Cystic fibrosis

Hypertonic saline prevents progression of lung disease in children with CF


Professor Stephen Stick (Telethon Institute)

An international trial has demonstrated world-first prevention of lung disease progression in young children with cystic fibrosis following treatment with inhaled hypertonic saline.

A study published this month in The Lancet Respiratory Medicine found nebulised hypertonic saline treatment generated a significant benefit in structural lung disease, as well as bronchiectasis and trapped air, after 48 weeks.

This was compared to children who were treated with inhaled isotonic saline.

The randomised, multicentre controlled trial known as Saline Hypertonic in Preschoolers (SHIP) was also the first for young children using chest CT to assess the primary outcome  of total lung volume occupied by abnormal airways (PRAGMA-CF %Disease).

Mean PRAGMA-CF %Disease at 48 weeks was 0·88% (95% CI 0·60–1·16) in the hypertonic saline group and 1·55% (1·25–1·84) in the isotonic saline group.

“The mean absolute difference between treatment groups in PRAGMA-CF %Disease [structural lung disease] at 48 weeks was 0·67% of total lung volume,” the paper reported.

“We therefore conclude that the difference in %Disease between treatment groups represents a clinically relevant difference in the severity of airways disease.”

One of the study’s authors, Professor Stephen Stick, Director of the Wal-yan Respiratory Research Centre, Telethon Kids Institute, Perth, said the clinical trial was the culmination of many years of research into the early origins of lung disease in cystic fibrosis research.

“We demonstrated that over the course of 48 weeks, it prevented the progression of structural lung disease, which is the characteristic problem that kids with cystic fibrosis get,” he said.

“It actually rehydrates the lining of the airways in the lung and actually prevents that stickiness.

“The impact is that there is a very cheap treatment now that is very safe. It is the first to show an effect of … improving lung damage in children under the age of five [years].

“This is the first treatment that actually demonstrates you can stop the progression of lung disease.”

Professor Stick said he expected the nebulised salt saline treatment – which had very few side effects and was cheap – would be incorporated into standard practice.

“We expect it will be very widely taken up,” he said. “I imagine it will eventually become standard practice in many centres around the world.”

The research paper recommended maintenance treatment with inhaled hypertonic saline in pre-school children with cystic fibrosis.

“SHIP-CT adds to the evidence base that twice daily nebulised hypertonic saline is an inexpensive and safe intervention with a positive effect on both respiratory function and on progression of structural lung disease,” the study authors said.

“We recommend that inhaled hypertonic saline be considered for all young children, particularly those without access to CFTR modulator therapy, to minimise progression of structural lung damage.”

The study built on existing knowledge that inhaled 7% hypertonic saline improved lung clearance for young children. The 116 participants in the trial were randomly assigned to receive two puffs of 100 μg salbutamol twice a day for 48 weeks. For one group this was followed by 4mL of 7% hypertonic saline, while the other group received 0·9% isotonic saline.

The most common adverse effects were cough, which was deemed mild. 12 patients dropped out of the study, seven in the hypertonic saline group and five in the isotonic saline group.

Professor Stick said while previous trials had demonstrated improvements in lung function for older patients, this was the first to show disease prevention in young children.

“This is something that people have assumed that there may be a beneficial effect, but I don’t think anyone anticipated it could actually halt the progression of lung disease,” he said.

“That is something quite unique.”

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