Therapy helps keep CF children’s lungs stable, reduces treatments
Study of Kaftrio also showed reduction was accompanied by drop in antibiotic use
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Children with cystic fibrosis (CF) on Kaftrio maintained stable lung function over one year despite reducing their use of hypertonic saline and other airway-clearance treatments, a study shows.
The reduction was accompanied by fewer days of antibiotic use. Respiratory symptoms and airway bacteria also remained stable, suggesting that taking airway clearance treatments less frequently may be feasible for some children on Kaftrio (sold as Trikafta in the U.S.).
“Reducing treatment frequency to once daily may therefore increase flexibility and support a more normal everyday life,” researchers wrote.
The study, “Once-daily hypertonic saline inhalation and airway clearance techniques in children with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor: a prospective multicentre study,” was published in the Journal of Cystic Fibrosis.
Evidence for reducing maintenance therapies remain limited
CF is caused by mutations in the CFTR gene, which result in an absent or dysfunctional CFTR protein. This leads to the accumulation of thick, sticky mucus in various organs, particularly the lungs, causing most CF symptoms.
The introduction of highly effective CFTR modulators such as Kaftrio (elexacaftor/tezacaftor/ivacaftor) has led to significant improvements in lung function, respiratory symptoms, and quality of life in children with CF.
This raises questions about the need to continue intensive maintenance therapies, including hypertonic saline and airway clearance techniques (ACTs). However, evidence supporting the reduction of these treatments remains limited.
Researchers in Sweden studied just that in children with CF, ages 6 to 17, who were followed between December 2023 and November 2024. The study enrolled 46 children (mean age 12.1 years, 63% boys) who had been receiving Vertex Pharmaceuticals‘ Kaftrio for a mean of 12.7 months.
Lung function remained stable after treatment reduction
Before study entry, all participants were prescribed nebulized (inhaled) hypertonic saline twice daily. A short-acting bronchodilator was recommended, followed by individually prescribed ACTs. Positive expiratory pressure mask therapy combined with huffing, a technique used to help loosen and clear mucus from the lungs, was the most common ACT.
Participants and their caregivers were offered the option to reduce the prescribed frequency of hypertonic saline and ACTs from twice daily to once daily for one year
Lung function remained stable over that period. Lung clearance index, a measure of how well air moves through the lungs, showed a small improvement, but the change was not statistically significant.
Forced expiratory volume in one second, which measures the amount of air a person can forcefully breathe out in one second, and forced vital capacity, the total amount of air a person can forcefully breathe out after a deep breath, showed no meaningful changes.
The number of days participants needed any antibiotics decreased by 39%, from 47 to 28.6 days per person for one year. A similar reduction was seen with oral antibiotics (33%). Use of inhaled and intravenous (into the vein) antibiotics was uncommon during both treatment periods.
[The findings suggest that] for clinically stable children with preserved lung function, treatment reduction may be considered as part of an individualised approach to CF care in the [Kaftrio] era.
The types of bacteria found in airway cultures were generally similar before and after reducing the treatments. Pseudomonas aeruginosa, a major contributor to lung disease in people with CF, was detected in two participants during the twice-daily period and in none after treatment reduction.
Respiratory symptoms also remained stable, as assessed with the respiratory domain of the Cystic Fibrosis Questionnaire-Revised.
According to the researchers, these findings suggest that “for clinically stable children with preserved lung function, treatment reduction may be considered as part of an individualised approach to CF care in the [Kaftrio] era.”
However, treatment decisions should be supported by “regular monitoring using sensitive measures to detect early deterioration in airway disease,” the researchers noted, highlighting the need for further research to confirm these findings.
Regarding study limitations, the team mentioned that the participants were mainly clinically stable children with preserved lung function, which means that the findings may not apply to children with more advanced disease.
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