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The impact of prophyactic treatment on children with severe haemophilia
R J Liesner1, K Khair, I M Hann
1The Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Trust, London.
Insights
Prophylactic factor concentrate significantly reduces bleeding episodes and joint damage in children with severe hemophilia. This treatment improves quality of life, reducing hospitalizations and school absences.
Area of Science:
- Pediatric Hematology
- Hemophilia Management
- Bleeding Disorders
Background:
- Severe hemophilia requires intensive management to prevent complications.
- Prophylaxis with factor concentrates is a cornerstone of modern hemophilia care.
- Evaluating the long-term effectiveness of prophylaxis is crucial for optimizing patient outcomes.
Purpose of the Study:
- To assess the overall effectiveness of prophylactic factor concentrate therapy in children with severe hemophilia.
- To evaluate the impact of prophylaxis on bleeding frequency, joint health, and quality of life.
Main Methods:
- Retrospective evaluation of 27 children with severe hemophilia on regular prophylactic factor concentrate.
- Analysis of bleeding history before and during prophylaxis.
- Assessment of joint status (arthropathy), need for walking aids, hospital admissions, and school days lost.
Main Results:
- Prophylaxis dramatically reduced annual bleeding episodes from a median of 14.7 to 1.5 (P<0.001).
- Significant improvement in arthropathy was observed, with eight children showing reversal of chronic joint damage.
- Reductions in hospital admissions, need for walking aids, and school days lost were reported.
Conclusions:
- Prophylactic factor concentrate therapy is highly effective in reducing bleeding and improving joint health in children with severe hemophilia.
- This treatment leads to substantial improvements in quality of life for affected children and their families.
- Prophylaxis represents a vital strategy in comprehensive hemophilia care, minimizing long-term morbidity.
Abstract:
Twenty-seven children with severe haemophilia receiving regular prophylactic factor concentrate were evaluated to examine the overall effectiveness of prophylaxis in modern haemophilia care. The median age at the start of prophylaxis was 6.2 years (range 1.3-15.9 years) and the cumulative length of follow-up was 808 months (mean 30, rang 7-76 months). Nine patients required a central venous catheter for venous access (age range 1.3-5.2 years), eight boys could cannulate themselves and in 10 the parents performed regular venepuncture. The mean dose of concentrate given at the time of study was 31.8 U/kg three times weekly (range 12.5.6 U/kg) or 4900 U/kg/year (range 1900-8200). None developed an inhibitor on prophylaxis, though four had previously had an antibody. The median average annual number of bleeds in the 27 patients prior to prophylaxis was 14.7 (range 3.7-35.4). On prophylaxis this fell to 1.5 (range 0-12.5) (P<0.001) and in the group as a whole the frequency of bleeds diminished in successive years on prophylaxis. All 20 children with evidence of arthropathy improved on prophylaxis and eight had reversal of chronic damage such that their joints appeared normal at the time of study. There were reductions in the need for walking aids, in hospital admissions, and in numbers of school days lost for bleeding episodes. All families feel that prophylaxis has brought about an improvement in quality of life.
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