Impact of CFTR Modulation on Intestinal pH, Motility, and Clinical Outcomes in Patients With Cystic Fibrosis and the G551D Mutation

• DOI: 10.1038/ctg.2017.10
• Publication Date: 2017-03-16T15:53:47Z
• Authors: Daniel Gelfond; Sonya Heltshe; Changxing Ma; Steven M Rowe; Carla Frederick; Ahmet Uluer; Leonard Sicilian; Michael Konstan; Elizabeth Tullis; Christine R N Roach; Katherine Griffin; Elizabeth Joseloff; Drucy Borowitz
• Keywords: 0301 basic medicine; 03 medical and health sciences; Original Contributions

A defect in bicarbonate secretion contributes to the pathophysiology of gastrointestinal complications patients with cystic fibrosis (CF). We measured pH, clinical outcomes, and intestinal transit profiles G551D mutation before after treatment ivacaftor, a CF transmembrane regulator channel (CFTR) potentiator.Observational studies ivacaftor effectiveness were conducted United States Canada. subset subjects ingested wireless motility capsule (n=10) that measures vivo both therapy 1 month treatment; values obtained compared for mean pH area under curve, regional motility. also queried about abdominal pain recorded body weight treatment.One administering significant increase was observed gastric emptying (P<0.05). Area curve analyses indicate increased mass (P<0.05 select 5 min intervals all segments >30 min); gain 1.1 kg (P=0.08). No difference or times seen.CFTR modulation improves proximal small profile CFTR we clinically relevant, contemporaneous gain, although it did not reach statistical significance. These data provide evidence is an important secretion, which may be translational link between function improvement.