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Evaluating the Mental Health Impact of a Promising New Treatment for Cystic Fibrosis

Evaluating the Mental Health Impact of a Promising New Treatment for Cystic Fibrosis 706 470 Pam Georgiana

Insights from Nationwide Children’s study on how Trikafta affects children with cystic fibrosis.

In response to the promising clinical benefits of elexacaftor/tezacaftor/ivacaftor (Trikafta) in adults with cystic fibrosis, a team in Nationwide Children’s Hospital Cystic Fibrosis Clinic recently investigated the effects of the drug in pediatric patients. Kimberly Pasley, PsyD, mental health coordinator in the Cystic Fibrosis Clinic, led the team. She was assisted by Anne E. May, MD, a physician in the Cystic Fibrosis Clinic and an assistant professor of Clinical Pediatrics for The Ohio State University College of Medicine. The findings were published in Pediatric Pulmonology.

“We have more than 500 pediatric and adult patients with cystic fibrosis at Nationwide Children’s. Many on our team had heard stories of this medication virtually eliminating symptoms in adults,” explains Dr. Pasley. “We had a list of patients ready to start taking the medication. But we needed to do our due diligence on how it might affect our younger patients.”

The FDA approved Trikafta for children as young as two in 2023. As Dr. Pasley says, the medication has been a “game changer” for many adults with cystic fibrosis, significantly improving lung function and overall quality of life. Some patients have been able to suspend most other therapies while taking Trikafta. However, previous studies on adolescent and adult patients have reported concerning mental health side effects such as insomnia, brain fog and depression, leading to caution among pediatric health care providers regarding its impact on younger patients.

The study tracked 81 pediatric cystic fibrosis patients taking the medication over 18 months. Dr. Pasley and team used standardized mental health screenings to evaluate changes in symptoms before and after starting the medication.

“Our goals were first to understand how the treatment affects our patients’ mental health and then to weigh those changes against the potential physical health benefits,” explains Dr. Pasley.

At the start of the study, most participants reported normal mental health. Only a small subset showed elevated depressive symptoms (13%), and a similar proportion reported anxiety (12.3%). Girls were more likely to have depressive symptoms at baseline. After 18 months on Trikafta, most patients (both boys and girls) showed significant improvements in baseline depressive and anxiety symptoms. No patients had elevated anxiety symptoms and only 3% experienced elevated depressive symptoms.

The findings suggest that, for most children with cystic fibrosis, depression and anxiety are not common. However, those who did experience symptoms showed marked improvement while taking Trikafta.

Despite these positive findings, the researchers emphasized that more long-term studies are needed to fully understand the lasting effects of Trikafta on young patients.

“This was a small subset of a larger five-year study looking at certain drugs, including Trikafta, and their impact on both physical and mental health,” Dr. Pasley explains. “We must explore the long-term benefits and potential drawbacks more comprehensively.”

As cystic fibrosis treatments continue to evolve, finding the right balance between physical and mental health will remain a crucial focus for health care teams.

“Because the life expectancy for cystic fibrosis patients has gone from 40 years to 61 years recently, it is hard to ignore the benefits of medications like Trikafta,” says Karen S. McCoy, MD, chief of the Division of Pulmonary Medicine at Nationwide Children’s, Nationwide Foundation Endowed Chair in Pulmonary Medicine, and professor of Pediatrics at The Ohio State University College of Medicine.

She adds this note of caution, “We do not want to give the impression that this drug cures cystic fibrosis. We still do not know much about the long-term damage caused by side effects or how it affects a patient’s clinical status. Providers must monitor any patient taking this drug very carefully.”

“Our job as a care team is to find the right balance to keep our patients’ physical and mental health going in an upward trajectory,” Dr. Pasley concludes.

 

References:

  1. Pasley K, Eisner M, Sliemers S, Young R, Dell ML, McCoy KS, May A. Mental Health of Pediatric Patients with Cystic Fibrosis after 18 Months on Elexacaftor-Tezacaftor-Ivacaftor Therapy. Pediatric Pulmonology, 2025;60:e27393.
  2. Quittner AL, Goldbeck L, Abbott J, et al. Prevalence of depression and anxiety in patients with cystic fibrosis and parent caregivers: results of The International Depression Epidemiological Study across nine countries. Thorax. 2014;69:1090-1097.
  3. https://www.cff.org/managing-cf/understanding-changes-life-expectancyl

About the author

Pam Georgiana is a brand marketing professional and writer located in Bexley, Ohio. She believes that words bind us together as humans and that the best stories remind us of our humanity. She specialized in telling engaging stories for healthcare, B2B services, and nonprofits using classic storytelling techniques. Pam has earned an MBA in Marketing from Capital University in Columbus, Ohio.

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