Investigators: Stephen Stick, Graham Hall, Oded Breuer, Tim Rosenow, Alana Harper, Sally McCappin, Clara Mok
External collaborators: Harm Tiddens (Erasmus University Medical Centre), Robert Trengrove (Murdoch University), Conor Murray (Perth Children's Hospital)
Plain language summary:
Sensitive and reliable tests are required to monitor lung disease severity in cystic fibrosis. A number of potential methods have emerged in recent years. We will investigate how these tests track an acute episode of lung disease and determine whether, following a typical course of treatment, there is evidence of residual damage that contributes to the overall progression of lung disease.
Project description:
For patients with cystic fibrosis (CF) who are older than six years there are convincing data that suggest pulmonary exacerbations play an important role in the progressive loss of functional lung tissue. Although the impact of respiratory tract exacerbations (RTE) on the progression of CF lung disease is less well established in children less than six years old, recent evidence suggests that the negative impact of RTE on the progression of CF lung disease in young children is similar. Therefore, the frequency of RTE is a relevant clinical endpoint and has been included as an outcome measure in a large number of studies.
Despite the common use of RTE as an outcome measure, there is a poor understanding of the pathobiology of RTE and whether current approaches to for treating a RTE reduces structural lung damage in the long term. There are limited tools available to measure the various components of CF lung disease and responses to therapy. Therefore, in order to better understand the impact of RTE on CF lung disease we need to:
- Develop sensitive measures to characterize RTE and responses to treatment
- Improve our understanding of structure-function changes during treatment of RTE
Although there are numerous emerging disease biomarkers, there are limited data regarding the variability of these in association with changes in disease status imposed by a RTE. Therefore, further insight regarding the relationships between RTE and putative biomarkers is needed.