Immunomodulating drugs in myelodysplastic syndromes | Aplastic Anemia and MDS International Foundation

Immunomodulating drugs in myelodysplastic syndromes

Journal Title: 
Hematology Am Soc Hematol Educ Program
Author(s): 
Adès L, Fenaux P.
Primary Author: 
Adès L
Original Publication Date: 
Thursday, December 1, 2011

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 patien

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