Toward Establishing Core Outcome Domains For Trials in Kidney Transplantation

Abstract

Background Treatment decisions in kidney transplantation requires patients and clinicians to weigh the benefits and harms of a broad range of medical and surgical interventions, but the heterogeneity and lack of patient-relevant outcomes across trials in transplantation makes these trade-offs uncertain, thus, the need for a core outcome set that reflects stakeholder priorities. Methods We convened 2 international SONG-Kidney Transplantation stakeholder consensus workshops in Boston (17 patients/caregivers; 52 health professionals) and Hong Kong (10 patients/caregivers; 45 health professionals). In facilitated breakout groups, participants discussed the development and implementation of core outcome domains for trials in kidney transplantation. Results Seven themes were identified. Reinforcing the paramount importance of graft outcomes encompassed the prevailing dread of dialysis, distilling the meaning of graft function, and acknowledging the terrifying and ambiguous terminology of rejection. Reflecting critical trade-offs between graft health and medical comorbidities was fundamental. Contextualizing mortality explained discrepancies in the prioritization of death among stakeholders – inevitability of death (patients), preventing premature death (clinicians), and ensuring safety (regulators). Imperative to capture patient-reported outcomes was driven by making explicit patient priorities, fulfilling regulatory requirements, and addressing life participation. Specificity to transplant; feasibility and pragmatism (long-term impacts and responsiveness to interventions); and recognizing gradients of severity within outcome domains were raised as considerations. Conclusions Stakeholders support the inclusion of graft health, mortality, cardiovascular disease, infection, cancer, and patient-reported outcomes (ie, life participation) in a core outcomes set. Addressing ambiguous terminology and feasibility is needed in establishing these core outcome domains for trials in kidney transplantation.

Associations with kidney transplant survival and eGFR decline in children and young adults in the United Kingdom: a retrospective cohort study

“…Initial transplant function was bidirectionally associated with both outcomes, with better initial function being associated with less risk of graft loss but a faster rate of eGFR decline. There are scarce data on eGFR decline among children and young adults, yet graft health is considered a core outcome for research [ 30 ]. Whilst it might be assumed that risk factors may be similar for eGFR decline and graft loss, this can now be confirmed.…”

Renal Outcomes of Antidiabetic Treatment Options for Type 2 Diabetes—A Proposed MARE Definition

“…54 Differences must be observed with regard to the treatment modality under evaluation. First considerations are available regarding hemodialysis, 55 , 56 peritoneal dialysis, 57 and kidney transplantation, 58 and more are underway.…”

Implementing core outcomes in kidney disease: report of the Standardized Outcomes in Nephrology (SONG) implementation workshop

“…The workshop also included specific reference to core outcome sets that had been established at the time of the workshop (Figure 1a and b) including for patients receiving hemodialysis (fatigue, cardiovascular disease, vascular access function, mortality) 9–12,35–38 and kidney transplant recipients (graft loss, cardiovascular disease, infection, life participation, cancer, mortality). 39–42 These core outcome sets were developed using an evidence- and consensus-based process (systematic review, focus groups with nominal group technique, stakeholder interviews, an international Delphi Survey, and a consensus workshop) involving more than 1300 patients, caregivers, and health professionals from more than 70 countries in each stream (i.e., hemodialysis, kidney transplantation). 10–12,37,38,40,41 The findings from the workshop will inform strategies and pathways for implementing core outcomes, with a focus on trials in nephrology.…”