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Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis
OBJECTIVES: To systematically analyze adverse events (AEs) in treatment of spinal muscular atrophy (SMA) with Nusinersen in children and adolescents. METHODS: The study is registered on PROSPERO (CRD42022345589). Databases were searched and literature relating to Nusinersen in the treatment of spina...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10167028/ https://www.ncbi.nlm.nih.gov/pubmed/37181426 http://dx.doi.org/10.3389/fped.2023.1152318 |
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author | Zhong, Zhi-Juan Zheng, Pi-Mei Dou, Hui-Hong Wang, Ji-Gan |
author_facet | Zhong, Zhi-Juan Zheng, Pi-Mei Dou, Hui-Hong Wang, Ji-Gan |
author_sort | Zhong, Zhi-Juan |
collection | PubMed |
description | OBJECTIVES: To systematically analyze adverse events (AEs) in treatment of spinal muscular atrophy (SMA) with Nusinersen in children and adolescents. METHODS: The study is registered on PROSPERO (CRD42022345589). Databases were searched and literature relating to Nusinersen in the treatment of spinal muscular atrophy in children from the start of database establishment to December 1, 2022, was retrospectively analyzed. R.3.6.3 statistical software was used, and random effects meta-analysis was performed to calculate weighted mean prevalence and 95% confidence intervals (CI). RESULTS: In total, 15 eligible studies were included, with a total of 967 children. Rate of definite Nusinersen-related AEs was 0.57% (95% CI: 0%–3.97%), and probable Nusinersen-related AEs 7.76% (95% CI: 1.85%–17.22%). Overall rate of AEs was 83.51% (95% CI: 73.55%–93.46%), and serious AEs 33.04% (95% CI: 18.15%–49.91%). For main specific AEs, fever was most common, 40.07% (95% CI: 25.14%–56.02%), followed by upper respiratory tract infection 39.94% (95% CI: 29.43%–50.94%), and pneumonia 26.62% (95% CI: 17.99%–36.25%). The difference in overall AE rates between the two groups (Nusinersen group and placebo group) was significant (OR = 0.27,95% CI: 0.08–0.95, P = 0.042). Moreover, incidence of serious adverse events, and fatal adverse events were both significantly lower than in the placebo group (OR = 0.47, 95%CI: 0.32–0.69, P < 0.01), and (OR = 0.37, 95%CI: 0.23–0.59, P < 0.01), respectively. CONCLUSION: Nusinersen direct adverse events are rare, and it can effectively reduces common, serious, and fatal adverse events in children and adolescents with spinal muscular atrophy. |
format | Online Article Text |
id | pubmed-10167028 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-101670282023-05-10 Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis Zhong, Zhi-Juan Zheng, Pi-Mei Dou, Hui-Hong Wang, Ji-Gan Front Pediatr Pediatrics OBJECTIVES: To systematically analyze adverse events (AEs) in treatment of spinal muscular atrophy (SMA) with Nusinersen in children and adolescents. METHODS: The study is registered on PROSPERO (CRD42022345589). Databases were searched and literature relating to Nusinersen in the treatment of spinal muscular atrophy in children from the start of database establishment to December 1, 2022, was retrospectively analyzed. R.3.6.3 statistical software was used, and random effects meta-analysis was performed to calculate weighted mean prevalence and 95% confidence intervals (CI). RESULTS: In total, 15 eligible studies were included, with a total of 967 children. Rate of definite Nusinersen-related AEs was 0.57% (95% CI: 0%–3.97%), and probable Nusinersen-related AEs 7.76% (95% CI: 1.85%–17.22%). Overall rate of AEs was 83.51% (95% CI: 73.55%–93.46%), and serious AEs 33.04% (95% CI: 18.15%–49.91%). For main specific AEs, fever was most common, 40.07% (95% CI: 25.14%–56.02%), followed by upper respiratory tract infection 39.94% (95% CI: 29.43%–50.94%), and pneumonia 26.62% (95% CI: 17.99%–36.25%). The difference in overall AE rates between the two groups (Nusinersen group and placebo group) was significant (OR = 0.27,95% CI: 0.08–0.95, P = 0.042). Moreover, incidence of serious adverse events, and fatal adverse events were both significantly lower than in the placebo group (OR = 0.47, 95%CI: 0.32–0.69, P < 0.01), and (OR = 0.37, 95%CI: 0.23–0.59, P < 0.01), respectively. CONCLUSION: Nusinersen direct adverse events are rare, and it can effectively reduces common, serious, and fatal adverse events in children and adolescents with spinal muscular atrophy. Frontiers Media S.A. 2023-04-25 /pmc/articles/PMC10167028/ /pubmed/37181426 http://dx.doi.org/10.3389/fped.2023.1152318 Text en © 2023 Zhong, Zheng, Dou and Wang. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY) (https://creativecommons.org/licenses/by/4.0/) . The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Pediatrics Zhong, Zhi-Juan Zheng, Pi-Mei Dou, Hui-Hong Wang, Ji-Gan Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title | Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title_full | Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title_fullStr | Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title_full_unstemmed | Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title_short | Adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: A systematic review and meta-analysis |
title_sort | adverse events in the treatment of spinal muscular atrophy in children and adolescents with nusinersen: a systematic review and meta-analysis |
topic | Pediatrics |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10167028/ https://www.ncbi.nlm.nih.gov/pubmed/37181426 http://dx.doi.org/10.3389/fped.2023.1152318 |
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