Telomere-related lung fibrosis is diagnostically heterogeneous but uniformly progressive

Eur Respir J. 2016 Dec;48(6):1710-1720. doi: 10.1183/13993003.00308-2016. Epub 2016 Aug 18.

Abstract

Heterozygous mutations in four telomere-related genes have been linked to pulmonary fibrosis, but little is known about similarities or differences of affected individuals.115 patients with mutations in telomerase reverse transcriptase (TERT) (n=75), telomerase RNA component (TERC) (n=7), regulator of telomere elongation helicase 1 (RTEL1) (n=14) and poly(A)-specific ribonuclease (PARN) (n=19) were identified and clinical data were analysed.Approximately one-half (46%) had a multidisciplinary diagnosis of idiopathic pulmonary fibrosis (IPF); others had unclassifiable lung fibrosis (20%), chronic hypersensitivity pneumonitis (12%), pleuroparenchymal fibroelastosis (10%), interstitial pneumonia with autoimmune features (7%), an idiopathic interstitial pneumonia (4%) and connective tissue disease-related interstitial fibrosis (3%). Discordant interstitial lung disease diagnoses were found in affected individuals from 80% of families. Patients with TERC mutations were diagnosed at an earlier age than those with PARN mutations (51±11 years versus 64±8 years; p=0.03) and had a higher incidence of haematological comorbidities. The mean rate of forced vital capacity decline was 300 mL·year-1 and the median time to death or transplant was 2.87 years. There was no significant difference in time to death or transplant for patients across gene mutation groups or for patients with a diagnosis of IPF versus a non-IPF diagnosis.Genetic mutations in telomere related genes lead to a variety of interstitial lung disease (ILD) diagnoses that are universally progressive.

Publication types

  • Observational Study
  • Research Support, N.I.H., Extramural

MeSH terms

  • Aged
  • DNA Helicases / genetics
  • Exoribonucleases / genetics
  • Female
  • Humans
  • Idiopathic Pulmonary Fibrosis / diagnosis*
  • Idiopathic Pulmonary Fibrosis / genetics*
  • Idiopathic Pulmonary Fibrosis / surgery
  • Linear Models
  • Lung Transplantation
  • Male
  • Middle Aged
  • Mutation
  • RNA / genetics
  • Telomerase / genetics
  • Telomere / genetics*
  • Texas
  • Tomography, X-Ray Computed

Substances

  • telomerase RNA
  • RNA
  • TERT protein, human
  • Telomerase
  • Exoribonucleases
  • poly(A)-specific ribonuclease
  • RTEL1 protein, human
  • DNA Helicases