L G Shaffer1, M D Phillips. 1. University of Texas-M.D. Anderson Cancer Research Center, Houston, USA.
Abstract
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.
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.
Authors: Peter Collins; Francesco Baudo; Angela Huth-Kühne; Jørgen Ingerslev; Craig M Kessler; Maria E Mingot Castellano; Midori Shima; Jean St-Louis; Hervé Lévesque Journal: BMC Res Notes Date: 2010-06-07
Authors: Angela Huth-Kühne; Francesco Baudo; Peter Collins; Jørgen Ingerslev; Craig M Kessler; Hervé Lévesque; Maria Eva Mingot Castellano; Midori Shima; Jean St-Louis Journal: Haematologica Date: 2009-04 Impact factor: 9.941