Endpoint selection and evaluation in hematology studies. Best Pract Res Clin Haematol 2023 Sep;36(3):101479
Date
08/24/2023Pubmed ID
37611997Pubmed Central ID
PMC10979628DOI
10.1016/j.beha.2023.101479Scopus ID
2-s2.0-85161313534 (requires institutional sign-in at Scopus site)Abstract
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.
Author List
Brazauskas R, Eapen M, Wang TAuthors
Ruta Brazauskas PhD Associate Professor in the Institute for Health and Equity department at Medical College of WisconsinMary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Tao Wang PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin
MESH terms used to index this publication - Major topics in bold
Disease ProgressionHematology
Humans
Quality of Life
