{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T00:28:51Z","timestamp":1778718531590,"version":"3.51.4"},"reference-count":14,"publisher":"Georg Thieme Verlag KG","issue":"03","license":[{"start":{"date-parts":[[2021,7,4]],"date-time":"2021-07-04T00:00:00Z","timestamp":1625356800000},"content-version":"vor","delay-in-days":3,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100016300","name":"Octapharma AG","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100016300","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["TH Open"],"published-print":{"date-parts":[[2021,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>\n          Background\u2003The efficacy and safety of wilate (human von Willebrand factor\/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD.<\/jats:p><jats:p>\n          Objectives\u2003The objectives of this observational, prospective, phase 4 study were to evaluate the safety, tolerability, and effectiveness of wilate in on-demand treatment of bleeding episodes (BEs), long-term prophylaxis, and surgical prophylaxis among patients with any type of VWD.<\/jats:p><jats:p>\n          Methods\u2003Patients were enrolled at 31 study centers in 11 countries and followed for up to 2 years. Safety endpoints included adverse drug reactions (ADRs) and drug tolerability. Effectiveness was assessed using annualized bleeding rates (ABRs) during prophylaxis and predefined criteria for the treatment of BEs and surgical prophylaxis.<\/jats:p><jats:p>\n          Results\u2003A total of 111 patients (76 [68%] female) including 41 (37%) children were treated with wilate. Twenty-five patients received prophylaxis, 29 on-demand treatment, and 62 surgical prophylaxis. Tolerability was rated by patients as \u201cexcellent\u201d for 96.2% of 6,497 infusions. No unexpected ADRs or thrombotic events were reported. Median ABR during prophylaxis was 1.9. Effectiveness was assessed as \u201cexcellent\u201d or \u201cgood\u201d by patients and investigators for 100% of BEs treated on-demand, 98% (patient rating) and 99% (investigator rating) of breakthrough BEs, and 99% of surgical procedures (investigator rating).<\/jats:p><jats:p>\n          Conclusion\u2003wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.<\/jats:p>","DOI":"10.1055\/s-0041-1730283","type":"journal-article","created":{"date-parts":[[2021,7,4]],"date-time":"2021-07-04T22:30:22Z","timestamp":1625437822000},"page":"e264-e272","source":"Crossref","is-referenced-by-count":7,"title":["Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease"],"prefix":"10.1055","volume":"05","author":[{"given":"Michelle","family":"Sholzberg","sequence":"additional","affiliation":[{"name":"Departments of Medicine and Laboratory Medicine & Pathobiology, St. Michael's Hospital, Li Ka Shing Knowledge Institute, University of Toronto, Toronto, Canada"},{"name":"Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, Ontario, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kate","family":"Khair","sequence":"additional","affiliation":[{"name":"Research in Children's Health, Illness and Disability (ORCHID), NIHR Biomedical Research Centre, Great Ormond Street Hospital for Children, London, United Kingdom"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hassan","family":"Yaish","sequence":"additional","affiliation":[{"name":"University of Utah School of Medicine, Salt Lake City, Utah, United States"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"George","family":"Rodgers","sequence":"additional","affiliation":[{"name":"University of Utah School of Medicine, Salt Lake City, Utah, United States"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Sol","family":"Cruz","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n de la Hemofilia de Salta, Salta, Argentina"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cesar Monta\u00f1o","family":"Mej\u00eda","sequence":"additional","affiliation":[{"name":"Hemolife and Universidad Tecnol\u00f3gica de Pereira, Pereira, Colombia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zuzana","family":"\u010cerm\u00e1kov\u00e1","sequence":"additional","affiliation":[{"name":"Blood Center, University Hospital Ostrava, Ostrava-Poruba, Czech Republic"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Davide","family":"Matino","sequence":"additional","affiliation":[{"name":"McMaster University Medical Center, Hamilton, Ontario, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jerome","family":"Teitel","sequence":"additional","affiliation":[{"name":"Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, Ontario, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alpha","family":"Barrie","sequence":"additional","affiliation":[{"name":"Research in Children's Health, Illness and Disability (ORCHID), NIHR Biomedical Research Centre, Great Ormond Street Hospital for Children, London, United Kingdom"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sylvia","family":"Werner","sequence":"additional","affiliation":[{"name":"Octapharma USA, Inc., Paramus, New Jersey, United States"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mario von Depka","family":"Prondzinski","sequence":"additional","affiliation":[{"name":"Werlhof-Institut GmbH, Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2021,7,4]]},"reference":[{"issue":"14","key":"ref1","doi-asserted-by":"crossref","first-page":"1531","DOI":"10.1007\/s40265-017-0793-2","article-title":"Current and emerging options for the management of inherited von Willebrand disease","volume":"77","author":"J M Heijdra","year":"2017","journal-title":"Drugs"},{"issue":"01","key":"ref2","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1182\/asheducation-2014.1.536","article-title":"Management of VWD","volume":"2014","author":"A T Neff","year":"2014","journal-title":"Hematology (Am Soc Hematol Educ Program)"},{"issue":"S 01","key":"ref3","doi-asserted-by":"crossref","first-page":"S23","DOI":"10.1097\/01.mbc.0000167659.23262.18","article-title":"Long-term prophylaxis in von Willebrand disease","volume":"16","author":"E Berntorp","year":"2005","journal-title":"Blood Coagul 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