{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,11]],"date-time":"2026-07-11T14:46:27Z","timestamp":1783781187974,"version":"3.55.0"},"reference-count":38,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2022,12,5]],"date-time":"2022-12-05T00:00:00Z","timestamp":1670198400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>To inform education and treatment discussions, it is important to understand how persons with haemophilia prefer to learn about and discuss new therapies and to identify variables that influence decision\u2010making.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>The aim of this study was to evaluate preferences and variables which influence decision\u2010making related to gene therapy and other novel haemophilia therapies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>An online survey was sent to men with severe haemophilia enrolled in the National Hemophilia Foundation Community Voices in Research online platform for patient\u2010powered research.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>One hundred four men completed the survey including 33% Hispanics, 96 who had had not gene therapy and 71\/96 (74%) who were on prophylaxis. Ninety\u2010five percent were somewhat or very familiar with gene therapy. Men with haemophilia obtain information about new therapies from several sources, most commonly their haemophilia treatment team, patient advocacy groups and self\u2010study. Participants identified safety and efficacy as well as other educational needs to inform decision\u2010making. Of those without prior gene therapy, 73% indicated a high likelihood of considering gene therapy. Hispanic ethnicity and government\u2010issued insurance were associated with a higher likelihood of considering gene therapy as a treatment option.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Haemophilia Treatment Centers and patient advocacy groups must be able to educate persons with haemophilia about aspects of novel therapies which are important to the individual, especially short\u2010 and long\u2010term safety and efficacy. Further research is needed to determine how patient activation and health literacy influence decision\u2010making and how to achieve equitable access and valid informed consent for novel therapies.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14706","type":"journal-article","created":{"date-parts":[[2022,12,5]],"date-time":"2022-12-05T21:25:11Z","timestamp":1670275511000},"page":"51-60","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Gene therapy preferences and informed decision\u2010making: Results from a National Hemophilia Foundation Community Voices in research survey"],"prefix":"10.1111","volume":"29","author":[{"given":"Jacqueline","family":"Limjoco","sequence":"first","affiliation":[{"name":"Hemophilia and Thrombosis Treatment Center Rady Children's Hospital San Diego  San Diego California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Agustin","family":"Calatroni","sequence":"additional","affiliation":[{"name":"Rho Federal Systems Division  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula","family":"Aristizabal","sequence":"additional","affiliation":[{"name":"Hemophilia and Thrombosis Treatment Center Rady Children's Hospital San Diego  San Diego California USA"},{"name":"Department of Pediatrics University of California San Diego Health Sciences  La Jolla California USA"},{"name":"Population Sciences, Disparities and Community Engagement University of California San Diego Moores Cancer Center and University of California San Diego Altman Clinical and Translational Research Institute Dissemination and Implementation Science Center  La Jolla California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney D.","family":"Thornburg","sequence":"additional","affiliation":[{"name":"Hemophilia and Thrombosis Treatment Center Rady Children's Hospital San Diego  San Diego California USA"},{"name":"Department of Pediatrics University of California San Diego Health Sciences  La Jolla California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,12,5]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019001140"},{"issue":"6","key":"e_1_2_12_4_1","first-page":"1436","article-title":"Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran\u2010Results of the phase 1 inhibitor cohort","volume":"19","author":"Pasi KJ","year":"2021","journal-title":"JTH"},{"key":"e_1_2_12_5_1","first-page":"221","article-title":"Recent advances in the treatment of hemophilia: a review","volume":"15","author":"Marchesini E","year":"2021","journal-title":"Biologics"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S159297"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-09-743385"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-07-820720"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.17225\/jhp00178"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620718784830"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1200\/JOP.2014.001434"},{"key":"e_1_2_12_12_1","article-title":"Improving patient informed consent for haemophilia gene therapy: the case for change","volume":"2","author":"Woollard L","year":"2021","journal-title":"TAIRD"},{"key":"e_1_2_12_13_1","unstructured":"Agency for Healthcare Research and Quality.The SHARE Approach.2016; . 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