title: Immunomodulating drugs in myelodysplastic syndromes.
abstract: Based on immune mechanisms that appear to play an important role in the pathophysiology of at least part of the lower-risk myelodysplastic syndrome (MDS), the immunomodulating drug (IMID) thalidomide and its derivative lenalidomide (LEN) have been used in MDS, principally in lower-risk MDS. LEN has become the first-line US Food and Drug Administration (FDA)-approved treatment for lower-risk MDS with 5q deletion (del5q), in which its main mechanism of action is probably a direct cytotoxic activity on the del5q clone. This possibly specific effect is currently being investigated in higher-risk MDS-and even acute myeloid leukemia (AML)-with del5q, but LEN has also demonstrated some efficacy in MDS and AML without del5q. Thalidomide also has some activity in lower-risk MDS without del5q, but its side effects limit its practical use in these patients.
authors.affiliation: Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris, Paris, France. pierre.fenaux@avc.aphp.fr
authors.forename: Lionel
authors.initials: L
authors.lastname: Adès
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
mesh_terms: D002872:Chromosome Deletion; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D000077269:Lenalidomide; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D012307:Risk Factors; D013792:Thalidomide / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D007155:Immunologic Factors; D013792:Thalidomide; D000077269:Lenalidomide