{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,19]],"date-time":"2026-04-19T06:32:14Z","timestamp":1776580334375,"version":"3.51.2"},"reference-count":31,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2021,6,5]],"date-time":"2021-06-05T00:00:00Z","timestamp":1622851200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2021,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>SCT800 is a recombinant human B\u2010domain\u2010deleted coagulation factor VIII (BDDrFVIII) developed in China.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To evaluate the repeat pharmacokinetics (PKs), efficacy, and safety of SCT800 in previously treated Chinese adolescent and adult patients with severe haemophilia A.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A phase III, multicentre, prospective, open\u2010label, single\u2010arm trial was conducted at 12 medical centres. Subjects received treatment for 24\u00a0weeks. PKs were assessed at the initial and repeated dosing 24\u00a0weeks later. The primary endpoint was annualized bleeding rate (ABR). Breakthrough bleeding episodes and inhibitor development were assessed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 71 of 73 patients completed the study, and 18 were enrolled for the repeat PK investigation. Total exposure was 5643 exposure days. Overall, SCT800 showed comparable repeat PK profiles. The total ABR was 2.82 (95% confidence interval 2.01\u20103.96). During prophylaxis, 43.8% of patients had no bleeding episodes. The majority (89.4%) of bleeding episodes were controlled with 1\u20132 injections of SCT800, the success rate (defined as \u2018excellent\u2019 or \u2018good\u2019 haemostatic response) for the treatment of bleeding episodes was 92.6%. The incidence of treatment\u2010related adverse events was 53.4%. Drug\u2010related AE incidence was 4.1%. The observed AEs were similar to those of other coagulation factor VIII, but lower in frequency. No subject developed an inhibitor, and no other safety concerns were identified.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>SCT800 has robust PK characteristics, and is safe and efficacious for the prophylaxis and treatment of bleeding episodes in previously treated adolescent and adult patients with severe haemophilia A.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14350","type":"journal-article","created":{"date-parts":[[2021,6,5]],"date-time":"2021-06-05T07:40:10Z","timestamp":1622878810000},"page":"814-822","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Pharmacokinetic, efficacy and safety evaluation of B\u2010domain\u2010deleted recombinant FVIII (SCT800) for prophylactic treatment in adolescent and adult patients with severe haemophilia A"],"prefix":"10.1111","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5947-3924","authenticated-orcid":false,"given":"Feng","family":"Xue","sequence":"first","affiliation":[{"name":"State Key Laboratory of Experimental Hematology Tianjin Laboratory of Blood Disease Gene Therapy CAMS Key Laboratory of Gene Therapy for Blood Diseases National Clinical Research Center for Blood Diseases Chinese Academy of Medical Sciences &amp; Peking Union Medical College Institute of Hematology &amp; Blood Diseases Hospital  Tianjin China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xielan","family":"Zhao","sequence":"additional","affiliation":[{"name":"Department of Hematology Xiangya Hospital Central South University  Changsha China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jing","family":"Sun","sequence":"additional","affiliation":[{"name":"Department of Hematology Nanfang Hospital Nanfang Medial University  Guangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaojing","family":"Zeng","sequence":"additional","affiliation":[{"name":"Department of Hematology The Affiliated Hospital of Guizhou Medical University  Guiyang China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fenge","family":"Yang","sequence":"additional","affiliation":[{"name":"Fujian Institute of Hematology Fujian Medical University Union Hospital  Fuzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ming","family":"Xu","sequence":"additional","affiliation":[{"name":"Pediatric Hematology&amp; Oncology Chengdu Women's &amp; Children's Central Hospital  Chengdu China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ziqiang","family":"Yu","sequence":"additional","affiliation":[{"name":"Department of Hematology The First Affiliated Hospital of Soochow University  Suzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Weiying","family":"Gu","sequence":"additional","affiliation":[{"name":"Department of Hematology The First People's Hospital of Changzhou  Changzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ying","family":"Feng","sequence":"additional","affiliation":[{"name":"Department of Hematology The Second Affiliated Hospital of Guangzhou Medical University  Guangzhou China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wenqian","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Hematology &amp; Rheumatology Qinghai Provincial People's Hospital  Qinghai China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Changcheng","family":"Zheng","sequence":"additional","affiliation":[{"name":"Department of Hematology The First Affiliated Hospital of University of Science and Technology of China  Hefei China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hui","family":"Bi","sequence":"additional","affiliation":[{"name":"Department of Hematology The Second Affiliated Hospital of Kunming Medical University  Kunming China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liangzhi","family":"Xie","sequence":"additional","affiliation":[{"name":"Beijing Engineering Research Center of Protein and Antibody Sinocelltech Ltd.  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wenlin","family":"Gai","sequence":"additional","affiliation":[{"name":"Beijing Engineering Research Center of Protein and Antibody Sinocelltech Ltd.  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Renchi","family":"Yang","sequence":"additional","affiliation":[{"name":"State Key Laboratory of Experimental Hematology Tianjin Laboratory of Blood Disease Gene Therapy CAMS Key Laboratory of Gene Therapy for Blood Diseases National Clinical Research Center for Blood Diseases Chinese Academy of Medical Sciences &amp; Peking Union Medical College Institute of Hematology &amp; Blood Diseases Hospital  Tianjin China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,6,5]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2013.06.002"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2010.03962.x"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12288-012-0209-0"},{"key":"e_1_2_9_5_1","first-page":"s116","article-title":"Advances in hemophilia and the role of current and emerging prophylaxis","volume":"22","author":"Acharya SS","year":"2016","journal-title":"Am J Manag Care"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa067659"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2516.2002.00581.x"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14101"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14029"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/aps.2015.121"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"e_1_2_9_13_1","unstructured":"European Medicines Agency. 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