{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T22:25:42Z","timestamp":1789424742844,"version":"build-2803163510"},"reference-count":23,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2025,6,12]],"date-time":"2025-06-12T00:00:00Z","timestamp":1749686400000},"content-version":"vor","delay-in-days":11,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["bpspubs.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacology Res & Perspec"],"published-print":{"date-parts":[[2025,6]]},"abstract":"ABSTRACT<\/jats:title>Aims were to reveal types of onasemnogene abeparvovec\u2010xioi (OA<\/jats:styled-content>)\u2010induced liver injury, their treatment patterns, utilization of healthcare, and treatment costs. This study employed secondary research to analyze OA<\/jats:styled-content>\u2010induced liver injury using data from the EudraVigilance<\/jats:styled-content> database, published case reports, cohort studies, and clinical trials. The extracted data were analyzed to define real\u2010life clinical entities that could be clearly outlined as syndromes resulting from the OA<\/jats:styled-content>\u2010induced 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<\/jats:styled-content>. From 1566 adverse event reports in the EudraVigilance<\/jats:styled-content> database following OA<\/jats:styled-content> therapy, 231 were hepatobiliary disorders, predominantly hypertransaminasaemia (30.7%; 71\/231). Liver injury largely manifested as mild\u2010to\u2010moderate biochemical abnormalities, rarely progressing to severe complications, and was effectively managed with corticosteroid therapy. Economic analysis highlights the manageable burden of OA<\/jats:styled-content>\u2010induced liver injury. In the EU<\/jats:styled-content>, mild\u2010to\u2010moderate cases cost \u20ac823.7, while severe cases average \u20ac1638.6. Medication costs range from \u20ac26.8 for prednisone to \u20ac695.4 for severe cases requiring additional immunosuppressive agents like tacrolimus and mycophenolate mofetil. To conclude, OA<\/jats:styled-content>\u2010induced 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<\/jats:styled-content>'s cost\u2010effectiveness, supporting its transformative role in spinal muscular atrophy treatment.<\/jats:p>","DOI":"10.1002\/prp2.70134","type":"journal-article","created":{"date-parts":[[2025,6,13]],"date-time":"2025-06-13T06:08:43Z","timestamp":1749794923000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Costs of Treating Onasemnogene Abeparvovec\u2010Xioi\u2010Induced Liver Injury"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7848-6600","authenticated-orcid":false,"given":"Andrej","family":"Belan\u010di\u0107","sequence":"first","affiliation":[{"name":"Department of Basic and Clinical Pharmacology and Toxicology Faculty of Medicine, University of Rijeka Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Branislava","family":"Rai\u010devi\u0107","sequence":"additional","affiliation":[{"name":"Faculty of Medical Sciences, University of Kragujevac Kragujevac Serbia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivana","family":"Stevi\u0107","sequence":"additional","affiliation":[{"name":"Department of Social Pharmacy and Pharmaceutical Legislation Faculty of Pharmacy, University of Belgrade Belgrade Serbia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dinko","family":"Vitezi\u0107","sequence":"additional","affiliation":[{"name":"Department of Basic and Clinical Pharmacology and Toxicology Faculty of Medicine, University of Rijeka Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Slobodan M.","family":"Jankovi\u0107","sequence":"additional","affiliation":[{"name":"Faculty of Medical Sciences, University of Kragujevac Kragujevac Serbia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,6,12]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms24021130"},{"issue":"3","key":"e_1_2_11_3_1","article-title":"Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)","volume":"15","author":"Ogbonmide T.","year":"2023","journal-title":"Cureus"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1177\/01926233231201408"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1590\/1413-81232023287.18122022en"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2020.09.021"},{"key":"e_1_2_11_7_1","unstructured":"\u201cEuropean Database of Suspected Adverse Drug Reaction Reports\u2014Search \u201dhttps:\/\/www.adrreports.eu\/en\/search.html."},{"key":"e_1_2_11_8_1","unstructured":"\u201cEuropean Database of Suspected Adverse Drug Reaction Reports\u2014Data Sources \u201dhttps:\/\/www.adrreports.eu\/en\/data_source.html."},{"key":"e_1_2_11_9_1","unstructured":"\u201cEuropean Database of Suspected Adverse Drug Reaction Reports\u2014Data Quality \u201dhttps:\/\/www.adrreports.eu\/en\/data_quality.html."},{"key":"e_1_2_11_10_1","unstructured":"\u201cEuropean Database of Suspected Adverse Drug Reaction Reports\u2014Disclaimer \u201dhttps:\/\/www.adrreports.eu\/en\/disclaimer.html."},{"key":"e_1_2_11_11_1","unstructured":"\u201cRulebook on the Prices of Health Services at the Secondary and Tertiary Level of Health Care \u201d Official Gazette of the Republic of Serbia No. 88\/2021\u201334 97\/2021\u2013250 109\/2021\u201393 132\/2021\u2013132 47\/2022\u2013258 82\/2022\u201311 123\/2022\u201310 128\/2022\u201356 1\/2023\u20137 59\/2023\u201377 96\/2023\u2013255 15\/2024\u2013142 13\/2025\u2013117."},{"key":"e_1_2_11_12_1","unstructured":"\u201cRulebook on the Approved List of Medicines Prescribed and Dispensed Under the Compulsory Health Insurance Scheme \u201d Official Gazette of the Republic of Serbia No. 40\/2022\u2013185 144\/2022\u2013148 40\/2023\u2013168 57\/2023\u2013127 66\/2023\u20137 67\/2023\u20135 86\/2023\u201389 104\/2023\u2013111 17\/2024\u2013134 74\/2024\u20135."},{"key":"e_1_2_11_13_1","unstructured":"\u201cAverage Currency Rate \u201d 2024 https:\/\/www.nbs.rs\/kursnaListaModul\/naZeljeniDan.faces;jsessionid=2AF3071B5AB3004A7D0E28AC28F2D896."},{"key":"e_1_2_11_14_1","unstructured":"F.Koechlin P.Konijn L.Lorenzoni andP.Schreyer \u201cComparing Hospital and Health Prices and Volumes Internationally: Results of a Eurostat\/ OECD Project \u201d (2014) OECD Health Working Papers No. 75 OECD Publishing https:\/\/doi.org\/10.1787\/5jxznwrj32mp\u2010en."},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2023.1230889"},{"key":"e_1_2_11_16_1","first-page":"S1","article-title":"Treatment of Spinal Muscular Atrophy With Onasemnogene Abeparvovec: Off\u2010Label Case Report and Follow\u2010Up Protocol Proposal","volume":"81","author":"Funck E. 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