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Poster Networking

Pediatric Hemostasis and Thrombosis

PB0613 - Real-World Use of Emicizumab in Chinese Children with Hemophilia A: Retrospective Data from a Comprehensive Care Center

Monday, June 24, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall

    Presenting Author(s)

  • QM

    Qianqian Mao

    Master student
    Beijing Children´s hospital
    Beijing, Beijing, China (People's Republic)

    • Co-Author(s)

  • Zhenping Chen

    Associate professor
    Hematologic Diseases Laboratory, Hematology Center, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, ChinaChildren’s Health, Beijing, 100045, China
    Beijing, Beijing, China (People's Republic)

  • Runhui Wu, PhD, MD

    Professor
    Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
    Beijing, Beijing, China (People's Republic)

  • MP

    Man-Chiu Poon

    Professor
    University of Calgary
    Calgary, Alberta, Canada

  • GL

    Guoqing Liu

    professor
    Beijing Children’s Hospital
    Beijing, Beijing, China (People's Republic)

  • GL

    Gang Li

    researcher
    Beijing Children's Hospital
    Beijing, Beijing, China (People's Republic)

  • Zekun Li, PhD

    Ph.D.
    Department II of Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
    Beijing, Beijing, China (People's Republic)

  • Zhengping Li, Medical Doctor in progress

    M.D. in progress
    zhengp_li@163.com
    Beijing, Beijing, China (People's Republic)

  • Wanru Yao, MA

    Doctor
    Haemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, China, 100045
    Beijing, Beijing, China (People's Republic)

  • DA

    Di Ai

    PhD student
    Beijing Children’s Hospital
    Beijing, Beijing, China (People's Republic)

  • YZ

    Yingzi Zhen

    nurse
    Beijing Children‘s Hospital’
    Beijing, Beijing, China (People's Republic)

  • XC

    Xiaoling Cheng

    Department of Pharmacy
    Beijing Children’s Hospital, Capital Medical University,
    Beijing, Beijing, China (People's Republic)

  • NW

    Nan Wang, Master student

    Department of Pharmacy
    Beijing Children’s Hospital, Capital Medical University,
    Beijing, Beijing, China (People's Republic)

Background: Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China.

Aims: To explore the possibility of using reduced-dosage EMI in Chinese HA children.

Methods: We conducted a retrospective study for HA children in our Comprehensive Care Center.

Results: Thirty-four HA children receiving EMI prophylaxis for median (range) 24.53 (2.53-47.9) months by June 2023. Of these, 25 (73.5%) were under 3 years age, 26 (76.5%) had severe hemophilia and 12 (35.3%) were minimally or previously treated patients (MTP/PUP). Thirty-one (91.2%) of the 34 patients received reduced-dosage EMI for economic reason. EMI concentration and FVIII equivalent activity measured showed a strong correlation. Overall, while on EMI, their annual treated bleeding rate (ATBR) and annual bleeding rate (ABR) decreased significantly (2 to 0) while their zero-bleeding rate (ZBR) increased significantly (11.5% to 65.4%). The ATBR, ABR and ZBR and were similar between MTP/PUP and previous treated patients and were similar whether inhibitors were present or not. However, patients on reduced maintenance dosage may have a significant decrease in ZBR. ROC curves suggested following cut-off values for zero bleeding: 4-weekly maintenance dosage 3.77 mg/kg, EMI-concentration 48.1 µg/ml and FVIII equivalent activity 15.4 IU/dl.

Conclusion(s): We showed EMI effectively prevented bleeding even at reduced dosages. However, bleeding risk maybe higher with EMI 4-weekly maintenance dosage < 3.77 mg/kg, EMI concentration < 48.1 µg/ml and FVIII equivalent activity < 15.4 IU/dl for zero bleeding. It is important that dosage reduction be done rationally. Dosage tailoring is possible.