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Developing Consensus-Based Priority Outcome Domains for Trials in Kidney Transplantation: A Multinational Delphi Survey With Patients, Caregivers, and Health Professionals

Sautenet, Bénédicte MD, PhD1,2,3,4,5; Tong, Allison PhD1,2; Manera, Karine E. MIPH1,2; Chapman, Jeremy R. FRCP6; Warrens, Anthony N. MD7; Rosenbloom, David8; Wong, Germaine MBBS, PhD1,2,6; Gill, John MD9; Budde, Klemens MD10; Rostaing, Lionel MD, PhD11; Marson, Lorna MD, FRCS12; Josephson, Michelle A. MD13; Reese, Peter P. MD, MSCE14; Pruett, Timothy L. MD15; Hanson, Camilla S. BPsych (Hons)1,2; O’Donoghue, Donal MB, ChB, FRCP16; Tam-Tham, Helen MSc17; Halimi, Jean-Michel MD, PhD3,4; Shen, Jenny I. MD18; Kanellis, John MBBS, PhD19; Scandling, John D. MD20; Howard, Kirsten PhD1; Howell, Martin PhD1,2; Cross, Nick MD, PhD21; Evangelidis, Nicole BSocSc1,2; Masson, Philip MBChB, PhD22; Oberbauer, Rainer MD, PhD23; Fung, Samuel MBBS, FRCP24; Jesudason, Shilpa MD, PhD25,26; Knight, Simon MA, MB, MChir FRCS27; Mandayam, Sreedhar MD28; McDonald, Stephen P. MD, PhD25,26,29; Chadban, Steve FRACP, PhD30; Rajan, Tasleem MD17; Craig, Jonathan C. MBChB, PhD1,2

doi: 10.1097/TP.0000000000001776
Original Clinical Science—General

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.

Clinical trials report a variety of clinical, surrogate, or patient-reported outcomes. This article describes the results of novel methods using iterative Delphi Surveys of not only health professionals but patients and caregivers to determine the most important clinical trial outcome endpoints among both groups. Supplemental digital content is available in the text.

1 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.

Received 26 January 2017. Revision received 21 February 2017.

Accepted 2 March 2017.

This project is supported by a National Health and Medical Research Council Project Grant (1128564) and Program Grant (1092597). AT is supported by a NHMRC Career Development Fellowship (1106716). JS is supported by a National Institutes for Health (NIH) K23 Career Development Grant (DK103972).

The authors declare no conflicts of interest.

B.S. participated in the research design, data collection, data analysis, and drafted the article. A.T. participated in the research design, data collection, data analysis, and drafted the article. K.E.M. participated in the research design, data collection, data analysis, and drafted the article. J.R.C. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. A.W. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. D.R. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. G.W. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. J.G. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. K.B. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. L.R. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. L.M. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. M.A.J. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. P.R. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. T.P. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. C.S.H. participated in the research design, and provided intellectual input on the article and contributed to article writing. D.O. participated in the research design, and provided intellectual input on the article and contributed to article writing. H.T. participated in the research design, and provided intellectual input on the article and contributed to article writing. J.M.H. participated in the research design, and provided intellectual input on the article and contributed to article writing. J.I.S. participated in the research design, and provided intellectual input on the article and contributed to article writing. J.K. participated in the research design, and provided intellectual input on the article and contributed to article writing. J.D.S. participated in the research design, and provided intellectual input on the article and contributed to article writing. K.H. participated in the research design, and provided intellectual input on the article and contributed to article writing. M.H. participated in the research design, and provided intellectual input on the article and contributed to article writing. N.C. participated in the research design, and provided intellectual input on the article and contributed to article writing. N.E. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. P.M. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. R.O. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. S.F. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. S.J. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. S.K. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. S.M. participated in the research design, data analysis, and provided intellectual input on the article and contributed to article writing. S.P.M. participated in the research design, and provided intellectual input on the article and contributed to article writing. S.C. participated in the research design, and provided intellectual input on the article and contributed to article writing. T.R. participated in the research design, and provided intellectual input on the article and contributed to article writing. J.C.C. participated in the research design, data collection, data analysis, and drafted the article.

Correspondence: Allison Tong, PhD, Centre for Kidney Research, The Children’s Hospital at Westmead, Westmead, NSW, Sydney 2145, Australia. (allison.tong@sydney.edu.au).

Supplemental digital content (SDC) is available for this article. Direct URL citations appear in the printed text, and links to the digital files are provided in the HTML text of this article on the journal’s Web site (www.transplantjournal.com).

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