# Differentiation syndrome

Source: https://onco.cc/terms/differentiation-syndrome/  
OnCo record `differentiation-syndrome` (Term). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

When a targeted drug makes leukaemia cells mature all at once, causing fever, fluid in the lungs, and weight gain. Treatable with steroids if caught early.

## Summary

Differentiation syndrome occurs when a targeted drug makes leukaemia cells mature all at once, producing fever, fluid in the lungs and weight gain; it is treatable with steroids if caught early. It was first described with all-trans retinoic acid in acute promyelocytic leukaemia and is also seen with arsenic trioxide. It is now a boxed-warning class effect of the IDH inhibitors ivosidenib, enasidenib and olutasidenib and of the menin inhibitors revumenib and ziftomenib, affecting a minority of treated patients, and the gilteritinib page also links here. Management is dexamethasone, hydroxyurea for leukocytosis and interruption of the drug. Readers meet it on those drug pages, in the AUGMENT-101 trial, in myelodysplastic syndromes and in the transcriptional addiction pathway.

## Fields

- Kind: Term
- Last checked: 2026-09-07

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Differentiation_syndrome
- Wikipedia: https://en.wikipedia.org/wiki/Differentiation_syndrome

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute promyelocytic leukaemia](https://onco.cc/cancers/apl/), [IDH1- and IDH2-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-idh/), [Myelodysplastic syndromes / neoplasms (MDS)](https://onco.cc/cancers/mds/), [NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia](https://onco.cc/cancers/aml-npm1-kmt2a/)
- targets: [IDH1 / IDH2](https://onco.cc/targets/idh/), [Menin](https://onco.cc/targets/menin/)
- drugs: [Arsenic trioxide](https://onco.cc/drugs/arsenic-trioxide/), [Enasidenib](https://onco.cc/drugs/enasidenib/), [Gilteritinib](https://onco.cc/drugs/gilteritinib/), [Ivosidenib](https://onco.cc/drugs/ivosidenib/), [Olutasidenib](https://onco.cc/drugs/olutasidenib/), [Revumenib](https://onco.cc/drugs/revumenib/), [Tretinoin (all-trans retinoic acid, ATRA)](https://onco.cc/drugs/tretinoin-atra/), [Ziftomenib](https://onco.cc/drugs/ziftomenib/)
- trials: [AUGMENT-101](https://onco.cc/trials/augment-101/)
- people: [Janet Rowley](https://onco.cc/people/janet-rowley/)
- pathways: [Transcriptional machinery & addiction](https://onco.cc/pathways/transcription-addiction/)

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