Clinical Pharmacogenetics Implementation Consortium (CPIC) Guidelines for Ivacaftor Therapy in the Context of CFTR Genotype. Issue 6 (5th March 2014)

• Record Type: Journal Article
• Authors: Clancy, J P; Johnson, S G; Yee, S W; McDonagh, E M; Caudle, K E; Klein, T E; Cannavo, M; Giacomini, K M
• Is Part Of: Clinical pharmacology & therapeutics. Volume 95:Issue 6(2014)
• Page Start: 592
• Page End: 597
• Publication Date: 2014-03-05
• Subjects: Pharmacology -- Periodicals; Therapeutics -- Periodicals; 615.5
• DOI: 10.1038/clpt.2014.54
• Languages: English
• ISSNs: 0009-9236

Abstract

<abstract abstract-type="main"> <title> <x xml:space="preserve">Abstract</x> </title> <p>Cystic fibrosis (CF) is a life‐shortening disease arising as a consequence of mutations within the <italic>CFTR</italic> gene. Novel therapeutics for CF are emerging that target CF transmembrane conductance regulator protein (CFTR) defects resulting from specific <italic>CFTR</italic> variants. Ivacaftor is a drug that potentiates CFTR gating function and is specifically indicated for CF patients with a particular <italic>CFTR</italic> variant, <italic>G551D‐CFTR</italic> (rs75527207). Here, we provide therapeutic recommendations for ivacaftor based on preemptive <italic>CFTR</italic> genotype results.</p> <p> <italic>Clinical Pharmacology &amp; Therapeutics</italic> (2014); <bold>95</bold> 6, 592–597. doi:<ext-link ext-link-type="doi" xlink:type="simple" xmlns:xlink="http://www.w3.org/1999/xlink">10.1038/clpt.2014.54</ext-link></p> </abstract>