Designing and conducting a clinical trial in blood and marrow transplantation. Best Pract Res Clin Haematol 2023 Jun;36(2):101471
Date
06/24/2023Pubmed ID
37353295Pubmed Central ID
PMC10845212DOI
10.1016/j.beha.2023.101471Scopus ID
2-s2.0-85159403396 (requires institutional sign-in at Scopus site)Abstract
Clinical trials form the cornerstone of the science-based approach to improving patient outcomes. A trial needs to be designed and performed carefully to provide valid evidence to inform medical science and to protect the safety and well-being of its participants. The development of a clinical trial involving blood and marrow transplant (BMT) requires special considerations, including the rare disease populations involved and transplant-specific outcomes of interest that necessitate appropriate analysis techniques to evaluate. This article reviews key considerations and best practices for the design and conduct of a clinical trial in BMT, including the selection of patient population, treatment groups, objectives and endpoints, targeted sample size, statistical analysis strategy, provisions for monitoring patient safety and trial progress, and dissemination of trial results. The practical application of these principles is demonstrated using BMT CTN 1301, a recently completed clinical trial evaluating regimens for chronic graft-versus-host disease prevention in transplant patients.
Author List
Martens MJ, Gao Y, Szabo AAuthors
Yan Gao PhD Assistant Professor in the Institute for Health and Equity department at Medical College of WisconsinMichael Martens PhD Assistant Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Aniko Szabo PhD 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
Bone MarrowBone Marrow Transplantation
Clinical Trials as Topic
Hematopoietic Stem Cell Transplantation
Humans
