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.