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Katsuyoshi Koh,1 Keiji Nogami,2 Harumi Kakuda,3 Yuri Okimoto,3 Moeko Hino,4 Yusuke Tanigawara,5,6 Zhaoyang Li,7 Chihiro Suzuki,8 Souhei Terashio,8 Hitoshi Ueda,9 Shouichi Ohga10 1Department of Hematology/Oncology, Saitama Children’s Medical Center, Saitama City, Saitama, Japan; 2Department of Pediatrics, Nara Medical University, Kashihara City, Nara, Japan; 3Department of Hematology/Oncology, Chiba Children’s Hospital, Chiba City, Chiba, Japan; 4Department of Pediatrics, Graduate School of Medicine, Chiba University, Chiba City, Chiba, Japan; 5Laboratory of Pharmacometrics and Systems Pharmacology, K-FRECS at Tonomachi, Keio University, Kawasaki City, Kanagawa, Japan; 6Quantitative Clinical Pharmacology, Takeda Development Center, Osaka City, Osaka, Japan; 7Clinical & Translational Sciences, PDT Business Unit, Takeda Development Center Americas, Inc., Cambridge, MA, USA; 8Plasma-Derived Therapies Strategy Unit, PDT R&D, Takeda Pharmaceutical Company Limited, Osaka City, Osaka, Japan; 9Japan Medical Office, Takeda Pharmaceutical Company Limited, Chuo-ku, Tokyo, Japan; 10Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka City, Fukuoka, JapanCorrespondence: Katsuyoshi Koh, Department of Hematology/Oncology, Saitama Children’s Medical Center, 1-2, Shintoshin, Chuo-ku, Saitama, 330-8777, Japan, Email ko.katsuyoshi@saitama-pho.jpPurpose: Severe congenital protein C deficiency (SCPCD) is a rare, life-threatening disorder. Plasma-derived protein C concentrate is recommended for the acute and long-term management of SCPCD; however, data in Japanese patients are lacking. In this study, the pharmacokinetics (PK) and safety of protein C concentrate were investigated in a Japanese population.Patients and Methods: This study was an open-label, phase 1/2, nonrandomized, noncontrolled, multicenter clinical trial in Japanese patients with SCPCD (ClinicalTrials.gov: NCT04984889). Patients received a single intravenous dose (80 IU/kg) of human plasma-derived protein C ... |