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This phase II\/III open-label study evaluated the safety and efficacy of recombinant porcine factor VIII (rpFVIII, susoctocog alfa) in adults with AHA and severe bleeding episodes in Japan (NCT04580407). The initial rpFVIII dose was 200 U\/kg, with subsequent doses based on clinical measures including plasma FVIII activity. The primary efficacy endpoint was the proportion of severe bleeding episodes with a positive response to rpFVIII therapy 24\u00a0h after treatment initiation. Five patients were eligible for, and completed, rpFVIII treatment (age group: 60s\u201380s; median hFVIII inhibitor: 52\u00a0BU\/mL; porcine FVIII [pFVIII] inhibitor: 3\/5 patients). The median (range) total dose\/patient was 548.4 (198\u20131803) U\/kg with a median 3.0\u00a0infusions\/patient. All patients responded positively to rpFVIII therapy at 24\u00a0h regardless of baseline pFVIII inhibitor status. rpFVIII treatment was well tolerated with no adverse events of special interest such as thromboembolic events or de novo pFVIII inhibitors. This study supports the use of rpFVIII as a novel therapy in the clinical management of patients with AHA in Japan. rpFVIII was approved for treating bleeding episodes in adults with AHA in Japan in 2024.<\/jats:p>","DOI":"10.1007\/s12185-024-03823-y","type":"journal-article","created":{"date-parts":[[2024,8,19]],"date-time":"2024-08-19T11:02:51Z","timestamp":1724065371000},"page":"482-491","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Efficacy and safety of recombinant porcine factor VIII in Japanese patients with acquired hemophilia A"],"prefix":"10.1007","volume":"120","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6220-8657","authenticated-orcid":false,"given":"Yoshinobu","family":"Seki","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshiyuki","family":"Ogawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takahide","family":"Kikuchi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emiko","family":"Sakaida","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuki","family":"Mizuta","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tadayuki","family":"Kitagawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kazuhiko","family":"Takemura","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yasuo","family":"Miyaguchi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keiji","family":"Nogami","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tadashi","family":"Matsushita","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,8,19]]},"reference":[{"key":"3823_CR1","doi-asserted-by":"publisher","first-page":"564","DOI":"10.1111\/hae.12138","volume":"19","author":"JY Borg","year":"2013","unstructured":"Borg JY, Guillet B, Le Cam-Duchez V, Goudemand J, Levesque H, Sacha Study Group. 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Ltd., and Takeda Pharmaceuticals; contracts for clinical trials from Chugai Pharmaceutical and Takeda Pharmaceuticals; consulting fees from Takeda Pharmaceuticals; and payment\/honoraria from Asahi Kasei, Chugai Pharmaceutical, CSL Behring, Novo Nordisk, Sanofi, and Takeda Pharmaceuticals. YO reports consulting fees and payment\/honoraria from Chugai Pharmaceutical. TKikuchi reports no conflicts of interest. ES is an editor of the International Journal of Hematology, and reports consulting fees from Bristol Myers Squibb Japan, Otsuka Pharmaceutical, Servier, and Ohara Pharmaceutical Co. Ltd.; and payment\/honoraria from Janssen Pharmaceuticals, Novartis, Pfizer, Sanofi, and Takeda Pharmaceuticals. YMizuta, TKitagawa, KT, and YMiyaguchi are all employees of Takeda Pharmaceuticals. KN reports grants and payment\/honoraria from Bayer, Chugai Pharmaceutical, CSL Ltd., Fujimoto Pharmaceutical Corp., KM Biologics, Novo Nordisk, Sanofi, Sekisui, Sysmex, and Takeda Pharmaceuticals. TM reports grants from Chugai Pharmaceuticals and Novo Nordisk; consulting fees from Bayer, Chugai Pharmaceuticals, Novo Nordisk, Pfizer, and Takeda Pharmaceuticals; and payment\/honoraria from Bayer, Chugai Pharmaceuticals, CSL, JB Pharma, KM Biologics, Novo Nordisk, Sanofi, Sysmex, and Takeda Pharmaceuticals.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"The study protocol, informed consent form, and all amendments were reviewed and approved by the local institutional review board at each investigator site before study initiation. The study was conducted in accordance with the Declaration of Helsinki and the principles and guidelines described in the study protocol.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}},{"value":"All patients provided written informed consent (either in person or via a legal decision maker) prior to entering the study and were free to withdraw from the study at any time.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed consent"}}]}}