Acute Myeloid Leukemia (AML)

Simple Summary Acute myeloid leukemia with NPM1 mutation (NPM1-mutated AML) is one of the most frequent and clinically relevant subtypes of acute myeloid leukemia. Over the last two decades, major advances in molecular biology have transformed the understanding of this disease…

ABSTRACT Introduction Menin inhibitors, including the FDA-approved agents revumenib and ziftomenib, have emerged as transformative targeted therapies in acute myeloid leukemia (AML), particularly in KMT2A-rearranged (KMT2Ar) and NPM1-mutated (NPM1m) disease. Despite high initial…

Key Points Automated NGS in Mexico reveals a distinct AML profile with high Fms-like tyrosine kinase 3 mutations and few age-related variants. AML risk assessment improved via genomics but survival was limited by restricted access to targeted therapies and HCT. Abstract…

Key Points Older adults with AML have different expectations compared to their oncologist regarding how their physical function will change. Future research is needed to understand the clinical impact of discordant expectations and how to support communication about functioning…

Abstract MR1-restricted T (MR1T) cells are a subset of unconventional T cells that recognize metabolite-derived antigens presented by the oligomorphic major histocompatibility complex class I–related molecule MR1. MR1T cells express a polyclonal T-cell receptor repertoire, can…

Abstract Post-transplant cyclophosphamide (PTCy) has transformed graft-versus-host disease (GVHD) prophylaxis but its role in matched-donor reduced-intensity allogeneic hematopoietic cell transplantation (allo-HCT) remains uncertain. We retrospectively analyzed adults with AML or…

Key Points Ziftomenib shows pronounced preclinical activity in the menin-KMT2A–dependent leukemia models, including patient-derived xenografts. Ziftomenib demonstrates potent activity against a subset of the drug-induced MEN1 mutations found in patients with acute myeloid…

Key Points MLL3 and MLL4 act redundantly in HSCs to sustain transcription factor and enhancer networks that support multipotency. Simultaneous loss of MLL3 and MLL4 drives hematopoietic progenitors into a uniform B-cell–like default state. Abstract Hematopoietic stem cells (HSCs)…