Advancing Care in AML: Exploring Novel Treatment Options

Panelists discuss how the pathophysiology of acute myeloid leukemia has evolved from a single phenotype understanding to recognizing diverse genetic events that lead to transformation at the hematopoietic stem cell level, with treatment decisions now increasingly integrated with specific genetic mutations despite the disease’s rapid progression timeline.

Panelists discuss how current risk stratification systems, particularly the European Leukemia Net 2022 classification for intensive therapy and the 2024 classification for lower intensity treatments, categorize patients based on complex cytogenetics and molecular features to guide prognosis and treatment selection.

Panelists discuss how fitness assessment has evolved beyond traditional age cutoffs to incorporate comprehensive geriatric assessments, frailty measures, and individualized evaluations, while questioning whether intensive therapy should automatically be given to fit patients given newer effective treatment options.

Panelists discuss how the greatest unmet needs include addressing poor outcomes in specific subsets like TP53-mutant disease, managing the increasingly older population with secondary mutations, and ensuring global access to targeted therapies and next-generation sequencing.

Panelists discuss how social determinants of health significantly impact AML care, particularly regarding transportation access, health literacy, and the intensive nature of treatment requiring frequent clinic visits for blood work and transfusions, which disproportionately affects patients living far from treatment centers.

Panelists discuss how current NCCN guidelines emphasize the importance of treating AML at experienced centers with proper infrastructure, while treatment decisions are based on intensive vs nonintensive therapy eligibility and specific genetic mutations like FLT3, IDH1, and TP53.

Panelists discuss how transplant eligibility has expanded beyond traditional intensive chemotherapy candidates to include patients receiving lower intensity regimens, with earlier transplant consultations and consideration of organ function preservation through less toxic induction approaches.

Panelists discuss how venetoclax has revolutionized AML treatment by providing effective therapy options for older patients with previously untreatable disease, enabling higher response rates, longer survival, and increased transplant eligibility while transforming the treatment landscape across all age groups.

Panelists discuss how combination trials like VIALE-A and VIALE-C have demonstrated venetoclax’s survival benefits when added to hypomethylating agents, opening doors for numerous combination studies while emphasizing the need for randomized trials to prove clinical benefit in different disease contexts.

Panelists discuss how the treatment landscape has evolved from limited options to include hypomethylating agents and venetoclax combinations, with emerging oral formulations promising greater accessibility while requiring careful consideration of patient selection and toxicity management.

Panelists discuss how IDH inhibitors, particularly ivosidenib combined with azacitidine, represent potentially the greatest advance in AML treatment due to significant overall survival improvements, though adoption challenges include waiting for mutation results and limited patient applicability.

Panelists discuss how venetoclax management requires standardized approaches to duration, bone marrow biopsy timing, growth factor use, and azole antifungal selection, with practices varying significantly between centers and the need for consistent protocols to optimize patient outcomes.

Panelists discuss how emerging therapies like menin inhibitors show promise based on strong preclinical data and clinical responses, while CD47 inhibitors have faced setbacks in phase 3 trials, with the greatest potential for novel agents likely in frontline combination settings.

Panelists discuss how oral azacitidine plus venetoclax represents a transformative advance toward completely oral regimens that could dramatically improve quality of life and accessibility, though challenges remain regarding cost, insurance coverage, and the complexity of managing multiple oral agents.