Novel approaches for myelodysplastic syndromes: beyond hypomethylating agents.
OBJECTIVE
Several novel therapeutic approaches exist for treatment of patients with myelodysplastic syndrome, with goals to improve quality of life and prolong survival. This review highlights new therapies from the last 18 months.


RESULTS
Immunosuppressants, erythropoiesis-stimulating agents in combination with granulocyte colony-stimulating factor or all-trans-retinoic-acid have shown improvement in decreasing the need for transfusions and improving quality-of-life and/or survival. Eltrombopag has shown promising results in the treatment of thrombocytopenia. However, determination of an optimal chemotherapeutic approach remains elusive and controversial. DNA methyltransferase inhibitors are well tolerated in outpatient settings, with azacitidine prolonging survival and decreasing time to acute myeloid leukemia progression in patients with high-risk myelodysplastic syndromes. A novel erythroid-specific gene expression profile may predict response to lenalidomide in patients who lack the deletion of 5q31.1. Tools such as the Charlson comorbidity index may help select appropriate patients for allogeneic stem cell transplant. A variety of promising new agents are under investigation.


CONCLUSIONS
This review focuses on recent advances in new strategies and targeted therapies for treatment of myelodysplastic syndrome.
Loaiza-Bonilla
Arturo
A
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, Baltimore, Maryland, USA.
Gore
Steven D
SD
Carraway
Hetty E
HE
D000970:Antineoplastic Agents / Q000627:therapeutic use; D001565:Benzoates / Q000627:therapeutic use*; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D000077269:Lenalidomide; D008780:Methyltransferases / Q000037:antagonists & inhibitors; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000503:physiopathology; D011720:Pyrazoles / Q000627:therapeutic use*; D011788:Quality of Life; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D001565:Benzoates; D004791:Enzyme Inhibitors; D006834:Hydrazines; D011720:Pyrazoles; D013792:Thalidomide; D008780:Methyltransferases; D000077269:Lenalidomide; C520809:eltrombopag