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
  • 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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