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Effectiveness and Safety of Prophylactic Low-Dose Emicizumab in Young Children With Hemophilia A: A Multicenter

Guichi Zhou1, Liya He2, Chunyan Wang1

  • 1Department of Pediatric Hematology & Oncology Shenzhen Children's Hospital Shenzhen People's Republic of China.

Ejhaem
|July 8, 2026
PubMed

Insights

Low-dose emicizumab significantly reduced bleeding episodes and improved quality of life in young children with hemophilia A. This treatment is effective and feasible, even in resource-limited settings.

Area of Science:

  • Pediatric Hematology
  • Pharmacology

Background:

  • Limited real-world data exists for low-dose emicizumab in pediatric hemophilia A.
  • Hemophilia A poses significant bleeding risks in young children.

Purpose of the Study:

  • To evaluate the real-world effectiveness and safety of individualized low-dose emicizumab in young children with hemophilia A.
  • To assess the impact on bleeding rates, quality of life, and FVIII equivalent levels.

Main Methods:

  • Multicenter retrospective study of 26 children (median age 2.2 years) receiving low-dose emicizumab.
  • Data collected from 2019-2025, analyzing bleeding rates, maintenance doses, FVIII equivalent levels, and quality of life (CHO-KLAT).

Main Results:

  • Annualized bleeding rate decreased from 5.31 to 0.60 (p < 0.001) over 48 months.
  • Quality of life scores improved significantly (57.2 to 69.8, p < 0.001).
  • No thrombotic events were reported; subclinical joint changes progressed despite bleeding control.

Conclusions:

  • Individualized low-dose emicizumab is effective in reducing bleeding and enhancing quality of life in young hemophilia A patients.
  • The treatment demonstrates feasibility for use in resource-limited settings.
  • Continued monitoring for joint health is advised.
Abstract

Keywords:
childrenemicizumabhemophilia Alow‐dose prophylaxisresource‐limited settings

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