A pilot study evaluating the benefits of Trikafta treatment for cystic fibrosis (CF) indicates a significant improvement in bone mineral density (BMD), body composition, and exercise capacity, according to a new article published in the journal Pediatric Pulmonology. 

The study design included 9 CF patients treated with Trikafta and 9 control CF patients who never took the medication. To evaluate the metabolic effects of Trikafta, the researchers primarily looked for changes in BMD and analyzed the weight, height, body mass index, and body composition parameters, including bone mineral content, lean body mass, fat percentage, and fat mass index. Spirometry, lung clearance index, and sweat test, 6-minute walk test, and cardiopulmonary exercise test results were secondary outcomes. 

The researchers compared the BMD and body composition values recorded 3 months after the initiation of Trikafta with data gathered 2 years earlier; the rest of the parameters were measured before and 3 months after the treatment initiation. The BMD measurements in the control group were tested 2 years apart. 

Patients treated with Trikafta had an increase of 0.08 g/cm2 in hip BMD and 0.06 g/cm2 in spine BMD, while the values remained steady in the control group. The body composition analysis indicated that treated patients gained an average of 2.5 kg, which correlated with a significant increase in body mass index, lean body mass, and fat parameters. There were also improvements in the results of the 6-minute walk test, the sweat test, and the cardiopulmonary exercise test, which showed a decrease in minute ventilation/carbon dioxide production, as well as an improvement of forced expiratory volume in 1 second. 

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“To the best of our knowledge, this is the first study evaluating the metabolic effects of Trikafta, and the initial results are encouraging,“ Gur and colleagues wrote. “Understanding the extra‐pulmonary effects of Trikafta may aid in managing other organs involved in CF, in the prevention of long‐term complications, and may enable dietary and exercise guidance for people on cystic fibrosis transmembrane conductance regulator modulators.“

Osteopenia and osteoporosis are often associated with CF. Although treatment with cystic fibrosis transmembrane conductance modulators such as Trikafta is known to improve pulmonary function, there is growing interest in its extra-pulmonary benefits. However, there is still a paucity of evidence about the effects of Trikafta on other organs.

Reference

Gur M, Bar-Yoseph R, Hanna M, et al. Effect of Trikafta on bone density, body composition, and exercise capacity in CF: a pilot study. Pediatr Pulmonol. Published online November 13, 2022. doi:10.1002/ppul.26243