Successful treatment of acquired hemophilia with oral immunosuppressive therapy

Background: Acquired autoantibodies to factor VIII in patients without hemophilia are rare, but they cause severe illness and death if not eradicated. Objective: To examine daily therapy with oral cyclophosphamide and prednisone for acquired hemophilia. Design: Case series. Setting: Academic medical center. Patients: Nine consecutive patients without hemophilia who had severe hemorrhage caused by high titers of factor VIII inhibitors. Interventions: Daily oral cyclophosphamide and prednisone. Coagulation factors were used only for bleeding. Measurements: Plasma titers of factor VIII inhibitor, factor VIII activity, and clinical evidence of bleeding. Results: All patients achieved complete remission, which was defined as loss of residual inhibitor activity and return to a normal titer of factor VIII. Therapy lasted a median of 12 weeks (range, 3 to 37 weeks), Bleeding resolved in a median of 3 weeks (range, 2 to 10 weeks). Median follow-up after discontinuation of therapy was 91 weeks (range, 61 to 164 weeks). Conclusion: Daily administration of oral cyclophosphamide and prednisone without empirical factor VIII therapy seems to be an effective and well-tolerated treatment for acquired hemophilia.