Acute myeloid leukemia, IDH-mutated

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Martin Schoen, MD, MPH
St. Louis, MO

Note: these are biomarker-specific regimens for patients with IDH-mutated AML, please see the main AML page for other regimens.

1 regimens on this page
1 variants on this page

IDH2 Relapsed or refractory, salvage therapy

Enasidenib monotherapy

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FDA-recommended dose
Study Evidence Efficacy
Stein et al. 2017 Phase I/II ORR: 40%

This is the dose used in the phase II expansion cohort; enrolled patients were required to have IDH2-mutated advanced myeloid malignancies.


28-day cycles


  1. Stein EM, DiNardo CD, Pollyea DA, Fathi AT, Roboz GJ, Altman JK, Stone RM, DeAngelo DJ, Levine RL, Flinn IW, Kantarjian HM, Collins R, Patel MR, Frankel AE, Stein A, Sekeres MA, Swords RT, Medeiros BC, Willekens C, Vyas P, Tosolini A, Xu Q, Knight RD, Yen KE, Agresta S, de Botton S, Tallman MS. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017 Aug 10;130(6):722-731. Epub 2017 Jun 6. link to original article contains verified protocol link to PMC article PubMed

Investigational agents

These are drugs under study with at least some promising results for this disease.


  1. DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, Swords R, Collins RH, Mannis GN, Pollyea DA, Donnellan W, Fathi AT, Pigneux A, Erba HP, Prince GT, Stein AS, Uy GL, Foran JM, Traer E, Stuart RK, Arellano ML, Slack JL, Sekeres MA, Willekens C, Choe S, Wang H, Zhang V, Yen KE, Kapsalis SM, Yang H, Dai D, Fan B, Goldwasser M, Liu H, Agresta S, Wu B, Attar EC, Tallman MS, Stone RM, Kantarjian HM. Durable Remissions with Ivosidenib in IDH1-Mutated Relapsd or Refractory AML. N Engl J Med. Epub 2018 Jun 2. link to original article contains verified protocol