Trikafta linked to fewer hospital stays for cystic fibrosis complications
Study finds treatment tied to reduced healthcare use beyond lungs
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Trikafta was associated with significant reductions in healthcare use, including hospitalizations related to cystic fibrosis (CF) complications affecting organs and systems outside the lungs, according to an insurance claims analysis.
“Results of this study suggest a multisystemic benefit of [Trikafta] therapy with reductions in extrapulmonary complication-related healthcare resource utilization following the initiation of treatment,” the researchers wrote.
Study examines Trikafta’s effects beyond the lungs
The study, “Effect of Elexacaftor/Tezacaftor/Ivacaftor Treatment on Extrapulmonary Complications of Cystic Fibrosis,” was published in Advances in Therapy. Vertex Pharmaceuticals, the maker of Trikafta, funded the work.
CF is best known for its effects on the lungs, but it’s a disease that affects the whole body.
Complications can arise in the digestive tract, liver, pancreas, and other organ systems. Some appear as early as birth, while others, such as electrolyte (salt) imbalances, kidney stones, and bowel blockages, can occur at any age. Long-term complications include CF-related liver disease and diabetes, and osteoporosis (bone weakening).
Together, these complications and their intensive management result in significantly higher healthcare use and costs than in the general population.
CFTR modulators are a class of medicines that boost the function of CFTR, a protein that is dysfunctional or missing in people with CF. Trikafta is one such treatment approved in the U.S. for CF patients ages 2 and older who carry at least one copy of the F508del mutation, the most common CF-causing mutation, or other mutations that are responsive to the medication.
In clinical trials, treatment with the therapy led to meaningful improvements in lung function and a reduction in pulmonary flare-ups (exacerbations). Real-world data have since supported these benefits and also shown reductions in overall healthcare use and costs.
Despite these gains, research into Trikafta’s effects on complications outside the lungs, including those involving the pancreas, intestines, liver, bones, and immune and endocrine systems, is still emerging.
Researchers analyze healthcare use before and after treatment
With this in mind, a team led by scientists at Vertex used real-world healthcare claims data to examine whether Trikafta is associated with changes in healthcare use related to complications beyond the lungs.
Specifically, they compared healthcare use for extrapulmonary (outside-the-lungs) complications before and after patients started treatment. The researchers focused on seven extrapulmonary systems: the lower gastrointestinal tract (intestines), the upper gastrointestinal tract (mouth, esophagus, stomach), the hepatobiliary system (liver and bile ducts), nutritional complications and electrolyte disturbances, CF-related diabetes, and the upper respiratory tract (nose and throat).
The study included 1,612 people with CF, with a mean age of 20.7 years. Most were ages 12 or older (80.1%), male (55.5%), and covered by commercial insurance (53.6%). Just over half (56.8%) had previously been on a different CFTR modulator, most commonly Symdeko (ivacaftor/tezacaftor).
According to the analysis, a higher proportion of CF patients had at least one medical claim for any extrapulmonary complication before starting Trikafta than afterward (75% vs. 64.5%).
The treatment effect was more pronounced for hospitalizations. The share of patients admitted to hospital for at least one such complication fell from 24.6% to 8.8%, a reduction of 64.2%. The mean number of hospital days related to these complications also dropped sharply, from 5.1 days before treatment to 1.2 days after, a 76.4% reduction.
Before treatment, the most common extrapulmonary complications across all care settings were lower gastrointestinal issues (59%), upper respiratory complications (27.4%), and nutritional issues (19.4%).
After starting Trikafta, statistically significant reductions in the proportion of CF patients with at least one claim were observed for complications related to electrolytes (41.2%), the upper respiratory system (32.8%), nutrition (28.1%), and the lower gastrointestinal system (17.8%).
Fewer hospitalizations seen for complications beyond the lungs
Within hospitals specifically, reductions exceeded 50% for lower gastrointestinal, nutritional, CF-related diabetes, upper respiratory, and upper gastrointestinal complications, with nutritional complications showing the largest drop at 77.2%.
Similar patterns were observed across several subgroups, including patients ages 12 and older, those with commercial or Medicaid insurance, and those with or without prior use of CFTR modulators. Among patients with three years of follow-up, benefits appeared relatively stable across successive years of treatment, though this subgroup was small.
“Results of this analysis confirm and extend prior findings indicating that [Trikafta] improves extrapulmonary outcomes in [CF],” the researchers concluded.
“These additional benefits of [Trikafta] therapy, beyond its substantial impact on management of pulmonary complications, stand to provide both direct and indirect economic benefits as individuals with CF will experience fewer complications, thus reducing healthcare expenditures and allowing them to engage more fully in society,” the team added.
