Advancing the Management of CH, MDS, and AML From the First Bridging the Gaps in Leukemia, Lymphoma, and Multiple Myeloma Conference.

Abstract

Purpose: The management of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) have evolved substantially in recent years with the development of targeted therapies and novel nontargeted approaches. However, many questions remain about how to best use current therapies, and there is a large unmet need for effective therapies, particularly for patients with higher-risk MDS, AML, and those with MDS/AML relapsed/refractory (R/R) to prior therapy.

Methods and results: A panel of experts was assembled to discuss current controversies and unanswered questions in the care of patients with MDS and AML. Workshop topics included: molecular testing and new classification systems, clonal hematopoiesis, treatment of MDS (lower-risk and higher-risk), frontline treatment of AML, treatment of special populations, treatment of R/R AML, and novel approaches.

Conclusions: We identified many areas of ongoing controversy in the diagnosis and management of MDS and AML related to classification and risk assessment, treatment selection, sequencing of therapies, and monitoring of responses. Many clinical trials are ongoing to further improve outcomes for patients with MDS and AML, and we noted potential areas of debate related to study design, selection of endpoints, and assessment of responses. The controversies and gaps in knowledge identified by this panel will inform a follow-up conference in 2025 that will employ a modified Delphi method with a goal of developing and publishing formal consensus recommendations that can provide actionable guidance to clinicians in practice.