Range and variability of outcomes reported in randomized trials conducted in patients with polycystic kidney disease: A systematic review
American Journal of Kidney Diseases Mar 22, 2020
Sautenet B, Cho Y, Gutman T, et al. - Given that despite an increase in the trials in autosomal dominant polycystic kidney disease (ADPKD), their influence on decision making has been limited, and since the likely reason be heterogeneity in reported outcomes, so, researchers evaluated their range as well as variability in ADPKD trials. For this purpose, they conducted a systematic review including adult participants in clinical trials in ADPKD. Trials with adults and those published in English were included. Experts analyzed 68 trials with 1,413 different outcome measures. They found 97 domains. The 3 most commonly reported domains fell in the surrogate category: kidney function, kidney and cyst volumes, and blood pressure; in the clinical category: infection, cardiovascular events, and kidney failure needing kidney replacement therapy; and in the patient-reported category: pain associated with ADPKD, pain for other reasons, and diarrhea/constipation/gas. A wide scope as well as high variability of the outcomes in ADPKD trials was reported. Most commonly reported ones were surrogate outcomes. Patient-reported outcomes were uncommon.
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