{"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":1776580334561,"version":"3.51.2"},"reference-count":24,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,3,13]],"date-time":"2023-03-13T00:00:00Z","timestamp":1678665600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["pericles.pericles-gcp.literatumonline.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>On\u2010demand treatment is the most common treatment strategy for haemophilia A in China.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>This study aims to evaluate the efficacy and safety of a human\u2010derived B\u2010domain\u2010deleted recombinant factor VIII (TQG202) in the on\u2010demand treatment of bleeding episodes in moderate\/severe haemophilia A patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This multicentre, single\u2010arm clinical trial enrolled moderate\/severe haemophilia patients previously treated with FVIII concentrates for \u226550 exposure days (EDs) from May 2017 to October 2019. TQG202 was injected intravenously on\u2010demand for bleeding episode management. The primary endpoints were the infusion efficiency at 15 and 60\u00a0min after the first administration and haemostatic efficacy of the first bleeding episode. Safety was also monitored.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifty\u2010six participants were enrolled with a median age of 24.5 (range: 12\u201364)\u00a0years old. The median total dose of TQG202 was 29,250 IU per participant (range: 1750\u20132,02,500), and the median number of administrations was 24.5 (2\u2013116). The median infusion efficiency at 15 and 60\u00a0min after the first administration was 155.4% and 145.2%, respectively. Among the 48 first bleeding episodes evaluated, 47 (83.9%, 95% CI: 71.7%\u201392.4%) had a haemostatic efficacy rating of excellent or good. Eleven (19.6%) participants had treatment\u2010related adverse events (TRAEs), but no \u2265grade 3 TRAE was observed. Inhibitor development (0.6BU) was observed in one participant (1.8%) after 22 EDs, but was undetectable after 43 EDs.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>TQG202 for on\u2010demand treatment in moderate\/severe haemophilia A shows effective control of the bleeding symptoms, with a low incidence of adverse events and inhibitors development.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14777","type":"journal-article","created":{"date-parts":[[2023,3,13]],"date-time":"2023-03-13T17:33:57Z","timestamp":1678728837000},"page":"997-1004","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Efficacy and safety of the B\u2010domain\u2010deleted TQG202 for on\u2010demand treatment in moderate and severe haemophilia A patients: A multicentre, single\u2010arm trial"],"prefix":"10.1111","volume":"29","author":[{"given":"Zimin","family":"Sun","sequence":"first","affiliation":[{"name":"Department of Hematology The Affiliated Provincial Hospital of Anhui Medical University  Hefei Anhui China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yaming","family":"Xi","sequence":"additional","affiliation":[{"name":"Department of Hematology The First Hospital of Lanzhou University  Lanzhou Gansu China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8340-0154","authenticated-orcid":false,"given":"Wei","family":"Liu","sequence":"additional","affiliation":[{"name":"State Key Laboratory of Experimental Hematology National Clinical Research Center for Blood Diseases Haihe Laboratory of Cell Ecosystem Tianjin Key Laboratory of Gene Therapy for Blood Diseases CAMS Key Laboratory of Gene Therapy for Blood Diseases Institute of Hematology &amp; Blood Diseases Hospital, Chinese Academy of Medical Sciences &amp; Peking Union Medical College  Tianjin China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2000-4087","authenticated-orcid":false,"given":"Linhua","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Hematology The Second Hospital of Shanxi Medical University  Taiyuan Shanxi China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xuefeng","family":"Wang","sequence":"additional","affiliation":[{"name":"Ruijin Hospital Shanghai Jiaotong University Medical School  Shanghai China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chenghao","family":"Jin","sequence":"additional","affiliation":[{"name":"Hemophilia Information Management Center of Jiangxi Province Jiangxi Provincial People's Hospital  Nanchang Jiangxi China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Haifei","family":"Jia","sequence":"additional","affiliation":[{"name":"R&amp;D Institute Chia Tai Tianqing Pharmaceutical Group Co. 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