RT Journal Article
SR Electronic
T1 Early intervention studies in infants and preschool children with cystic fibrosis: are we ready?
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP 527
OP 538
DO 10.1183/09031936.00108212
VO 42
IS 2
A1 Stephen Stick
A1 Harm Tiddens
A1 Paul Aurora
A1 Per Gustafsson
A1 Sarath Ranganathan
A1 Paul Robinson
A1 Margaret Rosenfeld
A1 Peter Sly
A1 Felix Ratjen
YR 2013
UL http://erj.ersjournals.com/content/42/2/527.abstract
AB 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 in this review; chest computed tomography to assess structural damage of the lung and multiple breath washout as a technique to quantify ventilation inhomogeneity. While not all questions regarding the utility of these outcome measures in infants and young children have been resolved, significant advances have been made and it now appears feasible to design and conduct adequately powered efficacy studies in this age group. This could be a crucial step to further improve outcomes in CF patients as initiating effective treatment early is considered essential to prevent permanent lung damage.