National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Research Methodology and Study Design Working Group Report.

The increasing numbers of hematopoietic cell transplantations (HCTs) performed each year, the changing demographics of HCT recipients, the introduction of new transplantation strategies, incremental improvement in survival, and the growing population of HCT survivors demand a comprehensive approach to examining the health and well-being 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 hoped that these concepts will promote continued research and facilitate the development of new approaches to address fundamental questions in transplantation outcomes.