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

 

 

 

 

HR-MDS:

Significant treatment innovations are lacking1,2

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

 

AML:

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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