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Successful Treatment of Acquired Hemophilia with Oral Immunosuppressive Therapy

Linda G. Shaffer, MD; and Martin D. Phillips, MD
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For author affiliations and current author addresses, see end of text. Acknowledgments: The authors thank Dr. Burton Dickey for critical review of the manuscript and helpful suggestions, Dr. Chul Ahn for statistical advice, and the physicians who referred their patients. Requests for Reprints: Martin D. Phillips, MD, University of Texas-Houston Medical School, Division of Hematology, 6431 Fannin Street, Room 5.278 MSB, Houston, TX 77030. Current Author Addresses: Dr. Shaffer: Hematology/Oncology Section, Wilford Hall Medical Center/PFP, 2200 Bergquist, Suite 1, Lackland AFB, TX 78236.

Copyright ©2004 by the American College of Physicians

Ann Intern Med. 1997;127(3):206-209. doi:10.7326/0003-4819-127-3-199708010-00005
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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.


Grahic Jump Location
Figure 1.
Titers of factor VIII inhibitor in all patients after initiation of therapy.

Therapy with cyclophosphamide and prednisone was started on day 0. Numbers in the key refer to patient numbers.

Grahic Jump Location




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