The immunomodulatory agents lenalidomide and thalidomide for treatment of the myelodysplastic syndromes: a clinical practice guideline

Background: Myelodysplastic syndromes (MDS) are clonal disorders that result in cytopenias and risk of acute myeloid leukemia. Incidence increases with age and more diagnoses are expected with the aging population. Treatment includes red blood cell transfusion for anemia. The immunomodulatory agents (imids) thalidomide and lenalidomide may induce transfusion independence. This guideline systematically reviews evidence on imids to treat MDS and makes evidence-based recommendations.

Methods: The literature and meeting abstracts were searched for phase 2-3 clinical trials. Data on efficacy, toxicity, and which patients benefit were extracted.

Results: 7019 citations on MDS management were identified. Thirteen publications and 9 meeting abstracts met eligibility criteria.

Conclusions: Lenalidomide is recommended as first line therapy in lower risk del5q MDS. There is insufficient evidence to recommend lenalidomide for treatment of higher risk del5q MDS or AML, or for any risk non-del5q MDS or AML. Combining lenalidomide with other agents is not recommended. Thalidomide is not recommended.