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Physical Activity Limitations In Children With Severe Haemophilia A. Does Emicizumab Make A Difference?

Ahmed Sobhi Hassan1, Laila Metwally Sherief2, Mona Hamdy3

  • 1Department of Pediatrics, Kafrelsheikh University,Egypt.

JPMA. the Journal of the Pakistan Medical Association
|July 24, 2023
PubMed

Insights

Emicizumab prophylaxis significantly improved physical activity in children with severe haemophilia A. This treatment enhanced the quality of life for young patients managing this rare bleeding disorder.

Area of Science:

  • Paediatric Haematology
  • Clinical Paediatrics
  • Pharmacology

Background:

  • Severe haemophilia A impacts children's physical activity and quality of life.
  • Current treatments aim to prevent bleeding episodes and improve patient outcomes.
  • Emicizumab offers a novel prophylactic option for severe haemophilia A.

Purpose of the Study:

  • To evaluate the impact of emicizumab prophylaxis on physical activity levels in children with severe haemophilia A.
  • To assess changes in physical activity using the Paediatric Haemophilia Activities List.
  • To determine the efficacy of emicizumab in improving daily functioning for these patients.

Main Methods:

  • Prospective cohort study conducted between October 2021 and April 2022.
  • Involved 29 children (aged 4-18 years) with severe haemophilia A receiving emicizumab prophylaxis.
  • Physical activity assessed at baseline and after six months using the Paediatric Haemophilia Activities List.

Main Results:

  • A significant increase in the Paediatric Haemophilia Activities List sum score was observed post-intervention (median 59.54 to 84, p<0.001).
  • The study included 29 boys, with a mean age of 8.7 years.
  • 58.62% of patients were negative for inhibitors.

Conclusions:

  • Emicizumab prophylaxis demonstrated a significant improvement in physical activity levels for children with severe haemophilia A.
  • This suggests emicizumab can positively impact the functional capacity and well-being of young patients.
  • Further research can explore long-term effects and broader quality-of-life improvements.
Abstract

Keywords:
Emicizumab, Haemophilia, Haematology, PedHAL.

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