2. Academic Validation
  3. Efficacy, safety and pharmacokinetics of recombinant human coagulation factor VIII (omfiloctocog alfa) in previously treated Chinese children with severe hemophilia A

Efficacy, safety and pharmacokinetics of recombinant human coagulation factor VIII (omfiloctocog alfa) in previously treated Chinese children with severe hemophilia A

  • Haemophilia. 2022 Nov;28(6):e199-e208. doi: 10.1111/hae.14622.
Runhui Wu 1,  Xiaoling Wang 1,  Xielan Zhao 2,  Yanli Cheng 3,  Zeping Zhou 4,  Jing Sun 5,  Ming Xu 6,  Wenqian Li 7,  Jianwen Xiao 8,  Fenge Yang 9,  Yun Chen 10,  Weiqun Xu 11,  Jing Huang 12,  Chuanrong Ma 13,  Wenlin Gai 13,  Liangzhi Xie 13,  Renchi Yang 14
Affiliations

Affiliations

  • 1 Beijing, Children's Hospital, Capital Medical University, Beijing, People's Republic of China.
  • 2 Xiangya Hospital Central South University, Hunan, People's Republic of China.
  • 3 Shanxi Provincial Children's Hospital, Shanxi, People's Republic of China.
  • 4 The Second Affiliated Hospital of Kunming Medical University, Yunnan, People's Republic of China.
  • 5 Nanfang Hospital Affiliated to Southern Medical University, Guangdong, People's Republic of China.
  • 6 Chengdu Women's & Children's Central Hospital, Sichuan, People's Republic of China.
  • 7 Qinghai Provincial People's Hospital, Qinghai, People's Republic of China.
  • 8 The Affiliated Children's Hospital of Chongqing Medical University, Chongqing, People's Republic of China.
  • 9 Fujian Medical University Union Hospital, Fujian, People's Republic of China.
  • 10 Jinan Central Hospital, Shandong, People's Republic of China.
  • 11 The Affiliated Children's Hospital of Zhejiang Medical University, Zhejiang, People's Republic of China.
  • 12 The Affiliated Hospital of Guizhou Medical University, Guangdong, People's Republic of China.
  • 13 Beijing Engineering Research Center of Protein and Antibody, Sinocelltech Ltd., Beijing, People's Republic of China.
  • 14 State Key Laboratory of Experimental Hematology, National Clinical Research Center for Hematological Disorders, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, People's Republic of China.
PMID: 35802040 DOI: 10.1111/hae.14622
Abstract

Introduction: Omfiloctocog alfa, the first China-developed recombinant Factor VIII (FVIII), demonstrated efficacy and safety of prophylaxis in previously treated patients (PTPs) aged ≥12 years with severe hemophilia A in China.

Aims: To investigate efficacy, safety and pharmacokinetics (PK) of omfiloctocog alfa in pediatric PTPs with severe hemophilia A in China.

Methods: PTPs (>50 exposure days [ED] for Chinese patients aged <6 years; >150 EDs for patients aged 6-12 years) were treated with omfiloctocog alfa at 25-50 IU/kg every Other day or three times per week for 24 weeks. PK was evaluated after single injection of 50 IU/kg. The primary efficacy endpoint was annualized bleeding rate (ABR).

Results: A total of 69 patients were enrolled (<6 years, n = 35; 6-12 years, n = 34) and mean exposure to omfiloctocog alfa was 78.9 days. Mean half-life was 6.7 and 10.2 h in children < 6 years and 6-12 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 IU/kg for these patients. 83% bleeding episodes were controlled with ≤2 injections. Adverse reactions occurred in 2.9% of the patients. One 2-year-old patient developed inhibitors after 12 EDs and it resolved with omfiloctocog alfa immune tolerance induction.

Conclusion: Omfiloctocog alfa was efficacious and well tolerated for the prevention and treatment of bleeding in Chinese pediatric PTPs with severe hemophilia A.

Keywords

children; hemophilia A; omfiloctocog alfa; pharmacokinetics; prophylaxis.

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