Thalidomide exerts in vitro heterogeneous biological effects on hematopoiesis which have supported its possible use in treating myelodysplastic syndromes (MDS). Some recent clinical trials have confirmed that thalidomide may improve anemia and, less frequently, other cytopenias, in a proportion of younger patients with low-risk MDS (11–56%, on intention-to-treat analysis). Of interest, erythroid responses may be achieved also in transfusion-dependent subjects with high serum levels of endogenous erythropoietin, a subset of MDS patients with little chance of responding to recombinant erythropoietin, alone or in combination with G-CSF.
Although the FDA currenlty apporvies thalidomide only for the 5q- deletion, there are many trials confirming effectiveness, albeit lesser effectiveness, in patients with MDS who do not have this deletion. NCCN mentions thalidomide and notes that it has greater effectiveness in the cases with 5q- deletion.Thus, it is recognized as a reasonably effective treatment for MDS, even without the 5q- diletion and there are more than 2 phase II trials that support it.
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