Core-Binding Factor Acute Myeloid Leukemia: Risk-Adapted Strategies from Biology to Real-World Practice

Core-binding factor acute myeloid leukemia (CBF-AML), defined by t(8;21)(q22;q22) or inv(16)/t(16;16), has long been categorized as “favorable-risk,” yet real-world data show substantial heterogeneity in outcomes. Recent clinical and translational studies, including cooperative efforts from Italian centers, have highlighted the prognostic impact of co-mutations (such as KIT and FLT3), the depth and kinetics of measurable residual disease (MRD), and the role of intensive chemotherapy in both younger and selected older patients. Phase II trials exploring the addition of targeted agents (for example, multikinase inhibitors like midostaurin) to high-dose cytarabine-based backbones, as well as long-term follow-up analyses, are redefining the therapeutic landscape. This Research Topic aims to integrate biological insights, MRD-driven management, and real-world experience to refine treatment strategies and improve long-term survival in CBF-AML.This Research Topic aims to address the unresolved clinical and biological challenges in CBF-AML by leveraging both prospective trials and robust real-world evidence. Although cure rates are higher than in other AML subtypes, relapse, treatment-related toxicity, and suboptimal management of high‑risk molecular subsets remain major concerns. Building on recent work from the Guest Editors and collaborators—on intensive chemotherapy in older patients, KIT/FLT3-mutated CBF-AML, midostaurin-containing regimens, and long-term outcome analyses—we seek contributions that clarify how genomic and MRD information should drive risk-adapted strategies.Key questions include: Which patients benefit most from intensive cytarabine-based approaches, and up to what age? How should co-mutations and MRD dynamics influence the use of targeted agents, maintenance strategies, or allogeneic transplantation? What is the real impact of current practices outside clinical trials? We particularly welcome studies that connect disease biology with pragmatic treatment algorithms, including management of relapse, late toxicities, and survivorship. By integrating translational data and large-scale clinical experience, this Topic aims to support a truly personalized and evidence-based approach to CBF-AML.We welcome Original Research, Review, Mini Review, and Perspective articles focusing on CBF acute myeloid leukemia, including—but not limited to—the following themes:• Biology, mutational landscape, and clonal evolution in CBF-AML• Prognostic impact of KIT, FLT3, and other co-mutations• Measurable residual disease (MRD) techniques, thresholds, and decision-making• Optimization of intensive chemotherapy (including high-dose cytarabine) in younger and older adults• Integration of targeted and epigenetic agents (e.g., multikinase inhibitors, maintenance strategies)• Role and timing of allogeneic stem cell transplantation and transplant-free approaches• Real-world outcomes, including multicenter series, toxicity profiles, and quality of life• Management of relapse, therapy-related CBF-AML, and special clinical scenariosPlease note: Manuscripts consisting solely of bioinformatics or computational analysis of public databases, without validation (independent cohort or biological validation in vitro or in vivo), are out of scope for this section and will not be accepted as part of this Research Topic. Topic editors: Roberto Cairoli, Michele Gottardi Source: https://www.frontiersin.org/research-topics/74428/core-binding-factor-acute-myeloid-leukemia-risk-adapted-strategies-from-biology-to-real-world-practice