Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury
ABSTRACT Aims were to reveal types of onasemnogene abeparvovec‐xioi (OA)‐induced liver injury, their treatment patterns, utilization of healthcare, and treatment costs. This study employed secondary research to analyze OA‐induced liver injury using data from the EudraVigilance database, published ca...
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| Format: | Article |
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Wiley
2025-06-01
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| Series: | Pharmacology Research & Perspectives |
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| Online Access: | https://doi.org/10.1002/prp2.70134 |
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| author | Andrej Belančić Branislava Raičević Ivana Stević Dinko Vitezić Slobodan M. Janković |
| author_facet | Andrej Belančić Branislava Raičević Ivana Stević Dinko Vitezić Slobodan M. Janković |
| author_sort | Andrej Belančić |
| collection | DOAJ |
| description | ABSTRACT Aims were to reveal types of onasemnogene abeparvovec‐xioi (OA)‐induced liver injury, their treatment patterns, utilization of healthcare, and treatment costs. This study employed secondary research to analyze OA‐induced liver injury using data from the EudraVigilance database, published case reports, cohort studies, and clinical trials. The extracted data were analyzed to define real‐life clinical entities that could be clearly outlined as syndromes resulting from the OA‐induced liver injury, and further used in guiding the development of healthcare utilization matrices. Serbian healthcare costs were calculated by multiplying utilization figures by local unit prices, converted to Euros using exchange rates and adjusted by price level indices. A spreadsheet model with uniform distributions simulated costs for 1000 virtual patients, providing mean values and standard deviations for Serbia and the EU. From 1566 adverse event reports in the EudraVigilance database following OA therapy, 231 were hepatobiliary disorders, predominantly hypertransaminasaemia (30.7%; 71/231). Liver injury largely manifested as mild‐to‐moderate biochemical abnormalities, rarely progressing to severe complications, and was effectively managed with corticosteroid therapy. Economic analysis highlights the manageable burden of OA‐induced liver injury. In the EU, mild‐to‐moderate cases cost €823.7, while severe cases average €1638.6. Medication costs range from €26.8 for prednisone to €695.4 for severe cases requiring additional immunosuppressive agents like tacrolimus and mycophenolate mofetil. To conclude, OA‐induced liver injury, though notable, is clinically manageable with immunosuppressive therapy and rarely causes severe complications like encephalopathy or liver failure. Its modest costs do not undermine OA's cost‐effectiveness, supporting its transformative role in spinal muscular atrophy treatment. |
| format | Article |
| id | doaj-art-630da63d122d426eb4e1786d6c3a7296 |
| institution | OA Journals |
| issn | 2052-1707 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Wiley |
| record_format | Article |
| series | Pharmacology Research & Perspectives |
| spelling | doaj-art-630da63d122d426eb4e1786d6c3a72962025-08-20T02:07:24ZengWileyPharmacology Research & Perspectives2052-17072025-06-01133n/an/a10.1002/prp2.70134Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver InjuryAndrej Belančić0Branislava Raičević1Ivana Stević2Dinko Vitezić3Slobodan M. Janković4Department of Basic and Clinical Pharmacology and Toxicology Faculty of Medicine, University of Rijeka Rijeka CroatiaFaculty of Medical Sciences, University of Kragujevac Kragujevac SerbiaDepartment of Social Pharmacy and Pharmaceutical Legislation Faculty of Pharmacy, University of Belgrade Belgrade SerbiaDepartment of Basic and Clinical Pharmacology and Toxicology Faculty of Medicine, University of Rijeka Rijeka CroatiaFaculty of Medical Sciences, University of Kragujevac Kragujevac SerbiaABSTRACT Aims were to reveal types of onasemnogene abeparvovec‐xioi (OA)‐induced liver injury, their treatment patterns, utilization of healthcare, and treatment costs. This study employed secondary research to analyze OA‐induced liver injury using data from the EudraVigilance database, published case reports, cohort studies, and clinical trials. The extracted data were analyzed to define real‐life clinical entities that could be clearly outlined as syndromes resulting from the OA‐induced liver injury, and further used in guiding the development of healthcare utilization matrices. Serbian healthcare costs were calculated by multiplying utilization figures by local unit prices, converted to Euros using exchange rates and adjusted by price level indices. A spreadsheet model with uniform distributions simulated costs for 1000 virtual patients, providing mean values and standard deviations for Serbia and the EU. From 1566 adverse event reports in the EudraVigilance database following OA therapy, 231 were hepatobiliary disorders, predominantly hypertransaminasaemia (30.7%; 71/231). Liver injury largely manifested as mild‐to‐moderate biochemical abnormalities, rarely progressing to severe complications, and was effectively managed with corticosteroid therapy. Economic analysis highlights the manageable burden of OA‐induced liver injury. In the EU, mild‐to‐moderate cases cost €823.7, while severe cases average €1638.6. Medication costs range from €26.8 for prednisone to €695.4 for severe cases requiring additional immunosuppressive agents like tacrolimus and mycophenolate mofetil. To conclude, OA‐induced liver injury, though notable, is clinically manageable with immunosuppressive therapy and rarely causes severe complications like encephalopathy or liver failure. Its modest costs do not undermine OA's cost‐effectiveness, supporting its transformative role in spinal muscular atrophy treatment.https://doi.org/10.1002/prp2.70134drug safetygene therapyliver injuryonasemnogene abeparvovec‐xioipharmacoeconomicsspinal muscular atrophy |
| spellingShingle | Andrej Belančić Branislava Raičević Ivana Stević Dinko Vitezić Slobodan M. Janković Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury Pharmacology Research & Perspectives drug safety gene therapy liver injury onasemnogene abeparvovec‐xioi pharmacoeconomics spinal muscular atrophy |
| title | Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury |
| title_full | Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury |
| title_fullStr | Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury |
| title_full_unstemmed | Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury |
| title_short | Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury |
| title_sort | costs of treating onasemnogene abeparvovec xioi induced liver injury |
| topic | drug safety gene therapy liver injury onasemnogene abeparvovec‐xioi pharmacoeconomics spinal muscular atrophy |
| url | https://doi.org/10.1002/prp2.70134 |
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