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Giving kids with CF a chance at a longer, healthier life
Diagnosed with cystic fibrosis (CF) at three weeks of age, Connor Barrett’s daily routine involves 25 separate doses of medicine and two sessions of chest physiotherapy.
It’s all necessary to help this happy, clever kid – who dreams of one day becoming a medical scientist – to breathe better and reduce the damage to his lungs and digestive system caused by what will be a lifelong disease.
Children living with CF have a build-up of thick mucus in their lungs, making them prone to severe lung infections. Whenever Connor becomes unwell with a lung infection, he must endure an extended spell in hospital being treated with IV antibiotics.
“It’s a lot for a little boy,” Connor’s mum Taryn Barrett said. “While we are grateful for the medical research of the past, which is keeping Connor as healthy as he can be, there is still so much work to be done.”
It is for this reason that Taryn became an advocate for cystic fibrosis research – and why she decided Connor should take part in the Telethon Kids Institute-led COMBAT CF trial.
COMBAT CF is one of two long-standing international trials which have resulted in new early intervention options that hold great promise for reducing progressive lung damage in kids living with CF.
The COMBAT CF study was co-led by Professor Stick, Director of the Walyan Respiratory Research Centre – a powerhouse partnership between Telethon Kids Institute, Perth Children’s Hospital Foundation and Perth Children’s Hospital – and Professor Peter Sly, of The University of Queensland.
Professor Stick is also a researcher at The University of Western Australia’s Medical School and a respiratory clinician at Perth Children’s Hospital COMBAT CF was funded by the Cystic Fibrosis Foundation and carried out in collaboration with the Child Health Research Centre at The University of Queensland, Griffith University, Erasmus University Medical Centre, Monash Children’s Hospital, The Children’s Hospital at Westmead, Women’s and Children’s Hospital, Starship Children’s Health, Sydney Children’s Hospital, Queensland Children’s Hospital, The Royal Children’s Hospital, and New York Presbyterian Hospital.
SHIP CT was funded by the Cystic Fibrosis Foundation and carried out in collaboration with Erasmus University Medical Centre, Seattle Children’s Hospital, Indiana University, and The Hospital for Sick Children
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Reducing hospital stays
The decade-long COMBAT CF trial followed 130 children from CF clinics across Australia and New Zealand, from the time they were diagnosed at birth until they turned three years old, to see if using the commonly prescribed broad spectrum antibiotic, azithromycin, as a preventive therapy could reduce lung inflammation – and thus alter the course of the disease. Lung inflammation is associated with progressive lung disease in young children with CF.
Children involved in the study were given either the antibiotic treatment or a placebo, with the trial finding that those who had received the antibiotics had less lung inflammation and did not get as sick as frequently as those in the control group. This meant they spent fewer days in hospital and received fewer antibiotics each year than children given the placebo.
“Importantly, we found this treatment reduced inflammation in the lungs of young children, which is incredibly significant because there are no other antiinflammatory medications that are currently available to treat the lung inflammation that we see in these kids,” Wal-yan Respiratory Research Centre Director, Professor Stephen Stick, said.
The research team also found long-term use of azithromycin was safe and improved respiratory symptoms.
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Effective, affordable treatment
The second trial, SHIP CT, aimed to identify effective and affordable therapies to prevent or slow development of structural lung disease which had already developed in a large population of children with CF aged between three and six years.
The study trialled the use of inhaled hypertonic saline – a saltwater solution with a higher concentration of salt than normal saline – as a treatment and found it was a safe, cheap, and effective option.
Using images of the lung from chest CT scans to measure results, the study showed children who inhaled hypertonic saline twice daily for 48 weeks exhibited less structural lung damage compared with children who inhaled isotonic saline – a saltwater solution with the same salt concentration as the body.
“This is the first evidence of a treatment that alters structural lung disease in children of this age group,” Professor Stick said.
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New options for younger kids
Professor Stick described both the COMBAT CF and SHIP CT trial findings as promising, with each offering a disease-altering early pathway to a group of children currently too young to access key medications – such as modulator therapies – which have transformed treatment in older children and adults with CF.
Modulator medication changes the way the CF gene works at a cellular level, making the body more efficient at clearing mucus so it does not build up – in turn giving the body a better chance at fighting infection. However, many young children in the world do not have access to these transformative therapies.
“As interventions, we now know that both azithromycin and hypertonic saline hold great promise in terms of reducing the progressive lung damage we see in early CF,” Professor Stick said.
“They therefore give doctors another weapon to treat these kids until they are old enough and eligible for modulator therapies.”
Pictured: Connor Barrett