Bénédicte Sautenet1, Yeoungjee Cho2, Talia Gutman3, Gopala Rangan4, Albert Ong5, Arlene B Chapman6, Curie Ahn7, Helen Coolican8, Juliana Tze-Wah Kao9, Kevin Fowler10, Ron T Gansevoort11, Claire Geneste12, Ronald D Perrone13, Tess Harris14, Vicente E Torres15, York Pei16, Jonathan C Craig17, Allison Tong3. 1. Service de Néphrologie-Hypertension, Dialyses, Transplantation Rénale, Hôpital de Tours, Tours, France; Université de Tours, Université de Nantes, INSERM, SPHERE U1246, Tours, France. Electronic address: benedicte.sautenet@univ-tours.fr. 2. Department of Nephrology, Princess Alexandra Hospital, Brisbane, Australia; Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia; Translational Research Institute, Brisbane, 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. 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. 5. Academic Nephrology Unit, Department of Infection Immunity & Cardiovascular Disease, University of Sheffield, United Kingdom. 6. Department of Medicine, The University of Chicago, Chicago, IL. 7. Division of Nephrology, Seoul National University Hospital, Seoul, South Korea. 8. Polycystic Kidney Disease Foundation of Australia, Sydney, Australia. 9. School of Medicine, Fu Jen Catholic University, Taiwan; Department of Internal Medicine, National Taiwan University Hospital, Taiwan. 10. Kidney Health Initiative, Patient Family Partnership Council, The Voice of the Patient, Elmhurst, Illinois. 11. Faculty of Medical Sciences, University Medical Center Groningen, the Netherlands. 12. Service de Néphrologie-Hypertension, Dialyses, Transplantation Rénale, Hôpital de Tours, Tours, France. 13. Division of Nephrology, Tufts Medical Center, Tufts University School of Medicine, Boston, MA. 14. Polycystic Kidney Disease International, London, United Kingdom. 15. Department of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota. 16. Division of Nephrology, University of Toronto, Toronto, Canada; Division of Genomic Medicine, University of Toronto, Toronto, Canada. 17. College of Medicine and Public Health, Flinders University, Adelaide, Australia.
Abstract
RATIONALE & OBJECTIVE: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials. STUDY DESIGN: Systematic review. SETTING & STUDY POPULATION: Adult participants in clinical trials in ADPKD. SELECTION CRITERIA FOR STUDIES: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling ≥50% of participants with ADPKD were included. DATA EXTRACTION: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures. ANALYTICAL APPROACH: Frequencies and characteristics of outcome measures were described. RESULTS: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures). LIMITATIONS: Outcome measures were assessed for only the top 3 domains in each category. CONCLUSIONS: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials.
RATIONALE & OBJECTIVE: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials. STUDY DESIGN: Systematic review. SETTING & STUDY POPULATION: Adult participants in clinical trials in ADPKD. SELECTION CRITERIA FOR STUDIES: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling ≥50% of participants with ADPKD were included. DATA EXTRACTION: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures. ANALYTICAL APPROACH: Frequencies and characteristics of outcome measures were described. RESULTS: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures). LIMITATIONS: Outcome measures were assessed for only the top 3 domains in each category. CONCLUSIONS: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials.
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