A semiquantitative MRI-Score can predict loss of lung function in patients with cystic fibrosis: Preliminary results.

Objectives: To evaluate the applicability of a semiquantitative MRI scoring system (MR-CF-S) as a prognostic marker for clinical course of cystic fibrosis (CF) lung disease.

Methods: This observational study of a single-centre CF cohort included a group of 61 patients (mean age 12.9 ± 4.

Long-Term Pulmonal Therapy of Cystic Fibrosis-Patients with Amitriptyline.

Background/aims: Several recent clinical studies revealed an accumulation of ceramide in bronchial epithelial cells of patients with cystic fibrosis (CF). Degradation of ceramide concentrations in lungs of CF patients employing the functional acid sphingomyelinase inhibitor amitriptyline revealed a benefit in lung function, weight and exacerbation rates.

Methods: To test for a beneficial effect of amitriptyline in vivo, we performed two phase II randomised, double-blind, placebo-controlled studies.

Microbial colonization and lung function in adolescents with cystic fibrosis.

With intensified antibiotic therapy and longer survival, patients with cystic fibrosis (CF) are colonized with a more complex pattern of bacteria and fungi. However, the clinical relevance of these emerging pathogens for lung function remains poorly defined. The aim of this study was to assess the association of bacterial and fungal colonization patterns with lung function in adolescent patients with CF.

Regulatory T-cell impairment in cystic fibrosis patients with chronic pseudomonas infection.

Rationale: Patients with cystic fibrosis (CF) lung disease have chronic airway inflammation driven by disrupted balance of T-cell (Th17 and Th2) responses. Regulatory T cells (Tregs) dampen T-cell activation, but their role in CF is incompletely understood.

Objectives: To characterize numbers, function, and clinical impact of Tregs in CF lung disease.

Expression and regulation of interferon-related development regulator-1 in cystic fibrosis neutrophils.

A genome-wide association study identified interferon-related development regulator-1 (IFRD1), a protein expressed by neutrophils, as a key modifier gene in cystic fibrosis (CF) lung disease. Here, we investigated the expression and regulation of IFRD1 in CF neutrophils. IFRD1 expression was quantified in peripheral blood and airway neutrophils from patients with CF, patients with non-CF lung disease, and healthy control subjects.

Noninvasive cardiac output measurement at rest and during exercise in pediatric patients after interventional or surgical atrial septal defect closure.

In the majority of patients, secundum atrial septal defects (ASDs) are treated interventionally or surgically, before the onset of clinical symptoms, between 3 and 6 years of age. Because right-ventricular dimensions usually normalize after ASD closure, it has been assumed that cardiac function and exercise performance also normalize at long-term follow-up. The aim of our study was to determine cardiac index (CI) at rest and during exercise at medium-term follow-up of children who had undergone surgical or interventional closure of ASD because no such reports have been published thus far.

Clinical and in vitro effect of dornase alfa in mechanically ventilated pediatric non-cystic fibrosis patients with atelectases.

Introduction: At present no evidence-based medical treatment for persistent atelectasis in pediatric non-cystic fibrosis (CF) patients is available.

Method: To evaluate the use of intratracheally instilled recombinant human deoxyribonuclease (rhDNase) in intubated and ventilated pediatric patients, we performed a single-center observational study on 46 pediatric intensive care patients who had received intratracheal DNase. Patients were classified, according to radiologic findings of atelectasis (group 1) or infiltrates.

DNA quantification and fragmentation in sputum after inhalation of recombinant human deoxyribonuclease.

Background: Inhaled rhDNase may improve sputum viscosity and mucociliary clearance by cleavage of extracellular DNA derived for instance from dead leukocytes in purulent, highly viscous patient sputum.

Methods: Here we established a method to quantify rhDNase-mediated DNA fragmentation in sputum using gel electrophoresis. Sputum of Pseudomonas aeruginosa colonized cystic fibrosis (CF) patients with (CF+) or without (CF-) rhDNase treatment or mechanically ventilated non-CF patients receiving rhDNase (non-CF+) or not (non-CF-) was analyzed.

Recombinant human deoxyribonuclease shortens ventilation time in young, mechanically ventilated children.

Recombinant human deoxyribonuclease I (dornase alfa) is currently used as an inhaled mucoactive agent in the treatment of cystic fibrosis. In a randomized, placebo-controlled, double-blind clinical study in 100 infants, we investigated whether the therapeutic use of dornase alfa can be extended to ventilated, fluid-restricted children to reduce reintubation rate, ventilation duration, pediatric intensive care unit (PICU) stay, and ventilation complications. While reintubation rates were similar for dornase alfa 7% vs.