National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: Consensus Recommendations for Patient-Centered Outcomes

Abstract In 2016 the National Institutes of Health (NIH) convened an expert consensus panel to address the research needs and best practices for patient-centered late effects of hematopoietic stem cell transplantation (HCT). The panel used a scoping review approach to efficiently survey the large body of literature in adult and pediatric HCT survivors over one year post-transplant. The goals of this paper are to 1) summarize the current literature describing patient-centered outcomes (PCO) in survivors including the various dimensions of healthrelated quality of life affected by HCT and describe interventions tested to improve these outcomes; 2) highlight areas with sufficient evidence allowing for integration into standard practice; 3) address methodological issues that restrict progress in this field; 4) identify major gaps to guide future research; and 5) specify priority research recommendations. PCOs were summarized within physical, psychological, social and environmental domains, as well as for adherence to treatment, and health behaviors. Interventions to improve outcomes were evaluated for evidence of efficacy, although few interventions have been tested in long-term HCT survivors. Methodologic issues defined included lack of consistency in the selection of PCO measures, along with the absence of a standard for timing, frequency, and mode of administration. Recommendations for HCT survivorship care included: integration of annual screening of PCOs; use of evidence based practice guidelines; and provision of treatment summaries and survivorship care plans after HCT. Three priority research recommendations included: 1) Design and test risk-targeted interventions with dose intensity modulation matching the needs of HCT survivors with priority domains including sexual dysfunction, fatigue, sleep disruption, non-adherence to medications & recommended health care, health behaviors including physical inactivity and healthy eating, and psychological dysfunction, with particular consideration of novel technologies to reach HCT survivors distant from their transplant centers, 2) Design a consensus based methodologic framework for outcomes evaluation, and 3) Evaluate and compare existing practices for integrating PCOs screening and interventions across HCT survivorship programs.