Thrombopoietin increases susceptibility for EVI1 + KMT2A-MLLT3-driven AML expressing stem cell genes linked to poor outcome.

Châtel-Soulet, Hugues-Étienne; Juge, Sabine; Pereira, Ana Luisa; Seguin, Jonathan; El Taher, Athimed; Valigi, Federica; Jevtic, Zivojin; Sivalingam, Rathick et al. · Nat Commun · 2025

basic_science · Level V

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Abstract

To address the cellular origin of ecotropic virus integration site 1 (EVI1)-expressing aggressive KMT2A-rearranged acute myeloid leukaemia (AML) we integrate an Evi1-GFP reporter allele in the inducible iKMT2A-MLLT3 mouse model. We observe that a single injection of thrombopoietin (TPO) selectively increases the number of cycling Evi1<sup>+</sup> haematopoietic stem cells (HSC) and accelerates AML initiation. Comparison of mouse Evi1<sup>+</sup> KMT2-MLLT3<sup>+</sup> AML originating from TPO-stimulated HSC with human EVI1<sup>+</sup>AML reveals higher expression of HSC genes including IL12Rβ2 and INPP4B linked to poor disease outcome of patients of four large AML cohorts. Knockdown experiments show exclusive MECOM-dependency of human EVI1<sup>high</sup> KMT2A-rearranged OCI-AML4 cells while reduction of IL12Rβ2 also impairs clonogenic growth of EVI1<sup>low</sup> MOLM-13, THP-1 or HL-60 AML cells. Collectively, we show that exogenous factors like TPO can increase the susceptibility for iKMT2A-MLLT3-driven HSC-originating Evi1<sup>+</sup> AML expressing stem cell genes linked to transformation maintenance of cell lines, and poor disease outcome of patients.

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