cause of mortality in infants receiving whole genome sequencing. npj Genom. Med. 5, 49 (2020). doi: 10.1038/s41525-020-00155-8 - DOI - PMC - PubMed
    1. D’Amico A, et al.. 2011. Spinal muscular atrophy. Orphanet Journal of Rare Diseases 6 (71) 1–10. doi: 10.1186/1750-1172-6-71 - DOI - PMC - PubMed

Publication types

MeSH terms