Investigators
A/Prof Kathryn Ramsey, Dr Adelaide Withers, Crystal Bourke, Alana Harper, Faith Mhembere, Tabitha Cleary, Kitty Obando
Project description
Cystic fibrosis is an inherited condition that results in chronic lung disease. In recent years, a new type of medication called CFTR modulators has become available. CFTR modulators correct the malfunctioning protein that causes cystic fibrosis and help the lung cells to function more normally.
Modulator treatments have significantly improved lung function and quality of life in adults and adolescents with cystic fibrosis. In Australia, these modulators have now been approved under the Pharmaceutical Benefits Scheme for children aged 2 and older who have at least one responsive CFTR gene mutation, including those with ultra-rare variants
Early modulator therapy in the first years of life has the potential to prevent the development of lung disease. The aim of this study is to understand changes in lung structure and function during the preschool years in children with and without modulator therapy.
Study design
This is a prospective, longitudinal cohort study of children with cystic fibrosis aged 2-6 years of age attending Perth Children’s Hospital. Specifically, we aim to:
- Determine whether lung structure and function are normal in preschool children with cystic fibrosis on modulator therapy from early preschool age
- Characterise the burden of disease and impact of quality of life
- Examine the impact of adherence to treatments on lung health outcomes
- Examine longitudinal associations between lung function, structural disease, respiratory events, patient/parent-reported outcomes, treatment adherence, and medical history
These data will inform the cystic fibrosis community of the long-term impacts of early modulator therapy and provide evidence for updated clinical care guidelines for this population.
Eligibility
All preschool-aged children with a cystic fibrosis attending Perth Children’s Hospital, with or without modulator therapy.
For more information, please contact CFResearch@thekids.org.au
Funders
National Health and Medical Research Council (NHMRC)