MECOM is a master repressor of myeloid differentiation through dose control of <i>CEBPA</i> in acute myeloid leukemia

D Dorien Pastoors (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) M Marije Havermans R Roger Mulet-Lazaro (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) L Leonie Smeenk (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) S Sophie Ottema (3Department of Clinical Genetics, University Medical Center Rotterdam, Rotterdam, The Netherlands) C Claudia Erpelinck-Verschueren (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) S Stanley van Herk (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) M Maikel Anthonissen (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) T Tim Grob (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) S Shruthi Subramanian (4School of Clinical Medicine, University of New South Wales, Sydney, Australia) J Julie A. I. Thoms J John E. Pimanda B Bas J. Wouters (1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands) B Berna Beverloo (3Department of Clinical Genetics, University Medical Center Rotterdam, Rotterdam, The Netherlands) T Torsten Haferlach (7Munich Leukemia Laboratory, Munich, Germany) C Claudia Haferlach (8Munich Leukemia Laboratory, Munich, Germany) J Johannes Zuber E Eric Bindels (Department of Hematology, Erasmus MC University Medical Center) R Ruud Delwel

Abstract

Abstract The transcription factor MECOM, located at 3q26, is essential for hematopoietic stem cells in healthy individuals. Enhancer translocations, due to 3q26 rearrangements, drive out-of-context MECOM expression in one of the most aggressive subtypes of acute myeloid leukemia (AML). Aberrantly expressed MECOM is essential for the survival and immature phenotype of these leukemia cells. Direct depletion of MECOM using an endogenous auxin-inducible degron immediately upregulates expression of CEBPA, which encodes a transcription factor required for neutrophil development and is frequently mutated in other AML subtypes. MECOM depletion is accompanied by a severe loss of CD34 and gain of mature myeloid cell surface marker CD15. MECOM exerts its inhibitory effect on differentiation by binding to the +42-kilobase CEBPA enhancer. This is partially dependent on the interaction between MECOM and its corepressor CTBP2. We demonstrate that CEBPA overexpression can bypass the MECOM-mediated block of differentiation. In addition, patients with AML with MECOM overexpression through enhancer hijacking show significantly reduced CEBPA levels. Our study directly connects 2 major players in normal and malignant hematopoiesis, MECOM and CEBPA, and unveils how MECOM maintains self-renewal by repressing CEBPA-induced differentiation.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 25
Published December 18, 2025
Pages 3098-3105
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (19)

D

Dorien Pastoors

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

M

Marije Havermans

R

Roger Mulet-Lazaro

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

L

Leonie Smeenk

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

S

Sophie Ottema

3Department of Clinical Genetics, University Medical Center Rotterdam, Rotterdam, The Netherlands

C

Claudia Erpelinck-Verschueren

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

S

Stanley van Herk

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

M

Maikel Anthonissen

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

T

Tim Grob

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

S

Shruthi Subramanian

4School of Clinical Medicine, University of New South Wales, Sydney, Australia

J

Julie A. I. Thoms

J

John E. Pimanda

B

Bas J. Wouters

1Department of Hematology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands

B

Berna Beverloo

3Department of Clinical Genetics, University Medical Center Rotterdam, Rotterdam, The Netherlands

T

Torsten Haferlach

7Munich Leukemia Laboratory, Munich, Germany

C

Claudia Haferlach

8Munich Leukemia Laboratory, Munich, Germany

J

Johannes Zuber

E

Eric Bindels

Department of Hematology, Erasmus MC University Medical Center

R

Ruud Delwel