The lack of fully effective therapies to alter the natural course of myelodysplastic syndromes (MDS) leads to chronic morbidity, mortality and affects quality of life (QoL). Since existing therapies outside of hematopoietic cell transplantation (HCT) are not curative, there is a growing interest in incorporating patient-reported outcomes (PROs) as meaningful endpoints for these patients in research and clinical practice.
Currently, there are limited numbers of studies reporting the impact of MDS therapeutics on PROs to guide clinical decision-making and increase patient satisfaction. However, clinical trials that have incorporated QoL outcomes have demonstrated positive results with the use of growth factors, hypomethylating agents, and lenalidomide. Here we review and highlight the importance of harnessing the power of PROs as part of a comprehensive efficacy evaluation to ultimately deliver superior patient-centered care across the spectrum of MDS. As our understanding of MDS continues to increase, adapting the metrics of these outcome measurements will be equally important as the alteration of the natural history in developing new therapies.