{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T14:09:37Z","timestamp":1790345377323,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,1,10]],"date-time":"2024-01-10T00:00:00Z","timestamp":1704844800000},"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":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>We sought to understand how people with haemophilia (PwH) and their caregivers value the potential benefits of novel prophylactic treatments. We conducted a discrete\u2010choice experiment (DCE) to quantify preferences for features of haemophilia treatments among adults and caregivers of children with haemophilia. A best\u2010worst scaling (BWS) exercise measured the perceived burden of treatment administration features.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A cross\u2010sectional, web\u2010based survey was administered to male adults (\u226518\u00a0years) and caregivers of male children (\u226417\u00a0years) living with haemophilia in the United States. Respondents evaluated eight pairs of hypothetical haemophilia treatment profiles defined by six attributes in the DCE and 15 features in the BWS.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In the DCE, both adults with haemophilia (<jats:italic>n<\/jats:italic>\u00a0=\u00a0151) and caregivers (<jats:italic>n<\/jats:italic>\u00a0=\u00a0151) prioritised avoiding the risk of developing inhibitor\/ anti\u2010drug antibodies and treatments that allowed for a more active life. They placed a lower priority on reducing the number of spontaneous bleeding episodes, route and frequency of administration, and avoiding the risk of hospitalisation due to adverse events. The BWS documented the burdensomeness of IV infusions and medications that require mixing and refrigeration.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>PwH and caregivers prefer treatments that enable a more active lifestyle with a lower risk of inhibitor development. Both groups valued the ability to lead an active life over reducing spontaneous bleeding, with caregivers placing the most weight on this attribute. As new treatments expand possibilities, healthcare professionals and PwH should continue to share decision\u2010making, incorporating clinical judgment and individual preferences.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14928","type":"journal-article","created":{"date-parts":[[2024,1,10]],"date-time":"2024-01-10T17:55:36Z","timestamp":1704909336000},"page":"375-387","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Patient and caregiver preferences for haemophilia treatments: A discrete\u2010choice experiment"],"prefix":"10.1111","volume":"30","author":[{"given":"Viridiana Cano","family":"Garcia","sequence":"first","affiliation":[{"name":"Global Medical Affairs Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carol","family":"Mansfield","sequence":"additional","affiliation":[{"name":"Health Preference Assessment RTI Health Solutions  Research Triangle Park North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Pierce","sequence":"additional","affiliation":[{"name":"Health Preference Assessment RTI Health Solutions  Research Triangle Park North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colton","family":"Leach","sequence":"additional","affiliation":[{"name":"Health Preference Assessment RTI Health Solutions  Research Triangle Park North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane Cavanaugh","family":"Smith","sequence":"additional","affiliation":[{"name":"Global Medical Affairs Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-7953-5954","authenticated-orcid":false,"given":"Marion","family":"Afonso","sequence":"additional","affiliation":[{"name":"Health Economics and Value Assessment Sanofi  Chilly\u2010Mazarin France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,1,10]]},"reference":[{"issue":"6","key":"e_1_2_12_2_1","first-page":"S112","article-title":"Current challenges in the management of hemophilia","volume":"21","author":"Bauer KA","year":"2015","journal-title":"Am J Manag Care"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13862"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2010.02411.x"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2008.01656.x"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2005.01159.x"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2008.01926.x"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S151812"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12011"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2010.02401.x"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2010.11.013"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2016.04.004"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2012.08.2223"},{"key":"e_1_2_12_15_1","unstructured":"FDA. Food and Drug Administration.Patient preference information\u2014voluntary submission review in premarket approval applications humanitarian device exemption applications and de novo requests and inclusion in decision summaries and device labeling\u2014guidance for industry Food and Drug Administration staff and other stakeholders.2016. Accessed March 8 2018.https:\/\/www.fda.gov\/downloads\/MedicalDevices\/DeviceRegulationandGuidance\/GuidanceDocuments\/UCM446680.pdf"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/HAE.14236"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1017\/CBO9780511805271"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/1-4020-3684-1_7"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00464\u2010014\u20104044\u20102"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ecolecon.2007.02.005"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.reseneeco.2010.01.006"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.14928","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,4,30]],"date-time":"2024-04-30T02:08:24Z","timestamp":1714442904000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.14928"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1,10]]},"references-count":20,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2024,3]]}},"alternative-id":["10.1111\/hae.14928"],"URL":"https:\/\/doi.org\/10.1111\/hae.14928","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,1,10]]},"assertion":[{"value":"2023-08-31","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-12-13","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-01-10","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}