Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor

Drucy Borowitz*, Barry Lubarsky, Michael Wilschanski, Anne Munck, Daniel Gelfond, Frank Bodewes, Sarah Jane Schwarzenberg

*Corresponding author for this work

    Research output: Contribution to journalArticleAcademicpeer-review

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    Abstract

    The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability.

    To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged a parts per thousand yen6 years with CF and the G551D mutation.

    Patients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).

    Studies included 213 patients (aged a parts per thousand currency sign 20 years, n = 105; aged > 20 years, n = 108). In patients a parts per thousand currency sign20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p <0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p <0.0001). In patients > 20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight.

    Nutritional status improved following treatment with ivacaftor for 48 weeks.

    Original languageEnglish
    Pages (from-to)198-207
    Number of pages10
    JournalDigestive Diseases and Sciences
    Volume61
    Issue number1
    DOIs
    Publication statusPublished - Jan-2016

    Keywords

    • Weight gain
    • Growth
    • Cystic fibrosis transmembrane conductance regulator
    • Potentiator
    • Bicarbonate
    • Kalydeco
    • TRANSMEMBRANE CONDUCTANCE REGULATOR
    • QUALITY-OF-LIFE
    • BICARBONATE SECRETION
    • ENERGY-EXPENDITURE
    • FAT MALABSORPTION
    • CFTR POTENTIATOR
    • FECAL ELASTASE-1
    • CHILDREN
    • THERAPY
    • DISEASE

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