title: 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 convened six working groups to address the research needs and best practices for late effects of hematopoietic stem cell transplantation 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 in survivors, including the various dimensions of health-related 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. Patient-centered outcomes 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 patient-centered outcome 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 patient-centered outcomes, use of evidence-based practice guidelines, and provision of treatment summaries and survivorship care plans after HCT. Three priority research recommendations included the following: (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, nonadherence to medications and 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 transplantation centers; (2) design a consensus-based methodologic framework for outcomes evaluation; and (3) evaluate and compare existing practices for integrating patient-centered outcome screening and interventions across HCT survivorship programs.
authors.affiliation: Nursing Department, National Institutes of Health Clinical Center, Bethesda, Maryland. Electronic address: mbevans@cc.nih.gov.
authors.forename: Margaret
authors.initials: M
authors.lastname: Bevans
authors.affiliation: Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Areej
authors.initials: A
authors.lastname: El-Jawahri
authors.affiliation: Division of Primary, Preventive and Community Medicine, Stanford University, Stanford, California.
authors.forename: D Kathryn
authors.initials: DK
authors.lastname: Tierney
authors.affiliation: Psychosocial Support and Research Program, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland.
authors.forename: Lori
authors.initials: L
authors.lastname: Wiener
authors.affiliation: Division of Hematology and Oncology, University of North Carolina, Durham, North Carolina.
authors.forename: William A
authors.initials: WA
authors.lastname: Wood
authors.affiliation: Department of Psychiatry, University of Michigan & Department of Psychology, Eastern Michigan University, Ann Arbor, Michigan.
authors.forename: Flora
authors.initials: F
authors.lastname: Hoodin
authors.affiliation: Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, Maryland.
authors.forename: Erin E
authors.initials: EE
authors.lastname: Kent
authors.affiliation: Psychosocial and Palliative Care Program, Moffitt Cancer Center, Tampa, Florida.
authors.forename: Paul B
authors.initials: PB
authors.lastname: Jacobsen
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
authors.affiliation: Molecular and Clinical Hematology Branch, National Institutes of Health, National Heart Lung and Blood Institute, Bethesda, Maryland.
authors.forename: Matthew M
authors.initials: MM
authors.lastname: Hsieh
authors.affiliation: National Marrow Donor Program/Center for International Blood and Marrow Transplant Research, Minneapolis, Minnesota.
authors.forename: Ellen M
authors.initials: EM
authors.lastname: Denzen
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
authors.forename: Karen L
authors.initials: KL
authors.lastname: Syrjala
mesh_terms: D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000379:methods / Q000592:standards; D006801:Humans; D009316:National Institutes of Health (U.S.); D017063:Outcome Assessment, Health Care / Q000379:methods / Q000592:standards*; D017410:Practice Guidelines as Topic; D011788:Quality of Life / Q000523:psychology; D012107:Research Design / Q000592:standards; D058028:Research Report; D017741:Survivors / Q000523:psychology; D014481:United States
keywords: Hematopoietic cell transplantation; Late effects; Patient-centered outcomes; Psychosocial; Quality of life; Survivorship