Jessica Altman, MD

Panelists emphasize that effective use of menin inhibitors for KMT2A-rearranged acute myeloid leukemia (AML) requires dispelling common misconceptions about oral targeted therapy, ensuring close monitoring for adverse effects, clarifying treatment goals—especially the role of transplant—and supporting patients through comprehensive education and multidisciplinary care.

Panelists highlight that the emergence of menin inhibitors is reshaping the treatment paradigm for KMT2A-rearranged acute myeloid leukemia (AML), with ongoing research focused on optimizing their use, overcoming resistance, expanding access, and integrating these targeted therapies into personalized and potentially curative treatment strategies across diverse patient populations.

Panelists emphasize that the approval of menin inhibitors has transformed the treatment landscape for KMT2A-rearranged acute myeloid leukemia (AML) by replacing historically limited and toxic chemotherapy-based strategies with a targeted, guideline-endorsed option that aligns with disease biology—offering renewed hope for both adult and pediatric patients, especially in the relapsed/refractory setting.

Panelists report that the AUGMENT-101 trial demonstrated meaningful clinical benefits of menin inhibitors in relapsed/refractory KMT2A-rearranged acute myeloid leukemia (AML)—with a 23% complete remission rate and manageable safety profile including differentiation syndrome—highlighting the importance of early recognition, patient education, and supportive care to optimize outcomes as these agents move toward becoming a new standard of care.

Panelists highlight that menin inhibitors represent a breakthrough in the treatment of KMT2A-rearranged AML by targeting the disease’s core epigenetic drivers, with early clinical trials like AUGMENT-101 showing promising results in heavily pretreated, high-risk patients and supporting the potential for a new therapeutic standard in this aggressive leukemia subtype.

Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is a biologically aggressive and diagnostically complex leukemia subtype—especially prevalent in pediatric and therapy-related cases—with variable prognostic implications depending on fusion partners, underscoring the need for early molecular identification, personalized treatment strategies, and expanded access to targeted therapies and advanced diagnostics.

Panelists emphasize that patients with KMT2A-rearranged acute myeloid leukemia (AML) often face steep educational challenges at diagnosis, requiring clear communication about the subtype’s adverse-risk classification, biological complexity, and treatment implications—highlighting the need for accessible education, academic-community collaboration, and early referral to specialized care for optimal outcomes.

Panelists explain that KMT2A-rearranged acute myeloid leukemia (AML) is an aggressive, molecularly distinct leukemia subtype marked by menin-dependent transcriptional dysregulation, poor response to standard therapy, diagnostic challenges, and high relapse risk—underscoring the urgency of RNA-based diagnostics, early transplant consideration, and emerging menin-targeted treatments to improve outcomes.