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Haemophilia prophylaxis in young patients--a long-term follow-up
T Löfqvist1, I M Nilsson, E Berntorp
1Department of Coagulation Disorders, University of Lund, Malmö, Sweden.
Insights
Long-term prophylactic factor treatment in young patients with severe hemophilia A and B effectively prevented joint damage. Most patients maintained zero joint scores, indicating successful prevention of hemophilic arthropathy.
Area of Science:
- Paediatric Hematology
- Orthopaedic Surgery
- Radiology
Background:
- Severe hemophilia A and B are inherited bleeding disorders.
- Joint damage (hemophilic arthropathy) is a major complication.
- Early intervention with prophylactic factor treatment is crucial.
Purpose of the Study:
- To evaluate the long-term orthopaedic and radiological outcomes of early prophylactic factor treatment in young patients with severe hemophilia A and B.
Main Methods:
- A cohort of 34 patients (29 with hemophilia A, 5 with hemophilia B) aged 7-22 years were studied.
- Patients initiated treatment between 1-4.5 years of age with factor concentrates (F VIII or F IX) at 25-40 IU/kg, 2-3 times weekly.
- Orthopaedic and radiological joint scores were assessed annually over a 5-year period (1990-1995) using World Federation of Haemophilia criteria.
Main Results:
- Orthopaedic and radiological joint scores remained stable in nearly all patients throughout the follow-up period.
- A significant majority (79%) of patients maintained zero joint scores, indicating no joint damage.
- The treatment regimen demonstrated effectiveness in preserving joint health.
Conclusions:
- Early, high-dose prophylactic factor treatment is highly effective in preventing hemophilic arthropathy.
- These findings support the international consensus on early prophylaxis for hemophilia management.
- Proactive treatment strategies are essential for long-term joint health in young hemophilia patients.
Objectives:
To review long-term prophylactic factor treatment in young patients with severe haemophilia A and B, focusing on the orthopaedic and radiological outcome.
Design:
We received 34 patients with severe haemophilia A (n = 29) and B (n = 5), aged 7-22 years. Age at start of treatment was 1-4.5 years. Dosages of factor concentrate (F VIII and F IX, respectively) were 25-40 IU/kg body weight, three times a week for haemophilia A and twice a week for haemophilia B. The patients had been checked annually over a 5-year period (1990-95). Orthopaedic and radiological joint scores were evaluated according to recommendations by the World Federation of Haemophilia.
Setting:
All results were obtained at the Department for Coagulation Disorders, University of Lund, Malmö University Hospital, Malmö, Sweden.
Results:
Orthopaedic and radiological joint scores were found to have remained unchanged during follow-up in almost all patients and to be still zero (i.e. no unaffected joints) in 79% (n = 27) of the patients.
Conclusion:
There is a growing international consensus haemophilic arthropathy can be prevented by administering early high-dose prophylaxis. The results of the present investigation strongly support this opinion.