Novel Endpoints in Solid Organ Transplantation: Targeting Patient-reported Outcome Measures

Abstract

Although solid organ transplantation improves survival and quality of life in many patients with organ failure, treatment complications and side effects can have debilitating consequences for patients. Patient-reported outcome measures (PROMs) capture how patients feel and function, including quality of life, symptoms, and side effects that are assessed directly by patients. Yet, they remain infrequently reported in trials in solid organ transplantation. Barriers to implementing PROMs in trials include uncertainty in selecting appropriate PROMs, concerns about resources limitations, patient burden, and limited evidence to support the psychometric robustness of measures for use in transplant recipients. In recent years, there have been increasing efforts to include patient-reported outcomes that are important to patients in trials to ensure that trials provide patient-centered information for decision-making. This article will provide an overview of PROMs, discuss PROs that are important to solid organ transplant recipients and those that are used in trials in solid organ transplantation, and outline approaches for selecting PROMs for clinical trials.

European Society for Organ Transplantation (ESOT) Consensus Statement on Outcome Measures in Liver Transplantation According to Value-Based Health Care

“…The nature of LT, encompassing the patient’s journey from the time of registration on the waiting list to long-term post-transplant survival, highlights the need for longitudinal health related quality of life data [ 43 ]. However, most studies reported to date are most often cross-sectional analyses and pay little attention to the phase post-transplant [ 27 , 44 ].…”

European Society of Organ Transplantation (ESOT) Consensus Statement on Prehabilitation for Solid Organ Transplantation Candidates

“…All stakeholders including solid organ transplant candidates and recipients, transplant professionals, and researchers in the field of transplantation strongly supported that a core outcome set be defined to facilitate comparative studies and give impetus to the field. A core outcomes set refers to a minimum set of outcome measures that are critical to patients, caregivers, and health professionals for decision making [71]. Selected outcomes that have so far not been considered but do carry clinical relevance during the pretransplant waiting time are health-related physical fitness parameters such as muscular fitness, motor fitness, body composition and (cardio)metabolic health, as well as patientreported outcomes such as fatigue, medication adherence and lifestyle, and clinical outcomes such as duration of intensive care stay, hospitalization, (re-)admissions, complications, graft function and survival, and waitlist and post-transplant mortality.…”