Myelodysplastic syndrome, IDH-mutated

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Section editor
Sanjay mohan.png
Sanjay R. Mohan, MD, MSCI
Vanderbilt University
Nashville, TN, USA

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

1 regimens on this page
1 variants on this page


Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.


IDH1 relapsed or refractory

Ivosidenib monotherapy


FDA-recommended dose
Study Dates of enrollment Evidence Efficacy
DiNardo et al. 2018 (AG120-C-001 r/r) 2014-03-12 to 2017-05-08 Phase 1/2 (RT) ORR: 42%

Biomarker eligibility criteria

  • Alteration: IDH1 mutation

Targeted therapy

28-day cycles


  1. AG120-C-001 r/r: 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 relapsed or refractory AML. N Engl J Med. 2018 Jun 21;378(25):2386-2398. Epub 2018 Jun 2. link to original article contains dosing details in abstract PubMed NCT02074839