{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,11]],"date-time":"2026-06-11T16:04:40Z","timestamp":1781193880831,"version":"3.54.1"},"reference-count":25,"publisher":"Becaris Publishing Limited","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2026,4]]},"abstract":"<jats:p>\n                    <jats:bold>Background:<\/jats:bold>\n                    Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce.\n                    <jats:bold>Aim:<\/jats:bold>\n                    To describe and compare patients' characteristics, treatment patterns, healthcare resource use and associated costs of patients with VWD using VWF concentrates.\n                    <jats:bold>Materials &amp; methods:<\/jats:bold>\n                    Using the French healthcare claims database, we included adult patients with \u22651 reimbursement for a replacement therapy (RT) containing VWF concentrate between 1 January 2017 and 30 September 2021 and followed them from first RT dispensation to 31 December 2021. Treatment patterns, healthcare resource use and associated costs of RT on-demand users were evaluated over each 30-days exposure period (EP) starting the first day of each hospital stay with \u22651 RT administration. In- and out-hospital RT doses and FVIII, number of general practitioner and nurse visits, in- and out-hospital RT dispensings and length of hospitalizations and their costs were described and compared across RTs using adjusted Generalized Estimating Equation models accounting for confounding factors.\n                    <jats:bold>Results:<\/jats:bold>\n                    Among 2540 on-demand RT users, WILFACTIN\n                    <jats:sup>\u00ae<\/jats:sup>\n                    was the main RT used, followed by VONCENTO\n                    <jats:sup>\u00ae<\/jats:sup>\n                    , VEYVONDI\n                    <jats:sup>\u00ae<\/jats:sup>\n                    , EQWILATE\n                    <jats:sup>\u00ae<\/jats:sup>\n                    and WILSTART\n                    <jats:sup>\u00ae<\/jats:sup>\n                    . Overall, the mean total RT dose was 12,962 IU and the mean cost was \u20ac21,034\/EP. Compared with VEYVONDI\n                    <jats:sup>\u00ae<\/jats:sup>\n                    -treated EP, WILFACTIN\n                    <jats:sup>\u00ae<\/jats:sup>\n                    -treated EP had significantly longer stay duration, had more out-hospital RT dose and had higher overall and in-hospital costs; VONCENTO\n                    <jats:sup>\u00ae<\/jats:sup>\n                    -treated EP had more overall and in-hospital RT dose, and had higher in-hospital and RT-related costs.\n                    <jats:bold>Conclusion:<\/jats:bold>\n                    This first real-world study suggests that VEYVONDI\n                    <jats:sup>\u00ae<\/jats:sup>\n                    seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence.\n                  <\/jats:p>","DOI":"10.57264\/cer-2025-0128","type":"journal-article","created":{"date-parts":[[2026,6,11]],"date-time":"2026-06-11T15:38:18Z","timestamp":1781192298000},"source":"Crossref","is-referenced-by-count":0,"title":["Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study"],"prefix":"10.57264","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1697-6249","authenticated-orcid":false,"given":"Benoit","family":"Polack","sequence":"first","affiliation":[{"name":"TIMC Laboratory, CNRS \u2013 Grenoble Alpes University, Grenoble, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0351-6040","authenticated-orcid":false,"given":"Virginie","family":"Nerich","sequence":"additional","affiliation":[{"name":"Universit\u00e9 de Franche-Comt\u00e9, CHU Besan\u00e7on, INSERM, EFS-BFC, UMR 1098, P\u00f4le Pharmacie, F-25030 Besan\u00e7on, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5635-8605","authenticated-orcid":false,"given":"Claire Marant","family":"Micallef","sequence":"additional","affiliation":[{"name":"PELyon, Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4072-839X","authenticated-orcid":false,"given":"Marc","family":"Trossa\u00ebrt","sequence":"additional","affiliation":[{"name":"Haemophilia center, CHU Nantes, Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Biron-Andreani","sequence":"additional","affiliation":[{"name":"Haemophilia center, CHU Montpellier, Montpellier, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nad\u00e8ge","family":"Bornier","sequence":"additional","affiliation":[{"name":"PELyon, Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Chatelanaz","sequence":"additional","affiliation":[{"name":"Takeda