{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T02:17:30Z","timestamp":1782958650200,"version":"3.54.5"},"reference-count":44,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100016387","name":"Grifols","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100016387","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Clin Appl Thromb Hemost"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:p>Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF\/FVIII-C) are the mainstay of treatment in von Willebrand disease (VWD). Real-world data on efficacy and safety of these pd-VWF\/FVIII-C are required. To retrospectively evaluate the efficacy and safety of pd-VWF\/FVIII-C (Fanhdi\u00ae and Alphanate\u00ae, Grifols) in clinical practice in Italy. A multicentric, observational, retrospective study at 10 Italian centers was conducted. Eligible patients diagnosed with inherited VWD (ISTH criteria) were treated with either Fanhdi\u00ae or Alphanate\u00ae for bleeding episodes, prevention of surgical bleeding and secondary long-term prophylaxis (SLTP) according to clinical practice with medical records collected from January 2007 to December 2019. Efficacy\/safety of pd-VWF\/FVIII-C was assessed according to FDA-agreed objective criteria following regulatory procedures. Fifty-seven patients (M\/F: 21\/36) were enrolled in the study with the following VWD types: VWD1 (n\u2009=\u200929, 52%), VWD2A (n\u2009=\u200910, 18%), VWD2B (n\u2009=\u20097, 12%), VWD2M (n\u2009=\u20092, 4%), VWD2N (n\u2009=\u20091, 2%), VWD2 unclassified (n\u2009=\u20091, 2%), and VWD3 (n\u2009=\u20097, 12%). These pd-VWF\/FVIII-C were used to manage 58 bleeding episodes (n\u2009=\u200924 patients), 100 surgeries (n\u2009=\u200947 patients), and 7 SLTP (n\u2009=\u20096 patients). Global clinical efficacy with these pd-VWF\/FVIII-C was reported to be excellent\/good in 85% of bleeding episodes, 98% of surgeries, and 100% of SLTP. As far as safety, no adverse-drug-related episodes, immunogenic or thrombotic events were reported. This study confirmed that Fanhdi\u00ae and Alphanate\u00ae were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries and for SLTP in Italian patients with inherited VWD.<\/jats:p>","DOI":"10.1177\/10760296241264541","type":"journal-article","created":{"date-parts":[[2024,7,21]],"date-time":"2024-07-21T09:45:05Z","timestamp":1721555105000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Real-World Efficacy and Safety of Plasma-Derived Von Willebrand Factor-Containing Factor VIII Concentrates in Patients With Von Willebrand Disease in Italy"],"prefix":"10.1177","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4325-0918","authenticated-orcid":false,"given":"Augusto B.","family":"Federici","sequence":"first","affiliation":[{"name":"Department of Haematology and Haemotherapy, Luigi Sacco Hospital, Milano, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rita Carlotta","family":"Santoro","sequence":"additional","affiliation":[{"name":"Haemophilia Centre, Haemostasis and Thrombosis Unit, Department of Haemato-Oncology, Azienda Ospedaliero-Universitaria Dulbecco, Catanzaro, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Santoro","sequence":"additional","affiliation":[{"name":"Department of Hematology, University Hospital Policlinico Umberto I, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa","family":"Pieri","sequence":"additional","affiliation":[{"name":"Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto Mario","family":"Santi","sequence":"additional","affiliation":[{"name":"SSD Thrombosis and Hemostasis Center, Azienda Ospedaliera \u201cSS Antonio e Biagio e C. Arrigo\u201d, Alessandria, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanni","family":"Barillari","sequence":"additional","affiliation":[{"name":"Center for Hemorrhagic and Thrombotic Diseases, Udine General and University Hospital \u201cS. Maria della Misericordia\u201d, Udine, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Borchiellini","sequence":"additional","affiliation":[{"name":"Regional Reference Center for Thrombotic and Haemorrhagic Disorders of Hematology, Division Department of Hematology and Oncology, A.O.U. Citt\u00e0 della Salute e della Scienza di Torino, Torino, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Tosetto","sequence":"additional","affiliation":[{"name":"Hemophilia and Thrombosis Center, Hematology Department, San Bortolo Hospital, Vicenza, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ezio","family":"Zanon","sequence":"additional","affiliation":[{"name":"Haemophilia Center, Department of Medicine, University of Padua Medical School, Padua, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raimondo","family":"De Cristofaro","sequence":"additional","affiliation":[{"name":"Thrombotic and Hemorrhagic Diseases Center and Department of Translational Medicine and Surgery, Catholic University of the S. Heart, School of Medicine, Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Esther","family":"Mairal","sequence":"additional","affiliation":[{"name":"Scientific and Medical Affairs Department, Grifols, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roser","family":"Mir","sequence":"additional","affiliation":[{"name":"Scientific and Medical Affairs Department, Grifols, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,7,21]]},"reference":[{"key":"bibr1-10760296241264541","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S389241"},{"key":"bibr2-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1601561"},{"key":"bibr3-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25656"},{"key":"bibr4-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2006.01847.x"},{"key":"bibr5-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1055\/s-0031-1281037"},{"key":"bibr6-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1111\/jth.14928"},{"key":"bibr7-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02916.x"},{"key":"bibr8-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02088.x"},{"key":"bibr9-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2006.02179.x"},{"key":"bibr10-10760296241264541","doi-asserted-by":"publisher","DOI":"10.1055\/s-2006-949667"},{"key":"bibr11-10760296241264541","volume-title":"von Willebrand Disease. 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