National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Patient-Centered Outcomes Working Group Report

Abstract

In 2015 the National Institutes of Health (NIH) convened six working groups to address the research needs and best practices for patient-centered late effects of hematopoietic stem cell transplantation (HCT) survivors. The Patient-Centered Outcomes Working Group, charged with summarizing the HRQOL evidence base, used a scoping review approach to efficiently survey the large body of literature in adult and pediatric HCT survivors over 1 year after transplantation. 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.

Effect of Inpatient Palliative Care During Hematopoietic Stem-Cell Transplant on Psychological Distress 6 Months After Transplant: Results of a Randomized Clinical Trial

“…21 Given the paucity of data, the National Institutes of Health Late Effects Initiative identified a critical need to address psychological distress, including depression and PTSD symptoms, in HCT survivors. 38 To our knowledge, the current study is the first to demonstrate that a brief inpatient intervention during HCT hospitalization leads to a reduction in patients’ depression and PTSD symptoms after transplant. We did not, however, detect significant intervention effects on anxiety symptoms at 6 months post-transplant.…”

Wound healing outcomes: Using big data and a modified intent‐to‐treat method as a metric for reporting healing rates

“…Compare this to Oncology where data exists not only on specific cancer types in clinical trials but also at the macro level there are indications of cancer outcomes changing over time in the aggregate . Oncology also has moved toward coordinated efforts between providers to gain consensus and standardize outcomes metrics and transition toward patient centric and patient reported data points . Oncology also employs a staging system that allow for risk stratification, prediction of outcomes and the appropriate selection of therapeutic treatments for a corresponding stage.…”

Engaging Patients in Setting a Patient-Centered Outcomes Research Agenda in Hematopoietic Cell Transplantation

“…Inconsistency in instrument choice for measuring physical health and fatigue and application across studies limits our understanding in this area [19–25]. Relatively few studies have attempted to correlate phenotype (self-reported) with biologic measures to identify causative pathways (e.g.…”