The treatment of cystic fibrosis (CF) with elexacaftor/tezacaftor/ivacaftor (ETI) might be associated with increased lung function and body mass index (BMI), fewer disease exacerbations, and only mild side effects, according to a study published in the journal Pharmacological Research & Perspectives.

This multicenter longitudinal observational study gathered 20 Dutch patients with CF who received ETI on a compassionate use basis. To examine the real-life benefits of this treatment modality, the authors looked for changes in Forced expiratory volume in 1 second (FEV1) and BMI and measured these indicators at the initiation of ETI therapy, as well as 1, 3, 6, and 12 months later.

The assessment of adverse events was carried out by also gathering data about the unfavorable occurrences in the year prior to ETI treatment. All participants were asked to fill in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) used to measure the quality of life across 12 domains and showcase improvement with an increase of 4 points. 

The statistical analysis concluded a mean absolute increase of FEV1 by 11.8%/13.7% and of BMI by 0.49/1.87 kg/m2. As for the CFQ-R questionnaire, researchers found significant improvements at follow-ups across respiratory, physical, treatment, health, social, and body domain scores.

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“For some patients the treatment made it possible to play more sports, or be more active with their kids, which would increase the quality of life scores. For others, the improved lung function did not lead to major changes in their daily lives,” Kos and colleagues noted.

The ETI treatment was well-tolerated, as neither patients nor physicians reported any significant side effects, namely, rash in 15% and stomach ache in 20% of the study population. The number of exacerbations and subsequent hospitalizations was found to be significantly lower compared with the year-long period before the treatment initiation.

“Our findings support the notion that severe CF lung disease patients should receive ETI,” the authors wrote. “Six patients were on the transplant list, and all of them had their status changed to inactive due to the effect of ETI.”

The most frequent cause of CF, a Phe508del mutation, qualifies patients for treatment by CFTR modulators. According to the compassionate use program, severe respiratory patients are eligible for ETI if their FEV1 is below 40% for at least 2 months prior and if they have applied for a lung transplantation surgery.

Reference

Kos R, Neerincx AH, Fenn DW, et al. Real-life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients. Pharmacol Res Perspect. Published on November 28, 2022. doi:10.1002/prp2.1015