{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T19:05:07Z","timestamp":1786993507225,"version":"build-2736575974"},"reference-count":31,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,8,11]],"date-time":"2025-08-11T00:00:00Z","timestamp":1754870400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100012112","name":"Swedish Orphan Biovitrum AB","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012112","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004339","name":"Sanofi US","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["jthjournal.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Thrombosis and Haemostasis"],"published-print":{"date-parts":[[2026,1]]},"DOI":"10.1016\/j.jtha.2025.07.013","type":"journal-article","created":{"date-parts":[[2025,7,18]],"date-time":"2025-07-18T15:35:16Z","timestamp":1752852916000},"page":"79-87","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":8,"title":["A phase 1, open-label study to assess the pharmacokinetics, safety, and tolerability of a single intravenous injection of efanesoctocog alfa in adults with type 2N or type 3 von Willebrand disease"],"prefix":"10.1016","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5953-163X","authenticated-orcid":false,"given":"Sophie","family":"Susen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Trossa\u00ebrt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moshe","family":"Zilberstein","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lara","family":"Mamikonian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Santagostino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abhimanyu","family":"Yarramaneni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy","family":"Wong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annemieke","family":"Willemze","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ekta","family":"Seth Chhabra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jtha.2025.07.013_bib1","doi-asserted-by":"crossref","first-page":"2067","DOI":"10.1056\/NEJMra1601561","article-title":"Von Willebrand\u2019s disease","volume":"375","author":"Leebeek","year":"2016","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.07.013_bib2","doi-asserted-by":"crossref","first-page":"1085","DOI":"10.1016\/j.hoc.2021.07.004","article-title":"Von Willebrand disease: current status of diagnosis and management","volume":"35","author":"Weyand","year":"2021","journal-title":"Hematol Oncol Clin North Am"},{"key":"10.1016\/j.jtha.2025.07.013_bib3","doi-asserted-by":"crossref","DOI":"10.1016\/j.rpth.2024.102599","article-title":"Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies","volume":"8","author":"van Kwawegen","year":"2024","journal-title":"Res Pract Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib4","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1038\/s41572-024-00536-8","article-title":"von Willebrand disease","volume":"10","author":"Seidizadeh","year":"2024","journal-title":"Nat Rev Dis Primers"},{"key":"10.1016\/j.jtha.2025.07.013_bib5","doi-asserted-by":"crossref","first-page":"1523","DOI":"10.1182\/blood-2018-10-876300","article-title":"The von Willebrand factor D\u2019D3 assembly and structural principles for factor VIII binding and concatemer biogenesis","volume":"133","author":"Dong","year":"2019","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.07.013_bib6","doi-asserted-by":"crossref","first-page":"1353","DOI":"10.1111\/j.1538-7836.2007.02572.x","article-title":"Clearance mechanisms of von Willebrand factor and factor VIII","volume":"5","author":"Lenting","year":"2007","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib7","doi-asserted-by":"crossref","first-page":"2007","DOI":"10.1182\/blood-2016-04-713289","article-title":"Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A","volume":"128","author":"Pipe","year":"2016","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.07.013_bib8","series-title":"GeneReviews","article-title":"von Willebrand Disease","author":"Goodeve","year":"2017"},{"key":"10.1016\/j.jtha.2025.07.013_bib9","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1182\/asheducation-2013.1.254","article-title":"von Willebrand disease: advances in pathogenetic understanding, diagnosis, and therapy","volume":"2013","author":"Lillicrap","year":"2013","journal-title":"Hematology Am Soc Hematol Educ Program"},{"key":"10.1016\/j.jtha.2025.07.013_bib10","doi-asserted-by":"crossref","first-page":"909","DOI":"10.1111\/jth.15247","article-title":"Von Willebrand disease type 2N: an update","volume":"19","author":"Seidizadeh","year":"2021","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib11","doi-asserted-by":"crossref","first-page":"2702","DOI":"10.1016\/j.jtha.2024.06.020","article-title":"Type 2N von Willebrand disease: genotype drives different bleeding phenotypes and treatment needs","volume":"22","author":"Daniel","year":"2024","journal-title":"J Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib12","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","year":"2021","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.07.013_bib13","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1007\/BF01836069","article-title":"Pharmacokinetics and hemostatic effect of different factor VIII\/von Willebrand factor concentrates in von Willebrand\u2019s disease type III","volume":"65","author":"Lethagen","year":"1992","journal-title":"Ann Hematol"},{"key":"10.1016\/j.jtha.2025.07.013_bib14","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1055\/s-0038-1649564","article-title":"Pharmacokinetics of monoclonally-purified and recombinant factor VIII in patients with severe von Willebrand disease","volume":"70","author":"Morfini","year":"1993","journal-title":"Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib15","first-page":"391","article-title":"A phase III study comparing secondary long-term prophylaxis versus on-demand treatment with vWF\/FVIII concentrates in severe inherited von Willebrand disease","volume":"17","author":"Peyvandi","year":"2019","journal-title":"Blood Transfus"},{"key":"10.1016\/j.jtha.2025.07.013_bib17","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1056\/NEJMoa2209226","article-title":"Efanesoctocog