Acute myeloid leukemia, IDH-mutated

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Note: these are biomarker-specific regimens for patients with IDH1 or IDH2-mutated AML, please see the main AML page for other regimens.

1 regimens on this page
1 variants on this page

Relapsed or refractory, salvage therapy

Enasidenib monotherapy

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Study Evidence ORR
Stein et al. 2017 Phase I/II 40%

This is the dose used in the 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