Promising results from a ten-year Australian clinical trial could drastically change the way we care for young children with cystic fibrosis, thanks to a commonly prescribed broad-spectrum antibiotic.
The 60-plus researchers demonstrated that when used as a preventive therapy, azithromycin reduced the inflammation associated with progressive lung disease in young children with CF, from birth to 36 months of age.
The findings, published June 2nd in The Lancet Respiratory Medicine, showed that long-term use was safe, improved respiratory symptoms, reduced hospital stays for respiratory needs, and reduced the number of courses of IV antibiotics to treat infections.
The decade-long multi-site trial, co-led by Professor Stephen Stick, a researcher at UWA’s Medical School and respiratory clinician at Perth Children’s Hospital, and Professor Peter Sly from UQ, followed 130 children from 10 CF clinics across Australia and NZ.
“Using azithromycin as a preventative treatment meant these kids didn’t get as sick as they otherwise would have in the first few years of their life, meaning they weren’t going into hospital as frequently, and therefore didn’t need to have stronger medications to treat infection flareups,” Professor Stick said.
“Those that did present to hospital had fewer days of strong IV antibiotics, so they were able to get back to being kids faster.”
A baby is born with cystic fibrosis every four days in Australia, and children living with CF are susceptible to severe bacterial infections and worsening lung inflammation, causing irreversible lung damage.
The severity of lung damage in patients with CF is an indication of the progression of the disease and these infections and their symptoms can result in children being admitted to hospital for lengthy periods of time for treatment with antibiotics.
“We found (azithromycin) treatment reduced inflammation in the lungs of young children, which is incredibly significant because there are no other anti-inflammatory medications that are currently available to treat the lung inflammation that we see in these kids,” Professor Stick said.
“As an intervention it holds great promise in terms of reducing the progressive lung damage we see in early CF, and therefore gives us an avenue to help these kids have a better a chance at a longer, healthier life.”
Professor Stick said that the findings may help change the trajectory of the disease in a group of children who are not yet able to access the new classes of modulator therapies, which change the way the CF gene works at a cellular level.
“Most modulators are approved for school-aged kids but not for those under four years old.
“So now we have a medication that is safe to use in early life that could slow the progression of the disease, until they are old enough for modulator therapies,” Professor Stick explained, noting that most young children in the world do not have access to these transformative therapies.
“Based on our study, treatment with azithromycin could significantly reduce the hospital cost for treating a young child with CF, which is about $12,000 a year.”