Abstract: SA-PO492
Disease Symptoms and Poor Quality of Life (QoL) in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Preserved Kidney Function
Session Information
- ADPKD: Clinical Studies
October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidney
- 1001 Genetic Diseases of the Kidney: Cystic
Authors
- Yarlas, Aaron, Optum Patient Insights, Johnston, Rhode Island, United States
- Oberdhan, Dorothee, Otsuka, Rockville, Maryland, United States
- Krasa, Holly, Otsuka, Rockville, Maryland, United States
- Maher, Stephen M., Optum Patient Insights, Johnston, Rhode Island, United States
- Bjorner, Jakob B., Optum Patient Insights, Johnston, Rhode Island, United States
Background
ADPKD is a hereditary progressive disease in which enlarging kidney cysts impair function and lead to kidney failure. In general, patients in early-stage chronic kidney disease [CKD] (eg, stages 1 and 2) tend to have normal QoL, but there is variability in disease burden with early-stage ADPKD. A subgroup of patients within an observational study had significant disease burden prior to renal function decline. We examine the characteristics of this subgroup.
Methods
Data were from a multi-center, longitudinal, observational study of 3,409 adult ADPKD patients (NCT01430494) with 990 and 956 patients in CKD stages 1 and 2, respectively. A subgroup of patients in stages 1 or 2 (n=42) was identified based on poor scores (≥3 points) on 2 of 3 ADPKD-Impact scale (ADPKD-IS) subscales (physical, emotional, fatigue). We examined associations of poor ADPKD-IS scores with clinical outcomes (eg, albuminuria, kidney stones, urinary tract infection [UTI]), biomarkers (eg, height-adjusted total kidney volume, estimated glomerular filtration rate, abdominal girth), and patient-reported outcome (PRO) measures (ADPKD-Urinary Impact Scale, SF-12v2® Health Survey, Brief Pain Inventory). Analyses used χ2 tests, receiver operating characteristic (ROC) curves, and Cohen’s d effect sizes for mean differences.
Results
Patients in the poor ADPKD-IS subsample were more likely than other early-stage CKD patients to have albuminuria, hematuria, kidney pain, kidney stones, and UTI (all p <.05). ROC analyses found that biomarkers did not distinguish between the poor ADPKD-IS subgroup and other early-stage patients: all areas under the curve (AUC) ≤.60, all p >.05. All PRO scores accurately differentiated the poor ADPKD-IS subgroup from the remaining early stage sample: AUCs from .83 to .95 (all p <.0001), with large mean sample differences (all d ≥ 1.6).
Conclusion
A subgroup of ADPKD patients in early-stage CKD experience poor symptomatology and QOL. These patients are more likely to have albuminuria, hematuria, kidney pain, kidney stones, or UTI.
Funding
- Commercial Support – Otsuka Pharmaceutical Development & Commercialization, Inc.