Treating Myelodysplastic Syndromes (MDS)

Treatment goals for MDS should follow a risk-adapted approach based on the risk category according to the Revised International Prognostic Scoring System (IPSS-R).1 IPSS-R improves the ability to predict survival and progression in MDS based on the identification of independent predictive variables, such as2:

  • Percentage of bone marrow (BM) blasts
  • Cytogenetic parameters (karyotype)
  • Number of cytopenias

Goals of therapy for MDS according to IPSS-R scores

Very Low, Low, and Intermediate Risk High and Very High Risk
Transfusion independence or decrease transfusion needs3 Prolong survival3
Decrease transformation to higher-risk disease or AML3 Slow disease course3
Improve survival4 Improve quality of life4

AML, acute myeloid leukemia.

Depending on patient prognosis, cytogenetics, and preferences, 2 distinct types of treatment may be selected: active therapy (may be used to alter disease progression) or supportive care (given to reduce disease symptoms).1,5

Active Therapy1 Supportive Care5

Hematopoietic stem cell transplant (HSCT)

  • Should be considered for patients who are candidates, have been exposed to multiple therapies (growth factors, immunomodulatory agents, azanucleosides, etc), and for younger patients with severe hypoplastic MDS. HSCT is currently the only curative option for MDS1

Hypomethylating agents (HMAs)

  • Approved options for all types of MDS, helps with improving hematologic parameters1


  • Immunomodulatory agents may be an option for patients with a del(5q) mutation1
  • Immunosuppressive therapy improves immune regulation in patients with MDS1


  • Used in AML, chemotherapy may also be considered in some patients with MDS with excess blasts5
NCCN Guidelines® recommend that all symptomatic patients receive appropriate supportive care.6

Growth factors

  • Growth factors exist to treat anemia, thrombocytopenia, and neutropenia; erythropoiesis-stimulating agents (ESAs) are specifically used to treat anemia and they are sometimes used in combination with granulocyte colony-stimulating factors (G-CSF)1,5

Blood transfusions

  • May also be used to address the symptoms of anemia5
  • Can be used as supportive care for bleeding events6


  • May be given to treat infection5

NCCN Guidelines® recommend that clinical trial enrollment should be considered for all patients.6

Aligning treatment considerations with treatment options

Treatment decisions in MDS should include patient-related characteristics, disease-related characteristics, and currently available treatment options.6-8

According to NCCN Guidelines®:

  • Treatment options for high-risk patients include allogeneic stem cell transplant, chemotherapy, clinical trial, and supportive care6
  • Risk stratification should be supplemented by molecular testing9-11
  • Treatment options for lower-risk patients include allogeneic stem cell transplant, DNA hypomethylators, immunosuppressive therapy, immunomodulatory agents, biologic response modifiers, clinical trial, and supportive care6

References: 1. Montalban-Bravo G, Garcia-Manero G. Myelodysplastic syndromes: 2018 update on diagnosis, risk stratification and management. Am J Hematol. 2018;93(1):129-147. 2. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079-2088. 3. Komrokji RS, Corrales-Yepez M, Al Ali N, et al. Validation of the MD Anderson Prognostic Risk Model for patients with myelodysplastic syndrome. Cancer. 2012;118(10):2659-2664. 4. Cheson BD, Greenberg PL, Bennett JM, et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006;108(2):419-425. 5. PDQ Adult Treatment Editorial Board. Myelodysplastic Syndromes Treatment (PDQM®): Patient Version. 2019 Mar 28. In: PDQ Cancer Information Summaries [Internet]. Bethesda, MD: National Cancer Institute (US); 2002-. Available from: Accessed August 29, 2019. 6. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myelodysplastic Syndromes V.1.2020. © National Comprehensive Cancer Network, Inc. 2019. All rights reserved. Accessed August 28, 2019. To view the most recent and complete version of the guideline, go online to NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 7. Kurtin SE, Demakos EP. An update on the treatment of myelodysplastic syndromes. Clin J Oncol Nurs. 2010;14(3):E29-44. 8. Kurtin SE. Current approaches to the diagnosis and management of myelodysplastic syndromes. J Adv Pract Oncol. 2011;2(suppl 2):7-18. 9. Steensma DP. Myelodysplastic syndromes current treatment algorithm 2018. Blood Cancer J. 2018;8(5):47. 10. Gangat N, Patnaik MM, Tefferi A. Myelodysplastic syndromes: contemporary review and how we treat. Am J Hematol. 2016;91(1):76-89. 11. Santini V. First-line therapeutic strategies for myelodysplastic syndromes. Clin Lymphoma Myeloma Leuk. 2017;17S:S31-S36.