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 J Schwarzenberg

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

Background: 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. Aim: To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation. Methods: 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). Results: Studies included 213 patients (aged ≤ 20 years, n = 105; aged > 20 years, n = 108). In patients ≤20 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/m2 (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. Conclusions: Nutritional status improved following treatment with ivacaftor for 48 weeks.

Original languageEnglish (US)
Pages (from-to)198-207
Number of pages10
JournalDigestive Diseases and Sciences
Volume61
Issue number1
DOIs
StatePublished - Jan 1 2016

Fingerprint

Nutritional Status
Cystic Fibrosis
Mutation
Weights and Measures
Body Mass Index
Placebos
Body Weight
Therapeutics
Cystic Fibrosis Transmembrane Conductance Regulator
Lung
Body Weight Changes
Aptitude
Sweat
Body Image
Ion Transport
Weight Gain
Chlorides
Eating
ivacaftor
Surveys and Questionnaires

Keywords

  • Bicarbonate
  • Cystic fibrosis transmembrane conductance regulator
  • Growth
  • Kalydeco
  • Potentiator
  • Weight gain

Cite this

Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor. / Borowitz, Drucy; Lubarsky, Barry; Wilschanski, Michael; Munck, Anne; Gelfond, Daniel; Bodewes, Frank; Schwarzenberg, Sarah J.

In: Digestive Diseases and Sciences, Vol. 61, No. 1, 01.01.2016, p. 198-207.

Research output: Contribution to journalArticle

Borowitz, Drucy ; Lubarsky, Barry ; Wilschanski, Michael ; Munck, Anne ; Gelfond, Daniel ; Bodewes, Frank ; Schwarzenberg, Sarah J. / Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor. In: Digestive Diseases and Sciences. 2016 ; Vol. 61, No. 1. pp. 198-207.
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AU - Gelfond, Daniel

AU - Bodewes, Frank

AU - Schwarzenberg, Sarah J

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