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Immunosuppressive therapies in the management of acquired immune-mediated marrow failures

Risitano, Antonio M.

Current Opinion in Hematology: January 2012 - Volume 19 - Issue 1 - p 3–13
doi: 10.1097/MOH.0b013e32834da9a4
MYELOID BIOLOGY: Edited by David C. Dale

Purpose of review Immunosuppression is a key treatment strategy for patients suffering from aplastic anemia or related immune-mediated bone marrow failure syndromes. Several attempts have been performed to improve the standard immunosuppression regimen of horse antithymocyte globulin (h-ATG) and cyclosporine A (CyA).

Recent findings The addition of a third immunosuppression agent to h-ATG + CyA did not result in any improvement. Antilymphocyte agents other than h-ATG have been investigated. A rabbit-ATG preparation, which was known to be more immunosuppressive than h-ATG, resulted in markedly inferior outcome in a large randomized study from the National Institutes of Health. These data seem to be confirmed in additional experiences with rabbit-ATG from other groups. Cyclophosphamide and alemtuzumab have been proven to be biologically active in small studies, but available data suggest inferior outcomes when compared with h-ATG. All these alternative agents result in a more pronounced lymphocyte depletion, suggesting that the actual mechanisms of action of immunosuppressive therapy in aplastic anemia are not fully understood.

Summary Immunosuppression by h-ATG and CyA remains the standard of care for aplastic anemia patients lacking a low-risk transplant procedure, resulting in a 60–70% response rate. Rabbit-ATG, cyclophosphamide and alemtuzumab demonstrated a biological activity, but resulted in inferior outcome as compared with h-ATG; thus, they are not recommended as front-line therapy of aplastic anemia.

Department of Biochemistry and Medical Biotechnologies, Federico II University of Naples, Naples, Italy

Correspondence to Antonio M Risitano, MD, PhD, Assistant Professor of Hematology, Head of Bone Marrow Transplant Clinical Unit, Department of Biochemistry and Medical Biotechnologies, Federico II University of Naples, Via Pansini 5, 80131 Naples, Italy. Tel: +39 081 7462037; fax: +39 081 7464728; e-mail:

© 2012 Lippincott Williams & Wilkins, Inc.