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Published on: April 12, 2021
Developing Consensus-Based Priority Outcome Domains for Trials in Kidney Transplantation: A Multinational Delphi
Bénédicte Sautenet1, Allison Tong, Karine E Manera
11 Sydney School of Public Health, The University of Sydney, Sydney, Australia.2 Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, Sydney, Australia.3 Faculté de Médecine, Université Francois Rabelais, Tours, France.4 Department of Nephrology and Clinical Immunology, Tours Hospital, Tours, France.5 INSERM, U1246, Tours, France.6 Centre for Transplant and Renal Research, Westmead Hospital, Sydney, Australia.7 School of Medicine and Dentistry, Queen Mary University of London, London, United Kingdom.8 ESRD Network 18, Los Angeles, CA.9 Division of Nephrology, University of British Columbia, Vancouver, Canada.10 Department of Nephrology, Charité - Universitätsmedizin Berlin, Germany.11 Department of Nephrology, Dialysis and Organ Transplantation, Centre Hospitalier Universitaire, Rangueil, Toulouse, France.12 Transplant Unit, University of Edinburgh, Edinburgh, United Kingdom.13 Department of Medicine, The University of Chicago, Chicago, IL.14 Renal Division, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.15 Department of Surgery, University of Minnesota, Minneapolis, MN.16 Department of Renal Medicine, Salford Royal NHS Foundation Trust, Salford, United Kingdom.17 Department Community Health Sciences, University of Calgary, Calgary, Canada.18 Department of Nephrology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Los Angeles, CA.19 Department of Nephrology, Monash Health and Centre for Inflammatory Diseases, Department of Medicine, Monash University, Clayton, Victoria, Australia.20 Department of Medicine, Stanford University School of Medicine, Stanford, CA.21 Department of Nephrology, Christchurch Hospital, Christchurch, New Zealand.22 Department of Renal Medicine, Royal Infirmary of Edinburgh, Edinburgh, Scotland, United Kingdom.23 Department of Internal Medicine, Division of Nephrology, University of Vienna, Austria.24 Jockey Club Nephrology and Urology Centre, Princess Margaret Hospital, Hong Kong.25 Central and Northern Adelaide Renal and Transplantation Service, Royal Adelaide Hospital, Adelaide, South Australia, Australia.26 Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, Australia.27 Centre for Evidence in Transplantation, Nuffield Department of Surgical Sciences, University of Oxford, Oxford, United Kingdom.28 Selzman Institute for Kidney Health, Section of Nephrology, Baylor College of Medicine, Houston, TX.29 ANZDATA Registry, South Australia Health and Medical Research Institute, Adelaide, Australia.30 Renal Medicine and Transplantation, Royal Prince Alfred Hospital, Sydney, Australia.
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 complications, 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.
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