| アブストラクト | The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma((R))), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma((R)) therapy has proven to lead to functional improvements in SMA children, some gaps in its safety profile still need to be investigated. To better characterize the Zolgensma((R)) safety profile, we conducted a retrospective observational study, analyzing all the Individual Case Safety Reports (ICSRs) referred to it and collected in the European pharmacovigilance database between 1 January 2019 and 22 September 2023. We found 661 ICSRs related to Zolgensma((R)), with a growing trend in the annual reporting. The majority of the reports were sent by healthcare professionals and referred to infant females. In more than 90% of the cases, Zolgensma((R)) was the only reported suspected drug. Out of a total of 2744 reported ADRs, increased hepatic enzymes, pyrexia, vomiting, and thrombocytopenia were the most commonly reported adverse reactions. Of these adverse reactions (ADRs), 56.9% were serious, causing or prolonging the patient's hospitalization. A total of 39 ICSRs related to cases with a fatal outcome. Alterations in the heart rhythm, acute hepatic failure, and hepatic cytolysis emerged among the cardiac and hepatic disorders, respectively. |
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| ジャーナル名 | Pharmaceuticals (Basel, Switzerland) |
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| 投稿日 | 2024/3/28 |
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| 投稿者 | Ruggiero, Rosanna; Balzano, Nunzia; Nicoletti, Maria Maddalena; di Mauro, Gabriella; Fraenza, Federica; Campitiello, Maria Rosaria; Rossi, Francesco; Capuano, Annalisa |
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| 組織名 | Department of Experimental Medicine, University of Campania "Luigi Vanvitelli",;80138 Naples, Italy.;Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region,;Department of Precision Medicine, University of Campania "Luigi Vanvitelli",;UOC Pharmacy, AORN Santobono-Pausilipon Children's Hospital, 80122 Naples, Italy.;Department of Obstetrics and Gynaecology and Physiopathology of Human;Reproduction, ASL Salerno, 84124 Salerno, Italy.;Department of Life Sciences, Health and Health Professions, Link Campus;University, 00165 Rome, Italy. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/38543180/ |
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