Patient-Reported Outcomes as Endpoints in Clinical Trials of Kidney Transplantation Interventions

Abstract

Patient-reported outcomes (PROs) that assess individuals’ perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient’s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.

A Woman’s Place Is in Theatre, but Are Theatres Designed with Women in Mind? A Systematic Review of Ergonomics for Women in Surgery

“…Importantly, a more diverse staff also helps patients from minority backgrounds to feel better valued during their hospital stay. In fact, it is not uncommon to observe—because of religious and sociocultural beliefs—challenges for women to be assisted by male doctors or associated staff; therefore, to have an impact on patient health outcomes and quality of life, the care the same patients experience and how they perceive their representation is essential [ 30 , 31 ].…”

Rationale for Surrogate Endpoints and Conditional Marketing Authorization of New Therapies for Kidney Transplantation

“…Given that patients listed for re-transplantation are a selected population deemed capable of receiving another graft, it seems likely that those who are not relisted (primarily because of comorbidity and unacceptable risk) will have worse outcomes on dialysis. In addition, none of these analyses considers the burdens of returning to dialysis after failed transplantation, such as the costs associated with treatment ( 27 ), decreased ability to work and participate in society ( 28 ), and the psychological impact of returning to dialysis ( 29 – 31 ) (see also article by Tong et al on patient reported outcome measures, in this Special Issue ( 32 )).…”

Are cultural or psychosocial factors associated with patient‐reported outcomes at the conclusion of kidney transplant evaluation?

“…[1][2][3] Previous research demonstrated that receiving a KT is associated with many benefits post-transplant, 4 including improvements in QOL. 5,6 An unexplored period in ESKD patients' clinical experience is the KT evaluation (KTE) process. It is a period with many demands and stressors placed on a patient, which may lead to poorer patient-reported outcomes (PROs), including poorer QOL and low satisfaction with care.…”

“…It reduces mortality, improves quality of life (QOL), and costs less than dialysis 1–3 . Previous research demonstrated that receiving a KT is associated with many benefits post‐transplant, 4 including improvements in QOL 5,6 . An unexplored period in ESKD patients’ clinical experience is the KT evaluation (KTE) process.…”

“…The KTE process is a unique period for understanding PROs and the characteristics that influence them. To date, there is little research on the factors that are associated with variation in PROs at the completion of KTE 6,13 . We hypothesized that patients’ cultural and psychosocial characteristics can mitigate poor PROs at the completion of KTE if they are not accepted for transplant (either by being rejected for transplant by the transplant team, or due to a state of uncertainty from a prolonged evaluation period).…”