{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T00:23:17Z","timestamp":1768782197766,"version":"3.49.0"},"reference-count":13,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2014,8,12]],"date-time":"2014-08-12T00:00:00Z","timestamp":1407801600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2015,1]]},"abstract":"Summary<\/jats:title>Children with von Willebrand disease (VWD<\/jats:styled-content>) in whom DDAVP<\/jats:styled-content> is ineffective or contraindicated require treatment with a coagulation factor concentrate containing von Willebrand factor (VWF<\/jats:styled-content>) and factor VIII<\/jats:styled-content> (FVIII<\/jats:styled-content>). The aim of this study was to monitor the safety, efficacy and tolerability of Wilate\u00ae<\/jats:sup> (a VWF<\/jats:styled-content>:FVIII<\/jats:styled-content> concentrate with a 1:1 ratio) used across the North London Paediatric Haemophilia Network since May 2010. In total, 47 children (aged 0.0\u201317.0\u00a0years) with type 1 (n\u00a0<\/jats:italic>=\u00a0<\/jats:italic>28), type 2 (n\u00a0<\/jats:italic>=\u00a0<\/jats:italic>7), type 3 (n\u00a0<\/jats:italic>=\u00a0<\/jats:italic>10) and acquired VWS<\/jats:styled-content> (n\u00a0<\/jats:italic>=\u00a0<\/jats:italic>2) have been treated for bleeds, surgery and\/or prophylaxis using 260\u00a0000\u00a0IU Wilate\u00ae<\/jats:sup>. Analysis of dose and frequency of treatment show expected responses to treatment with mean doses of 55, 50 and 50\u00a0IU\u00a0kg\u22121<\/jats:sup> for bleeds, surgery and prophylaxis respectively. Most bleeds responded to a single treatment. Surgical procedures were covered with clinician approved dosing schedules with 95% (39\/41) reported as having excellent or good efficacy. There was no accumulation of FVIII<\/jats:styled-content> or VWF<\/jats:styled-content> and no thromboembolic events. This case series confirms the efficacy, safety and tolerability of Wilate\u00ae<\/jats:sup> in neonates, children and adolescents when used on\u2010demand, prophylactically and in the surgical setting.<\/jats:p>","DOI":"10.1111\/hae.12497","type":"journal-article","created":{"date-parts":[[2014,8,12]],"date-time":"2014-08-12T10:29:22Z","timestamp":1407839362000},"source":"Crossref","is-referenced-by-count":19,"title":["Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience"],"prefix":"10.1111","volume":"21","author":[{"given":"K.","family":"Khair","sequence":"first","affiliation":[{"name":"Haemophilia Centre Great Ormond Street Hospital for Children NHS Foundation Trust London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.","family":"Batty","sequence":"additional","affiliation":[{"name":"The Royal London Haemophilia Centre Barts and The London School of Medicine and Dentistry The Royal London Hospital London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"Riat","sequence":"additional","affiliation":[{"name":"Haemophilia Centre Great Ormond Street Hospital for Children NHS Foundation Trust London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"L.","family":"Bowles","sequence":"additional","affiliation":[{"name":"St Bartholome w's Hospital Haemophilia Centre Barts and The London School of Medicine and Dentistry The Royal London Hospital London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C.","family":"Burgess","sequence":"additional","affiliation":[{"name":"Haemophilia Centre Great Ormond Street Hospital for Children NHS Foundation Trust London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Y. \u2010H.","family":"Chen","sequence":"additional","affiliation":[{"name":"The Royal London Haemophilia Centre London Barts and The London School of Medicine and Dentistry QMUL London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"D.","family":"Hart","sequence":"additional","affiliation":[{"name":"Barts and The London School of Medicine and Dentistry Haematology The Royal London Hospital London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"S.","family":"Platton","sequence":"additional","affiliation":[{"name":"The Royal London Haemophilia Centre Barts and The London School of Medicine and Dentistry The Royal London Hospital London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Pasi","sequence":"additional","affiliation":[{"name":"Barts and The London Centre for Haematology ICMS The Royal London Haemophilia Centre London Barts and The London School of Medicine and Dentistry QMUL London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"Liesner","sequence":"additional","affiliation":[{"name":"Haemophilia Centre Great Ormond Street Hospital for Children NHS Foundation Trust London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2014,8,12]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2006.02146.x"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra040403"},{"key":"e_1_2_8_4_1","doi-asserted-by":"crossref","first-page":"1449","DOI":"10.1182\/blood.V72.5.1449.1449","article-title":"Desmopressin: a nontransfusional form of treatment for congenital and acquired bleeding disorders","volume":"72","author":"Mannucci PM","year":"1988","journal-title":"Blood"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2008.01695.x"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.biologicals.2005.11.010"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH11-02-0057"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH10-10-0631"},{"key":"e_1_2_8_9_1","first-page":"597","article-title":"Long term secondary prophylaxis in children adolescents and young adults with von Willebrand disease. 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