Options for HR-MDS and AML are limited

Additional approaches are needed to improve options and patient outcomes1-3:


  • Stem cell transplant is the only potential cure, however it is high risk and not an option for most patients4
  • Challenges facing current standard-of-care therapies include poor overall survival (OS), limited duration of response, and singular mechanistic action5-8
    • Nontransplant options only prolong survival by months and the risk of death remains high6,8
    • Responses achieved with currently available treatment are often <1 year duration6,7
    • These therapies focus on either decreasing leukemic proliferation or limiting immune activation, but do not do both5,9-11
  • Many patients require blood transfusions for cytopenias and are at risk for hospitalization12

Patient characteristics and management options of HR-MDS and AML are often similar7,13-15

Clinical treatment goals and patient characteristics of HR-MDS and AML






Significant treatment innovations are lacking1,2

The risk of death and transformation to AML remain high,
despite treatment with hypomethylating agents (HMAs)2,4



Most innovations have been limited to specific driver mutations1-3

Despite recent advances, overall survival is <1 year13-15

Learn more about the role of TIM-3

*For AML, lower-intensity regimens for unfit patients can include low-dose cytarabine. For HR-MDS, lower-intensity regimens are based on hypomethylating agents.

AML, acute myeloid leukemia; HR-MDS, high-risk myelodysplastic syndrome; TIM-3, T cell immunoglobulin and mucin domain-3.


References: 1. Dombret H, Gardin C. An update of current treatments for adult acute myeloid leukemia. Blood. 2016;127(1):53-61. 2. Faber MG, Griffiths EA, Thota, S. Current state of myelodysplastic syndromes: standard treatment practices and therapeutic advances. J Clin Pathways. 2019;5(6):43-47. 3. Short NJ, Kantarjian H, Ravandi F, Daver N. Emerging treatment paradigms with FLT3 inhibitors in acute myeloid leukemia. Ther Adv Hematol. 2019; doi:10.1177/2040620719827310. 4. Gyurkocza B, Deeg HJ. Allogeneic hematopoietic cell transplantation for MDS: for whom, when and how? Blood Rev. 2012;26(6):247-254. 5. Kadia TM, Jabbour E, Kantarjian H. Failure of hypomethylating agent–based therapy in myelodysplastic syndromes. Semin Oncol. 2011;38(5):682-692. 6. Dinardo C, Pratz K,
Pullarkat V, et al. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019;133(1):7-17. 7. Platzbecker U. Treatment of MDS.  Blood. 2019;133(10):1096-1107. 8. Fenaux P, Mufti G, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009;10(3):223-232. 9. Hollenbach P, Nguyen A, Brady H, et al. A comparison of azacitidine and decitabine activities in acute myeloid leukemia cell lines. PLoS ONE. 2010;5(2):e9001. 10. Kordasti SY, Ingram W, Hayden J, et al. CD4+CD25high Foxp3+ regulatory T cells in myelodysplastic syndrome (MDS). Blood. 2007;110(3):847-850. 11. Li C, Chen X, Yu X, et al. Tim-3 is highly expressed in T cells in acute myeloid leukemia and associated with clinicopathological prognostic stratification. Int J Clin Exp Pathol. 2014;7(10):6880-6888. 12. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454-2465. 13. Dombret H, Seymour JF, Butrym A, et al. International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts. Blood. 2015;126(3):291-299. 14. Kantarjian HM, Thomas XG, Dmoszynska A, et al. Multicenter, randomized, open-label, phase III trial of decitabine versus patient choice, with physician advice, of either supportive care or low-dose cytarabine for the treatment of older patients with newly diagnosed acute myeloid leukemia. J Clin Oncol. 2012;30(21):2670-2677. 15. Döhner H, Estey E, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424-447.

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