Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
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Randomized Controlled Trial Multicenter Study
Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data.
We examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiator, in cystic fibrosis (CF) patients with aG551D mutation to evaluate standardized approaches to sweat chloride measurement and to explore the use of sweat chloride and nasal potential difference (NPD) to estimate CFTR activity. ⋯ Sweat chloride is useful in multicenter trials as a biomarker of CFTR activity and to test the effect of CFTR potentiators.
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Comparative Study
HRCT and MRI of the lung in children with cystic fibrosis: comparison of different scoring systems.
Chest imaging is essential in the assessment of respiratory disease in cystic fibrosis (CF). High-resolution computed tomography (HRCT) can detect progressive lung disease but involves significant delivered dose of ionizing radiation. Magnetic resonance imaging (MRI) is radiation-free but is rarely used in CF. Based on the limited information on the potential interest of chest MRI in CF pediatric patients, the aims of our study were: 1) to evaluate and compare the reproducibility of HRCT and MRI scores; and 2) to evaluate the agreement between HRCT and MRI scores using both Helbich and Eichinger scores. ⋯ We showed that, in CF children, MRI could adequately visualize lung morphologic changes when compared with the "gold-standard" HRCT. Regarding the potential cancer risks from associated ionizing radiation with HRCT, these results lead us to propose larger intervals of time between two lung HRCTs with realization of lung MRI in the meantime.