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Prophylactic therapy for hemophilia in a developing country, Turkey
K Kavakli1, G Nişli, Y Aydinok
1Department of Pediatric Hematology, Medicine Faculty of Ege University, Bornova, Izmir, Turkey.
Pediatric Hematology and Oncology
|March 1, 1997
Summary
Prophylaxis significantly reduced bleeding episodes and hospitalizations in children with severe hemophilia. This study suggests prophylaxis is effective and feasible, even with intermediate concentrates, for preventing disabilities in developing countries.
Area of Science:
- Hematology
- Pediatrics
- Public Health
Background:
- Prophylaxis for hemophilia is widely accepted but cost-prohibitive in developing nations.
- High cost remains a major barrier to implementing prophylaxis in countries like Turkey.
Purpose of the Study:
- To evaluate the efficacy, safety, and feasibility of prophylaxis in children with severe hemophilia.
- To assess the impact of prophylaxis on bleeding episodes, joint health, and hospitalization rates.
Main Methods:
- A controlled preliminary study involving seven boys (1.5-7 years) with severe hemophilia (A and B).
- Patients received 20-50 IU/kg factor twice weekly for 6-24 months.
- Data compared with historical control data from the same patients before prophylaxis and a group on demand treatment.
Main Results:
- Bleeding episodes decreased from 10.5 to 4.5 per patient.
- Orthopedic and radiologic joint scores remained stable; hospital days significantly reduced from 18 to 0.7.
- No new hepatitis A, B, or HIV infections; one patient seroconverted for hepatitis C.
Conclusions:
- Twice-weekly prophylaxis with intermediate concentrates is superior to on-demand treatment for severe hemophilia.
- Prophylaxis is effective, safe, and feasible for preventing hemophilia-related disabilities in children.
- Prophylaxis should be considered for selected severely hemophilic children in developing countries.