Review of antihemophilic factor injection for the routine prophylaxis of bleeding episodes and risk of joint damage in severe hemophilia A.
Individuals with severe factor VIII deficiency experience recurrent hemorrhages and develop progressive joint damage. Large retrospective, nonrandomized studies of patient cohorts followed over decades show that factor prophylaxis initiated at an early age before the onset of recurrent bleeding reduces the incidence of hemophilic arthropathy. Two recent prospective, multicenter, randomized trials conducted in Europe (the ESPRIT study) and the USA (the Joint Outcome Study) confirm the efficacy of prophylaxis in the prevention of hemarthroses and arthropathy. Regular prophylaxis initiated in early childhood enhances the quality of life for patients with severe hemophilia and reduces the risk of inhibitor development. The substantial costs of such preventative therapy may be offset by the reduced expenditures that the care of degenerative joint disease in adult hemophilia patients would otherwise require.
Rossbach
Hans-Christoph
HC
Division of Pediatric Hematology/Oncology, St. Joseph Children's Hospital, Tampa, FL 33607, USA. hanschristoph.rossbach@baycare.org
D000293:Adolescent; D000328:Adult; D002648:Child; D003029:Coagulants / Q000008:administration & dosage* / Q000037:antagonists & inhibitors; D005169:Factor VIII / Q000008:administration & dosage* / Q000502:physiology; D006395:Hemarthrosis / Q000517:prevention & control*; D006467:Hemophilia A / Q000150:complications*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007592:Joint Diseases / Q000517:prevention & control; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic; D055815:Young Adult
D003029:Coagulants; D005169:Factor VIII
arthropathy; bleeding; factor VIII; hemophilia; prophylaxis