Early intervention studies in infants and preschool children with cystic fibrosis: are we ready?
Stephen Stick, Harm Tiddens, Paul Aurora, Per Gustafsson, Sarath Ranganathan, Paul Robinson, Margaret Rosenfeld, Peter Sly, Felix Ratjen
EUROPEAN RESPIRATORY JOURNAL | EUROPEAN RESPIRATORY SOC JOURNALS LTD | Published : 2013
Cystic fibrosis (CF) lung disease starts early in life and progresses even in the absence of clinical symptoms. Therefore, sensitive outcome measures to quantify and track these early abnormalities in infants and young children are needed; both for clinical care and interventional trials. Currently, the efficacy of most therapeutic interventions in CF has not been tested in children under the age of 6 years and drug development programmes have focused on assessing safety rather than efficacy in this age group. This article summarises the current status for outcome measures that can be utilised in clinical trials in infants and children with CF. Two methodologies are specifically highlighted ..View full abstract