Study reveals persistent mental health concerns in children with cystic fibrosis despite Trikafta treatment

Despite physical improvements from Trikafta, children and adolescents with cystic fibrosis continue to face significant mental health and behavioural issues, highlighting the need for holistic care approaches, new research finds.

Children and adolescents with cystic fibrosis continue to show mental health and behavioural concerns even after starting Trikafta, a treatment that has transformed the physical outlook for many patients, according to a recent study in Pediatric Pulmonology. The research also found that anxiety and depression in caregivers were linked with symptoms in their children, reinforcing the idea that CF care needs to look beyond the lungs and address the family as a whole.

The study drew on the U.S. Your ETI project, which tracks people with cystic fibrosis taking elexacaftor/tezacaftor/ivacaftor, the triple-drug therapy sold as Trikafta. Between March and July 2024, researchers analysed 118 caregiver-youth pairs, including 48 children and 70 adolescents. Most of the caregivers were working and living with a partner, and most of the young people had been taking Trikafta for several years. Although average scores across the group remained in the non-clinical range, clinically meaningful symptoms were still common: anxiety appeared in 31.3% of children and 18.8% of adolescents, while depressive symptoms were seen in 27.1% and 28.6%, respectively.

Sleep stood out as a major issue. Half of the adolescents scored above the clinical threshold for sleep disturbance, and nearly 30% of children did as well. Researchers also reported significant attention difficulties and externalising behaviours, a term that covers problems such as hyperactivity, rule-breaking and disruptive conduct. Caregiver mental health mattered too: anxiety in caregivers was associated with children’s depression, anxiety and sleep problems, while caregiver depression was linked with depression in the child. Among adolescents, caregiver depression was weakly but significantly associated with all of the mental and behavioural measures the researchers assessed.

The findings fit a broader pattern seen in earlier research. A 2022 adult study found no major change in average anxiety or depression scores after Trikafta was started, even though some patients needed psychiatric medication changes and sleep problems were reported. A 2024 study of people with end-stage cystic fibrosis also suggested that while ETI brought physical gains, some patients developed new mental health symptoms. More recent work has focused on better screening tools for cystic fibrosis-specific mental health concerns, while older reviews have long warned that the condition carries a heavy psychological burden for patients and families. The authors of the new study said screening should broaden to include younger children and issues such as sleep and attention, and they cautioned that their sample was relatively small and not fully representative of all people with cystic fibrosis.