alfa prophylaxis for patients with severe hemophilia A","volume":"388","author":"von Drygalski","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.07.013_bib18","doi-asserted-by":"crossref","first-page":"1484","DOI":"10.1182\/blood.2019001292","article-title":"BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice","volume":"135","author":"Seth","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.07.013_bib19","doi-asserted-by":"crossref","first-page":"1018","DOI":"10.1056\/NEJMoa2002699","article-title":"BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A","volume":"383","author":"Konkle","year":"2020","journal-title":"N Engl J Med"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.07.013_bib20","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.1182\/blood-2021-148534","article-title":"Efanesoctocog alfa half-life and clearance are independent of von willebrand factor in severe hemophilia A: a post hoc analysis from phase 1\/2a studies","volume":"138","author":"Staber","year":"2021","journal-title":"Blood"},{"key":"10.1016\/j.jtha.2025.07.013_bib21","doi-asserted-by":"crossref","first-page":"214","DOI":"10.1111\/hae.14831","article-title":"A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical hemostasis laboratories","volume":"30","author":"Pipe","year":"2024","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.07.013_bib22","doi-asserted-by":"crossref","first-page":"1089","DOI":"10.1182\/bloodadvances.2021006119","article-title":"Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study","volume":"6","author":"Lissitchkov","year":"2022","journal-title":"Blood Adv"},{"key":"10.1016\/j.jtha.2025.07.013_bib23","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1056\/NEJMoa2312611","article-title":"Efanesoctocog alfa prophylaxis for children with severe hemophilia A","volume":"391","author":"Malec","year":"2024","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jtha.2025.07.013_bib24","doi-asserted-by":"crossref","first-page":"e173","DOI":"10.1111\/j.1365-2516.2012.02758.x","article-title":"Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies","volume":"18","author":"Coppola","year":"2012","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.07.013_bib25","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1111\/hae.14450","article-title":"Off-label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: a scoping review of the literature","volume":"28","author":"Thomas","year":"2022","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.07.013_bib26","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1111\/hae.14491","article-title":"Efficacy of emicizumab in von Willebrand disease (VWD) patients with and without alloantibodies to von Willebrand factor (VWF): report of two cases and review of literature","volume":"28","author":"Shanmukhaiah","year":"2022","journal-title":"Haemophilia"},{"key":"10.1016\/j.jtha.2025.07.013_bib27","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1055\/s-0044-1787662","article-title":"Emicizumab in Type 3 von Willebrand disease: report of a case with an alloantibody and literature review","volume":"51","author":"Giuffrida","year":"2025","journal-title":"Semin Thromb Hemost"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.07.013_bib28","first-page":"24","article-title":"Elevating levels of endogenous circulating von Willebrand factor (VWF): the potential of HMB-002 as a prophylactic treatment of von Willebrand disease (VWD) [Abstract]","volume":"31","author":"Ostergaard","year":"2025","journal-title":"Haemophilia"},{"issue":"Suppl 1","key":"10.1016\/j.jtha.2025.07.013_bib29","doi-asserted-by":"crossref","first-page":"3981","DOI":"10.1182\/blood-2024-208020","article-title":"A Phase Ia study of VGA039, a protein S-targeting monoclonal antibody, in individuals with von Willebrand disease demonstrates concentration-dependent increases in thrombin generation for reducing bleeding","volume":"144","author":"Millar","year":"2024","journal-title":"Blood"},{"issue":"Suppl 7","key":"10.1016\/j.jtha.2025.07.013_bib30","article-title":"OC 08.3 KB-V13A12, a novel nanobody-based therapeutic molecule for the treatment of von Willebrand disease [Abstract]","volume":"7","author":"Peyron","year":"2023","journal-title":"Res Pract Thromb Haemost"},{"key":"10.1016\/j.jtha.2025.07.013_bib31","doi-asserted-by":"crossref","first-page":"923","DOI":"10.3324\/haematol.2024.286076","article-title":"A fully humanized von Willebrand disease type 1 mouse model as unique platform to investigate novel therapeutic options","volume":"110","author":"McCluskey","year":"2025","journal-title":"Haematologica"},{"key":"10.1016\/j.jtha.2025.07.013_bib32","doi-asserted-by":"crossref","first-page":"3508","DOI":"10.1016\/j.jtha.2023.09.009","article-title":"Periprocedural management of type 2N von Willebrand disease with efanesoctocog alfa","volume":"21","author":"Ryu","year":"2023","journal-title":"J Thromb Haemost"}],"container-title":["Journal of Thrombosis and Haemostasis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625004374?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1538783625004374?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,1,5]],"date-time":"2026-01-05T08:39:24Z","timestamp":1767602364000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1538783625004374"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":31,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["S1538783625004374"],"URL":"https:\/\/doi.org\/10.1016\/j.jtha.2025.07.013","relation":{},"ISSN":["1538-7836"],"issn-type":[{"value":"1538-7836","type":"print"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A phase 1, open-label study to assess the pharmacokinetics, safety, and tolerability of a single intravenous injection of efanesoctocog alfa in adults with type 2N or type 3 von Willebrand disease","name":"articletitle","label":"Article Title"},{"value":"Journal of Thrombosis and Haemostasis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jtha.2025.07.013","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of International Society on Thrombosis and Haemostasis.","name":"copyright","label":"Copyright"}]}}