Immunomodulatory treatment of myelodysplastic syndromes: antithymocyte globulin, cyclosporine, and alemtuzumab.
It is now well accepted that a subgroup of patients with myelodysplastic syndromes (MDS) can recover from pancytopenia following immunosuppressive treatment (IST). For many years immunosuppression with antilymphocyte antibodies has been a standard treatment approach for patients with severe aplastic anemia (SAA). The initial concept of using immunosuppression to treat pancytopenic patients with MDS was based on the premise that MDS might share with SAA an autoimmune basis for the bone marrow failure common to both conditions. The idea was supported by reports of favorable outcomes in occasional cases of MDS treated with antithymocyte globulin (ATG). Today, various forms of IST have been successfully used to restore hematopoiesis in MDS in many centers worldwide. In this review we outline the rationale for use of IST in MDS, and describe studies which help to define the patients with MDS likely to respond to IST. We summarize 18 published clinical trials using IST for MDS and discuss how these studies have helped to define the MDS subgroups likely to respond to treatment, the nature and durability of the response, the impact of IST on long-term outcome, and the best treatment approach.
Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892-1202, USA.
Ankur R
AR
Parikh
Matthew J
MJ
Olnes
A John
AJ
Barrett
D000074323:Alemtuzumab; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use*; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000276:immunology* / Q000628:therapy*; D016896:Treatment Outcome
D061067:Antibodies, Monoclonal, Humanized; D000961:Antilymphocyte Serum; D000970:Antineoplastic Agents; D007155:Immunologic Factors; D007166:Immunosuppressive Agents; D000074323:Alemtuzumab; D016572:Cyclosporine