Helping children with cystic fibrosis

Researchers at the Queensland Institute of Medical Research are developing a new way to monitor the health of cystic fibrosis patients.

The Australian-first liver study could also ultimately eliminate the need for painful liver biopsies in children with the disease.

Professor Grant Ramm from QIMR’s Hepatic Fibrosis Laboratory and Dr Peter Lewindon from the Queensland Liver Transplant Service at the Royal Brisbane Children’s Hospital (RCH) are using a “Fibroscan” machine to measure the health of the child’s liver.

The machine uses ultrasound to locate the liver under the chest wall, and send a small vibration or pulse into the liver, measuring the rebounding pulse wave which determines the level of scarring on the liver.

Liver scarring is a crucial indicator of whether a child with cystic fibrosis will need a liver transplant, and biopsy is the current gold standard for assessing this. The biopsies are taken under general anaesthetic, but still leave the patient sore for several days.

Professor Ramm said the QIMR study would compare Fibroscan results in children.

“We’ll compare the scan results of healthy children, children with CF with healthy livers, and children with CF who have liver scarring. We also plan to compare these Fibroscan results against blood tests which may also prove useful in identifying children with CF who have liver scarring,” Professor Ramm said.

“It’ll provide benchmarks for doctors, and ultimately eliminate or reduce the need for biopsies.”

Dr Lewindon said the Fibroscan could also be used regularly, while doctors tried to limit the number of biopsies.

“You can imagine how distressing it is to take needle biopsies from children,” Dr Lewindon said.

“But doctors need the information, to establish how the child is faring, and how their treatment needs to be managed. The Fibroscan, on the other hand, is painless.

“We can get a more accurate picture of the development of scarring, and to pick up the early signs which normal ultrasounds can’t detect. Combined with the benchmarks this study with QIMR hopes to provide, Fibroscan could become the gold standard.” Dr Lewindon said.

“For most patients, we’ll be able to reassure them about the future health of their liver. When the screening is indicating more scarring, we can at least prepare those 5 – 10 % of children whose scarring indicates they may need a transplant.”

The $150,000 Fibroscan machine was donated by a member of the public, and is the only paediatric facility of its kind in Queensland.

Cystic fibrosis (CF) is the most common recessive genetic condition in Australian children. The life-threatening condition clogs the body’s organs – including the pancreas, lungs and liver – with a thick, sticky mucus, causing breathing difficulties and long-term liver damage.