PURPOSE OF REVIEW:
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.
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.
This review focuses on recent advances in new strategies and targeted therapies for treatment of myelodysplastic syndrome.