{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T18:31:30Z","timestamp":1791657090806,"version":"4.3.4"},"reference-count":23,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T00:00:00Z","timestamp":1750982400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T00:00:00Z","timestamp":1750982400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100004191","name":"Novo Nordisk","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004191","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Haemophilia is a bleeding disorder caused by a deficiency in coagulation factors VIII or IX. Great advances in haemophilia treatment have been achieved in recent decades, with a range of products for prophylaxis now available, the ability to individualise therapy, and resultant improvements in life expectancy and quality of life. Despite these advances, there remains an unresolved burden, both from the disease and its treatment. Real\u2010world data provide an opportunity to understand the experiences of people with haemophilia and gain insight into the areas of greatest unmet need.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To assess the treatment and disease burden of haemophilia using real\u2010world data.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Medical records data and patient\u2010reported outcomes from a self\u2010selected cohort of 446 people with haemophilia without inhibitors across the United States were combined using an online research platform. Participants\u2019 treatment and disease burdens were assessed using the Hemophilia\u2010Treatment Experience Measure (Hemo\u2010TEM) and the patient\u2010reported outcomes measurement information system (PROMIS)\u201029, respectively. Results were stratified by haemophilia type and disease severity.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>These real\u2010world data indicated that treatment and disease burden are experienced by people with haemophilia regardless of treatment regimen or severity of disease. The domains most affected by treatment burden were physical impact and emotional impact; the domains most affected by disease burden were physical functioning, pain interference, depression and anxiety.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This underscores how the perspectives of people with haemophilia in real\u2010world settings represent a valuable adjunct to clinical trial data. Such insights can potentially contribute to guide individualised treatment options and help address current unmet needs.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70078","type":"journal-article","created":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T13:45:53Z","timestamp":1751031953000},"page":"912-921","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Treatment and Disease Burden in a Cohort of People With Haemophilia Without Inhibitors in the United States"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3967-4873","authenticated-orcid":false,"given":"Allison P.","family":"Wheeler","sequence":"first","affiliation":[{"name":"Washington Center for Bleeding Disorders University of Washington  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3137-4817","authenticated-orcid":false,"given":"Emily","family":"Cibelli","sequence":"additional","affiliation":[{"name":"PicnicHealth  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gillian","family":"Hanson","sequence":"additional","affiliation":[{"name":"PicnicHealth  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cl\u00e9a","family":"Percier","sequence":"additional","affiliation":[{"name":"Novo Nordisk Health Care AG  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Porstmann","sequence":"additional","affiliation":[{"name":"Novo Nordisk Health Care AG  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naveen","family":"Shridhar","sequence":"additional","affiliation":[{"name":"Novo Nordisk Service Centre India Private Ltd  Bangalore India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2821-7159","authenticated-orcid":false,"given":"Amy","family":"Shapiro","sequence":"additional","affiliation":[{"name":"Indiana Hemophilia and Thrombosis Center  Indianapolis Indiana 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