Since the approval of Trikafta in 2019, the number of lung transplants among people in the U.S. with cystic fibrosis (CF) has significantly decreased, according to a new analysis published in the JHLT Open journal. The study reveals a notable reduction in both the number of CF patients undergoing lung transplantation and those being added to the transplant waitlist.
Impact of Trikafta on Lung Transplant Trends
The research team at University of Iowa Healthcare assessed data from the Organ Procurement & Transplantation (OPTN) database, comparing lung transplant statistics from 2009-2019 (before Trikafta's approval) with data from 2021-2023. The findings indicate a significant shift in the landscape of lung transplantation for individuals with CF.
While the overall number of lung transplants in the U.S. has increased, surpassing 3,000 by 2023, the trend among CF patients tells a different story. The average number of lung transplants performed annually in CF patients dropped more than fourfold, from 243 in the pre-Trikafta era to 56.7 in the years following its approval. Similarly, the average number of CF patients added to the lung transplant waitlist each year decreased from 295 to 55.6.
Improved Patient Outcomes and Reduced Need for Transplantation
"The decline in lung transplants for [people with CF] suggests that [Trikafta] may have played an important role in improving CF lung health, potentially contributing to fewer individuals reaching advanced stages of lung disease that would necessitate transplantation," the team wrote in their publication.
Notably, in 2020, 36 CF patients were removed from the transplant waitlist due to improved condition – the highest number reported for this reason in over a decade. This observation further supports the positive impact of Trikafta on CF patient outcomes.
Understanding Cystic Fibrosis and the Role of CFTR Modulators
Cystic fibrosis is a genetic disorder characterized by defects in the CFTR protein, leading to the buildup of thick mucus in the lungs and other organs. This mucus accumulation results in airway blockages, chronic infections, and inflammation, ultimately causing progressive lung damage. Lung transplantation is often considered for CF patients with advanced lung disease who do not respond to other available treatments.
Trikafta, developed by Vertex Pharmaceuticals, is a CFTR modulator designed to improve the folding and function of the defective CFTR protein. Clinical trials have demonstrated that Trikafta can improve lung function and reduce pulmonary exacerbations in CF patients, including those with advanced lung disease.
Considerations and Future Research
The researchers acknowledge that some CF patients who started CFTR modulator therapy after developing advanced lung disease may still require a transplant. Additionally, lung transplants will likely remain necessary for CF patients with genetic mutations that do not respond to current CFTR modulators.
"Future research should focus on evaluating the long-term outcomes of [Trikafta] therapy, particularly for patients who began treatment with advanced lung disease, as well as the sustained impact on transplantation trends," the researchers concluded. They also called for further studies of Trikafta as time goes on, to assess the treatment’s long-term use.
