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Expert Opin Investig Drugs. 2006 Jul;15(7):805-13.

Immunomodulatory drugs in myelodysplastic syndromes.

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  • 1The Radhey Khanna MDS Center, University of Massachusetts Medical Centre, Division of Hematology, Department of Medicine, 364 Plantation Street, LRB 208, Worcester, MA 01605, USA. naomi.galili@umassmed.edu


This review summarises the mechanism of action of immunomodulatory analogues of thalidomide and their use in myelodysplastic syndromes. Thalidomide was found to have a response rate of approximately 20% in these patients. Lenalidomide--which is more potent and less toxic than thalidomide--has been used in three clinical trials and produced the best responses (60 - > 90%) in low- and intermediate-1-risk transfusion-dependent patients with del(5q). The responses are purely erythroid in nature, and are associated with major cytogenetic responses in > 50% of the del(5q) patients. Non-del(5q) low- and intermediate-1-risk transfusion-dependent patients also had a approximately 25% incidence of transfusion independence following therapy with lenalidomide. Median time to response is approximately 4 weeks and 90% of patients respond within 12 weeks. The precise mechanism of action remains unknown but anticytokine, antiangiogenic and immunomodulatory properties are thought to play a role.

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