CPIC guideline for ivacaftor therapy based on CFTR genotype of cystic fibrosis patients

Cystic fibrosis (CF) therapy has traditionally been based on the treatment of symptoms and infections, rather than targeting the underlying defect caused by variants in the CFTRgene. Ivacaftor is the first FDA-approved drug to specifically target a particular defect of the CFTR protein. It is indicated for use in patients who have at least one allele carrying the G551Dvariant (rs75527207genotype AA or AG), which is present in around 4.4% of CF patients. CPIC have published therapeutic guidelines for ivacaftor based on CFTR genotype in the Clinical Pharmacology & Therapeutics journal. Interactive versions of these guidelines are available on PharmGKB, along with the manuscript and supplement. > View the guidelines> Read the article:  Clinical Pharmacogenetics ImplementationConsortium (CPIC) Guidelines for Ivacaftor Therapy in the context of CysticFibrosis Transmembrane Conductance Regulator (CFTR) Genotype.John P. Clancy, Samuel G. Johnson, Sook Wah Yee, Ellen M. McDonagh, Kelly E. Caudle, Teri E. Klein, Matthew Cannavo, Kathleen M Giacomini. Clinical Pharmacology & Therapeutics. (Accepted article preview).

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