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Genova, Italy

De Boeck K.,University Hospital of Leuven | Kent L.,University of Ulster | Davies J.,Imperial College London | Derichs N.,Charite - Medical University of Berlin | And 22 more authors.
European Respiratory Journal | Year: 2013

In patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers, such as sweat chloride concentration and/or nasal potential difference, are used as end-points of efficacy in phase-III clinical trials with the disease modifying drugs ivacaftor (VX-770), VX809 and ataluren. The aim of this project was to review the literature on reliability, validity and responsiveness of nasal potential difference, sweat chloride and intestinal current measurement in patients with cystic fibrosis. Data on clinimetric properties were collected for each biomarker and reviewed by an international team of experts. Data on reliability, validity and responsiveness were tabulated. In addition, narrative answers to four key questions were discussed and agreed by the team of experts. The data collected demonstrated the reliability, validity and responsiveness of nasal potential difference. Fewer data were found on reliability of sweat chloride concentration; however, validity and responsiveness were demonstrated. Validity was demonstrated for intestinal current measurement, but further information is required on reliability and responsiveness. For all three end-points, normal values were collected and further research requirements were proposed. This body of work adds useful information to support the promotion of CFTR biomarkers to surrogate end-points and to guide further research in the area. Copyright©ERS 2013. Source

Assael B.M.,Cystic Fibrosis Center | Assael B.M.,Centro Fibrosi Cistica | Pressler T.,Copenhagen University | Bilton D.,Royal Brompton Hospital | And 10 more authors.
Journal of Cystic Fibrosis | Year: 2013

Background: Open-label, parallel-group, international trial comparing aztreonam for inhalation solution (AZLI) and tobramycin nebulizer solution (TNS) for cystic fibrosis patients with airway Pseudomonas aeruginosa. Methods: 273 patients (≥6 years); randomized to three 28-day courses (AZLI 75. mg [three-times/day] or TNS 300 mg [twice/day]); 28 off-days separated each course. Results: 268 patients were treated (AZLI/TNS: 136/132). Mean baseline FEV1 was 52% predicted. Mean relative changes after 1 course (AZLI: 8.35%; TNS: 0.55%; p<0.001) and mean actual changes across 3 courses (AZLI: 2.05%; TNS: -0.66%; p=0.002) indicated AZLI statistical superiority vs. TNS. AZLI-treated patients had fewer respiratory hospitalizations (p=0.044) and respiratory events requiring additional antipseudomonal antibiotics (p=0.004); both treatments were well tolerated. 133 patients received 1 to 3 courses of AZLI treatment in the open-label extension-period (28-day courses separated by 28days off-treatment); lung function improvements were comparable regardless of whether patients had received TNS or AZLI in the preceding comparative period. Conclusions: AZLI demonstrated statistical superiority in lung function and a reduction in acute pulmonary exacerbations compared to TNS over 3 treatment courses (ClinicalTrials.gov: NCT00757237). © 2012 . Source

Castellania C.,Centro Fibrosi Cistica | Seiab M.,Laboratorio Of Genetica Medica
Rivista Italiana della Medicina di Laboratorio | Year: 2010

Cystic Fibrosis is an an autosomal recessive inherited disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This gene codes for a protein which is an ion transport protein in the epithelial surfaces of lungs, pancreas, liver, intestines, sweat ducts and vas deferens. The article describes and discusses prenatal, pre-conception and pre-implantation mutation analysis of the CFTR gene. Source

Tzani P.,Clinica Pneumologica | Pisi G.,Centro Fibrosi Cistica | Aiello M.,Clinica Pneumologica | Olivieri D.,Clinica Pneumologica | Chetta A.,Clinica Pneumologica
Respiration | Year: 2010

Patients with respiratory diseases may be at risk during flight because at cruising altitude an important hypobaric hypoxia may occur. The only absolute contraindications to flying in these patients are pneumothorax, bronchogenic cyst and severe pulmonary hypertension. In order to evaluate the risks related to air travel in patients with respiratory diseases, an evaluation of their fitness to fly, including the hypoxia altitude simulation test, is required. The fitness to fly evaluation can identify patients requiring supplemental oxygen during flight which is provided by most airlines when prescribed by the passenger's physician. This review deals with the cardiorespiratory effects of flight, the risks associated with respiratory diseases during air travel and the procedures to follow in order to assess fitness to fly in patients with respiratory disorders. © 2010 S. Karger AG, Basel. Source

Alicandro G.,University of Milan | Faelli N.,University of Milan | Gagliardini R.,Centro Fibrosi Cistica | Santini B.,University of Turin | And 13 more authors.
Prostaglandins Leukotrienes and Essential Fatty Acids | Year: 2013

Low plasma concentrations of docosahexaenoic acid (DHA) are reported in unsupplemented cystic fibrosis (CF) patients. Forty-one CF patients aged from 6 to 12 years were randomized to receive high-dose DHA (100. mg/kg/day in the first month and 1. g per day thereafter through a 12-month supplementation) or placebo (germ oil). Primary outcome was percentage change in plasma AA:DHA ratio. Secondary outcomes were changes in the number of pulmonary exacerbations compared to previous year, lung function, BMI, skinfold thicknesses, and body composition assessed by DXA and in serum concentrations of C-reactive protein, cytokines and vitamin (α-tocopherol and retinol). Compared to the control group plasma AA:DHA ratio decreased in the intervention group after 6 months (median percentage changes: -73% in the intervention group vs. -10% in the control group, P=0.001). No differences were detected between groups for secondary outcomes. Despite a decrease of the AA/DHA ratio, DHA supplementation for one year did not induce any significant biochemical and clinical improvement in CF patients. © 2012 Elsevier Ltd. Source

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