{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T03:29:33Z","timestamp":1775446173424,"version":"3.50.1"},"reference-count":21,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,1,30]],"date-time":"2026-01-30T00:00:00Z","timestamp":1769731200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,1,30]],"date-time":"2026-01-30T00:00:00Z","timestamp":1769731200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100016300","name":"Octapharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100016300","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>The WIL\u201031 study demonstrated efficacy and safety of prophylaxis with the plasma\u2010derived von Willebrand factor\/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on\u2010demand run\u2010in study as an intra\u2010individual comparator. This subgroup analysis examines the efficacy of wilate prophylaxis in patients with type 3 VWD in WIL\u201031.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients received 20\u201340\u2009IU\/kg wilate prophylaxis 2\u20133 times weekly for 12\u2009months.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Twenty\u2010two patients in WIL\u201031 had type 3 VWD. Mean total annualized bleeding rate (ABRs) during on\u2010demand versus prophylaxis was 37.1 versus 5.2 (86% reduction). During prophylaxis, 115 bleeds occurred, most (95%) of which were minor; the most common bleeding site was the nose (40% of bleeds). Mean overall spontaneous ABRs during on\u2010demand versus prophylaxis were 26.5 versus 2.8 (89% reduction); mean treated spontaneous ABRs were 20.3 versus 1.4, respectively (93% reduction). ABRs were reduced further during the second 6\u2009months of prophylaxis versus the first 6\u2009months. Results were consistent in subgroups by age. No serious adverse events related to study treatment and no thrombotic events were observed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Prophylaxis with wilate was effective and well tolerated in patients with type 3 VWD, in all age groups.<\/jats:p>\n                    <jats:p>\n                      <jats:bold>Trial Registration:<\/jats:bold>\n                      NCT04052698;\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT04052698\">https:\/\/clinicaltrials.gov\/study\/NCT04052698<\/jats:ext-link>\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ejh.70117","type":"journal-article","created":{"date-parts":[[2026,1,30]],"date-time":"2026-01-30T10:50:57Z","timestamp":1769770257000},"page":"674-681","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Efficacy and Safety of Prophylaxis With a Plasma\u2010Derived von Willebrand Factor\/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease\u2014A WIL\u201031 Study Sub\u2010Analysis"],"prefix":"10.1111","volume":"116","author":[{"given":"Claudia","family":"Djambas Khayat","sequence":"first","affiliation":[{"name":"Hotel Dieu de France Hospital Saint Joseph University  Beirut Lebanon"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leonid","family":"Dubey","sequence":"additional","affiliation":[{"name":"Communal Nonprofit Enterprise \u201cWestern Ukrainian Specialized Children's Medical Center\u201d of Lviv Regional Council  Lviv 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Haematology and Oncology University Hospital St. Marina\u2010Varna  Varna Bulgaria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9509-9415","authenticated-orcid":false,"suffix":"Jr","given":"Robert F.","family":"Sidonio","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Emory University School of Medicine  Atlanta Georgia USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ali T.","family":"Taher","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Hematology\u2010Oncology American University of Beirut Medical Center  Beirut Lebanon"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kateryna V.","family":"Vilchevska","sequence":"additional","affiliation":[{"name":"National Specialized Children's Hospital Okhmatdyt, Center of Hemostasis Pathologies  Kyiv Ukraine"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vladimir","family":"Vdovin","sequence":"additional","affiliation":[{"name":"Morozovskaya Children's City Clinical Hospital  Moscow Russia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ana","family":"Boban","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine University Hospital Centre Zagreb  Zagreb Croatia"},{"name":"University of Zagreb School of Medicine  Zagreb 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