Rethinking clinical trial endpoints in myelodysplastic syndromes

The myelodysplastic syndromes (MDS) are a heterogeneous collection of clonal, hematopoietic disorders primarily affecting an older population, making successful drug development a complicated process. A sole focus on response rate in clinical trials is likely not clinically meaningful if not accompanied by substantive response duration, improvement in quality of life, and ideally prolongation of survival. The process of receiving a new therapy should not be more burdensome than the MDS sequela it is intended to ameliorate. We review challenges in drug development in MDS with respect to aligning trial endpoints for lower and higher risk patients with treatment goals meaningful to patients.