INTRODUCTION

MECOM-rearranged (MECOMr) AML is highly chemorefractory, with CR rates around 30% following conventional induction. Allogeneic HSCT remains the only curative option.1-3 We report a strategy in which MECOMr AML patients proceed directly to allo-HSCT with individualized venetoclax-based conditioning without achieving pre-transplant CR. 

AIM

To evaluate the efficacy and toxicity of direct-to-transplant strategy in MECOMr AML patients. 

METHOD

A single-center retrospective study (2023–2026) with 15 adult MECOMr AML patients, who proceeded directly to allo-HSCT without achieving pre-transplant CR. Patients received individualized venetoclax-based sequential conditioning — De-FLAG-Ida-Ven, DC-ACTIVE, or ARCHIVE-based regimens with alkylator-based myeloablation. Main outcome measures: MLFS at allo-HSCT; best posttransplant response; MRD negativity (flow <0.1%); OS; relapse; day-30/60 mortality; grade 3–4 acute GVHD; grade ≥3 infections; and engraftment kinetics.

CONCLUSIONS

Upfront allo-HSCT with individualized venetoclax-based conditioning, without requiring pre-transplant CR, is feasible in MECOMr AML and yields high post-transplant CR/CRp and MRD negativity rates with low early mortality. Relapses and infectious complications remain the main drivers of mortality. Findings warrant cautious interpretation given the small retrospective cohort.