CFTR modulators tied to lower anxiety in children with cystic fibrosis
Parents also had lower anxiety, depression scores in small study
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Children with cystic fibrosis (CF) who were using CFTR modulators had lower anxiety scores than those not using the treatments, while their parents had lower anxiety and depression scores, according to a small, single-center study in Turkey. The findings suggest these treatments may be linked to mental health benefits for families, in addition to easing physical symptoms.
“These findings suggest a potential early psychosocial benefit of CFTR modulator therapy for both patients and caregivers,” researchers wrote in the study, “Early effect of CFTR modulators on the mental health of patients with cystic fibrosis and parents,” published in the European Journal of Pediatrics.
How CFTR modulators treat CF
CF is a genetic disease that causes breathing and digestive problems, among other symptoms. It is caused by mutations in a gene called CFTR, which codes for a protein that normally helps control the movement of salt and water in and out of cells. CFTR modulators are designed to improve the function of the faulty CFTR protein, easing physical symptoms.
“CFTR modulator therapies have been increasingly used in recent years with demonstrated beneficial effects on clinical outcomes,” the researchers wrote. Despite these benefits, CF still carries a heavy emotional burden. However, the researchers added, “the effects of CFTR modulator therapies on mental health are not yet clearly defined.”
To understand whether CFTR modulators may have mental health benefits, the researchers evaluated 53 children with CF and one parent for each child. Of the 53 children, 24 were receiving CFTR modulators for a median of 17 months, while 29 had never used such treatments. The children had a median age of 9 years, and the two groups were similar in age, sex, and age at diagnosis.
Most children on CFTR modulators (87.5%) were receiving Trikafta (elexacaftor/tezacaftor/ivacaftor); of the remaining children, one was receiving Kalydeco (ivacaftor), one Symdeko (tezacaftor/ivacaftor), and the other Orkambi (lumacaftor/ivacaftor). Most children who were not receiving CFTR modulators (82.7%) had genetic mutations that made them ineligible for available CFTR modulator treatments.
The researchers used several questionnaires to measure mental health and quality of life. Because some questionnaires were designed for specific age groups, not every child completed every questionnaire. Anxiety was measured using the Screen for Child Anxiety-Related Emotional Disorders (SCARED), while depression was measured using the Children’s Depression Inventory (CDI). Quality of life was assessed using the CF Questionnaire-Revised (CFQ-R), which evaluates how CF affects daily life.
Children on modulators had lower anxiety scores
Children who were not receiving CFTR modulators had significantly higher scores on SCARED than those receiving CFTR modulators, indicating higher levels of anxiety. However, there were no significant differences in the CDI and CFQ-R between the two groups, suggesting no clear differences in depression scores or quality of life.
The study also included 53 parents, of whom 34 were mothers and 19 were fathers. Parents also completed a series of questionnaires. Anxiety was measured using the Beck Anxiety Inventory (BAI), depression using the Beck Depression Inventory (BDI), and quality of life using the World Health Organization Quality of Life-Short Form (WHOQOL-BREF).
Parents of children who were not receiving CFTR modulators scored significantly higher on the BAI and BDI, indicating higher levels of both anxiety and depression. Parents of children receiving CFTR modulators also had somewhat higher quality-of-life scores, but this difference was not large enough to be considered statistically significant.
Higher levels of anxiety in children were linked to higher levels of anxiety and depression in parents. In addition, better parental quality of life was linked to lower levels of anxiety in both parents and children, and of depression in parents. Overall, the findings suggest that CFTR modulator use may be associated with lower anxiety in children with CF and lower anxiety and depression in their parents.
However, because this was a small, observational study at a single center, without before-and-after treatment assessments, it cannot prove that CFTR modulators directly caused the mental health differences. “Despite these limitations, our study contributes to the limited literature evaluating mental health and [quality of life] in both pediatric patients with CF and their parents,” the researchers wrote.
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