Zhou X, Davenport EK, Ouyang J, Krasa H, Garbinsky D, Agarwal I, Oberdhan D. Population characteristics across a pooled database of multiple Autosomal Dominant Polycystic Kidney Disease (ADPKD) clinical studies. Poster presented at the 2020 National Kidney Foundation Spring Clinical Meeting; March 2020. New Orleans, LA. [abstract] Am J Kidney Dis. 2020 Apr; 75(4):663.

ADPKD is the most common hereditary kidney disease and the 4th leading cause of kidney failure. Over the past 2 decades, various studies were conducted to characterize the natural history of ADPKD and investigate impacts of various treatments on disease progression. We created a pooled longitudinal database of unique subjects from available studies to evaluate outcomes. Here we describe the baseline characteristics of the pooled population.

Data from 9 ADPKD studies (sponsored by Otsuka or NIH from 2001-2016) were combined and divided into 2 groups, tolvaptan (TOL) and standard of care (SOC). The TOL group consisted of trial subjects initiating treatment in one of 6 trials (156-04-250, 156-04- 251, 156-06-260, 156-09-284, 156-09-290, 156-08-271). The SOC group included subjects from placebo arms of 2 tolvaptan randomized trials, all standard blood pressure control arms in the HALT-PKD trials, and subjects from 2 observational studies (156-10-291, CRISP).

The pooled analysis included 5626 eligible subjects (TOL: 1437; SOC: 4189) with 21% having at least 5 years of follow up. The two cohorts were different in most demographics and disease characteristics. The TOL group was younger (mean age 40 vs 44 yrs) with a higher baseline eGFR (79 vs 70 mL/min/1.73 m2) and more frequent history of signs of rapid disease progression (eg. nephrolithiasis, hematuria, urinary tract infection). Among those with total kidney volume (TKV) assessments, mean TKV was higher in the TOL group (1,817 mL) compared with SOC (1,627 mL).

In this large longitudinal database of unique ADPKD subjects, distinct differences exist in the baseline characteristics of the TOL and SOC groups. These differences are expected to impact the observed outcomes related to progression of ADPKD.

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