Recent Advances in the Management of Cystic Fibrosis
There is a large amount of work underway investigating treatment options at the molecular level and further downstream. For patients with CF aged 6 years and over, there are several new treatment options available for use and more on the horizon. But what about younger children? The problem in infants and young children with CF is the lack of sensitive and reliable clinical markers to track changes of disease in intervention trials. There are now a number of trials examining LCI as a more sensitive marker of lung disease, but this is a challenging test to perform in the preschool child and potentially needs sedation in the infant. Chest CT scans are being used as an endpoint for the first time in COMBAT-CF but require a cooperative child or general anaesthetic to ensure comparable images for use in clinical trials and involve ionising radiation. This age group is particularly important when examining the further use of the small molecule drugs and gene therapy as these could minimise the need for the end-organ treatment options within the next generation of patients with CF. Paediatricians working in CF need to address this deficiency and work together to design pragmatic and feasible clinical trials for young children, acceptable both to families and regulatory agencies.
Future Challenges
There is a large amount of work underway investigating treatment options at the molecular level and further downstream. For patients with CF aged 6 years and over, there are several new treatment options available for use and more on the horizon. But what about younger children? The problem in infants and young children with CF is the lack of sensitive and reliable clinical markers to track changes of disease in intervention trials. There are now a number of trials examining LCI as a more sensitive marker of lung disease, but this is a challenging test to perform in the preschool child and potentially needs sedation in the infant. Chest CT scans are being used as an endpoint for the first time in COMBAT-CF but require a cooperative child or general anaesthetic to ensure comparable images for use in clinical trials and involve ionising radiation. This age group is particularly important when examining the further use of the small molecule drugs and gene therapy as these could minimise the need for the end-organ treatment options within the next generation of patients with CF. Paediatricians working in CF need to address this deficiency and work together to design pragmatic and feasible clinical trials for young children, acceptable both to families and regulatory agencies.