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Purification of the Cystic Fibrosis Transmembrane Conductance Regulator Protein Expressed in Saccharomyces cerevisiae
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A Review on the Use of Cystic Fibrosis Transmembrane Conductance Regulator Gene Modulators in Pediatric Patients

Michael Bitonti, Laura Fritts, Tsz-Yin So

    Journal of Pediatric Health Care : Official Publication of National Association of Pediatric Nurse Associates & Practitioners
    |April 29, 2019
    PubMed

    Insights

    New cystic fibrosis transmembrane conductance regulator (CFTR) targeted therapies are showing promise in pediatric patients. Research is expanding to include younger children and various CFTR mutations for broader treatment benefits.

    Area of Science:

    • Pediatric Pulmonology
    • Pharmacogenomics
    • Genetic Medicine

    Background:

    • Cystic Fibrosis (CF) management traditionally focused on symptom control and infection prevention.
    • Recent advancements shift treatment paradigms towards addressing the underlying genetic defect in CF.

    Purpose of the Study:

    • To review clinical studies on CFTR-targeted pharmacotherapies in pediatric populations.
    • To assess the safety and efficacy of specific CFTR modulators in younger patients.

    Main Methods:

    • Review of clinical trial data for ivacaftor, ivacaftor/lumacaftor, and ivacaftor/tezacaftor.
    • Analysis of studies involving pediatric and adolescent patient groups.

    Main Results:

    • CFTR-targeted therapies, initially studied in adults, are increasingly evaluated in pediatric cohorts.
    • Evidence is emerging for the safety and efficacy of these modulators in younger individuals.

    Conclusions:

    • CFTR-targeted pharmacotherapies offer a new approach to treating cystic fibrosis by addressing the genetic cause.
    • Further research in diverse pediatric age groups and CFTR mutations will broaden therapeutic access.

    Keywords:
    Cystic fibrosiscystic fibrosis transmembrane conductance regulatorpediatrics

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