CFTR modulators may ease anxiety, depression in children with CF

Continued treatment with CFTR modulators was linked to lower anxiety and depression scores in children with cystic fibrosis (CF) along with improvements in lung function and parents’ mental health and quality of life, a study found.

The study, “Mental health, sleep, and quality of life following CFTR modulator therapy: a longitudinal study of children with cystic fibrosis and caregivers,” was published in the European Journal of Pediatrics by researchers in Turkey.

The findings are in line with an earlier Turkish study that linked CFTR modulator use to better mental health outcomes in children with CF and their parents. The benefits of CFTR modulators “may extend beyond clinical outcomes to the psychosocial well-being of families,” the researchers wrote.

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CFTR modulators target the underlying protein defect

CF causes thick, sticky mucus to build up in the lungs and other organs in the body. CFTR modulators are medications that target the CFTR protein, whose function is disrupted by mutations that cause CF. These treatments are known to ease lung symptoms, but their longer-term effects on mental health, sleep, and quality of life have been less clear.

The researchers looked at whether continued use of CFTR modulators was linked to changes in anxiety, depression, daytime sleepiness, and quality of life in children with CF. They also looked at whether parents experienced changes in their own mental health, quality of life, and the burden of caring for a child with CF.

Their study included 24 children diagnosed with CF and their parents. The children had a median age of 10 years at the beginning of the study, and more than half (54.1%) were girls. Most children (87.5%) were taking Trikafta (elexacaftor/tezacaftor/ivacaftor), while one child each was taking Kalydeco (ivacaftor), Symdeko (tezacaftor/ivacaftor), or Orkambi (lumacaftor/ivacaftor). All these therapies are sold by Vertex Pharmaceuticals.

Most children (91.6%) had pancreatic insufficiency, meaning their pancreas did not produce or release enough digestive enzymes to properly absorb nutrients from food. These children were receiving pancreatic enzyme replacement treatment. During the 18-month follow-up, their median body mass index, a measure of weight relative to height, increased from 15.2 to 16.9.

Nineteen children were able to complete spirometry, a standard breathing test. Their percent predicted forced expiratory volume in one second, which measures the amount of air forcefully exhaled in the first second, improved significantly, as did their percent predicted forced vital capacity, which measures the total amount of air they can forcefully exhale.

Mental health and caregiver burden improved over time

The researchers used several questionnaires to assess mental health and sleep. Over the 18-month follow-up, scores on the Screen for Child Anxiety Related Emotional Disorders (SCARED), completed by 17 children, and Child Depression Inventory (CDI), completed by 18, were significantly lower, indicating less severe symptoms of anxiety and depression. Daytime sleepiness, an unusual tendency to feel sleepy during the day, also decreased.

The CF Quality of Life Questionnaire (CFQ-R) was used to measure how CF affects areas such as physical activity, social functioning, and breathing-related symptoms. In children ages 6 to 13 years, scores for physical functioning, social functioning, and breathing-related symptoms improved, yet these improvements did not remain statistically significant after adjustment for multiple comparisons.

After 18 months, parents had significantly lower scores on the Beck Anxiety Inventory and Beck Depression Inventory, indicating milder symptoms of anxiety and depression. Parents also had lower scores on the Zarit Burden Interview, which measures the strain associated with caring for another person. At the same time, they reported better quality of life.

The results were not identical for every child or parent, with individual responses varying. For example, five children had slightly higher daytime sleepiness scores at follow-up. However, all five already had very low scores, their scores rose by only one point, and none reached the threshold for excessive daytime sleepiness.

Nevertheless, the findings suggest that continued CFTR modulator use may be associated with improvements beyond physical health, including the psychosocial well-being of children with CF and their families. However, this was a small study without a comparison group, which means the researchers could not determine whether the modulators caused the observed changes. “Larger prospective studies with pre- and post-treatment assessments and control groups are needed to confirm these findings,” the researchers concluded.