The escalating burden of end-stage kidney disease has spurred an increase in randomised trials in this population, yet health outcomes and quality of life remain poor. The causes of this may in part be attributable to fundamental flaws in the design and reporting of these studies, particularly outcome selection. Problems include using outcomes of little patient relevance, under-use of outcomes of high patient relevance, highly variable outcomes selection and outcomes reporting bias. Core outcome sets have been developed to ensure consistent reporting of outcomes in a specified area of health or condition.
Through the Standardised Outcomes in Nephrology (SONG) Initiative, core outcomes have been established for trials in haemodialysis (fatigue, cardiovascular disease, mortality, vascular access) and kidney transplantation (graft loss, life participation, cardiovascular disease, cancer, infection). This thesis provides novel and comprehensive evidence on priorities and preferences of patients with end-stage kidney disease regarding the PROMs that should be used in trials to assess outcomes that are important to them – specifically fatigue in haemodialysis, and life participation in kidney transplantation. This directly informs the identification and validation of core patient-reported outcome measures for fatigue and life participation. This thesis aims to develop and establish core patient-reported outcome measures (PROMs) for fatigue and life participation.
Implementing core outcome measures that are psychometrically robust can ultimately improve the value of trial-based evidence to support decision based on outcomes that are meaningful and important to patients with end-stage kidney disease.