{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:51:07Z","timestamp":1787014267513,"version":"build-2736575974"},"reference-count":23,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T00:00:00Z","timestamp":1657238400000},"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":[[2022,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Omfiloctocog alfa, the first China\u2010developed recombinant factor VIII (FVIII), demonstrated efficacy and safety of prophylaxis in previously treated patients (PTPs) aged \u226512 years with severe hemophilia A in China.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>To investigate efficacy, safety and pharmacokinetics (PK) of omfiloctocog alfa in pediatric PTPs with severe hemophilia A in China.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>PTPs (&gt;50 exposure days [ED] for Chinese patients aged &lt;6 years; &gt;150 EDs for patients aged 6\u201312 years) were treated with omfiloctocog alfa at 25\u201350\u00a0IU\/kg every other day or three times per week for 24\u00a0weeks. PK was evaluated after single injection of 50\u00a0IU\/kg. The primary efficacy endpoint was annualized bleeding rate (ABR).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      A total of 69 patients were enrolled (&lt;6 years,\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u00a035; 6\u201312 years,\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u00a034) and mean exposure to omfiloctocog alfa was 78.9 days. Mean half\u2010life was 6.7 and 10.2\u00a0h in children\u00a0&lt;\u00a06 years and 6\u201312 years, respectively. Estimated mean ABRs of all patients were 4.05 for overall bleeding episodes and 1.38 for spontaneous bleeding episodes. Of 127 bleeding episodes, the success rate was 92.1%. 39.7% patients did not experience any bleeding episodes and the mean weekly dose of FVIII was 109.1\u00a0IU\/kg for these patients. 83% bleeding episodes were controlled with \u22642 injections. Adverse reactions occurred in 2.9% of the patients. One 2\u2010year\u2010old patient developed inhibitors after 12 EDs and it resolved with omfiloctocog alfa immune tolerance induction.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Omfiloctocog alfa was efficacious and well tolerated for the prevention and treatment of bleeding in Chinese pediatric PTPs with severe hemophilia A.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.14622","type":"journal-article","created":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T10:26:18Z","timestamp":1657275978000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Efficacy, safety and pharmacokinetics of recombinant human coagulation factor VIII (omfiloctocog alfa) in previously treated Chinese children with severe hemophilia A"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4030-209X","authenticated-orcid":false,"given":"Runhui","family":"Wu","sequence":"first","affiliation":[{"name":"Beijing Children's Hospital Capital Medical University  Beijing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoling","family":"Wang","sequence":"additional","affiliation":[{"name":"Beijing Children's Hospital Capital Medical University  Beijing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xielan","family":"Zhao","sequence":"additional","affiliation":[{"name":"Xiangya Hospital Central South University  Hunan People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanli","family":"Cheng","sequence":"additional","affiliation":[{"name":"Shanxi Provincial Children's Hospital  Shanxi People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zeping","family":"Zhou","sequence":"additional","affiliation":[{"name":"The Second Affiliated Hospital of Kunming Medical University  Yunnan People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Sun","sequence":"additional","affiliation":[{"name":"Nanfang Hospital Affiliated to Southern Medical University  Guangdong People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ming","family":"Xu","sequence":"additional","affiliation":[{"name":"Chengdu Women's &amp; Children's Central Hospital  Sichuan People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wenqian","family":"Li","sequence":"additional","affiliation":[{"name":"Qinghai Provincial People's Hospital  Qinghai People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianwen","family":"Xiao","sequence":"additional","affiliation":[{"name":"The Affiliated Children's Hospital of Chongqing Medical University  Chongqing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fenge","family":"Yang","sequence":"additional","affiliation":[{"name":"Fujian Medical University Union Hospital  Fujian People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yun","family":"Chen","sequence":"additional","affiliation":[{"name":"Jinan Central Hospital  Shandong People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weiqun","family":"Xu","sequence":"additional","affiliation":[{"name":"The Affiliated Children's Hospital of Zhejiang Medical University  Zhejiang People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Huang","sequence":"additional","affiliation":[{"name":"The Affiliated Hospital of Guizhou Medical University  Guangdong People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuanrong","family":"Ma","sequence":"additional","affiliation":[{"name":"Beijing Engineering Research Center of Protein and Antibody Sinocelltech Ltd.  Beijing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wenlin","family":"Gai","sequence":"additional","affiliation":[{"name":"Beijing Engineering Research Center of Protein and Antibody Sinocelltech Ltd.  Beijing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liangzhi","family":"Xie","sequence":"additional","affiliation":[{"name":"Beijing Engineering Research Center of Protein and Antibody Sinocelltech Ltd.  Beijing People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renchi","family":"Yang","sequence":"additional","affiliation":[{"name":"State Key Laboratory of Experimental Hematology National Clinical Research Center for Hematological Disorders Institute of Hematology &amp; Blood Diseases Hospital Chinese Academy of Medical Sciences &amp; Peking Union Medical College  Tianjin People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,7,8]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa067659"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-09-457036"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000658"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14101"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14029"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14350"},{"key":"e_1_2_10_9_1","unstructured":"European Medicines Agency.Guideline on the clinical investigation of recombinant and human plasma\u2010derived factor VIII products.2018.https:\/\/www.ema.europa.eu\/en\/documents\/scientific\u2010guideline\/guideline\u2010clinical\u2010investigation\u2010recombinant\u2010human\u2010plasma\u2010derived\u2010factor\u2010viii\u2010products\u2010revision\u20102_en.pdf"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"e_1_2_10_11_1","unstructured":"European Medicines Agency.Guideline on core SmPC for human plasma derived and recombinant coagulation factor VIII products rev. 3.2019.https:\/\/www.ema.europa.eu\/en\/documents\/scientific\u2010guideline\/guideline\u2010core\u2010smpc\u2010human\u2010plasma\u2010derived\u2010recombinant\u2010coagulation\u2010factor\u2010viii\u2010products\u2010revision\u20103_en.pdf"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0038-1653759"},{"key":"e_1_2_10_13_1","doi-asserted-by":"crossref","unstructured":"LeeM MorfiniM SchulmanS IngerslevJ.Factor VIII\/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis. 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