EVALUATING THE IMPACT OF ELEXACAFTOR/TEZACAFTOR/ IVACAFTOR ON ANXIETY IN SCHOOL-AGED CHILDREN

Abstract

Introduction/Aim: Elexacaftor/tezacaftor/ivacaftor (ETI), is associated with significantly improved clinical outcomes, but also increased anxiety in a small number of adolescents and adults with cystic fibrosis (CF). This has led to concerns around potential adverse mental health effects in younger children. We aimed to explore the effect of ETI on anxiety scores in school-aged children. Methods: 108 children aged 6-11 yrs were recruited from the CF Clinic at the Queensland Children's Hospital. Symptoms of anxiety were measured using parent- and child-reported versions of the Spence Child Anxiety Scale (SCAS) before commencing (baseline) and 6-months after ETI initiation. Scores were categorised as normal or clinically significant using established thresholds. Results: 78 parents (72%) and 71 children (66%) completed questionnaires at both timepoints. No statistically significant changes were observed between baseline and +6-months for parent-reported (diff = 1 [95% CI -4, 4], p = 0.11) or child-reported scores (diff = 1 [95% CI -3, 5], p = 0.19, Table 1). Parent-reported scores in 70 (90%) children remained unchanged throughout; 63 (81%) with normal scores, 7 (9%) as clinically significant. Change of category was observed in 8 (10%); 4 (5%) normal to clinically significant, and 4 (5%) clinically significant to normal. Similarly in child-reported data; 61 (86%) unchanged; 59 (83%) remaining normal and 2 (3%) remaining clinically significant. Change of category occurred in 10 (14%); 5 (7%) normal to clinically significant, and 5 (7%) clinically significant to normal. Concordance between parentand child-reported scores was 84.1% at baseline and 87.0% at 6-months. Conclusion: No statistically significant changes were seen in SCAS scores for both groups between baseline and 6-months post ETI commencement. Caregivers and children reported similar numbers of children whose scores improved and declined after 6-months of ETI therapy. These data are reassuring but need validation in other cohorts before drawing firm conclusions. Long-term follow-up will explore change in anxiety over the first 12-months of ETI use.