{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T04:23:02Z","timestamp":1790310182921,"version":"4.1.0"},"reference-count":41,"publisher":"Elsevier BV","issue":"7","license":[{"start":{"date-parts":[[2025,7,1]],"date-time":"2025-07-01T00:00:00Z","timestamp":1751328000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,7,1]],"date-time":"2025-07-01T00:00:00Z","timestamp":1751328000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,3,21]],"date-time":"2025-03-21T00:00:00Z","timestamp":1742515200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["jthjournal.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Thrombosis and Haemostasis"],"published-print":{"date-parts":[[2025,7]]},"DOI":"10.1016\/j.jtha.2025.02.042","type":"journal-article","created":{"date-parts":[[2025,3,14]],"date-time":"2025-03-14T12:21:40Z","timestamp":1741954900000},"page":"2086-2094","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":12,"title":["Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance"],"prefix":"10.1016","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3959-8797","authenticated-orcid":false,"given":"Barbara A.","family":"Konkle","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flora","family":"Peyvandi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Graham R.","family":"Foster","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cedric","family":"Hermans","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincenzo","family":"La Mura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew D.","family":"Leavitt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Lillicrap","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johnny","family":"Mahlangu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margareth C.","family":"Ozelo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven","family":"Pipe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Recht","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alok","family":"Srivastava","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guy","family":"Young","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang","family":"Miesbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jtha.2025.02.042_bib1","doi-asserted-by":"crossref","first-page":"782","DOI":"10.1089\/hum.2023.112","article-title":"Hemophilia gene therapy: the end of the beginning?","volume":"34","author":"De Wolf","year":"2023","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jtha.2025.02.042_bib2","doi-asserted-by":"crossref","first-page":"2519","DOI":"10.1056\/NEJMoa1708483","article-title":"AAV5-factor VIII gene transfer in severe hemophilia A","volume":"377","author":"Rangarajan","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib3","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1111\/hae.15071","article-title":"Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A","volume":"30","author":"Symington","year":"2024","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib4","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1056\/NEJMoa1908490","article-title":"Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A","volume":"382","author":"Pasi","year":"2020","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib5","doi-asserted-by":"crossref","first-page":"947","DOI":"10.1111\/hae.14391","article-title":"Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A","volume":"27","author":"Pasi","year":"2021","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib6","doi-asserted-by":"crossref","first-page":"1013","DOI":"10.1056\/NEJMoa2113708","article-title":"Valoctocogene roxaparvovec gene therapy for hemophilia A","volume":"386","author":"Ozelo","year":"2022","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib7","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1056\/NEJMoa2211075","article-title":"Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A","volume":"388","author":"Mahlangu","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib8","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging issues in AAV-mediated in vivo gene therapy","volume":"8","author":"Colella","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.jtha.2025.02.042_bib9","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1056\/NEJMoa2211644","article-title":"Gene therapy with etranacogene dezaparvovec for hemophilia B","volume":"388","author":"Pipe","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib10","doi-asserted-by":"crossref","first-page":"796","DOI":"10.1182\/blood.2022018971","article-title":"Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1\/2 Alta study","volume":"143","author":"Leavitt","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.02.042_bib11","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1089\/hum.2022.29199.ida","article-title":"Rational use of immunosuppressive corticosteroids in liver-directed adeno-associated virus gene therapy studies","volume":"33","author":"Dasgupta","year":"2022","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jtha.2025.02.042_bib12","first-page":"18","article-title":"Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results","volume":"30","author":"Ozelo","year":"2024","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib13","doi-asserted-by":"crossref","first-page":"1880","DOI":"10.1016\/j.jtha.2024.04.001","article-title":"Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A","volume":"22","author":"Madan","year":"2024","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.02.042_bib14","doi-asserted-by":"crossref","DOI":"10.1016\/j.rpth.2024.102615","article-title":"Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial","volume":"8","author":"Leavitt","year":"2024","journal-title":"Res Pract Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.02.042_bib15","first-page":"709","article-title":"Mildly elevated liver transaminase levels: causes and evaluation","volume":"96","author":"Oh","year":"2017","journal-title":"Am Fam Physician"},{"key":"10.1016\/j.jtha.2025.02.042_bib16","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib17","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1056\/NEJMoa1708538","article-title":"Hemophilia B gene therapy with a high-specific-activity factor IX variant","volume":"377","author":"George","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib18","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","article-title":"Long-term safety and efficacy of factor IX gene therapy in hemophilia B","volume":"371","author":"Nathwani","year":"2014","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib19","doi-asserted-by":"crossref","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","article-title":"Adenovirus-associated virus vector\u2013mediated gene transfer in hemophilia B","volume":"365","author":"Nathwani","year":"2011","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib20","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1016\/j.ymthe.2020.01.001","article-title":"Immune responses to viral gene therapy vectors","volume":"28","author":"Shirley","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.jtha.2025.02.042_bib21","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1007\/s40259-023-00585-7","article-title":"Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer","volume":"37","author":"Arjomandnejad","year":"2023","journal-title":"BioDrugs"},{"key":"10.1016\/j.jtha.2025.02.042_bib22","article-title":"Immunotherapy