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What is AML?

Defining AML

Acute myeloid leukemia (AML) is a heterogeneous clonal malignancy of the hematopoietic system.1 AML is the most common type of acute leukemia in adults and accounts for more deaths from leukemia annually than any other type of leukemia.2

Epidemiology of AML

The Surveillance, Epidemiology, and End Results Program (SEER) estimates that in 2023, the rate of new cases of AML will be ~4.1 per 100,000 men and women.3


Estimated new cases of AML4


Estimated mortalities of AML4

5-Year relative survival rate decreases with age in AML5

5-year AML relative survival rate graph 5-year AML relative survival rate graph
The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) notes that as the population ages, the incidence of AML along with MDS is rising.6

Role of IDH1 mutations in AML7-9

IDH1 mutations block normal differentiation of myeloblasts in AML

Environment of a myeloblast graphic

The myeloblast’s metabolic environment is abnormal

Overproduction of 2-HG causes DNA and histone hypermethylation

Myeloblast image

Normal myeloblast differentiation is obstructed

Multi myeloblast image

Undifferentiated myeloblasts proliferate uncontrollably

Isocitrate dehydrogenase-1 (IDH1) is a key enzyme involved in cellular metabolism, epigenetic regulation, and DNA repair.10 Mutated IDH1 (mIDH1) catalyzed the production of D-2-hydroxyglutarate (2-HG), which interferes with cellular metabolism and epigenetic regulation and contributes to oncogenesis.11

mIDH1 is a driver mutation in up to 14% of AML cases10,12
mIDH1 AML may be associated with a poor prognosis11
mIDH1 is an actionable target for treatment in AML patients6
Both the NCCN Guidelines® and the ASH-CAP Guidelines recommend performing molecular and cytogenetic analyses to identify IDH1 mutations in patients with AML. Further, the NCCN Guidelines recommends expediting these analyses.6,13
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ASH-CAP, American Society of Hematology-College of American Pathologists.

References: 1. Koenig K, Mims A. Relapsed or primary refractory AML: moving past MEC and FLAG-ida. Curr Opin Hematol. 2020;27(2):108-114. doi:10.1097/MOH.0000000000000561 2. Siegel RL, Miller KD, Jemal A. Cancer statistics, 2020. CA Cancer J Clin. 2020;70(1):7-30. doi:10.3322/caac.21590 3. Cancer stat facts: leukemia—acute myeloid leukemia (AML). National Cancer Institute Surveillance, Epidemiology, and End Results Program. Updated June 8, 2023. Accessed July 19, 2023. 4. Key statistics for acute myeloid leukemia (AML). American Cancer Society. Accessed July 19, 2023. 5. Acute myeloid leukemia (AML): SEER 5-year relative survival rates, 2013-2019. National Cancer Institute Surveillance, Epidemiology, and End Results Program. Updated June 8, 2023. Accessed July 19, 2023. 6. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia V.4.2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed August 14, 2023. To view the most recent and complete version of the guideline, go online to 7. Figueroa ME, Abdel-Wahab O, Lu C, et al. Leukemic IDH1 and IDH2 mutations result in a hypermethylation phenotype, disrupt TET2 function, and impair hematopoietic differentiation. Cancer Cell. 2010;18(6):553-567. doi:10.1016/j.ccr.2010.11.015 8. Birendra KC, DiNardo CD. Evidence for clinical differentiation and differentiation syndrome in patients with acute myeloid leukemia and IDH1 mutations treated with the targeted mutant IDH1 inhibitor, AG-120. Clin Lymphoma Myeloma Leuk. 2016;16(8):460-465. doi:10.1016/j.clml.2016.04.006 9. McMurry H, Fletcher L, Traer E. IDH inhibitors in AML—promise and pitfalls. Curr Hematol Malig Rep. 2021;16(2):207-217. doi:10.1007/s11899-021-00619-3 10. Molenaar RJ, Maciejewski JP, Wilmink JW, van Noorden CJF. Wild-type and mutated IDH1/2 enzymes and therapy responses. Oncogene. 2018;37(15):1949-1960. doi:10.1038/s41388-017-0077-z [Erratum in Oncogene. 2018;37(43):5810] 11. Roboz GJ, DiNardo CD, Stein EM, et al. Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia. Blood. 2020;135(7):463-471. doi:10.1182/blood.2019002140 12. Megías-Vericat JE, Ballesta-López O, Barragán E, Montesinos P. IDH1-mutated relapsed or refractory AML: current challenges and future prospects. Blood Lymphat Cancer. 2019;9:19-32. doi:10.2147/BLCTT.S177913 13. Arber DA, Borowitz MJ, Cessna M, et al. Initial diagnostic workup of acute leukemia: guideline from the College of American Pathologists and the American Society of Hematology. Arch Pathol Lab Med. 2017;141(10):1342-1393. doi:10.5858/arpa.2016-0504-CP