Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid malignancies characterized by multilineage cytopenias and risk of progression to acute myeloid leukemia.1,2 Patients with MDS often have recurring cytogenetic abnormalities3 and somatic mutations4 and exhibit widespread aberrant DNA hypermethylation.5 In addition to the accumulation of genetic and epigenetic abnormalities in myeloid progenitor cells, MDS disease pathogenesis is also driven by defects in the bone marrow microenvironment that further contribute to dysregulation of hematopoiesis.6

The safety and efficacy of the agents and/or uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated


  1. Cazzola M, Malcovati L. N Engl J Med. 2005;352:536-538.
  2. Heaney ML, Golde DW. N Engl J Med. 1999;340:1649-1660.
  3. Schanz J, et al. J Clin Oncol. 2012;30:820-829.
  4. Haferlach T, et al. Leukemia. 2014;28:241-247.
  5. Figueroa ME, et al. Blood. 2009;114:3448-3458.
  6. Bulycheva E, et al. Leukemia. 2015;29:259-268.