Myelodysplastic syndromes (MDS) consists of a group of disorders that are heterogeneous in cause and manifestations but share the common features of aberrant hematopoiesis and deteriorating cytopenias. Patients with MDS may suffer from anemia, neutropenia, and thrombocytopenia, with outcomes such as extreme fatigue, high rates of infections, excessive bleeding, and shortened life span.
This report provides an overview of the MDS landscape featuring a comprehensive analysis of patient populations, current therapies and medical practices, and opportunities for emerging therapies. The report identifies a very high level of unmet need for new, disease-modifying MDS therapies and provides expert insight on potential drug targets. At this time, the only potentially curative therapy for MDS is allogeneic hematopoietic stem cell transplant (HSCT). However, interviewed experts report that approximately three-quarters of the MDS patient population is ineligible for HSCT due to advanced age and/or poor overall health status. Current treatment options for MDS are very limited and frequently ineffective. Moreover, there are no good therapeutic options for treatment-refractory patients.