Trends in Clinical Investigation for Myelodysplastic Syndromes

Myelodysplastic syndrome (MDS) paradigms have been dramatically changed over the last 10 years by major breakthroughs on both pathophysiologic and therapeutic aspects. It is currently a field of intense clinical investigation as new challenges have emerged in both low-risk and high-risk populations. In low-risk MDS, long-term control of anemia is a major issue, and second-line treatments after failure of erythropoiesis-stimulating agents are warranted. Several promising therapies are available, and there are many open questions on how to select the most adapted agent and/or sequence of agents in a specific individual. For high-risk MDS patients, improvement of frontline treatment (namely hypomethylating agents) and identification of valid treatments for relapsed/refractory patients are of paramount importance. This review attempts to define these challenges, summarize the results of the most recent and promising investigational strategies in the field, and to describe the future directions.