{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T01:26:24Z","timestamp":1788830784630,"version":"build-2803163510"},"reference-count":93,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,12,27]],"date-time":"2025-12-27T00:00:00Z","timestamp":1766793600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003196","name":"Ministry of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003196","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":[[2026,3]]},"DOI":"10.1016\/j.jtha.2025.12.011","type":"journal-article","created":{"date-parts":[[2025,12,24]],"date-time":"2025-12-24T00:15:41Z","timestamp":1766535341000},"page":"837-851","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"title":["Overview of gene therapy for hemophilia: questions and answers to navigate the innovation"],"prefix":"10.1016","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0009-0009-1308-7524","authenticated-orcid":false,"given":"Pascal","family":"Hoffmann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5667-6857","authenticated-orcid":false,"given":"Niccol\u00f2","family":"Bitto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4685-7184","authenticated-orcid":false,"given":"Vincenzo","family":"La Mura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4506-0061","authenticated-orcid":false,"given":"Wolfgang","family":"Miesbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jtha.2025.12.011_bib1","doi-asserted-by":"crossref","first-page":"540","DOI":"10.7326\/M19-1208","article-title":"Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries","volume":"171","author":"Iorio","year":"2019","journal-title":"Ann Intern Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib2","doi-asserted-by":"crossref","first-page":"1935","DOI":"10.1111\/jth.12672","article-title":"Definitions in hemophilia: communication from the SSC of the ISTH","volume":"12","author":"Blanchette","year":"2014","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib3","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1055\/s-0037-1615621","article-title":"Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis","volume":"85","author":"White","year":"2001","journal-title":"Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib4","first-page":"2411","article-title":"Expert opinion for defining a severe bleeding phenotype to guide prophylaxis in patients with nonsevere hemophilia","author":"Pfrepper","year":"2024","journal-title":"H\u00e4mostaseologie"},{"key":"10.1016\/j.jtha.2025.12.011_bib5","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/hae.14046","article-title":"WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH guidelines for the management of hemophilia, 3rd edition","volume":"26","author":"Srivastava","year":"2020","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib6","doi-asserted-by":"crossref","first-page":"2038","DOI":"10.1182\/blood-2015-01-528414","article-title":"Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens","volume":"125","author":"Oldenburg","year":"2015","journal-title":"Blood"},{"issue":"Suppl 2","key":"10.1016\/j.jtha.2025.12.011_bib7","first-page":"S11","article-title":"Young adults with hemophilia in the U.S.: demographics, comorbidities, and health status","volume":"90","author":"Curtis","year":"2015","journal-title":"Am J Hematol"},{"key":"10.1016\/j.jtha.2025.12.011_bib8","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1111\/hae.13308","article-title":"Ageing successfully with haemophilia: a multidisciplinary programme","volume":"24","author":"Boccalandro","year":"2018","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib9","first-page":"CD010215","article-title":"Psychological interventions for people with hemophilia","volume":"3","author":"Palareti","year":"2020","journal-title":"Cochrane Database Syst Rev"},{"key":"10.1016\/j.jtha.2025.12.011_bib10","doi-asserted-by":"crossref","first-page":"2112","DOI":"10.1111\/jth.15444","article-title":"Hemophilic arthropathy: current knowledge and future perspectives","volume":"19","author":"Gualtierotti","year":"2021","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib11","doi-asserted-by":"crossref","first-page":"2313","DOI":"10.1056\/NEJMoa1305074","article-title":"Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B","volume":"369","author":"Powell","year":"2013","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib12","doi-asserted-by":"crossref","first-page":"1018","DOI":"10.1056\/NEJMoa2002699","article-title":"BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A","volume":"383","author":"Konkle","year":"2020","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib13","doi-asserted-by":"crossref","first-page":"e295","DOI":"10.1016\/S2352-3026(19)30054-7","article-title":"Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study","volume":"6","author":"Pipe","year":"2019","journal-title":"Lancet Haematol"},{"key":"10.1016\/j.jtha.2025.12.011_bib14","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1056\/NEJMoa2209226","article-title":"Efanesoctocog alfa prophylaxis for patients with severe hemophilia A","volume":"388","author":"von Drygalski","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib15","doi-asserted-by":"crossref","first-page":"1484","DOI":"10.1182\/blood.2019001292","article-title":"BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice","volume":"135","author":"Seth