Myelodysplastic syndrome (MDS) is a group of myeloid malignancies characterized by ineffective hematopoiesis, inflammation, and immune dysregulation in the setting of multiple genomic disruptions. Disease burdens associated with MDS encompass complications of the disease itself as well as those associated with transformation to acute myeloid leukemia (AML). Patients with high-risk MDS (HR-MDS) face even greater challenges, particularly when treated in community-based settings. This risk stems from the presence of greater numbers of cytogenetic abnormalities, which are unstable and associated with increased transformation to AML. Patients with HR-MDS and AML may not be candidates for allogeneic stem hematopoietic cell transplantation due to age or comorbidities. Hypomethylating agents (HMAs) remain as the standard of care for HR-MDS; however, responses to HMA do not exceed 50% and are short-lived. The efficacy of HMAs in some patients with MDS and their long-standing role in therapy have led to the development of novel treatment approaches that make use of HMA combination therapies with immune-based therapies, such as those that target the TIM-3 pathway.
This educational activity will focus on assessing MDS risk and its relation to treatment choices, the rationale and mechanism of action for novel immune-based therapies and integrating emerging combination therapies into clinical practice.