Early Prophylaxis Effective for Children With Hemophilia

Children initiated on prophylaxis by 36 months of age have less hemarthroses and no arthropathy

THURSDAY, Feb. 3 (HealthDay News) -- Prophylactic treatment of bleeding and arthropathy in children with hemophilia A is effective, particularly when initiated early in life, according to a study published online Jan. 21 in the Journal of Thrombosis and Haemostasis.

Alessandro Gringeri, M.D., from the University of Milan in Italy, and colleagues conducted a 10-year randomized controlled study of 1- to 7-year-old children with severe hemophilia A who had negative clinical-radiological joint scores at entry and at least one bleed during the preceding six-month period. The efficacy of prevention of hemarthroses and joint damage by prophylaxis with 25 IU/kg recombinant factor VIII three times a week (21 children) was compared with episodic therapy (19 children) with up to 25 IU/kg every 12 or 24 hours until bleeding resolution.

The researchers found that children on prophylaxis had significantly fewer hemarthroses than the children receiving episodic treatment (0.20 versus 0.52 events/patient/month). Signs of arthropathy, assessed by plain-film radiology, were demonstrated in 29 percent of the patients in the prophylaxis group and 74 percent of patients treated episodically. Prophylaxis was more effective when initiated early, at or before 36 months, resulting in fewer joint bleeds and no radiologic signs of arthropathy.

"Early prophylaxis is more efficacious than delayed prophylaxis, even though the latter still generates substantial benefits to these patients. The higher costs of prophylaxis compared to episodic therapy are balanced by a better orthopedic outcome and hence a better quality of life," the authors write.

This study was supported by an unrestricted grant from Baxter Italy; several authors disclosed financial relationships with pharmaceutical companies, including Baxter.

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