| アブストラクト | Background/Objectives: The approval of disease-modifying therapies has significantly improved outcomes for patients with spinal muscular atrophy (SMA), yet their long-term safety profiles remain under continuous evaluation. This study aimed to assess trends in the reporting of suspected adverse drug reactions (ADRs) associated with nusinersen, onasemnogene abeparvovec, and risdiplam across the European Union. Methods: We conducted a secondary analysis of annual suspected ADR data reported to EudraVigilance from 2017 to 2024 for the three approved disease-modifying therapies for SMA. On top of general reporting trend, specific adverse reactions of interest included post-lumbar puncture syndrome for nusinersen, liver toxicity and elevated serum troponin for onasemnogene abeparvovec, and respiratory and gastrointestinal reactions for risdiplam. Joinpoint regression analysis was used to evaluate annual percent changes and identify statistically significant trend segments for each medicine. Results: The reporting of suspected ADRs for nusinersen showed an initial increase, followed by a significant decline after 2019. Onasemnogene abeparvovec exhibited a continued but decelerating increase in suspected ADRs, while risdiplam demonstrated a consistent upward trend across all reported reactions. Conclusions: Diverging patterns in adverse reaction reporting suggest a stabilizing safety profile for nusinersen and potential emerging safety signals for risdiplam and onasemnogene abeparvovec, underscoring the need for ongoing continued pharmacovigilance (e.g., post-authorization studies and spontaneous reporting). |
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| ジャーナル名 | Neurology international |
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| Pubmed追加日 | 2025/10/28 |
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| 投稿者 | Belancic, Andrej; Mas, Petar; Stevic, Ivana; Vitezic, Dinko; Jankovic, Slobodan |
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| 組織名 | Department of Basic and Clinical Pharmacology and Toxicology, Faculty of;Medicine, University of Rijeka, 51000 Rijeka, Croatia.;Agency for Medicinal Products and Medical Devices, 10000 Zagreb, Croatia.;Faculty of Pharmacy, University of Belgrade, 11000 Belgrade, Serbia.;Faculty of Medical Sciences, University of Kragujevac, 34000 Kragujevac, Serbia. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/41149786/ |
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