Markedly fewer CF lung transplants since Trikafta approval: Study
Findings suggest therapy has helped improve lung health in US patients
Since its approval in 2019, treatment with Trikafta has markedly reduced the number of lung transplants among people in the U.S. with cystic fibrosis (CF), with more than 35 patients removed from a transplant waitlist the year after the therapy was approved, according to a new analysis.
Moreover, although the number of individuals on a waitlist for lung transplants has increased over the years, the number of CF patients on that list dropped substantially after Trikafta became available, the researchers found.
“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.
“The findings of this study reveal a significant shift in the landscape of lung transplantation for [people with CF] in the United States, coinciding with the approval and widespread adoption of [Trikafta] therapy,” the researchers added.
The findings, “Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States,” were published in the journal JHLT Open.
Investigating US transplant stats before, after Trikafta approval
In CF, defects in the production or function of the CFTR protein result in the abnormal buildup of thick and sticky mucus in various organs, most notably the lungs. This mucus buildup can cause airway blockages, chronic infection, and inflammation — all leading to a progressive loss of lung function and, in some cases, advanced lung disease and lung failure.
Lung transplants are indicated for CF patients with advanced lung disease whose illness fails to respond to available medical treatments, and who are not expected to live long without a transplant.
Developed by Vertex Pharmaceuticals, Trikafta is a CFTR modulator, or a medication that improves how the CFTR protein folds and functions. CF patients treated with the therapy — including those with advanced lung disease — have shown improved lung function and reduced pulmonary exacerbations, which are sudden increases in lung symptoms.
However, few clinical trials assessing CFTR modulators like Trikafta have enrolled CF patients with advanced lung disease, or those who required a transplant.
Thus, a research team at University of Iowa Healthcare conducted a study to evaluate Trikafta’s effect on the need for lung transplants among CF patients before and after its U.S. approval in October 2019.
The team accessed data from the Organ Procurement & Transplantation (OPTN) database on lung transplants performed 10 years before Trikafta’s approval — between 2009 and 2019 — and compared it with 2021-2023 data.
Lung transplants for CF patients have dropped fourfold since approval
Beginning in 2009, the number of lung transplants in the U.S. increased each year, surpassing 3,000 by 2023. On average, there were 2,085 transplants per year between 2009 and 2019, and 2,747 per year in the three years following Trikafta’s approval and assumed use.
However, among CF patients, the average number of lung transplants per year was more than four times lower in the three years after Trikafta’s approval than in the 10 years before, dropping from 243 to 56.7.
Likewise, the average number of individuals added to the lung transplant waitlist per year increased from 2,687 between 2009-2019 to 3,191 in 2021-2023. Nonetheless, the number of CF patients added to the list annually was more than five times lower after Trikafta’s approval than before, dropping from 295 to 55.6.
The sharp reduction in lung transplants and the declining number of [CF patients] added to the waitlist underscore the transformative impact of [Trikafta].
In an important change noted by the researchers, 36 CF patients were removed from the transplant waitlist in 2020 due to improved condition — the highest number reported for this reason in over a decade.
The team acknowledged that those who started CFTR modulator therapy after developing advanced lung disease may still require a transplant. Also, lung transplants will likely remain necessary for CF patients with genetic mutations that don’t respond to current CFTR modulators.
However, “the sharp reduction in lung transplants and the declining number of [CF patients] added to the waitlist underscore the transformative impact of [Trikafta],” the researchers concluded.
The researchers also called for further studies of Trikafta as time goes on, to assess the treatment’s long-term use.
“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 team wrote.
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