Yeoungjee Cho1, Gopala Rangan2, Charlotte Logeman3, Hyunjin Ryu4, Benedicte Sautenet5, Ronald D Perrone6, Annie-Claire Nadeau-Fredette7, Reem A Mustafa8, Htay Htay9, Michel Chonchol10, Tess Harris11, Talia Gutman3, Jonathan C Craig12, Albert C M Ong13, Arlene Chapman14, Curie Ahn4, Helen Coolican15, Juliana Tze-Wah Kao16, Ron T Gansevoort17, Vicente Torres18, York Pei19, David W Johnson20, Andrea K Viecelli21, Armando Teixeira-Pinto3, Martin Howell3, Angela Ju3, Karine E Manera3, Allison Tong3. 1. Department of Nephrology, Princess Alexandra Hospital, Brisbane, Australia; Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia; Translational Research Institute, Brisbane, Australia. Electronic address: yeoungjee.cho@health.qld.gov.au. 2. Centre for Transplant and Renal Research, Westmead Institute for Medical Research, The University of Sydney, Sydney, Australia; Department of Renal Medicine, Westmead Hospital, Western Sydney Local Health District, Sydney, Australia. 3. Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, Australia. 4. Department of Internal Medicine, Seoul National University Hospital, Seoul, South Korea. 5. Department of Nephrology Hypertension, Dialysis, Kidney Transplantation, Tours Hospital, SPHERE - INSERM 1246, University of Tours and Nantes, Tours, France. 6. Division of Nephrology, Tufts Medical Center, Tufts University School of Medicine, Boston, MA. 7. Department of Nephrology, Hopital Maisonneuve-Rosemont, Montreal, Canada. 8. Department of Internal Medicine, Division of Nephrology and Hypertension, University of Kansas Medical Center, Kansas City, KS. 9. Department of Renal Medicine, Singapore General Hospital, Bukit Merah, Singapore. 10. Department of Nephrology, University of Colorado, Denver, CO. 11. Polycystic Kidney Disease International, London, United Kingdom. 12. College of Medicine and Public Health, Flinders University. 13. Academic Nephrology Unit, Department of Infection Immunity & Cardiovascular Disease, University of Sheffield, Sheffield, United Kingdom. 14. Department of Medicine, The University of Chicago, Chicago, IL. 15. Polycystic Kidney Disease Foundation of Australia, Roseville, NSW, Australia. 16. School of Medicine, Fu Jen Catholic University and Fu Jen Catholic University Hospital, Taiwan; Department of Internal Medicine, National Taiwan University Hospital, Taipei City, Taiwan. 17. Faculty of Medical Sciences, University Medical Center Gronigen, Groningen, the Netherlands. 18. Department of Nephrology and Hypertension, Mayo Clinic, Rochester, MN. 19. Division of Nephrology and Division of Genomic Medicine, University of Toronto, Toronto, Canada. 20. Department of Nephrology, Princess Alexandra Hospital, Brisbane, Australia; Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia; Translational Research Institute, Brisbane, Australia. 21. Department of Nephrology, Princess Alexandra Hospital, Brisbane, Australia; Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia; Department of Nephrology, Mater Hospital, Brisbane, Australia.
Abstract
RATIONALE & OBJECTIVE: Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD. STUDY DESIGN: An international 2-round online Delphi survey was conducted in English, French, and Korean languages. SETTING & PARTICIPANTS: Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. ANALYTICAL APPROACH: The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically. RESULTS: 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst-related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities. LIMITATIONS: Study design precluded involvement from those without access to internet or limited computer literacy. CONCLUSIONS: Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst-related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making.
RATIONALE & OBJECTIVE: Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD. STUDY DESIGN: An international 2-round online Delphi survey was conducted in English, French, and Korean languages. SETTING & PARTICIPANTS: Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. ANALYTICAL APPROACH: The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically. RESULTS: 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst-related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities. LIMITATIONS: Study design precluded involvement from those without access to internet or limited computer literacy. CONCLUSIONS: Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst-related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making.
Authors: Patrizia Natale; Ronald D Perrone; Allison Tong; Tess Harris; Elyssa Hannan; Angela Ju; Eva Burnette; Niek F Casteleijn; Arlene Chapman; Sarah Eastty; Ron T Gansevoort; Marie Hogan; Shigeo Horie; Bertrand Knebelmann; Richard Lee; Reem A Mustafa; Richard Sandford; Amanda Baumgart; Jonathan C Craig; Gopala K Rangan; Bénédicte Sautenet; Andrea K Viecelli; Noa Amir; Nicole Evangelidis; Chandana Guha; Charlotte Logeman; Karine Manera; Andrea Matus Gonzalez; Martin Howell; Giovanni F M Strippoli; Yeoungjee Cho Journal: Clin Kidney J Date: 2021-07-06
Authors: Ilene L Hollin; Jonathan Paskett; Anne L R Schuster; Norah L Crossnohere; John F P Bridges Journal: Pharmacoeconomics Date: 2022-07-15 Impact factor: 4.558
Authors: Tess Harris; Hannah R Bridges; Wendy D Brown; Natasha L O'Brien; Ann C Daly; Bharat K Jindal; Gillian S Mundy; Albert Ong; Albert J Power; Richard N Sandford; John Sayer; Roslyn J Simms; Patricia D Wilson; Paul J D Winyard; Maryrose Tarpey Journal: BMJ Open Date: 2022-06-15 Impact factor: 3.006