Chebib FT, Zhou X, Garbinsky D, Davenport E, Nunna S, Oberdhan D, Fernandes A. Tolvaptan and kidney function decline in older individuals with autosomal dominant polycystic kidney disease: a pooled analysis of randomized clinical trials and observational studies. Kidney Med. 2023 Jun;5(6):100639. doi: 10.1016/j.xkme.2023.100639

BAKCGROUND: Autosomal dominant polycystic kidney disease (ADPKD) can impose substantial burden on health-related quality of life (HRQL) as disease progresses. This analysis describes disease burden and progression of ADPKD in young adults using data from the OVERTURE (NCT01430494) study.

METHODS: OVERTURE was an observational, longitudinal study of ADPKD patients which assessed total kidney volume (TKV), estimated glomerular filtration rate (eGFR), and other disease outcomes including patient-reported HRQL measures (12-Item Short Form Health Survey [SF-12v2] Mental and Physical Component Summary, MCS and PCS) and Brief Pain Inventory [BPI]) and ADPKD-specific questionnaires (ADPKD–Impact Scale [IS] and ADPKD Urinary Impact Scale [UIS]). Data were summarized by age groups, sex, and visits.

RESULTS: 93 eligible 18 to <22-year-old patients and 132 22 to <26-year-old patients at baseline were included. Approximately 60% were female per age group. While kidney function was normal for most patients in both groups (mean eGFR, 115.2 and 107.4 ml/min/1.73m2), TKV was enlarged (mean, 814.7 and 980.9 ml). Nearly half of patients (48.8% vs 50.4%) were in Mayo class 1D to 1E. At month 12, mean eGFR decline from baseline was 2.3 (18 to <22 years; female, 0.9; male, 4.3), and 1.7 (22 to <26 years; female, 1.8; male, 1.6) ml/min/1.73m2; mean TKV grew 5.2% (18 to <22 years; female, 3.2%; male, 8.2%) and 6.5% (22 to < 26 years; female, 5.7%; male, 7.6%) from baseline. For HRQOL at baseline, mean SF-12v2 PCS (53.1 and 54.2) and MCS (47.6 and 48.4) were close to normal. On average, no to very mild pain was reported (mean composite pain severity of BPI, 0.9 and 0.8). Similarly, no to little impact was reported on average for IS and UIS. Results were similar for male and female patients and retained at month 12.

CONCLUSION: These data improve understanding of disease burden and natural history of ADPKD in young adults. Patients with ADPKD aged 18 to <26 experience early enlarged TKV and eGFR decline. While there is no obvious impact on HRQOL, they may benefit from treatments that reduce the growth rate of TKV and eGFR decline.

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