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

• Type of Material: Article
• Authors: Linda J. Burns, National Marrow Donor Program; Beatrice Abbetti, Leukemia & Lymphoma Society; Staci D. Arnold, Emory University; Jeffrey Bender, Orlando, FL; Susan Doughtie, Georgia Cancer Center; Areej El-Jawahiri, Massachusetts General Hospital; Gloria Gee, Orlando, FL; Theresa Hahn, Roswell Park Cancer Institute; Mary M. Horowitz, Medical College of Wisconsin; Shirley Johnson, Roswell Park Cancer Institute; Mark Juckett, University of Wisconsin; Lakshmanan Krishnamurit, Emory University; Susan Kullberg, Minneapolis, MN; C. Fred LeMaistre, Sara Cannon; Alison Loren, University of Pennsylvania; Navneet S. Majhail, Cleveland Clinic; Elizabeth A. Murphy, National Marrow Donor Program; Doug Rizzo, Medical College of Wisconsin; Alva Roche-Green, Mayo Clinic; Wael Saber, Medical College of Wisconsin; Barry A. Schatz, Loyola University; Kim Schmit-Pokorny, Nebraska Medicine; Bronwen E. Shaw, Medical College of Wisconsin; Karen L. Syrjala, Fred Hutchinson Cancer Research Center; D. Kathryn Tierney, Stanford Health Care; Christina Ullrich, Dana Farber Cancer Institute; David J. Vanness, University of Wisconsin; William A. Wood, University of North Carolina; Ellen M. Denzen, National Marrow Donor Program
• Language: English
• Date: 2018-06-01
• Publisher: Elsevier: 12 months
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2018 American Society for Blood and Marrow Transplantation
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1016/j.bbmt.2018.01.029
• Title of Journal or Parent Work: Biology of Blood and Marrow Transplantation
• ISSN: 1083-8791
• Volume: 24
• Issue: 6
• Start Page: 1111
• End Page: 1118
• Grant/Funding Information: This project was supported in part by a Patient-Centered Outcomes Research Institute Eugene Washington Engagement Award (PCORI EAIN-2956).
• Keywords: Hematopoietic cell transplantation; EDUCATION; RANDOMIZED CONTROL TRIAL; Science & Technology; CAREGIVERS; FATIGUE; Hematology; ACUTE MYELOID-LEUKEMIA; Immunology; Life Sciences & Biomedicine; HEMATOLOGICAL CANCER; FERTILITY PRESERVATION; Transplantation; QUALITY-OF-LIFE; Survivorship; Patient-centered outcomes; BONE-MARROW-TRANSPLANTATION; SURVIVORS
• Research Categories: Health Sciences, Oncology; Health Sciences, Pathology

Abstract

The goal of patient-centered outcomes research (PCOR) is to help patients and those who care for them make informed decisions about healthcare. However, the clinical research enterprise has not involved patients, caregivers, and other nonproviders routinely in the process of prioritizing, designing, and conducting research in hematopoietic cell transplantation (HCT). To address this need the National Marrow Donor Program/Be The Match engaged patients, caregivers, researchers, and other key stakeholders in a 2-year project with the goal of setting a PCOR agenda for the HCT community. Through a collaborative process we identified 6 major areas of interest: (1) patient, caregiver, and family education and support; (2) emotional, cognitive, and social health; (3) physical health and fatigue; (4) sexual health and relationships; (5) financial burden; and (6) models of survivorship care delivery. We then organized into multistakeholder working groups to identify gaps in knowledge and make priority recommendations for critical research to fill those gaps. Gaps varied by working group, but all noted that a historical lack of consistency in measures use and patient populations made it difficult to compare outcomes across studies and urged investigators to incorporate uniform measures and homogenous patient groups in future research. Some groups advised that additional pre-emptory work is needed before conducting prospective interventional trials, whereas others were ready to proceed with comparative clinical effectiveness research studies. This report presents the results of this major initiative and makes recommendations by working group on priority questions for PCOR in HCT.