The purpose of this study is to further characterize early CF lung disease in newborns, infants and toddlers with cystic fibrosis (CF).
Newly diagnosed patients with Cystic Fibrosis (CF). Diagnosis of CF: at least one of the following three international accepted criteria is fulfilled: i) sweat chloride ≥ 60mEq/L and/or ii) 2 CF-causing mutations in the CFTR gene and/or iii) changes typical for CF in the transepithelial potential difference in nasal or rectal epithelium.
Early diagnosed (ED): Initial diagnosis following CF-NBS or for other reasons in the first 4 months of life (in preterms corrected age of 4 months) after January 1st, 2006. Other reasons could be prenatal diagnostics, meconium ileus or positive family history.
Late diagnosed (LD): Diagnosed after the fourth month of life due to clinical symptoms; initial diagnosis after January 1st, 2006.
Preterms <30th week of gestation
Longer period of mechanical ventilation in first 3 months of life
A significant medical disease or condition other than CF likely to interfere with the child's ability to complete the entire protocol
Previous major surgery except for meconium ileus or atresia of the intestine
Patients with CF diagnosed in the first 4 months of life (corrected age of 4 months in preterms) not before January 1st, 2006 build up the early diagnosed (ED) group. Early identification can be achieved by newborn screening, clinical diagnosis (e.g. patients with meconium ileus), due to positive family history or prenatal diagnosis. Patients with CF diagnosed after the first 4 months of life and after January 1st, 2006, are included as a comparison group with clinically diagnosed patients (late diagnosed, LD). Both groups (ED and LD) are investigated after the same investigational plan with all investigations that are part of the annual check-up and additional, study-related monthly telephone interviews on bronchopulmonary symptoms, quarterly assessment of QoL and voluntarily yearly bronchoscopy with broncho-alveolar lavage.
Cohort
Prospective
Not listed
Proportion with morphological and/or perfusion changes due to CF lung disease after chest MRI score in both groups
Proportion of patients with impairments in pulmonary function tests (e.g. multiple breath washout (MBW)) in both groups
Rate of protocol-defined pulmonary exacerbations in both groups (ED vs. LD) that are necessitating an antibiotic therapy orally, intravenously or per inhalation
Spontaneous development of infection or spectrum of pathogens, respectively, in throat and nose swabs as well as other airway secretions from routine diagnostics and if applicable bronchoalveolar lavage fluid (BALF)
From the patients in whom PsA or other CF pathogens could not be isolated at the beginning of their participation, comparison of the portion of patients with a positive culture during participation in both groups (ED and LD)
Time to first detection of a CF pathogen in both groups
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Heidelberg University
Lead sponsor
German Center for Lung Research
Collaborator
