In a development for the management of mild pulmonary exacerbations (PEx) in children with cystic fibrosis (CF), a pilot study led by Don B. Sanders, MD, a pediatric pulmonologist at Riley Children’s Health, yields hopeful outcomes.
The study, STOP PEDS (Streamlined Treatment of Pulmonary Exacerbations in Pediatrics), assesses outpatient exacerbation management, specifically comparing immediate oral antibiotics to tailored therapy, where antibiotics are given only if symptoms don’t respond to airway clearance alone.
Cystic fibrosis, a rare genetic disease marked by episodes of acute cough and other respiratory symptoms, is life-threatening and affects about 30,000 people in the U.S. These acute episodes of cough and other respiratory symptoms are commonly treated with antibiotics and increased airway clearance. With ongoing concerns about the overuse of these antibiotics, however, STOP PEDS aims to redefine that treatment approach.
Enrolling 121 participants aged six to 18 years at 10 U.S. CF centers, the study analyzed if tailored therapy, involving increased airway clearance with oral antibiotics added only for specific criteria, could reduce antibiotic exposure. Of the 33 study participants assigned to tailored therapy, 10 patients received oral antibiotics, and 29 of 30 patients assigned to immediate antibiotics also received oral antibiotics. By the end of the trial, results showed that 70% of participants in the tailored therapy arm avoided oral antibiotics during the 28 days after randomization. This outcome suggests heightened potentials for reduced antibiotic exposure in CF management.
“Caregivers and clinicians often voice ongoing concerns about the long-term impacts of antibiotics, especially in the ear of highly effective CFTR modulators,” Dr. Sanders said. “However, with this study, it reveals valuable insights into new possibilities for avoiding antibiotics for mild respiratory symptoms in children with CF.”
In addition to the study’s positive trajectory, the feasibility goals of the study, including enrollment, early symptom detection and the ability to randomize were successfully met. Importantly, the tailored therapy method demonstrated not only feasibility but also a clearer understanding to the treatment landscape for children with CF experiencing acute respiratory symptoms.
As a first-of-its-kind study, Dr. Sanders and his team of researchers will plan on spearheading a larger trial in the near future to delve deeper into optimal treatment strategies for CF. For now, the study’s success is an important step in addressing pediatric CF with its new findings around mitigating antibiotics.
About Pediatric Pulmonology
Alongside Dr. Sanders, the pulmonology program at Riley Children’s provides advanced clinical care for children experiencing respiratory and sleep disorders. Notably, it is home to one of the largest cystic fibrosis programs in the nation (accredited by the Cystic Fibrosis Foundation), and it is one of only nine centers in the nation to be accredited as a Pulmonary Hypertension Center of Comprehensive Care.