Clinical Pharmacogenetics Implementation Consortium (CPIC) Guidelines for Ivacaftor Therapy in the Context of CFTR Genotype

Clinical Pharmacogenetics Implementation Consortium (CPIC) Guidelines for Ivacaftor Therapy in the Context of CFTR Genotype

Cystic fibrosis (CF) is a life‐shortening disease arising as a consequence of mutations within the CFTR gene. Novel therapeutics for CF are emerging that target CF transmembrane conductance regulator protein (CFTR) defects resulting from specific CFTR variants. Ivacaftor is a drug that potentiates C...

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Veröffentlicht in:Clinical pharmacology and therapeutics 2014-06, Vol.95 (6), p.592-597
Hauptverfasser: Clancy, J P, Johnson, S G, Yee, S W, McDonagh, E M, Caudle, K E, Klein, T E, Cannavo, M, Giacomini, K M
Format: Artikel
Sprache:eng
Schlagworte:
Aminophenols - therapeutic use
Biological and medical sciences
CPIC Guidelines
Cystic Fibrosis - drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Genetic Testing
Humans
Medical sciences
Pharmacogenetics
Pharmacology. Drug treatments
Quinolones - therapeutic use
Risk Assessment
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Zusammenfassung:Cystic fibrosis (CF) is a life‐shortening disease arising as a consequence of mutations within the CFTR gene. Novel therapeutics for CF are emerging that target CF transmembrane conductance regulator protein (CFTR) defects resulting from specific CFTR variants. Ivacaftor is a drug that potentiates CFTR gating function and is specifically indicated for CF patients with a particular CFTR variant, G551D‐CFTR (rs75527207). Here, we provide therapeutic recommendations for ivacaftor based on preemptive CFTR genotype results. Clinical Pharmacology & Therapeutics (2014); 95 6, 592–597. doi:10.1038/clpt.2014.54
ISSN:0009-9236
1532-6535
DOI:10.1038/clpt.2014.54