{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T15:30:33Z","timestamp":1790263833727,"version":"4.1.0"},"reference-count":35,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:00:00Z","timestamp":1773273600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:00:00Z","timestamp":1773273600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Valoctocogene roxaparvovec, an adeno\u2010associated virus (AAV)\u2010based gene therapy, enables endogenous factor VIII (FVIII) expression in patients with severe hemophilia A without the need for regular FVIII infusions. Long\u2010term follow\u2010up assesses durability, safety, and immune\u2010related challenges following gene therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>Evaluation of six years of post\u2010infusion outcomes of a patient receiving valoctocogene roxaparvovec, focusing on FVIII expression, immune response management, and adverse events (AEs).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      A 44\u2010year\u2010old male with severe hemophilia A received a single infusion of valoctocogene roxaparvovec (6 \u00d7 10\n                      <jats:sup>13<\/jats:sup>\n                      vector genomes\/kg) in the GENEr8\u20101 study. Follow\u2010up assessments included FVIII levels, liver function tests, and immune suppression management.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Post\u2010infusion, the patient experienced an initial ALT elevation and FVIII decline, requiring oral prednisolone (60\u00a0mg\/day) at week five. ALT elevation grade 3 (peaking at 555\u00a0U\/L) was treated with intravenous methyl\u2010prednisolone (week 6), followed by oral prednisolone. To mitigate cortisone side effects, prednisolone was switched to budesonide therapy at week 19 and continued for 6 months. FVIII activity (measured with a chromogenic FVIII assay) reached a peak of 202\u00a0IU\/dL at month 6 and declined subsequently to levels of 50\u201370\u00a0IU\/dL. After six years, FVIII activity remained normal, thus eliminating prophylactic FVIII infusions and bleeding episodes. A total of 20 AEs occurred, including two serious adverse events related to ALT elevation and a traumatic acetabulum fracture.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Valoctocogene roxaparvovec can offer durable FVIII expression in severe hemophilia A. This case provides valuable insights into personalised immunosuppression therapy with a favourable outcome for long\u2010term efficacy and safety.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70261","type":"journal-article","created":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T18:19:53Z","timestamp":1773339593000},"page":"731-738","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels"],"prefix":"10.1111","volume":"32","author":[{"given":"Kerstin","family":"Herbst","sequence":"first","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Behnaz","family":"Pezeshkpoor","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Klein","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"},{"name":"Center For Rare Diseases Bonn (ZSEB) University Clinic Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thilo","family":"Albert","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philipp","family":"Lutz","sequence":"additional","affiliation":[{"name":"Department of General Internal Medicine I University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Strassburg","sequence":"additional","affiliation":[{"name":"Department of General Internal Medicine I University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrich","family":"Spengler","sequence":"additional","affiliation":[{"name":"Department of General Internal Medicine I University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Georg","family":"Goldmann","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"},{"name":"Center For Rare Diseases Bonn (ZSEB) University Clinic Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine University of Bonn  Bonn Germany"},{"name":"Center For Rare Diseases Bonn (ZSEB) University Clinic Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,3,12]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0042-1756188"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12185-018-2548-6"},{"issue":"51","key":"e_1_2_12_6_1","first-page":"887","article-title":"Gene Therapy for Hemophilia\u2010Opportunities and Risks","volume":"119","author":"Miesbach W.","year":"2022","journal-title":"Deutsches Arzteblatt International"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms231810228"},{"key":"e_1_2_12_8_1","unstructured":"BioMarin Pharmaceutical Inc (2022). 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