Sinonasal function and quality of life may be improved in children and young adults with cystic fibrosis (CF) following the combination therapy of ivacaftor, tezacaftor, and elexacaftor (ELX/TEZ/IVA; brand name, Trikafta®), according to a study published in the International Forum of Allergy & Rhinology.
After an average of 521 days of treatment with ELX/TEZ/IVA therapy, patients ≥14 years old (n=11) showed a decrease in the 22-item Sinonasal Outcomes Test scores from an average of 46.55 to 36.09 (P =.04). A decrease was also observed in the Sinus and Nasal Quality of Life Survey from 13.67 to 7.67 for patients aged between 12 and 13 years (n=3) after 249 days of treatment, however, the decrease was not significant (P =.037).
Improvement in the “Sniffin’ Sticks” threshold and discrimination tests was observed after ELX/TEZ/IVA therapy. Average smell discrimination increased from 9.33 pretreatment to 9.56 after treatment and the smell threshold increased from 5.81 to 7.44. Neither of these improvements was statistically significant (P =.081 and P =.24), however, they trended toward published values in healthy patients for this age group (12.69 and 8.52).
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Adolescent patients aged 14 years and over had statistically significant increases in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) from 62.77 pretreatment to a mean of 90.3 an average of 522 days after ELX/TEZ/IVA therapy initiation. Children aged 12 and 13 years also showed an improvement in CFQ-R scores from 59.26 pretreatment to 94.44 post-treatment but the changes were not significant (P =.16), most likely due to the small patient number (n=3).
“Our findings indicate that [ELX/TEZ/IVA] therapy may improve sinonasal function and quality of life in children with CF,” the authors concluded. “Further study with additional patient recruitment will be needed to substantiate our results on the effects of [ELX/TEZ/IVA] therapy on smell threshold and discrimination, quality of life, and self-reported respiratory function in pediatric CF patients.”
During the study, a total of 23 patients with CF between the ages of 12.1 and 21.7 years (median, 16.3 years) were enrolled in the study. The patients were 39% female and 43% Hispanic.
Castellanos CX, Osterbauer B, Hasday S, Keens TG, Koempel J, Ference EH. Improvement in sinonasal quality of life indicators for pediatric patients with cystic fibrosis treated with elexacaftor-tezacaftor- ivacaftor. Int Forum Allergy Rhinol. Published online May 29, 2022. doi:10.1002/alr.23036