ADPKD in Young Adults: Burden and Progression

By Victoria Socha - Last Updated: April 11, 2023

Health-related quality of life (HRQoL) is adversely affected in patients with autosomal dominant polycystic kidney disease (ADPKD) as the disease progresses. Eric Davenport, MEcon, MStat, and colleagues conducted an analysis to describe disease burden and progression of ADPKD in young adults.

Results were reported during a poster session at the National Kidney Foundation Spring Clinical Meetings 2023. The poster was titled Disease Burden and Disease Progression of Young Adult Patients With Autosomal Dominant Polycystic Kidney Disease.

The analysis included data from the OVERTURE (NCT01430494) study. OVERTURE was an observational, longitudinal study of patients with ADPKD that assessed total kidney volume (TKV), estimated glomerular filtration rate (eGFR), and other disease outcomes, as well as patient-reported measures of HRQoL. Measures of HRQoL were obtained using the 12-Item Short Form Health Survey (SF-12v2) Mental and Physical Component Summaries (MCS and PCS) and the Brief Pain Inventory (BPI) and ADPKD-specific questionnaires (ADPKD Impact Scale [ADPKD-IS] and ADPKD Urinary Impact Scale [ADPKD-UIS]). The data were summarized by age group, sex, and number of visits.

The analysis cohort included 93 eligible patients 18 to <22 years of age and 132 patients 22 to <26 years of age at baseline. In each group, approximately 60% were female. Kidney function was normal for most patients in both groups (mean eGFR, 115.2 and 107.4 mL/min/1.73 m2); TKV was enlarged (mean, 814.7 and 980.9 mL). Close to half of the patients (48.8% and 50.4%) were in Mayo class 1D to 1E.

At month 12, mean decline in eGFR from baseline was 2.3 mL/min/1.73 m2 in the 18 to <22 group (female, 0.9; male, 4.3) and 1.7 mL/min/1.73 m2 in the 22 to <26 group (female, 1.8; male, 1.6). Mean TKV grew 5.2% in the 18 to <22 group (female, 3.2%; male, 8.2%) and 6.5% in the 22 to <26 group (female, 5.7%; male, 7.6%) from baseline.

At baseline, mean scores on the SF-12v2 MCS (47.6 and 48.4) and PCS (53.1 and 54.2) were close to normal. On average, participants reported no-to-very-mild pain (mean composite pain severity of BPI, 0.9 and 0.8). On average, participants reported no-to-little impact on average for ADPKD-IS and ADPKD-UIS. Results were similar for male and female patients and retained at month 12.

In conclusion, the authors said, “These data improve understanding of disease burden and natural history of ADPKD in young adults. Patients with ADPKD ages 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 and eGFR decline.”

Source: Davenport E, Zhou X, Wang J, et al. Disease burden and disease progression of young adult patients with autosomal dominant polycystic kidney disease. Poster #351. Abstract of a poster presented at the National Kidney Foundation Spring Clinical Meetings; April 11-15, 2023; Austin, Texas.

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