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Results of secondary prophylaxis in children with severe hemophilia
M J Manco-Johnson1, R Nuss, S Geraghty
1Department of Pediatrics, University of Colorado Health Sciences Center, Denver 80262.
Insights
Aggressive factor replacement therapy for hemophilic arthropathy in children stabilizes joint disease but does not reverse existing damage. Further research is needed to compare this with early prophylaxis before joint bleeding occurs.
Area of Science:
- Pediatric Hematology
- Orthopedic Surgery
- Rheumatology
Background:
- Severe hemophilia in children often leads to arthropathy due to recurrent joint hemorrhages.
- Established hemophilic arthropathy presents a significant clinical challenge, impacting quality of life.
Purpose of the Study:
- To evaluate the efficacy of routine replacement infusions of factor VIII or IX in treating established arthropathy in children with severe hemophilia.
- To assess the impact of aggressive factor concentrate therapy on the clinical course and radiographic findings of hemophilic joint disease.
Main Methods:
- Retrospective analysis of 13 children with severe hemophilia receiving factor VIII or IX prophylaxis.
- Dosage adjustments and monitoring of trough factor levels were performed.
- Clinical and radiographic assessments were used to evaluate joint disease progression.
Main Results:
- More than half of the children showed stabilization of joint disease.
- No reversal of abnormal radiographic findings was observed.
- Factor replacement therapy altered the clinical course but did not reverse established joint damage.
Conclusions:
- Aggressive factor concentrate therapy in children with established hemophilic arthropathy can stabilize the condition.
- This intervention does not reverse existing joint disease.
- Future studies comparing this approach with primary prophylaxis are warranted.
Abstract:
In this study, 13 children with severe hemophilia were given routine replacement infusions of factor VIII or IX to treat arthropathy. The children who had a mean age of 6.9 years (range 2.0-12.5) at initiation of prophylaxis had experienced an average of 43 acute hemorrhages (range 8-127) in the year prior to prophylaxis, of which a mean of 24 (range 5-46) were into joints. Therapy was begun in five children, using factor VIII concentrate at 20 U/kg three times a week, and one boy received factor IX concentrate 40 U/kg twice a week. This dose schedule was inadequate for three factor VIII-deficient boys and for the one factor IX-deficient boy. Two of three factor VIII-deficient boys responded to an increase to 30 U/kg prior to the 3-day interval. The dose frequency was increased to three times a week for the factor IX-deficient boy, but he continued to bleed and was taken to synovectomy. One of the original five factor VIII-deficient boys plus seven other factor VIII-deficient boys were begun on factor VIII 20 U/kg every other day; 3 boys ceased bleeding. Trough factor VIII levels were measured 24 hr after an infusion in the five boys who continued to bleed. Factor VIII dosage was adjusted to achieve a trough level of > 1%; 4 responded to an increase in the dose of factor VIII; 1 had an adequate trough but, due to compliance issues, was taken to synovectomy. Serial clinical and radiographic assessments determined stabilization of joint disease in more than one-half of the boys. No child showed reversal of abnormal radiographic findings. Institution of aggressive factor VIII and IX concentrate in children with established hemophilic arthropathy does not reverse joint disease but may alter the clinical course of hemophilia. Future studies to compare this intervention with primary prophylaxis instituted prior to the onset of recurrent joint hemorrhage are warranted.