France SAS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-9519-7759","authenticated-orcid":false,"given":"Franck","family":"Favre-Besse","sequence":"additional","affiliation":[{"name":"Takeda France SAS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julien","family":"Beoletto","sequence":"additional","affiliation":[{"name":"Takeda France SAS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7016-501X","authenticated-orcid":false,"given":"M\u00e9lanie","family":"N\u00e9e","sequence":"additional","affiliation":[{"name":"PELyon, Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9478-5464","authenticated-orcid":false,"given":"Manon","family":"Belhassen","sequence":"additional","affiliation":[{"name":"PELyon, Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0005-5127-2617","authenticated-orcid":false,"given":"Cinira","family":"Lef\u00e8vre","sequence":"additional","affiliation":[{"name":"Takeda France SAS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5941-6823","authenticated-orcid":false,"given":"G\u00e9rard de","family":"Pouvourville","sequence":"additional","affiliation":[{"name":"Professor Emeritus, Department of Economics, ESSEC Business School, Cergy-Pontoise, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"key":"e_1_3_11_2_2","volume-title":"Protocole National de Diagnostic et de Soins (PNDS): Maladie de Willebrand","author":"Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand","year":"2018","unstructured":"Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand. Protocole National de Diagnostic et de Soins (PNDS): Maladie de Willebrand. Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand, Lille, France (2018)."},{"issue":"1","key":"e_1_3_11_3_2","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1182\/bloodadvances.2020003264","article-title":"ASH ISTH NHF WFH 2021 guidelines on the management of Von Willebrand disease","volume":"5","author":"Connell NT","year":"2021","unstructured":"Connell NT, Flood VH, Brignardello-Petersen R ASH ISTH NHF WFH 2021 guidelines on the management of Von Willebrand disease. Blood Adv. 5(1), 301\u2013325 (2021).","journal-title":"Blood Adv."},{"key":"e_1_3_11_4_2","volume-title":"Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand","author":"Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand","year":"2018","unstructured":"Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand. Protocole National de Diagnostic et de Soins (PNDS): Maladie de Willebrand \u2013 Argumentaire. Centre de R\u00e9f\u00e9rence de la Maladie de Willebrand, Lille, France (2018)."},{"key":"e_1_3_11_5_2","article-title":"Avis \u2013 Commission de la Transparence: vonicog alfa (facteur Von Willebrand recombinant humain)","author":"Haute Autorit\u00e9 de Sant\u00e9","year":"2018","unstructured":"Haute Autorit\u00e9 de Sant\u00e9. Avis \u2013 Commission de la Transparence: vonicog alfa (facteur Von Willebrand recombinant humain). VEYVONDI, 2018.","journal-title":"VEYVONDI"},{"issue":"4","key":"e_1_3_11_6_2","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1055\/s-0039-1692688","article-title":"Replacement therapy in patients with Von Willebrand disease-indications and monitoring","volume":"39","author":"Nowak-G\u00f6ttl U","year":"2019","unstructured":"Nowak-G\u00f6ttl U, Miesbach W, Koscielny J Replacement therapy in patients with Von Willebrand disease-indications and monitoring. 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Haemophilia 27(2), 270\u2013276 (2021).","journal-title":"Haemophilia"},{"key":"e_1_3_11_10_2","first-page":"107602962210743","article-title":"Clinical efficacy and safety of Fanhdi(\u00ae), a plasma-derived VWF\/factor VIII concentrate, in Von Willebrand disease in Spain: a retrospective study","volume":"28","author":"Jim\u00e9nez-Yuste V","year":"2022","unstructured":"Jim\u00e9nez-Yuste V, Alvarez-Rom\u00e1n MT, Palomo Bravo \u00c1 Clinical efficacy and safety of Fanhdi(\u00ae), a plasma-derived VWF\/factor VIII concentrate, in Von Willebrand disease in Spain: a retrospective study. Clin. App. Thromb. Hemost. 28, 10760296221074348 (2022).","journal-title":"Clin. App. Thromb. 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Haemophilia 22(5), 739\u2013751 (2016).","journal-title":"Haemophilia"},{"issue":"6","key":"e_1_3_11_12_2","doi-asserted-by":"crossref","first-page":"1115","DOI":"10.1111\/j.1538-7836.2007.02562.x","article-title":"Treatment of severe Von Willebrand disease with a high-purity Von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients","volume":"5","author":"Borel-Derlon A","year":"2007","unstructured":"Borel-Derlon A, Federici AB, Roussel-Robert V Treatment of severe Von Willebrand disease with a high-purity Von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients. J. Thromb. Haemost. 