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Low dose prophylaxis in Tunisian children with haemophilia

E Gouider1,2, L Jouini2, M Achour1,2

  • 1Hemophilia Center Aziza Othmana Hospital, Tunis, Tunisia.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|December 13, 2016
PubMed

Insights

Low dose prophylaxis for children with haemophilia is effective, reducing bleeding episodes and improving quality of life. This approach is recommended for resource-limited settings as a starting point for treatment.

Area of Science:

  • Pediatric Hematology
  • Bleeding Disorders
  • Public Health

Background:

  • Limited resources in many countries necessitate cost-effective haemophilia treatment strategies.
  • Low-dose prophylaxis offers a potential solution for managing haemophilia in underserved populations.

Purpose of the Study:

  • To evaluate the efficacy and outcomes of low-dose prophylaxis in children with haemophilia.
  • To assess the impact of prophylaxis on bleeding frequency, joint health, functional independence, and quality of life.

Main Methods:

  • A single-center study included 55 children with haemophilia.
  • Participants received a median weekly dose of 30 IU/kg, administered once, twice, or thrice weekly.
  • Outcomes were measured by bleeding episodes, Haemophilia Joint Health Score (HJHS), Functional Independence Score in Haemophilia (FISH), and Haemo-QoL.

Main Results:

  • A significant reduction in bleeding episodes was observed in all patients.
  • Post-prophylaxis assessments showed satisfactory Haemophilia Joint Health Score (HJHS), Functional Independence Score in Haemophilia (FISH), and Haemo-QoL scores.

Conclusions:

  • Low-dose prophylaxis is an effective treatment for paediatric haemophilia, outperforming on-demand therapy.
  • This regimen is a recommended starting point for prophylaxis in resource-limited countries.
Abstract

Keywords:
functional independence score in haemophiliahaemophiliahaemophilia joint health scorelow dose prophylaxisprophylaxis

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