Kaftrio, a combination of elexacaftor, tezacaftor, and ivacaftor marketed as Trikafta in the U.S., may relieve much of the discomfort that’s sometimes experienced by people with cystic fibrosis (CF) in the nose and the air-filled cavities around it, called sinuses, a German study found.
The study, “Effects of CFTR-modulator triple therapy on sinunasal symptoms in children and adults with cystic fibrosis,” was published in the European Archives of Oto-Rhino-Laryngology.
People with CF may experience sinonasal symptoms, such as a runny or stuffy nose, an infection of the sinuses, and a loss of sense of smell, in part due to a buildup of thick and sticky mucus along the airways. Such symptoms can make it difficult to breathe through the nose, and add to often already existing breathing trouble.
Vertex Pharmaceuticals’ Kaftrio is a combination of three different medications that work by making mucus less thick and sticky, thereby helping to relieve symptoms of CF. Previous work has suggested the triple combination may help ease sinonasal symptoms in people with CF who have advanced lung disease.
Now, a team of researchers in Germany wanted to watch for changes in sinonasal symptoms in children and adults with CF who had just started treatment with Kaftrio.
The study included 43 people with CF who were about to start treatment with Kaftrio. Their mean age was 32 years, and six (14%) were children. Their mean forced expiratory volume in 1 second (FEV1) percent predicted, a measure of lung function, was 62.7%.
Before they were started on Kaftrio (and then again at a mean 9.3 months later), patients were asked to fill in the 22-item Sinonasal Outcome Test (SNOT-22) and rate the severity of their sinonasal symptoms from zero (no problem) to five (worst possible problem).
Sinonasal symptoms included problems in the nose or ears, sleep, and emotional challenges such as feeling restless or less productive. The maximum total score is 110 points, and a higher total score indicates worse symptoms.
Improvements seen with SNOT-22 scores
On average, the SNOT-22 total score was down by 17 points after treatment with Kaftrio (from 32.7 to 15.7 points). The decrease was “greater than the minimally clinically significant difference for the SNOT-22,” the researchers wrote.
There also were significant improvements in each of the set of items in the SNOT-22. After being on Kaftrio, people scored 9.3 points less in problems related to the nose and 0.5 points less in problems related to the ears. Sleep improved by 3.7 points, and emotional challenges were down by 3.5 points.
Children started off with a lower SNOT-22 total score compared with adults (9.4 points). After treatment with Kaftrio, it went down by 7.2 points to 2.2 points. However, this difference did not reach statistical significance, the researchers noted.
Eight patients were seen by an otorhinolaryngologist — a doctor who specializes in diseases of the ear, nose, and throat — before and after they were started on Kaftrio.
All eight had chronic rhinosinusitis, which is a long-lasting infection of the nose and sinuses that makes them become inflamed. Five (62.5%) had nasal polyps, which are soft growths on the lining of the nose.
Nearly all (87.5%) said they experience less severe sinonasal symptoms, and four (50%) said their sense of smell had improved. An endoscopic evaluation to examine the inside of the nose and sinuses revealed clinical improvement in five (62.5%) of the patients.
Although the study’s sample size was relatively small, these findings add evidence Kaftrio may have “benefits on upper airway symptoms in CF patients,” the team concluded.
The study also included 20 people with CF (six children) who hadn’t started or were ineligible for treatment with Kaftrio as controls, but they only filled out the SNOT-22 at one single point during the study.