Autosomal Dominant Polycystic Kidney Disease: Clinical Assessment of Rapid Progression

Am J Nephrol. 2018;48(4):308-317. doi: 10.1159/000493325. Epub 2018 Oct 22.

Abstract

Background: Autosomal dominant polycystic kidney disease (ADPKD) causes the development of renal cysts and leads to a decline in renal function. Limited guidance exists in clinical practice on the use of tolvaptan. A decision algorithm from the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Working Groups of Inherited Kidney Disorders and European Renal Best Practice (WGIKD/ERBP) has been proposed to identify candidates for tolvaptan treatment; however, this algorithm has not been assessed in clinical practice.

Methods: Eighteen-month cross-sectional, unicenter, observational study assessing 305 consecutive ADPKD patients. The ERA-EDTA WGIKD/ERBP algorithm with a stepwise approach was used to assess rapid progression (RP). Subsequently, expanded criteria based on the REPRISE trial were applied to evaluate the -impact of extended age (≤55 years) and estimated glomerular filtration rate (eGFR; ≥25 mL/min/1.73 m2).

Results: Historical eGFR decline, indicative of RP, was fulfilled in 26% of 73 patients who were candidates for RP assessment, mostly aged 31-55 years. Further tests including ultrasound and MRI measurements of kidney volume plus genetic testing enabled the evaluation of the remaining patients. Overall, 15.7% of patients met the criteria for rapid or likely RP using the algorithm, and the percentage increased to 27% when extending age and eGFR.

Conclusions: The ERA-EDTA WGIKD/ERBP algorithm provides a valuable means of identifying in routine clinical practice patients who may be eligible for treatment with tolvaptan. The impact of a new threshold for age and eGFR may increase the percentage of patients to be treated.

Keywords: Autosomal dominant polycystic kidney disease; Chronic kidney disease; Gene; Magnetic resonance imaging; Prediction; Rapid progression; Total kidney volume; Ultrasound.

Publication types

  • Research Support, Non-U.S. Gov't
  • Validation Study

MeSH terms

  • Adult
  • Age Factors
  • Algorithms
  • Antidiuretic Hormone Receptor Antagonists / therapeutic use*
  • Clinical Decision-Making / methods*
  • Cross-Sectional Studies
  • Disease Progression
  • Female
  • Glomerular Filtration Rate
  • Humans
  • Kidney / diagnostic imaging
  • Kidney / pathology
  • Kidney / physiopathology
  • Magnetic Resonance Imaging
  • Male
  • Middle Aged
  • Organ Size
  • Patient Selection*
  • Polycystic Kidney, Autosomal Dominant / drug therapy*
  • Polycystic Kidney, Autosomal Dominant / pathology
  • Predictive Value of Tests
  • Retrospective Studies
  • Tolvaptan / therapeutic use*
  • Ultrasonography

Substances

  • Antidiuretic Hormone Receptor Antagonists
  • Tolvaptan