{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T02:49:49Z","timestamp":1792464589280,"version":"4.5.0"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T00:00:00Z","timestamp":1774828800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T00:00:00Z","timestamp":1774828800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"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>Despite available prophylactic therapies for haemophilia A, breakthrough bleeding and consequent pain and joint damage still occur.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To provide insights into the unmet treatment needs of people with moderate\u2010to\u2010severe haemophilia A in the US.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>For this descriptive, observational cohort study, eligible participants diagnosed with moderate\u2010to\u2010severe haemophilia A were enrolled in the PicnicHealth database on or before 3 October 2022. Three patient groups were established to identify: (1) treatment patterns; (2) clinical outcomes by therapy class (standard half\u2010life [SHL] factor VIII [FVIII]; extended half\u2010life FVIII [EHL]; emicizumab); (3) health\u2010related quality of life (HRQoL) using the Patient\u2010Reported Outcomes Measuring Instruments System (PROMIS)\u201029 survey and a supplemental pain survey. Patients switching treatment classes were included in multiple treatment groups.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of 211 participants in the \u2018treatment patterns population\u2019, 123 (58.3%) used SHL FVIII prophylaxis, 81 (38.4%) EHL FVIII prophylaxis, and 83 (39.3%) emicizumab prophylaxis. In the \u2018clinical outcomes population\u2019, 63 participants (70.8%) receiving SHL, 43 (61.4%) EHL and 36 (62.1%) emicizumab had \u22651 bleed at Year 1. Pain\u2010related events within the past year occurred in 18 (12.8%), 16 (18.2%) and 10 (12.2%) participants, respectively. The most frequent patient\u2010reported symptoms were \u2018sleep disturbance\u2019 (84.5%), \u2018inability to participate in social roles and activities\u2019 (69.7%) and \u2018fatigue\u2019 (66.9%). The most impacted PROMIS scores were anxiety and pain interference.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Regardless of the prophylaxis product class used, most participants experienced \u22651 bleeding event per year and reduced QoL. Further treatment optimisation may be needed to prevent bleeding episodes and improve patient QoL.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70271","type":"journal-article","created":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T09:05:12Z","timestamp":1774861512000},"page":"760-771","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Treatment of Haemophilia A Without Inhibitors: Real\u2010World Treatment Patterns and Clinical Outcomes in the US"],"prefix":"10.1111","volume":"32","author":[{"given":"Allison P.","family":"Wheeler","sequence":"first","affiliation":[{"name":"Washington Center for Bleeding Disorders  Seattle Washington USA"},{"name":"Department of Paediatric Haematology\u2010Oncology and Haematology\u2010Oncology University of Washington  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren E.","family":"Amos","sequence":"additional","affiliation":[{"name":"University of Missouri\u2010Kansas City School of Medicine  Kansas City Missouri USA"},{"name":"Division of Haematology\/Oncology Children's Mercy Kansas City  Kansas City Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shveta","family":"Gupta","sequence":"additional","affiliation":[{"name":"Arnold Palmer Hospital For Children  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvie","family":"Bozzi","sequence":"additional","affiliation":[{"name":"Sanofi  Gentilly France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gandarvaka","family":"Miles","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kalyani","family":"Hawaldar","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts 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