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Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
I M Nilsson1, E Berntorp, T Löfqvist
1Department of Coagulation Disorders, University of Lund, Malmö General Hospital, Sweden.
Insights
Prophylactic treatment for severe hemophilia in boys, initiated early, effectively prevents joint damage and allows normal lives. Continuous prophylaxis maintains factor levels above 1%, significantly improving patient outcomes.
Area of Science:
- Hematology
- Pediatric Medicine
- Orthopedics
Background:
- Severe hemophilia historically leads to significant joint damage and disability.
- Prophylactic treatment aims to mitigate the severe manifestations of hemophilia.
- Early intervention is crucial for long-term patient health outcomes.
Purpose of the Study:
- To evaluate the long-term efficacy of early prophylactic treatment in severe hemophilia.
- To assess the impact of prophylaxis on joint health and patient quality of life.
- To determine optimal prophylactic regimens for hemophilia A and B.
Main Methods:
- Retrospective analysis of 60 severe hemophiliacs (3-32 years old) receiving continuous prophylaxis since infancy.
- Assessment of orthopedic and radiological joint scores using World Federation of Hemophilia criteria.
- Monitoring of Factor VIII (F VIII) and Factor IX (F IX) levels.
Main Results:
- 29 out of 35 subjects (aged 3-17) achieved zero joint scores.
- Older patients exhibited only minor joint defects.
- Factor VIII:C and IX:C levels generally remained above 1% of normal.
- All 60 patients reported leading normal lives.
Conclusions:
- Effective continuous prophylaxis initiated in early childhood can prevent hemophilic arthropathy.
- Maintaining factor levels above 1% is critical for preventing joint disease.
- Early prophylactic treatment enables individuals with severe hemophilia to lead normal lives.
Abstract:
In Sweden, prophylactic treatment of boys with severe haemophilia has been practised since 1958 in an attempt to convert the disease from a severe to a milder form. The present study population consisted of 60 severe haemophiliacs (52 A, 8 B), aged 3-32 years. Treatment is started when the boys are 1-2 years of age, the regimens used being 24-40 IU F VIII kg-1 three times weekly in haemophilia-A cases (i.e. greater than 2000 IU kg-1 annually) and 25-40 IU F IX kg-1 twice weekly in haemophilia-B cases. The orthopaedic and radiological joint scores (maximum scores of 90 and 78, respectively) are evaluated as recommended by the World Federation of Haemophilia. Of those subjects aged 3-17 years, 29 out of 35 individuals had joint scores of zero. The oldest group had only minor joint defects. The VIII:C and IX:C concentrations had usually not fallen below 1% of normal. All 60 patients are able to lead normal lives. In conclusion, it appears to be possible to prevent haemophilic arthropathy by giving effective continuous prophylaxis from an early age, and preventing the VIII:C or IX:C concentration from falling below 1% of normal.