{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T22:29:36Z","timestamp":1773008976327,"version":"3.50.1"},"reference-count":23,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,10,14]],"date-time":"2025-10-14T00:00:00Z","timestamp":1760400000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,10,14]],"date-time":"2025-10-14T00:00:00Z","timestamp":1760400000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2025,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Although clinical experience of a triple\u2010secured, plasma\u2010derived, von Willebrand factor (pdVWF), almost devoid of Factor VIII (FVIII) in adults with von Willebrand disease (VWD), is widely reported, its use in children is less documented.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To explore the safety and efficacy of this concentrate in real\u2010life, in children &lt;12 years old.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Data from 30 paediatric patients enrolled in a prospective, 3\u2010year observational, post\u2010marketing study in France were analysed. Efficacy and safety were assessed in two cohorts: 0\u20136 and 6\u201311 years old.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The population included 14 children &lt;6 years of age and 16 children aged 6\u201311 years. Most patients (80%) were severely affected (von Willebrand factor ristocetin cofactor activity [VWF:RCo] \u2264 15\u00a0IU\/dL), including 30% with Type 3 VWD. Children received pdVWF for 16 major bleeds, 138 minor bleeds, 7 major surgeries, 8 minor surgeries and 12 invasive procedures. Efficacy was rated excellent or good in 100% of major bleeds or surgeries. The dose per infusion was approximately 50\u00a0IU\/kg. By age group, median doses per infusion varied from 49 to 73\u00a0IU\/kg for patients &lt;6 years and from 46 to 59\u00a0IU\/kg for patients 6\u201311 years, according to clinical situation. FVIII coadministration\/correction was more frequent in Type 3 VWD, regardless of patient's age. Of five children receiving long\u2010term prophylaxis, breakthrough bleeding occurred in 1.6% of infusions and median annualised bleeding rate was 0.8. No safety concerns were raised.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This analysis enlarges clinical experience of an FVIII\u2010poor pdVWF in the paediatric population. This concentrate offers safe and effective treatment regardless of VWD severity.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70129","type":"journal-article","created":{"date-parts":[[2025,10,14]],"date-time":"2025-10-14T13:05:34Z","timestamp":1760447134000},"page":"1250-1260","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Management of von Willebrand Disease With a Factor VIII\u2010Poor von Willebrand Factor Concentrate: Results From the Paediatric Cohort of a Prospective Observational Post\u2010Marketing Study"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3019-3299","authenticated-orcid":false,"given":"Jenny","family":"Goudemand","sequence":"first","affiliation":[{"name":"Department of Haemostasis and Transfusion Lille University Hospital  Lille France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7368-4624","authenticated-orcid":false,"given":"Annie","family":"Borel\u2010Derlon","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Centre University Hospital of Caen  Caen France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"S\u00e9gol\u00e8ne","family":"Claeyssens","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center Purpan Hospital  Toulouse France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Herv\u00e9","family":"Chambost","sequence":"additional","affiliation":[{"name":"AP\u2010HM, Department of Paediatric Haematology Oncology Institut National De La Sant\u00e9 Et De La Recherche m\u00e9dicale, Institut national De La Recherche Agronomique Centre Recherche En CardioVasculaire et Nutrition Children Hospital La Timone &amp; Aix Marseille University  Marseille France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guillaume","family":"Mourey","sequence":"additional","affiliation":[{"name":"Medical Biology Laboratory Biological Haemostasis Department CHU Besan\u00e7on  Besan\u00e7on France"},{"name":"CHU Besan\u00e7on SINERGIES (UR 4662) Universit\u00e9 Marie et Louis Pasteur  Besan\u00e7on France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Annie","family":"Harroche","sequence":"additional","affiliation":[{"name":"AP\u2010HP Haemophilia Treatment Centre Hospital Necker  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6785-4332","authenticated-orcid":false,"given":"Sandrine","family":"Meunier","sequence":"additional","affiliation":[{"name":"Hospices Civils de Lyon French Reference Center For Haemophilia Clinical Haemostasis Unit Lyon Louis Pradel Hospital  Bron France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C\u00e9line","family":"Henriet","sequence":"additional","affiliation":[{"name":"Clinical Development Laboratoire Fran\u00e7ais Du Fractionnement et Des Biotechnologies (LFB)  Les Ulis France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thierry","family":"Leroi","sequence":"additional","affiliation":[{"name":"Medical Operation France Laboratoire Fran\u00e7ais Du Fractionnement et Des Biotechnologies (LFB)  Les Ulis France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sophie","family":"Susen","sequence":"additional","affiliation":[{"name":"Department of Haemostasis and Transfusion Lille University Hospital  Lille France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3809-3858","authenticated-orcid":false,"given":"Yohann","family":"Repesse","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Centre University 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