A multi-centre, randomised, controlled trial of BAL directed therapy in young children with cystic fibrosis.

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The management of children with cystic fibrosis (CF) aims to delay the inevitable progression of lung disease that results from chronic lower respiratory tract infection (LRTI) and inflammation. Flexible bronchoscopy (FB) and bronchoalveolar lavage (BAL) are increasingly used as research and clinical tools in the management of LRTI in CF infants and are integral to the monitoring of future drug trials and gene therapy. Early LRTI particularly with Pseudomonas aeruginosa (Pa) in CF is associated with a decline in pulmonary function and an increase in morbidity and mortality. Many CF centres internationally now treat young children with CF using aggressive antibiotic protocols in an attempt to eradicate infection. Most centres use oropharyngeal specimens to diagnose LRTI with a sensitivity of around 45% and specificity of around 90%. Thus many children miss out on treatment or are exposed unnecessarily to antibiotics. The use of antibiotics themselves may increase the risk of infection with resistant organisms thus complicating the design of drug trials in young children as monitoring for the emergence of resistant new organisms requires BAL. It is thus of key importance that the safety and value of FB and BAL is examined and long term outcomes are obtained. The financial costs of managing patients with CF in Australia may be estimated at more than $85 million-annum. Early intervention strategies may reduce health costs because of improved morbidity or may increase costs due to the intervention. This will be the first time an economic evaluation of early management and the cost effectiveness of an intervention in children with CF has been undertaken which will enable responsible health care planning for this important group of patients. This trial provides a unique opportunity to study the relationship between LRTI and inflammation and long term outcomes such as lung function and radiological scores and will provide key evidence for designing future trials.