Trikafta Aids Quality of Life With CF in Multitude of Ways: Study

Phase 3 trials show gains in overall health and well-being beyond lung function

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Trikafta (elexacaftor/tezacaftor/ivacaftor) may bring health-related quality of life benefits to people with cystic fibrosis (CF) that go beyond those already reported for breathing, a study found.

This expectation stems from a new look by an international team of researchers into data from the two Phase 3 clinical trials that notched Trikafta’s U.S. approval. They indicate gains in quality of life that are both general and specific to the disease.

“These findings demonstrate that [Trikafta] improves a range of CF-specific symptoms and general functioning and well-being,” the researchers wrote.

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The study, “Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor,” was published as a short communication in the Journal of Cystic Fibrosis.

CF is caused by mutations in the CFTR gene, which codes for a protein of the same name. The mutations result in no CFTR being made, an unstable protein being produced in low amounts, or a misfolded CFTR protein that does not work the way it should.

When this happens, a sticky mucus builds in the lungs and other organs, causing breathing problems, increasing the risk of lung infections, and making digestion hard.

Trikafta, patients report, helps in social, physical and emotional ways

Vertex Pharmaceuticals’ Trikafta — sold as Kaftrio in Europe — is a triple combination of elexacaftor and tezacaftor, which help CFTR fold correctly, and ivacaftor, a potentiator that helps the protein work in a more effective way.

These actions combine to make mucus less thick and help to ease disease symptoms.

Its initial approval was for patients ages 12 and older who have at least one copy of the F508del mutation, which is the most common type of mutation causing CF. The approval was later expanded to cover other mutations and children starting at 6 years old.

The two pivotal AURORA program trials, in patients ages 12 and older, showed that taking Trikafta significantly improved lung function compared to one or two of the medications taken separately or to a placebo. Its use brought about better quality of life related to breathing, as seen by an increase in the scores of the respiratory domain of the Cystic Fibrosis Questionnaire–Revised (CFQ-R).

The CFQ-R is a CF-specific tool designed to measure patient perceptions of how the disease changes overall health, perceived well-being, and symptoms.

The researchers, with institutions in the U.S and Europe and at Vertex, looked at how these patients scored across the 11 non-respiratory domains of the CFQ-R. These domains cover general health-related quality of life (vitality, health perceptions, and physical, role, emotional, and social functioning) as well as those specific to the disease — body image, eating problems, treatment burden, weight, and digestive symptoms. Scores range from 0 to 100 for each domain, with higher scores indicating better quality of life.

Their study drew on data from 403 patients (194 female and 209 male) who carried one copy of the F508del mutation plus one copy of a minimal function mutation; 200 patients took Trikafta and 203 others were given a placebo as part of the AURORA F/MF Phase 3 clinical trial (NCT03525444).

After 24 weeks (about six months) of treatment, patients on Trikafta scored higher in all 11 non-respiratory CFQ-R domains than they had at the trial’s start, a baseline measure. Mean increases ranged from 2.1 points for digestive symptoms to 13.2 points for weight, and they were significantly greater than those seen in the placebo group for all domains expect digestive symptoms.

Data covering another 107 patients (59 female and 48 male) with two copies of the F508del mutation came from another Phase 3 clinical trial called AURORA F/F (NCT03525548). Of these, 55 patients took Trikafta and the remaining 52 took Symdeko (tezacaftor/ivacaftor; sold as Symkevi in Europe), a double combination also approved for CF.

After four weeks of Trikafta treatment, scores in all 11 non-respiratory domains rose relative to baseline scores. Increases ranged from a mean 1.1 points for digestive symptoms to a mean 9.9 points for physical functioning. Trikafta was also significantly better than Symdeko in bringing about higher scores in seven of these 11 non-respiratory domains.

“Treatment [with Trikafta] extends clinical benefit beyond previously reported improvements in lung function, respiratory symptoms, and CFTR function to broad benefits in patient-reported disease-related symptoms and general functioning and well-being,” the researchers concluded.

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