Prof Scott Bell: cystic fibrosis care moves into adulthood

By Mardi Chapman

2 Oct 2019

Professor Scott Bell

Professor Scott Bell,
QIMR Berghofer

It’s time to rethink cystic fibrosis as a paediatric condition and start considering the implications of an ageing CF population, in terms of their future clinical care and the delivery of that care.

This is one of the key challenges facing CF care providers as outlined in the Lancet Respiratory Medicine Commission on the Future of CF Care: A Global Perspective launched at the 2019 ERS Congress in Madrid.

The Commission, led by Australian CF expert Professor Scott Bell and Toronto-based expert Dr Felix Rajten and involving 38 authors from 18 countries, represents a comprehensive approach to a significant global health problem.

It attempts to go beyond the status quo – be futuristic, aspirational and change norms – and addresses the full health and economic costs of CF care over the next three decades.

Co-author Dr Susan Madge from the CF Centre at the UK’s Royal Brompton Hospital, told the congress the increasing life expectancy for CF already meant more than half of the CF population were adults.

The forecast in Europe was for a 75% increase in the adult CF population by 2025

That had implications for the delivery of routine and emergency CF care and in particular, highlighted the need for training and skill development for the multidisciplinary team in adult care.

Dr Madge said comorbidities in adults with CF paralleled those in the ageing non-CF population – including hearing, respiratory complications, diabetes, bowel and GI issues, poor nutrition, high cholesterol, arthropathy, liver disease, renal disease, GI malignancy and mental issues.

She said they were already seeing more demand for reproductive support and surrogacy from adult patients.

The Congress was told the adult centres would need to grow but the critical size for centres could not yet be predicted.

It was complicated by the as yet unknown impact of the CFTR modulator therapies. They may result in less demand for inpatient beds in the future but there will certainly still be a need for long-term monitoring of patients.

Professor Bell, recipient of the 2019 ERS Excellence Award for Research in CF, said making the expensive but highly effective drugs more widely available was a cornerstone recommendation of the Commission.

“These CFTR modulator therapies have the potential to revolutionise the care of CF by targeting the cause for the first time rather than managing symptoms. They could effectively stop the disease from becoming fatal for as many as 80% of patients,” Professor Scott said.

“Up to now treatment has always centred on the downstream consequences and symptoms such as mucus-clearing drugs and treatments, antibiotics and anti-inflammatories.”

The Congress was told other novel therapies included PTC suppressors, mRNA replacement, full gene replacement and gene editing.

But while there was a robust drug development pipeline of therapies to restore CFTR function, there was still a high need for more effective therapies to address mucociliary clearance, inflammation and bacterial infection in patients with chronic lung damage.

One of the other global challenges highlighted by the Commission is the changing epidemiology of the disease.

Newborn screening and increasingly, pre-conception carrier screening were complicated by the wide variety of CFTR mutations and their different prevalence in different countries.

It was for example, difficult to find a suitable panel of gene mutations for screening where there was little accurate data or where there was change due to patterns of migration.

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