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Editorial
. 2022 May 20:35:10134.
doi: 10.3389/ti.2022.10134. eCollection 2022.

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

Allison Tong  1 , Rainer Oberbauer  2 , Maria Irene Bellini  3 , Klemens Budde  4 , Fergus J Caskey  5 , Fabienne Dobbels  6 , Liset Pengel  7 , Lionel Rostaing  8 , Stefan Schneeberger  9 , Maarten Naesens  10
Affiliations
Editorial

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

Allison Tong et al. Transpl Int. .

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.

Keywords: PROMIS®; SONG-Tx; adherence; life participation; patient perspective; patient-reported outcome measure (PROM).

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Conflict of interest statement

RO has received grants/research support from Amgen, Astellas, and Chiesi; and speakers’ bureaux/honoraria from Amgen, Astellas, Chiesi, Hansa, Neovii, Novartis, and Teva. KB has received honoraria and/or research funding from Alexion, Astellas, Bristol Myers Squibb, Chiesi, Fresenius, Hansa, Hexal, Merck, Novartis, Otsuka, Pfizer, Roche, Sandoz, Siemens, and Veloxis. SS has received grants/research support from Bridge to Life, Chiesi, Neovii, Novartis, Organ Recovery Systems, and Sandoz; speakers’ bureaux/honoraria from Astellas, BMS, Chiesi, Novartis, OrganOx, and Sanofi; and consulting fees from Astellas, Atara, Merck, NefroHealth, Novartis, Sandoz, and Teva. The remaining authors declare that the work was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

FIGURE 1
FIGURE 1
SONG-Tx core outcomes. Reprinted from Kidney Int. Vol 94. Tong A, et al. (13). Implementing core outcomes in kidney disease. Report of the Standardized Outcomes in Nephrology, with permission from Elsevier.
See this image and copyright information in PMC

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