What constitutes meaningful improvement in myelodysplastic syndromes?

Myelodysplastic syndromes (MDS) are a heterogeneous spectrum of clonal stem cell disorders characterized by ineffective hematopoiesis and resulting peripheral blood cytopenias. Clinical manifestations of the disorder are varied and range from mild asymptomatic anemia to a markedly truncated life expectancy, underscoring the importance of risk-adapted therapy. MDS therapy for both lower- and higher-risk patients is an area of immense therapeutic opportunity. This is partly due to the diversity of treatment goals between patients and defining what constitutes meaningful benefit from a patient's viewpoint. Correlating metrics such as hematologic improvement or response rate to develop novel therapies can be falsely reassuring to physicians and patients, specifically if the interventions are burdensome, disconnected from a patient's sense of wellbeing and do not correlate with an improvement in quality of life or longevity. We discuss aligning drug development goals with patient goals that will lead to meaningful improvement in MDS clinical care.