Chhabra","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib16","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1080\/17474086.2018.1419862","article-title":"A contemporary look at FVIII inhibitor development: still a great influence on the evolution of hemophilia therapies","volume":"11","author":"Santagostino","year":"2018","journal-title":"Expert Rev Hematol"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.12.011_bib17","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/j.1365-2516.2007.01497.x","article-title":"International workshop on immune tolerance induction: consensus recommendations","volume":"13","author":"DiMichele","year":"2007","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib18","doi-asserted-by":"crossref","first-page":"1702","DOI":"10.3324\/haematol.2019.221093","article-title":"Hemophilia A and B: molecular and clinical similarities and differences","volume":"104","author":"Castaman","year":"2019","journal-title":"Haematologica"},{"key":"10.1016\/j.jtha.2025.12.011_bib19","doi-asserted-by":"crossref","first-page":"546","DOI":"10.1182\/blood-2006-04-017988","article-title":"A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study","volume":"109","author":"Astermark","year":"2007","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib20","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1056\/NEJMoa1703068","article-title":"Emicizumab prophylaxis in hemophilia A with inhibitors","volume":"377","author":"Oldenburg","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib21","doi-asserted-by":"crossref","first-page":"811","DOI":"10.1056\/NEJMoa1803550","article-title":"Emicizumab prophylaxis in patients who have hemophilia A without inhibitors","volume":"379","author":"Mahlangu","year":"2018","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib22","doi-asserted-by":"crossref","first-page":"1355","DOI":"10.1182\/blood.2023021832","article-title":"Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial","volume":"143","author":"Pipe","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib23","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.3390\/ph15101183","article-title":"Updates on novel non-replacement drugs for hemophilia","volume":"15","author":"Gualtierotti","year":"2022","journal-title":"Pharmaceuticals (Basel)"},{"key":"10.1016\/j.jtha.2025.12.011_bib24","doi-asserted-by":"crossref","first-page":"e891","DOI":"10.1016\/S2352-3026(24)00307-7","article-title":"Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial","volume":"11","author":"Chowdary","year":"2024","journal-title":"Lancet Haematol"},{"key":"10.1016\/j.jtha.2025.12.011_bib25","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1056\/NEJMoa2216455","article-title":"Phase 3 trial of concizumab in hemophilia with inhibitors","volume":"389","author":"Matsushita","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib26","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1007\/s40265-025-02203-y","article-title":"Fitusiran: first approval","volume":"85","author":"Lee","year":"2025","journal-title":"Drugs"},{"key":"10.1016\/j.jtha.2025.12.011_bib27","doi-asserted-by":"crossref","first-page":"2966","DOI":"10.1182\/blood.2024027008","article-title":"Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study","volume":"145","author":"Young","year":"2025","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib28","doi-asserted-by":"crossref","first-page":"e367","DOI":"10.1111\/hae.13003","article-title":"Adherence to prophylactic treatment in patients with haemophilia in Germany","volume":"22","author":"Miesbach","year":"2016","journal-title":"Haemophilia"},{"issue":"Suppl 3","key":"10.1016\/j.jtha.2025.12.011_bib29","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1111\/hae.13008","article-title":"NHF-McMaster guideline on care models for haemophilia management","volume":"22","author":"Pai","year":"2016","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib30","doi-asserted-by":"crossref","first-page":"897","DOI":"10.2147\/JBM.S325031","article-title":"Integrated hemophilia patient care via a national network of care centers in the United States: a model for rare coagulation disorders","volume":"12","author":"Valentino","year":"2021","journal-title":"J Blood Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib31","doi-asserted-by":"crossref","first-page":"736","DOI":"10.1016\/S0140-6736(24)02139-1","article-title":"Haemophilia","volume":"405","author":"Chowdary","year":"2025","journal-title":"Lancet"},{"key":"10.1016\/j.jtha.2025.12.011_bib32","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1182\/hematology.2022000388","article-title":"Gene therapy for hemophilia","volume":"2022","author":"Nathwani","year":"2022","journal-title":"Hematol Am Soc Hematol Educ Program"},{"key":"10.1016\/j.jtha.2025.12.011_bib33","doi-asserted-by":"crossref","first-page":"2963","DOI":"10.1182\/blood-2002-10-3296","article-title":"AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B","volume":"101","author":"Manno","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib34","doi-asserted-by":"crossref","first-page":"2670","DOI":"10.1182\/blood.V99.8.2670","article-title":"Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy","volume":"99","author":"Mount","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib35","doi-asserted-by":"crossref","first-page":"5957","DOI":"10.1182\/bloodadvances.2024014111","article-title":"Adeno-associated