Low clinical trial accrual of patients with myelodysplastic syndromes: Causes and potential solutions.

Abstract

Despite few effective therapies, only a small percentage of patients diagnosed with myelodysplastic syndromes (MDS) in the United States are enrolled in prospective, interventional clinical trials. MDS-specific barriers to trial accrual include a high frequency of elderly patients with comorbid conditions, atypical disease features and uncertainty regarding the diagnosis (because other nonclonal processes also can cause dysplasia and cytopenias), a history of another nonmyeloid neoplasm resulting in therapy-related MDS, rapid disease recurrence after allogeneic stem cell transplantation, and an arbitrary division between MDS and acute myeloid leukemia. In addition, barriers to accrual that are common to other oncology populations, such as difficulty traveling to clinical trial enrollment sites and narrow trial eligibility criteria, also prevent patients with MDS from enrolling in studies. Collectively these barriers must be assessed systematically, and creative solutions are needed to improve outcomes for this needy patient population.