Endpoint selection and evaluation in hematology studies

Observational studies and clinical trials in hematology aim to examine treatments for blood disorders. The outcomes being studied must address the goals of the study and provide meaningful information about treatment course, disease progression, describe patients’ survival experience and quality of life. Endpoints are the specific measures of these outcomes, and much consideration should be given to their selection. In this review, we describe the outcomes and endpoints frequently used in studying hematologic diseases and provide general guidelines for their statistical analysis. The main focus is on clinical outcomes which are commonly used in establishing treatment safety and efficacy. We also briefly discuss the role surrogate and composite endpoints play in hematology studies. The importance of patient reported outcomes to comprehensive assessment of the treatment effectiveness is highlighted. Provided practical considerations for choosing primary and secondary endpoints may be helpful in designing hematology clinical trials.