Grant: Project Grant - Priority Announcement: Cystic fibrosis across the lifespan

Cystic fibrosis (CF) is a genetic disease that used to shorten lives dramatically. Thanks to better care and new medications, people with CF are now living longer than ever before. The most significant recent advance has been the introduction of highly effective CFTR modulator therapies, such as elexacaftor/tezacaftor/ivacaftor (ETI), which can treat the root cause of CF in about 90% of people with the disease. Early studies suggest that ETI has reduced deaths from CF and dramatically decreased the need for lung transplantation. However, these findings come from individual countries using different methods, making it difficult to compare results or fully understand the global impact. It is also unclear how survival trends have been affected by other factors, such as the COVID-19 pandemic or differences in healthcare systems and access to ETI. Furthermore, while fewer people may now need lung transplants, the characteristics of those still requiring transplant have changed. Transplant patients today may have different genetics, more complex medical histories, or other risk factors that could affect survival after transplant. This project will bring together CF data from 96,000 people with CF living in 45 countries to answer three important questions: (1) How has survival changed globally in the era of ETI, and do these improvements differ between countries based on access to the drug? (2) How has the profile of people receiving lung transplants changed? (3) What are the current outcomes after lung transplant, and how do they compare to the past? The results of this study will help people with CF, healthcare providers, and policy makers understand the long-term outlook for people living with CF in this new era of care. It will also help guide future research, transplant decisions, and advocacy efforts to ensure equitable care around the world.