Trikafta helps cystic fibrosis patients reduce supportive treatments: Survey
Researchers back development of comprehensive recommendations
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A new survey conducted in Germany showed that, in addition to easing symptoms and improving quality of life, Trikafta enables most patients with cystic fibrosis (CF) to reduce the use of supportive therapies, according to a study.
However, this is being done without clear guidelines. “We think it best to develop comprehensive recommendations for both individuals with CF and their healthcare providers, guiding them toward optimal, individualized therapy approaches,” researchers wrote.
The study, “Discontinuation of supportive therapies in people with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor – A patient-reported outcomes’ study,” was published in the journal Respiratory Medicine.
Effect of modern medications on treatment burden a key topic of interest
Hallmark CF symptoms include breathing and digestive issues. Many people with CF also experience exocrine pancreatic insufficiency, when the pancreas cannot release enough digestive enzymes. CF is caused by mutations in the CFTR gene, which lead to the production of no or faulty CFTR, a protein that normally helps regulate the movement of certain salt molecules and water in and out of cells.
CFTR modulators are a class of treatments that aim to increase the protein’s function in people with specific CFTR mutations. In 2019, Trikafta became the first approved triple CFTR modulator therapy in the U.S., following clinical trial data that showed significant benefits, including improved lung function, better nutritional status, and reduced respiratory and digestive symptoms.
Whether modern medications can reduce the daily treatment burden associated with supportive therapies has become a central question in CF, as surveys have shown that such a reduction is a key priority for these patients. However, formal guidance remains limited. And because Trikafta does not fully restore CFTR function and long-term effects of reducing supportive therapies remain uncertain, experts have urged caution.
“Currently, the extent to which pwCF [people with CF] are reducing supportive therapies, in collaboration with their physicians or independently, remains unknown and would provide a valuable source of information for physicians to counsel pwCF accordingly,” the scientists wrote.
German researchers surveyed 400 CF patients
To better understand whether — and to what extent — people with CF are reducing their supportive therapies, researchers in Germany surveyed patients registered with the German Cystic Fibrosis Association.
A total of 400 participants were included in the analysis (58% women; 32% over age 40). Most participants (76%) were using or had previously used CFTR modulators, with Trikafta being the most commonly used (91%). The majority of these patients were using the medication for more than two years.
Relative to patients not treated with any CFTR modulator, those on Trikafta reported a significantly lower treatment burden (6.5% versus 18.8%). While severe burden was similar between the groups (3.3% vs. 3.1%), people on Trikafta were more likely to report no treatment burden at all (17.8% vs. 6.2%).
Participants who did not use modulators more frequently expressed a need to increase their treatment regimen — including antibiotics, high-calorie foods, inhalations, physiotherapy, insulin, and pancreatic enzymes — when compared with those on Trikafta.
Overall, respondents on Trikafta reported improvements in quality of life, physical abilities, and daily tasks, and nearly half experienced no side effects. Trikafta eased cough in 93% and sputum (phlegm) in 91% — the most pronounced improvements reported. Bowel symptoms, however, remained unchanged or worsened for some.
Notably, 80.4% of Trikafta users reported they had reduced at least one supportive therapy. The greatest decreases — more than 50% — were seen in mucus-thinning inhalations and systemic (body-wide) antibiotics. Inhaled antibiotics, physiotherapy, and high-calorie diets were reduced by about one-third. Smaller reductions were reported for insulin (14.4%) and pancreatic enzymes (11.7%).
Supportive treatment reductions generally linked to decreased symptoms
After starting Trikafta, 59.6 % of patients reported that they no longer needed intravenous, or into-the-vein, antibiotics, and 55.2% said they required fewer hospital stays. However, only 2.8% were able to stop pancreatic enzyme replacement.
Supportive treatment reductions were generally linked to decreased symptoms, with adults, especially those ages 30 to 40, more likely to reduce inhaled antibiotic usage than teenagers (49% vs. 21%).
People with better lung function — a percent predicted forced expiratory volume in 1 second (ppFEV1) above 50% — were significantly more likely to reduce mucus-thinning treatments when compared with those with a ppFEV1 of 30% to 50%. Participants whose ppFEV1 was higher than 80% were significantly less likely to reduce inhaled antibiotics than all other groups. Notably, ppFEV1 measures the amount of air that can be forcefully exhaled in one second.
We suggest that decisions regarding the (dis-)continuation of supportive therapies are influenced by multiple factors beyond physicians’ recommendations, including the desire to reduce therapy burden, the wish to improve health, and responses to alleviated symptoms.
Quality of life increased along with lower use of inhaled antibiotics. Results also showed that the likelihood of systemic antibiotic reduction decreased as therapy burden increased.
Overall, the survey confirms the positive impact of Trikafta on symptoms and day-to-day life for people with CF, while adding to the existing evidence of CF patients already reducing their supportive therapies.
“We suggest that decisions regarding the (dis-)continuation of supportive therapies are influenced by multiple factors beyond physicians’ recommendations, including the desire to reduce therapy burden, the wish to improve health, and responses to alleviated symptoms,” the team concluded.
“Our study does not aim to make recommendations regarding the continuation or discontinuation of supportive therapies,” the researchers noted, “and it does not imply that such recommendations should be included in future guidelines. It is important to keep in mind that discontinuation of supportive therapies may carry significant risks.”
