Funded Projects

Cystic fibrosis (CF) is a genetic disorder caused by a dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is a complex chronic illness which most notably affects the lungs and GI tract. Over time there has been significant improvement in health outcomes in CF. The development of highly effective modulator therapy (HEMT) was first approved in 2012 for a minority of patients, but in late 2019 a new HEMT became available for the majority of individuals with CF changing the course of the disease. Despite advances, CF still requires a large burden of care. Individuals with CF and their caregivers are 2-3 times more likely to experience symptoms of anxiety and depression as compared to the general population. These symptoms are a concern because of the associated negative health outcomes including worse adherence, lower pulmonary function, and lower health related quality of life. This study will collect mental health and clinical data from patients with CF surrounding the approval of HEMT. The objective of this study is to assess the impact HEMT has had on various aspects of life. It is an observational study of people with CF >12 yo. Current data (post-HEMT) will be compared to previously collected data and data in the CF Foundation Registry Database. Clinical data will be gathered and recorded per standard of care at clinic visits during the study period. Survey data collected via RedCap will include depression screening (Patient Health Questionanire-9), anxiety screening (Generalized Anxiety Disorder-7 (GAD-7), quality of life (CFQR) and open-ended questions on patient’s perspectives of the impact of HEMT. Change in mental and physical health outcomes will be examined over 24 months.