CFTR modulator therapy in patients with cystic fibrosis and an organ transplant

Paediatr Respir Rev. 2018 Jun:27:6-8. doi: 10.1016/j.prrv.2018.04.003. Epub 2018 Apr 25.

Abstract

CFTR modulators are a class of drugs which directly target the defective CFTR protein in cystic fibrosis (CF), improving its function with resultant clinical improvements. Currently these drugs are confined to people with a limited selection of genetic mutations. New modulators are in development which will lead to the majority of patients with CF becoming eligible for treatment. CFTR modulators are currently considered contraindicated in patients with a solid organ transplant. This excludes many patients who may benefit from the multisystem effects of CFTR modulator treatment. In this review, we discuss issues regarding drug interactions, organ transplantation and CFTR modulation.

Keywords: CFTR modulators; Cystic fibrosis; Drug interactions; Organ transplantation.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Aminophenols / therapeutic use*
  • Aminopyridines / therapeutic use*
  • Benzodioxoles / therapeutic use*
  • Chloride Channel Agonists / therapeutic use
  • Cystic Fibrosis / complications
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / surgery*
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Drug Combinations
  • Humans
  • Male
  • Molecular Targeted Therapy*
  • Organ Transplantation*
  • Quinolones / therapeutic use*
  • Young Adult

Substances

  • Aminophenols
  • Aminopyridines
  • Benzodioxoles
  • Chloride Channel Agonists
  • Drug Combinations
  • Quinolones
  • lumacaftor, ivacaftor drug combination
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor
  • lumacaftor