{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,11]],"date-time":"2026-07-11T04:19:16Z","timestamp":1783743556649,"version":"3.55.0"},"reference-count":35,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,3,10]],"date-time":"2024-03-10T00:00:00Z","timestamp":1710028800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008322","name":"CSL Behring","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008322","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>For people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health\u2010related quality of life (HRQoL). The pivotal phase 3 HOPE\u2010B trial investigating the adeno\u2010associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels \u22642%.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>Assess how EDZ affects HRQoL in HOPE\u2010B trial participants.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>HRQoL was evaluated using generic and disease\u2010specific patient reported outcomes (PROs) including the EQ\u20105D\u20105L and the Hem\u2010A\u2010QoL questionnaires. Mean domain and total scores were compared 6 months pre\u2010 and the first 2 years post\u2010EDZ administration using repeated measures linear mixed models. The percentage of participants with minimal clinically important improvements in HRQoL was also evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Two years post\u2010EDZ, there were nominally significant increases in the least squares (LS) mean score for the EQ\u20105D\u20105L Index Value (.04; <jats:italic>p<\/jats:italic>\u00a0=\u00a0.0129), reflecting better HRQoL. Nominally significant decreases in the LS mean scores, reflecting better HRQoL, were also found for the Hem\u2010A\u2010QoL total score (\u22126.0; <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.0001) and the Treatment (\u221213.94; <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.0001), Feelings (\u22129.01; <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.0001), Future (\u22126.45; <jats:italic>p<\/jats:italic>\u00a0=\u00a0.0004) and Work\/School (\u22125.21; <jats:italic>p<\/jats:italic>\u00a0=\u00a0.0098) domains. The percentage of participants with \u226515\u2010point improvement ranged from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% (95% CI: 4.67%, 29.50%) for Family Planning. Results were similar for Year 1.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>In conclusion, gene therapy with EDZ improved HRQoL in the first and second years in several Hem\u2010A\u2010QoL domains, including Treatment, Feelings, Work\/School and Future domains, whereas improvement in other aspects of HRQoL were not demonstrated.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14977","type":"journal-article","created":{"date-parts":[[2024,3,11]],"date-time":"2024-03-11T06:24:50Z","timestamp":1710138290000},"page":"709-719","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE\u2010B trial 2 years after gene therapy"],"prefix":"10.1111","volume":"30","author":[{"given":"Robbin","family":"Itzler","sequence":"first","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1954-4385","authenticated-orcid":false,"given":"Tyler W.","family":"Buckner","sequence":"additional","affiliation":[{"name":"Division of Hematology University of Colorado School of Medicine  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5677-1371","authenticated-orcid":false,"given":"Frank W. G.","family":"Leebeek","sequence":"additional","affiliation":[{"name":"Department of Hematology Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Miller","sequence":"additional","affiliation":[{"name":"Everest Clinical Research  New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2805-1016","authenticated-orcid":false,"given":"Michael","family":"Recht","sequence":"additional","affiliation":[{"name":"National Bleeding Disorders Foundation  New York New York USA"},{"name":"Yale University School of Medicine  New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglass","family":"Drelich","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul E.","family":"Monahan","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2558-2089","authenticated-orcid":false,"given":"Steven W.","family":"Pipe","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Pathology University of Michigan  Ann Arbor Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,3,10]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_11_3_1","volume-title":"Report on the Annual Global Survey 2020","author":"World Federation of Hemophilia","year":"2021"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13769"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14492"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620718796429"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40271-020-00442-7"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24220"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14098"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01774-9"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2011.02682.x"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.3402\/qhw.v10.28915"},{"key":"e_1_2_11_13_1","volume-title":"Gene therapy for hemophilia b and an update on gene therapy for hemophilia a: effectiveness and value","author":"Tice J","year":"2022"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2211644"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2022-166135"},{"key":"e_1_2_11_16_1","unstructured":"EuroQoL Group.EQ\u20105D\u20105L about.1990; 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