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This prospective, open-label phase 2\/3 study evaluated caplacizumab 10\u00a0mg administered daily during plasma exchange and for 30\u00a0days afterward, in combination with immunosuppressive treatment, in Japanese adults with a clinical diagnosis of iTTP (new or recurrent). The primary endpoint was prevention of iTTP recurrence; key secondary endpoints included time to platelet count response, time to organ damage normalization, and safety. Among 21 treated patients, 1 of 15 (6.7%) evaluable patients developed iTTP recurrence. Median time to normalization was 2.79\u00a0days for platelet count and 2.65\u00a0days for organ damage markers (<jats:italic>n<\/jats:italic>\u2009=\u200915). Treatment-emergent adverse events (TEAEs) were mostly mild to moderate in severity; the most frequently reported caplacizumab-related TEAEs were increased alanine aminotransferase, epistaxis, and gastrointestinal hemorrhage (all in 9.5% of patients). At least one bleeding event was reported in 7 of 21 patients (33%). Caplacizumab was effective in Japanese patients with iTTP, with a low rate of iTTP recurrence, rapid normalization of platelet counts and organ damage markers, and no unexpected TEAEs. Trial registration: ClinicalTrials.gov identifier, NCT04074187.<\/jats:p>","DOI":"10.1007\/s12185-022-03495-6","type":"journal-article","created":{"date-parts":[[2022,11,25]],"date-time":"2022-11-25T10:05:03Z","timestamp":1669370703000},"page":"366-377","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":21,"title":["The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2\/3 study"],"prefix":"10.1007","volume":"117","clinical-trial-number":[{"clinical-trial-number":"nct04074187","registry":"10.18810\/clinical-trials-gov"}],"author":[{"given":"Yoshitaka","family":"Miyakawa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazunori","family":"Imada","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Satoshi","family":"Ichikawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hitoji","family":"Uchiyama","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasunori","family":"Ueda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akihito","family":"Yonezawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shigeki","family":"Fujitani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshiyuki","family":"Ogawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tadashi","family":"Matsushita","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidesaku","family":"Asakura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenji","family":"Nishio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kodai","family":"Suzuki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasuhiro","family":"Hashimoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidenori","family":"Murakami","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sayaka","family":"Tahara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tomoyuki","family":"Tanaka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7243-3126","authenticated-orcid":false,"given":"Masanori","family":"Matsumoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,11,24]]},"reference":[{"key":"3495_CR1","doi-asserted-by":"publisher","first-page":"17020","DOI":"10.1038\/nrdp.2017.20","volume":"3","author":"JA Kremer Hovinga","year":"2017","unstructured":"Kremer Hovinga JA, Coppo P, Lammle B, Moake JL, Miyata T, Vanhoorelbeke K. 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KI received honoraria from Celgene Co., Ltd., Bristol-Myers Squibb K.K., Astellas Pharma Inc., Chugai Pharmaceutical Co., Ltd., Sumitomo Dainippon Pharma Co., Ltd., Otuka Pharmaceutical Co. Ltd., Takeda Pharmaceutical Co. Ltd., Novartis Pharma K.K, Meiji Seika Pharma Co. Ltd., Ono Pharmaceutical Co. Ltd., Janssen Pharmaceutical K.K., Nippon Shinyaku Co., Ltd., and AstraZeneca K.K. SI received honoraria from Sanofi K.K., AstraZeneca K.K, and Chugai Pharmaceutical Co., Ltd. TM served on advisory boards for Takeda Pharmaceutical Co. Ltd. (Baxalta\/Shire), Bayer Yakuhin, Ltd., Novo Nordisk Pharma Ltd., Chugai Pharmaceutical Co., Ltd., and Pfizer Inc., received educational and investigational support from Chugai Pharmaceutical Co., Ltd. and Novo Nordisk Pharma Ltd., and received honoraria from Takeda Pharmaceutical Co. Ltd. (Shire), Bayer Yakuhin, Ltd., Sanofi K.K. (Bioverative), Chugai Pharmaceutical Co., Ltd., CSL Behring K.K., Japan Blood Products Organization, KM Biologics Co., Ltd., Kyowa Kirin Co., Ltd., Nichiyaku, Novo Nordisk Pharma Ltd., Octapharma AG, and Sysmex Corporation. MM received honoraria from Asahi Kasei Pharma Corporation, Chugai Pharmaceutical Co., Ltd., and Alexion Pharma Inc., has served as a consultant for Sanofi K.K., Takeda Pharmaceutical Co. Ltd., and Alexion Pharma Inc., received research funding from Asahi Kasei Pharma Corporation and Chugai Pharmaceutical Co., Ltd., and received patents and royalties from Alfesa Pharma Corporation. YH, ST, HM, and TT are currently employed by Sanofi K.K. HU, YU, AY, SF, YO, HA, KN, and KS declare that they have no conflicts of interest.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}