National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: Consensus Recommendations for Research Methodology and Study Design.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2016)


The increasing numbers of HCTs performed each year, the changing demographics of HCT recipients, the introduction of new transplant strategies, incremental improvement in survival and the growing population of HCT survivors demands a comprehensive approach to examining the health and wellbeing of patients throughout life after HCT. This report summarizes strategies for the conduct of research on late effects after transplantation, including consideration of the study design and analytic approaches; methodologic challenges in handling complex phenotype data; an appreciation of the changing trends in the practice of transplantation, and the availability of biospecimens to support laboratory-based research. It is the hope that these concepts will promote continued research and facilitate the development of new approaches to address fundamental questions in transplant outcomes.