急性髓系白血病的新型治疗策略

Acute myeloid leukemia (AML) is a molecularly diverse hematologic malignancy, with clinical outcomes driven by patient fitness, disease biology, and the depth of remission. Over the past decade, the increasing use of molecular studies has enabled genotype-directed therapy and accelerated the incorporation of targeted agents into both intensive and lower-intensity treatment regimens. FLT3 inhibitors are now well established across a wide disease spectrum, including frontline settings, relapsed/refractory disease, and post-allogeneic hematopoietic cell transplantation maintenance, with emerging evidence supporting measurable residual disease (MRD)-adapted maintenance approaches. IDH1/2 inhibitors offer therapy for specific molecular subsets, particularly in older or unfit patients, and are increasingly being evaluated in doublet and triplet combinations.

Venetoclax-based regimens have become a cornerstone for older or unfit patients with AML and are being optimized through response-adapted dosing and careful partner selection to mitigate cytopenias while deepening remission. Menin inhibitors represent a major therapeutic advance for KMT2A-rearranged and NPM1-mutated AML and are rapidly moving into combination and earlier-line treatment strategies.

In parallel, immune and cellular therapies, including antibody-drug conjugates, immune checkpoint modulation, bispecific engagers, and chimeric antigen receptor T-cell and chimeric antigen receptor natural killer constructs, are under investigation, although durable, practice-changing benefits outside select settings remain limited to date.

This review synthesizes pivotal clinical data and translational principles informing emerging targeted, immune-based, and cellular strategies in AML, with an emphasis on remission depth, MRD-driven decision-making, and relapse prevention.