virus-based gene therapy for hemophilia A and B: a systematic review and meta-analysis","volume":"8","author":"Deshpande","year":"2024","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.12.011_bib36","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1182\/blood-2008-10-181479","article-title":"Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy","volume":"113","author":"Niemeyer","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib37","article-title":"on behalf of the ISTH SSC Working Group on Gene Therapy. Real world data out of clinical trials","author":"Miesbach","year":"2025","journal-title":"EHC Conference"},{"key":"10.1016\/j.jtha.2025.12.011_bib38","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1182\/blood.2019003777","article-title":"Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues","volume":"138","author":"Leebeek","year":"2021","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib39","doi-asserted-by":"crossref","first-page":"3450","DOI":"10.1016\/j.jtha.2023.08.032","article-title":"Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1","volume":"21","author":"O\u2019Mahony","year":"2023","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib40","doi-asserted-by":"crossref","DOI":"10.1002\/rth2.12567","article-title":"Living with a \u201chemophilia-free mind\u201d\u2014The new ambition of hemophilia care?","volume":"5","author":"Krumb","year":"2021","journal-title":"Res Pract Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib41","doi-asserted-by":"crossref","first-page":"951","DOI":"10.1111\/hae.14807","article-title":"Towards achieving a haemophilia-free mind","volume":"29","author":"Hermans","year":"2023","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib42","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1016\/j.ymgme.2003.08.016","article-title":"Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer","volume":"80","author":"Raper","year":"2003","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jtha.2025.12.011_bib43","doi-asserted-by":"crossref","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","article-title":"Adenovirus-associated virus vector-mediated gene transfer in hemophilia B","volume":"365","author":"Nathwani","year":"2011","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib44","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib45","doi-asserted-by":"crossref","first-page":"2945","DOI":"10.1016\/j.jtha.2024.07.012","article-title":"Adeno-associated viral vector integration: implications for long-term efficacy and safety","volume":"22","author":"Batty","year":"2024","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib46","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1056\/NEJMoa2410597","article-title":"Lentiviral gene therapy with CD34+ hematopoietic cells for hemophilia A","volume":"392","author":"Srivastava","year":"2025","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib47","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1056\/NEJMe2414214","article-title":"Could lentivirus overcome the AAV gene-therapy challenges in hemophilia A?","volume":"392","author":"Mahlangu","year":"2025","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib48","doi-asserted-by":"crossref","first-page":"1671","DOI":"10.1056\/NEJMoa0904377","article-title":"X-linked thrombophilia with a mutant factor IX (factor IX Padua)","volume":"361","author":"Simioni","year":"2009","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib49","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.2025.12.011_bib50","doi-asserted-by":"crossref","first-page":"e265","DOI":"10.1016\/S2352-3026(24)00006-1","article-title":"Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial","volume":"11","author":"Coppens","year":"2024","journal-title":"Lancet Haematol"},{"key":"10.1016\/j.jtha.2025.12.011_bib51","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1056\/NEJMoa2119913","article-title":"Phase 1-2 trial of AAVS3 gene therapy in patients with hemophilia B","volume":"387","author":"Chowdary","year":"2022","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib52","doi-asserted-by":"crossref","first-page":"1108","DOI":"10.1056\/NEJMoa2302982","article-title":"Gene therapy with fidanacogene elaparvovec in adults with hemophilia B","volume":"391","author":"Cuker","year":"2024","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib53","doi-asserted-by":"crossref","first-page":"7193","DOI":"10.1038\/s41467-024-51296-8","article-title":"Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice","volume":"15","author":"Sternberg","year":"2024","journal-title":"Nat Commun"},{"key":"10.1016\/j.jtha.2025.12.011_bib54","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1111\/hae.14936","article-title":"Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment","volume":"30","author":"Symington","year":"2024","journal-title":"Haemophilia"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.12.011_bib55","doi-asserted-by":"crossref","first-page":"1054","DOI":"10.1182\/blood-2023-179422","article-title":"Four-year