Impact of availability of a highly effective cystic fibrosis treatment (elexacaftor/tezacaftor/ivacaftor) on lung transplant waitlist and lung transplantation trends in the US.

Cystic fibrosis (CF) is a genetic disease that often leads to progressive lung disease and lung transplantation. CF transmembrane conductance regulator modulators (CFTRm) improve lung function in people with CF. The US Scientific Registry of Transplant Recipients (SRTR) data were used to assess rates of lung transplant waitlisting, waitlist removal, pre-transplant mortality, and lung transplantation in people with CF (CF cohort) compared to those with other respiratory conditions (non-CF cohort) across three time periods: (i) prior to approval of any CFTRm (Pre-CFTRm era); (ii) from approval of ivacaftor to pre-approval of elexacaftor/tezacaftor/ivacaftor (Pre-ETI CFTRm era); and (iii) after approval of ETI (ETI era). Among the CF cohort, new waitlistings decreased by 78 % in ETI era compared to Pre-ETI CFTRm era while rates increased in the non-CF cohort. Among the CF cohort, waitlist removal for improving condition increased 18-fold in ETI era compared to Pre-ETI CFTRm era; rates remained stable among the non-CF cohort. Lung transplants decreased by 72 % in ETI era compared to pre-ETI CFTRm era; rates increased among the non-CF cohort. These results suggest the availability of ETI is associated with reductions in demand for lung transplants for people with CF, increasing availability of donor lungs for non-CF candidates.