5(6), 1115\u20131124 (2007).","journal-title":"J. Thromb. 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Haemophilia 23(2), 264\u2013272 (2017).","journal-title":"Haemophilia"},{"issue":"1","key":"e_1_3_11_15_2","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1007\/s40261-023-01323-1","article-title":"Key drivers of coagulation factor use in Von Willebrand disease during hospitalization: an overview of the French BERHLINGO cohort","volume":"44","author":"Horvais V","year":"2024","unstructured":"Horvais V, Beurrier P, Cussac V Key drivers of coagulation factor use in Von Willebrand disease during hospitalization: an overview of the French BERHLINGO cohort. Clin. Drug. Invest. 44(1), 35\u201349 (2024).","journal-title":"Clin. Drug. Invest."},{"key":"e_1_3_11_16_2","first-page":"107602962211205","article-title":"The cost of Von Willebrand disease in Europe: the CVESS Study","volume":"28","author":"Morgan G","year":"2022","unstructured":"Morgan G, Brighton S, Laffan M The cost of Von Willebrand disease in Europe: the CVESS Study. Clin. App. Thromb. Hemost. 28, 10760296221120583 (2022).","journal-title":"Clin. App. Thromb. Hemost."},{"issue":"4","key":"e_1_3_11_17_2","doi-asserted-by":"crossref","first-page":"S149","DOI":"10.1016\/j.respe.2017.05.004","article-title":"Value of a national administrative database to guide public decisions: from the systeme national d'information interregimes de l'Assurance Maladie (SNIIRAM) to the systeme national des donnees de sante (SNDS) in France","volume":"65","author":"Tuppin P","year":"2017","unstructured":"Tuppin P, Rudant J, Constantinou P Value of a national administrative database to guide public decisions: from the systeme national d'information interregimes de l'Assurance Maladie (SNIIRAM) to the systeme national des donnees de sante (SNDS) in France. Rev. Epidemiol. Sante Publique 65(Suppl. 4), S149\u2013S167 (2017).","journal-title":"Rev. Epidemiol. 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Pharm."},{"issue":"2","key":"e_1_3_11_23_2","first-page":"152","article-title":"Effectiveness and safety of hFVIII\/VWF concentrate (Voncento(\u00ae)) in patients with inherited Von Willebrand disease requiring surgical procedures: the OPALE multicentre observational study","volume":"19","author":"Rugeri L","year":"2021","unstructured":"Rugeri L, d'Oiron R, Harroche A Effectiveness and safety of hFVIII\/VWF concentrate (Voncento(\u00ae)) in patients with inherited Von Willebrand disease requiring surgical procedures: the OPALE multicentre observational study. Blood Transfus. 19(2), 152\u2013157 (2021).","journal-title":"Blood Transfus."},{"issue":"1","key":"e_1_3_11_24_2","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1111\/ejh.13778","article-title":"Effectiveness of long-term prophylaxis using pdFVIII\/VWF concentrate in patients with inherited Von Willebrand disease","volume":"109","author":"Rugeri L","year":"2022","unstructured":"Rugeri L, Harroche A, Repess\u00e9 Y Effectiveness of long-term prophylaxis using pdFVIII\/VWF concentrate in patients with inherited Von Willebrand disease. Eur. J. Haematol. 109(1), 109\u2013117 (2022).","journal-title":"Eur. J. Haematol."},{"key":"e_1_3_11_25_2","article-title":"Cohorte fran\u00e7aise des patients vivant avec une maladie h\u00e9morragique constitutionnelle. Dispositif FranceCoag. Donn\u00e9es descriptives 2021","author":"FranceCoag","year":"2020","unstructured":"FranceCoag. Cohorte fran\u00e7aise des patients vivant avec une maladie h\u00e9morragique constitutionnelle. 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Congr\u00e8s Fran\u00e7ais d'H\u00e9motase, Saint-Malo, France. 10\u201312 May 2023."}],"container-title":["Journal of Comparative Effectiveness Research"],"original-title":[],"language":"en","deposited":{"date-parts":[[2026,6,11]],"date-time":"2026-06-11T15:38:22Z","timestamp":1781192302000},"score":1,"resource":{"primary":{"URL":"https:\/\/becarispublishing.com\/doi\/10.57264\/cer-2025-0128"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4]]},"references-count":25,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2026,4]]}},"alternative-id":["10.57264\/cer-2025-0128"],"URL":"https:\/\/doi.org\/10.57264\/cer-2025-0128","relation":{},"ISSN":["2042-6305","2042-6313"],"issn-type":[{"value":"2042-6305","type":"print"},{"value":"2042-6313","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4]]},"article-number":"e250128"}}