# Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory acute myeloid leukaemia

Source: https://onco.cc/key-papers/paper-ivosidenib-idh1-dinardo-nejm-2018/  
OnCo record `paper-ivosidenib-idh1-dinardo-nejm-2018` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The oral IDH1 inhibitor ivosidenib put about a third of patients with relapsed or refractory IDH1-mutated acute myeloid leukaemia into remission, with some clearing the mutation altogether, leading to its approval.

## Summary

Phase 1 dose-escalation and expansion study of 258 patients with IDH1-mutated advanced haematological cancers, including 179 with relapsed or refractory AML treated at 500 mg daily.

In the primary efficacy population the rate of complete remission or complete remission with partial haematological recovery was 30.4 percent, median duration of response 8.2 months, and 21 percent of responders had no detectable IDH1 mutation. Differentiation syndrome occurred in about one in ten.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: New England Journal of Medicine
- Year: 2018
- DOI: 10.1056/NEJMoa1716984
- Authors: DiNardo CD, Stein EM, de Botton S, et al.
- Findings: Complete remission or complete remission with partial haematological recovery in 30.4 percent; overall response 41.6 percent.; Median overall survival 8.8 months in relapsed or refractory AML.
- What it means: IDH1 testing at diagnosis and relapse now directs patients to a targeted oral drug; ivosidenib went on to be combined with azacitidine in newly diagnosed disease (AGILE).
- Caveats: Single-arm study; responses were partial for most and relapse common.; Differentiation syndrome and QT prolongation need monitoring.

## Sources

- N Engl J Med 2018: https://doi.org/10.1056/NEJMoa1716984
- PubMed: https://pubmed.ncbi.nlm.nih.gov/29860938/

## Connected records

- cancers: [IDH1- and IDH2-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-idh/)
- drugs: [Ivosidenib](https://onco.cc/drugs/ivosidenib/)
- journals: [New England Journal of Medicine](https://onco.cc/journals/nejm/)

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