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Is Low Dose a New Dose to Initiate Hemophilia A Prophylaxis? - A Systematic Study in Eastern India

Shazia Gulshan1, Prakas Kumar Mandal2, Abhijit Phukan1

  • 1Department of Hematology, NRS Medical College, Kolkata, 700014, India.

Indian Journal of Pediatrics
|February 13, 2020
PubMed

Insights

Low-dose prophylaxis significantly reduced joint bleeds and school absenteeism in children with Hemophilia A. This treatment improved daily life and joint health, offering a cost-effective option for developing nations.

Area of Science:

  • Pediatric Hematology
  • Bleeding Disorders
  • Pharmacology

Background:

  • Hemophilia A is a severe genetic bleeding disorder.
  • Children with severe Hemophilia A often experience frequent joint bleeds, leading to chronic joint damage and reduced quality of life.
  • Current treatment often involves on-demand factor replacement, which may not prevent long-term complications.

Purpose of the Study:

  • To evaluate the efficacy of low-dose secondary/tertiary prophylaxis using recombinant Fc-fusion long-acting Factor VIII (ELOCTATE) in children with severe Hemophilia A.
  • To assess the impact of this prophylaxis on daily life, including joint health, school attendance, and overall activity levels.

Main Methods:

  • A prospective study involving 30 children (≤12 years) with severe Hemophilia A (Factor VIII <2%) and minimal joint bleeds.
  • Patients received ELOCTATE at 10 IU/kg twice weekly for one year, following a minimum six-month period of on-demand FVIII therapy.
  • Outcomes measured included annual bleeding rate, Hemophilia Joint Health Score (HJHS), school absenteeism, and activity scores.

Main Results:

  • Significant reduction in annual joint bleeds by 85.76% (from 14.5 to 2.2) and school absenteeism by 86% (from 17.38 to 2.42 days/month).
  • Moderate improvement in daily activity scores (43%) and mild improvement in School Activity Participation Score (57%).
  • Mean HJHS score improved, and annual hospitalization rate decreased from 8.7 to 1.1.

Conclusions:

  • Low-dose secondary/tertiary prophylaxis with ELOCTATE is effective in reducing joint bleeds and improving joint health and daily activity in children with severe Hemophilia A.
  • The reduced factor consumption offers a positive cost-benefit effect, making it a feasible treatment option, particularly in developing countries.
Abstract

Keywords:
Children up to 12 yDeveloping countriesHemophilia ALow doseSecondary/Tertiary prophylaxis

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