Background.
Inconsistencies in outcome reporting and frequent omission of patient-centered outcomes can diminish the value
of trials in treatment decision making. We identified critically important outcome domains in kidney transplantation based on the
shared priorities of patients/caregivers and health professionals.
Methods.
In a 3-round Delphi survey, patients/caregivers
and health professionals rated the importance of outcome domains for trials in kidney transplantation on a 9-point Likert scale
and provided comments. During rounds 2 and 3, participants rerated the outcomes after reviewing their own score, the distribution
of the respondents' scores, and comments. We calculated the median, mean, and proportion rating 7 to 9 (critically important),
and analyzed comments thematically.
Results.
One thousand eighteen participants (461 [45%] patients/caregivers and 557
[55%] health professionals) from 79 countries completed round 1, and 779 (77%) completed round 3. The top 8 outcomes that
met the consensus criteria in round 3 (mean,
=
7.5; median,
=
8; proportion, >85%) in both groups were graft loss, graft function,
chronic rejection, acute rejection, mortality, infection, cancer (excluding skin), and cardiovascular disease. Compared with health
professionals, patients/caregivers gave higher priority to 6 outcomes (mean difference of 0.5 or more): skin cancer, surgical com-
plications, cognition, blood pressure, depression, and ability to work. We identified 5 themes: capacity to control and inevitability,
personal relevance, debilitating repercussions, gaining awareness of risks, and addressing knowledge gaps.
Conclusions.
Graft complications and severe comorbidities were critically important for both stakeholder groups. These stakeholder-
prioritized outcomes will inform the core outcome set to improve the consistency and relevance of trials in kidney transplantation.
Bénédicte Sautenet; Allison Tong; Karine E. Manera; Jeremy R. Chapman; Anthony N. Warrens; David Rosenbloom; Germaine Wong; John Gill; Klemens Budde; Lionel Rostaing; Lorna Marson; Michelle A. Josephson; Peter P. Reese; Timothy L. Pruett; Camilla S. Hanson; Donal O’Donoghue; Helen Tam-Tham; Jean-Michel Halimi; Jenny I. Shen; John Kanellis; John D. Scandling; Kirsten Howard; Martin Howell; Nick Cross; Nicole Evangelidis; Philip Masson; Rainer Oberbauer; Samuel Fung; Shilpa Jesudason; Simon Knight; Sreedhar Mandayam; Stephen P. McDonald; Steve Chadban; Tasleem Rajan; Jonathan C. Craig
Disease Category: Kidney disease
Disease Name: Kidney Transplant
Age Range: 18 - 71100
Sex: Either
Nature of Intervention: Surgery
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Policy makers
- Regulatory agency representatives
- Researchers
- Patient perspectives
- Delphi process