Clinical trials in infants with cystic fibrosis detected following newborn screening

Despite the wide implementation of newborn screening for cystic fibrosis there have been few clinical trials aimed at reducing the burden of lung disease in screened infants. Emerging tools such as infant lung function and low-dose computed tomography (CT) provide early indicators of lung disease and could be effective outcome measures in randomized controlled trials (RCTs) of interventions that aim to delay or prevent the onset of bronchiectasis. The most recent data suggest that strategies to reduce neutrophilic inflammation and prevent infection are good candidates for RCTs. However, cooperation between centres is needed if they are to be large enough to detect differences due to the intervention that otherwise may be masked by subtle differences in management practices between centres, and to detect rare but significant adverse effects.