WS03.03 Mental health and sleep quality in children receiving elexacaftor/tezacaftor/ivacaftor therapy

Abstract

Objectives Despite the clinical benefits of elexacaftor/tezacaftor/ivacaftor (ETI) modulator therapy for patients with cystic fibrosis (CF), adverse mental health and sleep outcomes have been observed in some adolescent and adult populations. ETI became available for children aged 6–11 yrs in Australia on May 1st, 2023. Currently, only one study has looked at ETI impact on children aged ≥10 yrs, no information is available in children <10 yrs. We aimed to evaluate the impact of ETI on mental health, sleep, and behaviour in this cohort. Methods 108 children aged 6–11 yrs were recruited from the CF Clinic at the Queensland Children’s Hospital, Australia. Sleep, behavioural and mental health outcomes were measured using parent reported Paediatric Symptom Checklist (PSC) and Sleep Disturbance Scale for Children (SDSC) before commencing ETI (baseline) and after 1-month of therapy. Results Baseline questionnaires were completed by N = 101 caregivers. N = 72 caregivers completed the 1-month post ETI questionnaires. No statistically significant differences were observed between PSC baseline and 1-month scores when examined as an entire cohort (difference 0 [95% CI-1,2], p = 0.71) and as matched data (difference 0 [95%CI-2,2], p = 0.55). Similar results were seen for SDSC scores as an entire cohort (difference 1 [95%CI-4,2], p = 0.59) and as matched data (difference 0 [95%CI-3,3], p = 0.95). When comparing matched data (baseline and 1-month post) for the PSC, there were five instances where scores became abnormal and eight where scores became normal. The SDSC had four instances become abnormal, and five instances become normal. [Formula presented] Conclusion Our preliminary data suggest there are no significant changes in mental health and sleep scores in the first month of ETI therapy in children aged 6–11yrs. A larger longitudinal study is planned to address this question more fully. Grant support This work has been supported by the Cystic Fibrosis Foundation and Child Health Foundation.