Sinonasal quality of life in cystic fibrosis pediatric patients undergoing treatment with trikafta

SESSION TITLE: Pediatrics Posters SESSION TYPE: Original Investigation Posters PRESENTED ON: 10/10/2023 12:00 pm - 12:45 pm PURPOSE: Evaluating the impact of the highly effective CFTR modulator, elexacaftor/tezacaftor/ivacaftor (Trikafta) on sinonasal quality of life and symptoms of pediatric cystic fibrosis patients utilizing validated Sinonasal Quality of Life Survey (SN-5). METHODS: This is a prospective observational study of pediatric cystic fibrosis patients and their response to Trikafta (elexacaftor/tezacaftor/ivacaftor), and the temporal effects of that drug on sinonasal quality of life through the perception of the child’s parent or primary caregiver. The SN-5 is a 6 question survey (sinus infection, nasal obstruction, allergy symptoms, emotional distress, activity limitation, overall quality of life as results of sinus or nose problems) that is validated to assess quality of life for pediatric patients with persistent sinonasal symptoms. It has been proven to correlate to direct estimate of clinical change if comparing answers of the same patient at various times. A baseline SN-5 from the parents or primary caregivers will be obtained prior to start of Trikafta and then a post SN-5 survey will be given after at least 6 weeks of start of treatment. The primary outcome measure will be the change from baseline in SN-5 scores. Inclusion criteria is cystic fibrosis patients beginning the medication Trikafta based on FDA approval of cystic fibrosis genetics and age. The exclusion criteria is any Cystic fibrosis patient not on Trikafta. The baseline results will serve as the control pre-treatment with Trikafta, and the follow up survey will be statistically analyzed. In the statistical analysis the variables were summarized with mean (standard deviation) and median (range). Signed rank test was used to compare pre and post treatment survey scores. All tests were two-sided with p value <0.05 considered statistially significant. RESULTS: A total of 23 patients who completed both pre- and post-TRIKAFTA treatment were included in the analysis. The median age was 13 years (range:2-20 years) and the post-treatment survey was filled out at median time of 13 months post treatment (range: 3-42 months). There were statistically significant drop at post-treatment time in all survey scores except for question 4 (emotional distress). The average score was also significantly lower at post treatment time compared to pre-treatment time. One main limitation of the study that is important to acknowledge is that, the post treatment survey score was collected at different time point, which ranged from 3-42 months, and the sample size did not allow us to sort out if survey score changes when time post treatment gets longer. CONCLUSIONS: Trikafta shows subjective improvement in quality of life as measured through the Sinonasal Quality of Life Survey (SN-5) upon continuous treatment with Trikafta as compared to pre-treatment measures of quality of life. CLINICAL IMPLICATIONS: Treatment with Trikafta will improve sinus and nasal symptoms in pediatric patients with cystic fibrosis. DISCLOSURES: No relevant relationships by Giselle Guerrero No relevant relationships by Zhuo Li No relevant relationships by Floyd Livingston No relevant relationships by Rick Schoppe No disclosure on file for Julie Wei