and gene therapy: new challenges in the diagnosis and management of drug-induced liver injury","volume":"12","author":"Delire","year":"2021","journal-title":"Front Pharmacol"},{"issue":"Supplement 3","key":"10.1016\/j.jtha.2025.02.042_bib23","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1111\/hae.14023","article-title":"Uncertainty in an era of transformative therapy for haemophilia: addressing the unknowns","volume":"27","author":"Pierce","year":"2021","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib24","doi-asserted-by":"crossref","first-page":"e540","DOI":"10.1097\/HS9.0000000000000540","article-title":"Hemophilia gene therapy: approaching the first licensed product","volume":"5","author":"Batty","year":"2021","journal-title":"Hemasphere"},{"key":"10.1016\/j.jtha.2025.02.042_bib25","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/j.jhep.2023.10.029","article-title":"Adeno-associated viruses for gene therapy \u2013 clinical implications and liver-related complications, a guide for hepatologists","volume":"80","author":"M\u00fccke","year":"2024","journal-title":"J Hepatol"},{"key":"10.1016\/j.jtha.2025.02.042_bib28","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1016\/j.jhep.2021.05.025","article-title":"EASL clinical practice guidelines on non-invasive tests for evaluation of liver disease severity and prognosis\u20132021 update","volume":"75","year":"2021","journal-title":"J Hepatol"},{"key":"10.1016\/j.jtha.2025.02.042_bib29","doi-asserted-by":"crossref","first-page":"5817","DOI":"10.1182\/bloodadvances.2023010723","article-title":"Residual burden of liver disease after HCV clearance in hemophilia: a word of caution in the era of gene therapy","volume":"7","author":"La Mura","year":"2023","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.02.042_bib30","doi-asserted-by":"crossref","first-page":"3629","DOI":"10.1016\/j.jtha.2024.08.018","article-title":"The management of liver disease in people with congenital bleeding disorders: guidance from European Association for Haemophilia and Allied Disorders, European Haemophilia Consortium, ISTH, and World Federation of Hemophilia","volume":"22","author":"La Mura","year":"2024","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.02.042_bib31","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1016\/j.jhep.2024.04.007","article-title":"EASL position paper on clinical follow-up after HCV cure","volume":"81","author":"Reiberger","year":"2024","journal-title":"J Hepatol"},{"key":"10.1016\/j.jtha.2025.02.042_bib32","doi-asserted-by":"crossref","first-page":"5725","DOI":"10.1182\/bloodadvances.2024013750","article-title":"Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice","volume":"8","author":"La Mura","year":"2024","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.02.042_bib33","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1097\/MCG.0b013e31826df40a","article-title":"Diurnal variation in serum alanine aminotransferase activity in the US population","volume":"47","author":"Ruhl","year":"2013","journal-title":"J Clin Gastroenterol"},{"key":"10.1016\/j.jtha.2025.02.042_bib34","first-page":"196","article-title":"Gene therapy of hemophilia: recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)","volume":"43","author":"Miesbach","year":"2023","journal-title":"Hamostaseologie"},{"key":"10.1016\/j.jtha.2025.02.042_bib35","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1038\/s41591-022-01751-0","article-title":"Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A","volume":"28","author":"Fong","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/j.jtha.2025.02.042_bib36","first-page":"18","article-title":"Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy","volume":"30","author":"Ismail","year":"2024","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib37","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1111\/hae.14709","article-title":"Laying the foundations for gene therapy in Italy for patients with haemophilia A: a Delphi consensus study","volume":"29","author":"Castaman","year":"2023","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib38","doi-asserted-by":"crossref","first-page":"1430","DOI":"10.1111\/hae.14867","article-title":"Delivery of gene therapy in haemophilia treatment centres in the United States: practical aspects of preparedness and implementation","volume":"29","author":"Pipe","year":"2023","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib39","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1684\/hma.2023.1779","article-title":"Mod\u00e8le hub & spoke en France: organisation des Soins et parcours des patients pour la th\u00e9rapie g\u00e9nique de l\u2019h\u00e9mophilie","volume":"29","author":"Dargaud","year":"2023","journal-title":"H\u00e9matologie"},{"key":"10.1016\/j.jtha.2025.02.042_bib40","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1111\/hae.14015","article-title":"World federation of hemophilia gene therapy registry","volume":"26","author":"Konkle","year":"2020","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib41","doi-asserted-by":"crossref","first-page":"3074","DOI":"10.1111\/jth.15023","article-title":"Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: communication from the SSC of the ISTH","volume":"18","author":"Konkle","year":"2020","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.02.042_bib42","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1111\/hae.14912","article-title":"Landmark endorsement of a global registry: the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard","volume":"30","author":"Konkle","year":"2024","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.02.042_bib43","doi-asserted-by":"crossref","first-page":"1510","DOI":"10.1016\/j.jtha.2023.12.039","article-title":"Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy","volume":"22","author":"Miesbach","year":"2024","journal-title":"J Thromb Haemost"}],"container-title":["Journal of Thrombosis and Haemostasis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625001503?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625001503?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,7]],"date-time":"2026-05-07T07:29:17Z","timestamp":1778138957000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1538783625001503"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,7]]},"references-count":41,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2025,7]]}},"alternative-id":["S1538783625001503"],"URL":"https:\/\/doi.org\/10.1016\/j.jtha.2025.02.042","relation":{},"ISSN":["1538-7836"],"issn-type":[{"value":"1538-7836","type":"print"}],"subject":[],"published":{"date-parts":[[2025,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance","name":"articletitle","label":"Article Title"},{"value":"Journal of Thrombosis and Haemostasis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jtha.2025.02.042","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of International Society on Thrombosis and Haemostasis.","name":"copyright","label":"Copyright"}]}}