Boston, MA – Treatment with Trikafta is associated with substantial reductions in healthcare use among people with cystic fibrosis (CF), including fewer hospitalizations for complications affecting organs beyond the lungs, according to a new analysis of insurance claims data.
“Results of this study suggest a multisystem benefit of Trikafta therapy, with reductions in healthcare resource use related to extrapulmonary complications after treatment initiation,” the researchers reported.
Looking Beyond the Lungs
The study, “Effect of Elexacaftor/Tezacaftor/Ivacaftor Treatment on Extrapulmonary Complications of Cystic Fibrosis,” published in Advances in Therapy and funded by Vertex Pharmaceuticals, explored how Trikafta impacts the many systems affected by CF.
While CF is widely recognized as a lung disease, it is in fact a multisystem condition. It can affect the digestive tract, liver, pancreas, and other organs. Some complications appear early in life, while others—such as electrolyte imbalances, kidney stones, or intestinal blockages—can develop later. Long-term issues may include CF-related diabetes, liver disease, and osteoporosis.
These complications often require intensive care, contributing to significantly higher healthcare use and costs compared with the general population.
Trikafta and CFTR Modulators
Trikafta belongs to a class of treatments known as CFTR modulators, which improve the function of the defective CFTR protein in people with CF. It is approved in the U.S. for patients aged 2 and older who carry at least one copy of the F508del mutation—the most common CF-causing genetic variant—or other responsive mutations.
Clinical trials have already shown that Trikafta improves lung function and reduces pulmonary exacerbations. Real-world evidence has reinforced these findings, also pointing to lower overall healthcare use and costs.
However, its impact on complications outside the lungs has been less well understood—until now.
Study Design and Patient Population
To address this gap, researchers analyzed real-world healthcare claims from 1,612 individuals with CF, with an average age of 20.7 years. Most participants were aged 12 or older, and just over half had previously used another CFTR modulator, most commonly Symdeko (ivacaftor/tezacaftor).
The team compared healthcare use before and after patients began Trikafta, focusing on seven extrapulmonary systems:
- Lower gastrointestinal tract (intestines)
- Upper gastrointestinal tract (mouth, esophagus, stomach)
- Hepatobiliary system (liver and bile ducts)
- Nutritional complications and electrolyte imbalances
- CF-related diabetes
- Upper respiratory tract (nose and throat)
Significant Reductions in Complications and Hospitalizations
After starting Trikafta, the proportion of patients with at least one healthcare claim for extrapulmonary complications dropped from 75% to 64.5%.
The most striking improvements were seen in hospitalizations:
- The percentage of patients hospitalized for these complications fell from 24.6% to 8.8%—a 64.2% reduction
- Average hospital days decreased from 5.1 to 1.2 days—a 76.4% drop
Before treatment, the most common complications were:
- Lower gastrointestinal issues (59%)
- Upper respiratory complications (27.4%)
- Nutritional problems (19.4%)
Following treatment, statistically significant reductions were observed in:
- Electrolyte-related complications (−41.2%)
- Upper respiratory issues (−32.8%)
- Nutritional complications (−28.1%)
- Lower gastrointestinal problems (−17.8%)
Broad and Consistent Benefits
Within hospital settings, reductions exceeded 50% for several complication types, including gastrointestinal, nutritional, diabetes-related, and upper respiratory conditions. Nutritional complications showed the largest decline, at 77.2%.
These benefits were consistent across multiple patient groups, including those with different insurance types and those with or without prior CFTR modulator use. Among patients followed for three years, improvements appeared stable over time, although this subgroup was relatively small.
Implications
The findings reinforce and expand previous evidence that Trikafta provides benefits beyond lung health in CF.
“These additional benefits…may translate into both direct and indirect economic advantages,” the researchers noted. “By reducing complications and healthcare utilization, patients may face lower medical costs and gain greater ability to participate in daily life.”
Contact
Vertex Pharmaceuticals