National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: Developing Recommendations to Improve Survivorship and Long-term Outcomes

摘要

Continual advances in hematopoietic cell transplantation (HCT) have greatly improved early transplant related mortality and broadened the applicability of this intense but curative therapy. With growing success there is increasing awareness of late complications, occurring ≥ 1-year post treatment, and their associated morbidity and mortality in HCT survivors. These late effects occur with a wide spectrum in terms of latency, intensity, reversibility and lethality. There is a need to understand the biology, surveillance, management and patient experience of HCT-related effects, as well as the healthcare and research infrastructure to manage this growing population. To address these needs, the National Cancer Institute (NCI) and National Heart, Lung and Blood Institute (NHLBI) co-sponsored a 12-month initiative to identify barriers and knowledge gaps and to formulate research and practice recommendations. Six major areas of interest were identified: research methodology and study design, subsequent neoplasms, patient centered outcomes, immune dysregulation and pathobiology, cardiovascular disease and associated risk factors, and healthcare delivery. These findings were presented during the 2016 workshop and revised based on public response. This report provides an overview of the National Institutes of Health HCT Late Effects Initiative process and recommendations.