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Unmet Needs and Emerging Trends in AML: ASCO 2025 Insights
A panelist discusses how the highest unmet needs in acute myeloid leukemia include treatments for patients with refractory and relapsed disease, addressing poor outcomes in patients with TP53 mutations and MECOM rearrangements, while emerging trends focus on combination therapies (doublets, triplets, quadruplets), the shift toward more convenient oral therapies, increased emphasis on minimal residual disease negativity as an end point, and expanded transplant eligibility for older patients aged into their mid to late 70s.
The most significant unmet needs in acute myeloid leukemia (AML) center on patients with refractory and relapsed disease, who continue to experience poor outcomes despite available salvage therapies. While these treatments can produce initial responses, they fail to achieve high cure rates except through bone marrow transplantation, which remains inaccessible to many patients due to age or comorbidities. Particularly challenging are specific patient subgroups, including those with TP53 mutations and MECOM rearrangements, who demonstrate persistently poor prognosis despite the numerous drug approvals and therapeutic advances in AML over the past decade.
Current treatment trends in AML management reflect the expanding arsenal of available drugs and investigational agents. The primary trend involves combination therapies using doublets, triplets, and potentially quadruplets in both intensive and nonintensive chemotherapy settings. Standard of care combinations now include azacitidine-venetoclax for older unfit patients and intensive chemotherapy with FLT3 inhibitors for fit patients with FLT3 mutations. Building on these foundations, researchers are exploring additional combinations with azacitidine triplets and augmenting traditional 7+3 chemotherapy with agents like venetoclax, menin inhibitors, and immunotherapies to improve response rates, deepen responses, increase minimal residual disease (MRD) negativity, and ultimately enhance survival outcomes.
The evolution toward oral therapies represents another significant trend, offering greater patient convenience while reducing infusion clinic burden. Studies have demonstrated promising results with oral combination therapies, such as oral decitabine with venetoclax for older unfit patients, and combinations of IDH or FLT3 inhibitors with oral hypomethylating agents. Additionally, there is increasing emphasis on achieving MRD negativity as a treatment end point, particularly for patients receiving intensive chemotherapy or azacitidine-based regimens. The expanding accessibility of bone marrow transplantation, now feasible for patients aged into their mid to late 70s due to improved donor matching and posttransplant protocols, further enhances treatment options across diverse patient populations.
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