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February 12, 2024
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Outcomes reported by adults with autosomal dominant PKD predicted hospitalizations

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Key takeaways:

  • Patients with poor baseline autosomal dominant polycystic kidney disease scores were more likely to experience symptoms.
  • Severe chronic kidney disease stages showed a connection with worse scores.

Patient-reported outcome scores effectively predicted clinical and health-economic outcomes in adults with autosomal dominant polycystic kidney disease, a study found.

“In autosomal dominant polycystic kidney disease (ADPKD), the formation and growth of fluid-filled cysts causes increased kidney volume and gradual loss of kidney function,” Dorothee Oberdhan, MS, of Otsuka Pharmaceutical Development and Commercialization Inc. in Rockville, Maryland, wrote. “Disease progression is associated with burdensome and anxiety-inducing symptoms, such as kidney pain, gross hematuria and urinary tract infection, as well as systemic complications, such as hypertension. In advanced disease, patients experience severe chronic kidney disease and, ultimately, kidney failure.”

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Patients with poor baseline autosomal dominant polycystic kidney disease scores were more likely to experience symptoms. Source: Adobe Stock.

Researchers conducted a prospective observational study analyzing data from the observational Overture study to characterize disease impact on patients’ quality of life and to determine if perceived burden may predict outcomes. Overall, 3,409 adults with ADPKD aged 12 to 78 years in CKD stages G1 to G5 and Mayo risk subclasses 1A to 1E were included.

Patient-reported outcome tools, such as the ADPKD-Impact Scale (ADPKD-IS) and ADPKD-Urinary Impact Scale, as well as generic measures, were used to assess scores. Investigators also evaluated clinical variables, such as height-adjusted total kidney volume (htTKV), eGFR, abdominal girth and health-economic outcomes. The study involved patients from 20 countries.

Findings showed that baseline CKD stage and Mayo risk classification had little correlation with baseline patient-reported outcome (PRO) scores, but more severe CKD stages did demonstrate a connection with worse scores on disease-specific instruments and measures of physical functioning.

PRO scores also predicted hospitalizations and sick days at 6 through 18 months, with the ADPKD-IS having the strongest associations. Although scores were not associated with htTKV and eGFR, poor scores were linked to greater abdominal girth.

Patients with poor baseline ADPKD-IS scores were more likely to experience ADPKD-related symptoms up to 18 months, including kidney pain, hematuria and urinary tract infection, according to the study.

PRO scores “predicted clinical and health-economic outcomes, such as hospitalization and absence from work, underscoring the importance of quality of life assessment of individuals with ADPKD,” Oberdhan and colleagues wrote.