About Autosomal Dominant Polycystic Kidney Disease
Autosomal dominant polycystic kidney disease is the most common inherited kidney disorder, caused by mutations in PKD1 or PKD2 encoding polycystin-1 and polycystin-2 respectively, leading to progressive bilateral renal cyst development and enlargement. It is the fourth leading cause of end-stage renal disease globally, with extrarenal manifestations including intracranial aneurysms, hepatic cysts, and mitral valve prolapse. PKD1 mutations cause a more severe phenotype than PKD2, with earlier onset of renal failure.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Autosomal Dominant Polycystic Kidney Disease trials.
Total kidney volume (TKV) measured by MRI or CT is the primary imaging biomarker and is used for Mayo Clinic Classification (class 1A–1E); document TKV and height-adjusted TKV before applying.
Tolvaptan eligibility criteria typically require rapidly progressive disease (Mayo class 1C–1E or historical TKV growth >5% per year); gather longitudinal imaging if available.
Current eGFR and rate of decline over the preceding 1–3 years are key eligibility metrics; ensure creatinine history is compiled from your GP or nephrologist.
Patient Resources
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