Myelodysplastic Syndromes

Pathophysiology

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 Furthermore, ineffective erythropoiesis characterized by abnormal erythroid differentiation and defects in erythroid progenitor growth contributes to the pathophysiology of MDS.7

Landscape

Due to heterogeneous outcomes among patients with MDS based on cytogenetics and other factors, research is focused on risk stratification and the development of tailored treatment approaches, including individualized optimization of doses and schedules.8-10 Investigational therapies include novel hypomethylating agents, small-molecule inhibitors, anti-apoptotic protein inhibitors, immunotherapeutic strategies, and agents that could potentially address ineffective erythropoiesis.9,10

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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.

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Clinical trial information is sourced from ClinicalTrials.gov. Information is updated manually as clinical trials are published. The efficacy and safety 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.

References

  1. Cazzola M, Malcovati L. N Engl J Med. 2005;352:536-538. PMID: 15703418
  2. Heaney ML, Golde DW. N Engl J Med. 1999;340:1649-1660. PMID: 10341278
  3. Schanz J, et al. J Clin Oncol. 2012;30:820-829. PMID: 22331955
  4. Haferlach T, et al. Leukemia. 2014;28:241-247. PMID: 24220272
  5. Figueroa ME, et al. Blood. 2009;114:3448-3458. PMID: 19652201
  6. Bulycheva E, et al. Leukemia. 2015;29:259-268. PMID: 25394715
  7. Fontenay-Roupie M, et al. Br J Haematol. 1999;106:464-473 PMID: 10460607
  8. Steensma DP. Blood Cancer J. 2018;8:47. PMID: 29795386
  9. Brunner AM, Steensma DP. Clin Adv Hematol Oncol. 2018;16:56-66. PMID: 29741506
  10. Tobiasson M, Kittang AO. J Intern Med. 2019 Mar 14. [Epub ahead of print]. PMID: 30869816