follow-up of the alta study, a phase 1\/2 study of giroctocogene fitelparvovec (PF-07055480\/SB-525) gene therapy in adults with severe hemophilia A","volume":"142","author":"Harrington","year":"2023","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib56","doi-asserted-by":"crossref","first-page":"3543","DOI":"10.1182\/bloodadvances.2024015291","article-title":"Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years","volume":"9","author":"von Drygalski","year":"2025","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.12.011_bib57","doi-asserted-by":"crossref","first-page":"1508","DOI":"10.1056\/NEJMoa2307159","article-title":"Fidanacogene elaparvovec for hemophilia B\u2014a multiyear follow-up study","volume":"392","author":"Rasko","year":"2025","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib58","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.2025.12.011_bib59","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"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.12.011_bib60","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1182\/blood-2024-208486","article-title":"Efficacy and safety of giroctocogene fitelparvovec in adults with moderately severe to severe hemophilia A: primary analysis results from the phase 3 AFFINE gene therapy trial","volume":"144","author":"Leavitt","year":"2024","journal-title":"Blood"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.12.011_bib61","doi-asserted-by":"crossref","first-page":"1055","DOI":"10.1182\/blood-2023-187624","volume":"142","author":"Pipe","year":"2023","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib62","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1016\/j.jtha.2022.11.026","article-title":"Liver-related aspects of gene therapy for hemophilia: need for collaborations with hepatologists","volume":"21","author":"Miesbach","year":"2023","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib63","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.2025.12.011_bib64","doi-asserted-by":"crossref","first-page":"1961","DOI":"10.1056\/NEJMoa2104205","article-title":"Multiyear factor VIII expression after AAV gene transfer for hemophilia A","volume":"385","author":"George","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib65","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1146\/annurev-med-043021-033013","article-title":"Adeno-associated virus gene therapy for hemophilia","volume":"74","author":"Samelson-Jones","year":"2023","journal-title":"Annu Rev Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib66","doi-asserted-by":"crossref","first-page":"785","DOI":"10.3390\/cells12050785","article-title":"Various AAV serotypes and their applications in gene therapy: an overview","volume":"12","author":"Issa","year":"2023","journal-title":"Cells"},{"key":"10.1016\/j.jtha.2025.12.011_bib67","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"},{"issue":"Suppl_1","key":"10.1016\/j.jtha.2025.12.011_bib68","doi-asserted-by":"crossref","first-page":"2059","DOI":"10.1182\/blood-2019-122535","article-title":"Stable FIX expression and durable reductions in bleeding and factor IX consumption for up to 4 years following AMT-060 gene therapy in adults with severe or moderate-severe hemophilia B","volume":"134","author":"Miesbach","year":"2019","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib69","doi-asserted-by":"crossref","first-page":"3578","DOI":"10.1182\/blood-2024-198379","article-title":"Stable factor IX expression and sustained reductions in factor IX use 8 years after gene therapy with CSL220 (formerly AMT-060) in adults with haemophilia B","volume":"144","author":"Leebeek","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib70","doi-asserted-by":"crossref","first-page":"2226","DOI":"10.1056\/NEJMoa2414783","article-title":"Sustained clinical benefit of AAV gene therapy in severe hemophilia B","volume":"392","author":"Reiss","year":"2025","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.12.011_bib71","doi-asserted-by":"crossref","first-page":"2086","DOI":"10.1016\/j.jtha.2025.02.042","article-title":"Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance","volume":"23","author":"Konkle","year":"2025","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.12.011_bib72","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1182\/asheducation-2010.1.191","article-title":"The aging patient with hemophilia: complications, comorbidities, and management issues","volume":"2010","author":"Philipp","year":"2010","journal-title":"Hematology"},{"key":"10.1016\/j.jtha.2025.12.011_bib73","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1182\/blood-2020-138861","article-title":"All cause mortality and causes of death in people with hemophilia: a systematic review and meta analysis","volume":"136","author":"Alam","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.12.011_bib74","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.2025.12.011_bib75","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.2025.12.011_bib76","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1016\/j.jhep.2024.04.031","article-title":"EASL\u2013EASD\u2013EASO Clinical Practice Guidelines on the management of metabolic dysfunction-associated steatotic liver disease (MASLD)","volume":"81","year":"2024","journal-title":"J Hepatol"},{"key":"10.1016\/j.jtha.2025.12.011_bib77","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.2025.12.011_bib78","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.2025.12.011_bib79","doi-asserted-by":"crossref","first-page":"967","DOI":"10.1111\/hae.14420","article-title":"Delivery of AAV-based gene therapy through haemophilia centres\u2014a need for re-evaluation of infrastructure and comprehensive care: a Joint publication of EAHAD and EHC","volume":"27","author":"Miesbach","year":"2021","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.12.011_bib80","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/j.jhep.2023.10.029","article-title":"Adeno-associated viruses for gene therapy\u2014clinical 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.2025.12.011_bib83","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1038\/ajg.2016.517","article-title":"ACG clinical guideline: evaluation of abnormal liver chemistries","volume":"112","author":"Kwo","year":"2017","journal-title":"Am J Gastroenterol"},{"key":"10.1016\/j.jtha.2025.12.011_bib84","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\u20142021 update","volume":"75","year":"2021","journal-title":"J Hepatol"},{"key":"10.1016\/j.jtha.2025.12.011_bib85","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1155\/2011\/954812","article-title":"Non-invasive diagnostic and prognostic evaluation of liver cirrhosis and portal hypertension","volume":"31","author":"Berzigotti","year":"2011","journal-title":"Dis Markers"},{"key":"10.1016\/j.jtha.2025.12.011_bib86","doi-asserted-by":"crossref","first-page":"1717","DOI":"10.1053\/j.gastro.2019.01.042","article-title":"Accuracy of FibroScan controlled attenuation parameter and liver stiffness measurement in assessing steatosis and fibrosis in patients with nonalcoholic fatty liver disease","volume":"156","author":"Eddowes","year":"2019","journal-title":"Gastroenterology"},{"key":"10.1016\/j.jtha.2025.12.011_bib87","doi-asserted-by":"crossref","first-page":"1966","DOI":"10.1038\/s41587-024-02523-6","article-title":"AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion","volume":"43","author":"Kim","year":"2025","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jtha.2025.12.011_bib88","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1053\/j.gastro.2014.12.004","article-title":"Herbal products and the liver: a review of adverse effects and mechanisms","volume":"148","author":"Seeff","year":"2015","journal-title":"Gastroenterology"},{"key":"10.1016\/j.jtha.2025.12.011_bib89","doi-asserted-by":"crossref","first-page":"1036","DOI":"10.1002\/hep.32689","article-title":"AASLD practice guidance on drug, herbal, and dietary supplement\u2013induced liver injury","volume":"77","author":"Fontana","year":"2023","journal-title":"Hepatology"},{"key":"10.1016\/j.jtha.2025.12.011_bib90","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.2025.12.011_bib91","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.2025.12.011_bib92","doi-asserted-by":"crossref","DOI":"10.1016\/j.tvir.2021.100795","article-title":"Transjugular liver biopsy","volume":"24","author":"Kaufman","year":"2021","journal-title":"Tech Vasc Interv Radiol"},{"key":"10.1016\/j.jtha.2025.12.011_bib93","doi-asserted-by":"crossref","first-page":"4966","DOI":"10.1182\/bloodadvances.2023009876","article-title":"Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B","volume":"7","author":"Schmidt","year":"2023","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.12.011_bib94","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1016\/j.jhep.2024.08.028","article-title":"EASL Clinical Practice Guidelines on the management of hepatocellular carcinoma","volume":"82","year":"2025","journal-title":"J Hepatol"},{"issue":"Suppl_1","key":"10.1016\/j.jtha.2025.12.011_bib95","doi-asserted-by":"crossref","first-page":"3347","DOI":"10.1182\/blood-2019-124091","article-title":"Efficacy and safety in 15 hemophilia B patients treated with the AAV gene therapy vector fidanacogene elaparvovec and followed for at least 1 year","volume":"134","author":"George","year":"2019","journal-title":"Blood"}],"container-title":["Journal of Thrombosis and Haemostasis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625009158?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625009158?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,6,14]],"date-time":"2026-06-14T16:04:45Z","timestamp":1781453085000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1538783625009158"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3]]},"references-count":93,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,3]]}},"alternative-id":["S1538783625009158"],"URL":"https:\/\/doi.org\/10.1016\/j.jtha.2025.12.011","relation":{},"ISSN":["1538-7836"],"issn-type":[{"value":"1538-7836","type":"print"}],"subject":[],"published":{"date-parts":[[2026,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Overview of gene therapy for hemophilia: questions and answers to navigate the innovation","name":"articletitle","label":"Article Title"},{"value":"Journal of Thrombosis and Haemostasis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jtha.2025.12.011","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"}]}}