Genomic diagnostics in polycystic kidney disease: an assessment of real-world use of whole-genome sequencing

Eur J Hum Genet. 2021 May;29(5):760-770. doi: 10.1038/s41431-020-00796-4. Epub 2021 Jan 12.

Abstract

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is common, with a prevalence of 1/1000 and predominantly caused by disease-causing variants in PKD1 or PKD2. Clinical diagnosis is usually by age-dependent imaging criteria, which is challenging in patients with atypical clinical features, without family history, or younger age. However, there is increasing need for definitive diagnosis of ADPKD with new treatments available. Sequencing is complicated by six pseudogenes that share 97% homology to PKD1 and by recently identified phenocopy genes. Whole-genome sequencing can definitively diagnose ADPKD, but requires validation for clinical use. We initially performed a validation study, in which 42 ADPKD patients underwent sequencing of PKD1 and PKD2 by both whole-genome and Sanger sequencing, using a blinded, cross-over method. Whole-genome sequencing identified all PKD1 and PKD2 germline pathogenic variants in the validation study (sensitivity and specificity 100%). Two mosaic variants outside pipeline thresholds were not detected. We then examined the first 144 samples referred to a clinically-accredited diagnostic laboratory for clinical whole-genome sequencing, with targeted-analysis to a polycystic kidney disease gene-panel. In this unselected, diagnostic cohort (71 males :73 females), the diagnostic rate was 70%, including a diagnostic rate of 81% in patients with typical ADPKD (98% with PKD1/PKD2 variants) and 60% in those with atypical features (56% PKD1/PKD2; 44% PKHD1/HNF1B/GANAB/ DNAJB11/PRKCSH/TSC2). Most patients with atypical disease did not have clinical features that predicted likelihood of a genetic diagnosis. These results suggest clinicians should consider diagnostic genomics as part of their assessment in polycystic kidney disease, particularly in atypical disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Child
  • Child, Preschool
  • Female
  • Gene Frequency*
  • Genetic Testing / methods*
  • Genetic Testing / standards
  • Glucosidases / genetics
  • HSP40 Heat-Shock Proteins / genetics
  • Hepatocyte Nuclear Factor 1-alpha / genetics
  • Humans
  • Infant
  • Male
  • Middle Aged
  • Polycystic Kidney Diseases / diagnosis
  • Polycystic Kidney Diseases / genetics*
  • Receptors, Cell Surface / genetics
  • Sensitivity and Specificity
  • TRPP Cation Channels / genetics
  • Tuberous Sclerosis Complex 2 Protein / genetics
  • Whole Genome Sequencing / methods*
  • Whole Genome Sequencing / standards

Substances

  • DNAJB11 protein, human
  • HNF1A protein, human
  • HSP40 Heat-Shock Proteins
  • Hepatocyte Nuclear Factor 1-alpha
  • PKHD1 protein, human
  • Receptors, Cell Surface
  • TRPP Cation Channels
  • TSC2 protein, human
  • Tuberous Sclerosis Complex 2 Protein
  • polycystic kidney disease 1 protein
  • polycystic kidney disease 2 protein
  • GANAB protein, human
  • Glucosidases