Shifts in Standard of Care for Adults and Children With KMT2A AML

EP: 1.KMT2A AML Pathogenesis and Presentation

EP: 2.Prognosis and Perception of KMT2A AML

EP: 3.Patient Profiles: Demographics and Disease Biology

EP: 4.Targeting Menin: A New Frontier in AML

EP: 5.Efficacy, Safety, and Experience With Revumenib

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EP: 6.Shifts in Standard of Care for Adults and Children With KMT2A AML

The AUGMENT-101 trial provided important insights into the efficacy and safety of a menin inhibitor in patients with relapsed or refractory KMT2A-rearranged AML, a group largely composed of younger patients with a median age of 35 years. The updated data from 2024 included 116 patients in the safety population and 97 evaluable for efficacy. Results showed a combined complete remission rate of approximately 23%, with an overall response rate including morphologic leukemia-free survival reaching about two-thirds. While the duration of response averaged just over 6 months, this represents a meaningful clinical benefit in a heavily pretreated population where durable responses are historically rare. Safety data revealed that more than half of patients experienced grade 3 or higher treatment-related adverse events, but these were consistent with expectations for this challenging patient population.

A key safety concern in this study was differentiation syndrome, a known class effect of menin inhibitors. About 15% of patients experienced grade 3 or higher differentiation syndrome, but importantly, no deaths or treatment discontinuations due to this condition were reported. Differentiation syndrome manifests with symptoms such as fever, weight gain, shortness of breath, and fluid accumulation, and can sometimes be confused with infection, which is a common concern in immunocompromised patients. Effective management relies on early recognition and prompt intervention with corticosteroids, alongside supportive care including antimicrobials and sometimes hydroxyurea to manage white blood cell counts. The evolving experience with differentiation syndrome highlights the importance of educating both health care providers and patients on its signs to ensure timely treatment.

This evolving understanding of differentiation syndrome underscores the critical need for awareness and communication, especially for patients who may be managed outside specialized centers. Educational tools like wallet cards and individualized care plans are becoming valuable resources to empower patients and emergency providers unfamiliar with AML-specific complications. These initiatives aim to bridge the gap between clinical expertise and community care, promoting rapid recognition and treatment to improve patient outcomes. Overall, the trial results and safety management strategies demonstrate the promise of menin inhibitors as a new standard of care, while emphasizing the necessity for continued education and support in clinical practice.