{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:51:40Z","timestamp":1787014300730,"version":"build-2736575974"},"reference-count":25,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,11,27]],"date-time":"2024-11-27T00:00:00Z","timestamp":1732665600000},"content-version":"vor","delay-in-days":26,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Treatment options are expanding for von Willebrand disease (VWD). A core outcome set (COS)\u2014a minimum set of agreed\u2010upon outcomes to be used in every clinical trial for a given condition\u2014provides guidance on which outcomes are most important to measure to ensure necessary data is collected for a variety of stakeholders and enable comparison across products and trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>coreVWD aimed to develop a COS for trials for prophylaxis and perioperative treatments for VWD.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A modified Delphi consensus process was used to condense\/prioritize a long list of potential outcomes. Over three Delphi rounds, a multi\u2010stakeholder panel (patients, clinicians, pharmaceutical company representatives, HTA organizations, payer, and government organization representatives) rated each outcome from 1 (not important to include in a COS) to 9 (essential to include). Outcomes were eliminated or retained based on pre\u2010determined criteria; a special provision to elevate patient priorities was included. An in\u2010person consensus meeting was held after Delphi round 2.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Thirty\u2010nine panellists participated. The final COS for prophylaxis treatment included 18 outcomes, seven of which are part of a special subset selected for women, girls and people with the potential to menstruate. There were 11 outcomes in the final perioperative branch COS. Six outcomes overlapped both COS.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The coreVWD COS represents a consensus list of outcomes for clinical trials for both factor and non\u2010factor VWD therapies. These outcomes will be useful across the lifecycle of a product, from clinical development through regulatory and market access phases and into patient\u2010provider decision\u2010making.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.15122","type":"journal-article","created":{"date-parts":[[2024,11,28]],"date-time":"2024-11-28T00:20:01Z","timestamp":1732753201000},"page":"1357-1365","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["A core outcome set for prophylaxis and perioperative treatment of von Willebrand disease: The coreVWD initiative"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1789-6635","authenticated-orcid":false,"given":"Elizabeth","family":"Clearfield","sequence":"first","affiliation":[{"name":"Institute for Policy Advancement Ltd Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Kim","sequence":"additional","affiliation":[{"name":"Vega Therapeutics Inc South San Francisco USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Ford","sequence":"additional","affiliation":[{"name":"Canadian Hemophilia Society Montreal Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4100-7826","authenticated-orcid":false,"given":"Nathan T.","family":"Connell","sequence":"additional","affiliation":[{"name":"Department of Medicine Brigham and Women's Hospital Boston USA"},{"name":"Department of Medicine Harvard Medical School Boston USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2029-677X","authenticated-orcid":false,"given":"Maria E.","family":"Santaella","sequence":"additional","affiliation":[{"name":"National Bleeding Disorders Foundation New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2999-4216","authenticated-orcid":false,"given":"Michelle","family":"Lavin","sequence":"additional","affiliation":[{"name":"National Coagulation Centre St. James's Hospital Dublin Ireland"},{"name":"Irish Centre for Vascular Biology School of Pharmacy and Biomolecular Sciences, RCSI Dublin Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manon E. L.","family":"Degenaar\u2010Dujardin","sequence":"additional","affiliation":[{"name":"European Haemophilia Consortium Brussels Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"Ayoub","sequence":"additional","affiliation":[{"name":"World Federation of Hemophilia Montreal Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veronica H.","family":"Flood","sequence":"additional","affiliation":[{"name":"Pediatric Hematology\/Oncology Medical College of Wisconsin Milwaukee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3354-3880","authenticated-orcid":false,"given":"Dawn","family":"Rotellini","sequence":"additional","affiliation":[{"name":"National Bleeding Disorders Foundation New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0934-0680","authenticated-orcid":false,"given":"Mark W.","family":"Skinner","sequence":"additional","affiliation":[{"name":"Institute for Policy Advancement Ltd Washington District of Columbia USA"},{"name":"Department of Health Research Methods, Evidence, and Impact McMaster University Hamilton Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula","family":"James","sequence":"additional","affiliation":[{"name":"Department of Medicine Queen's University Kingston Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the coreVWD 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