June 2023

What do you do?

I’m a consultant respiratory paediatrician at Royal Brompton Hospital. I came here 10 years ago after having already been a consultant at Royal London Hospital for 15 years.

In my role, I care for children with a range of lung conditions. I have a special interest in suppurative lung diseases, a group of disorders which result in chronic lung infections, leading to long term lung damage. My special interests include cystic fibrosis and primary ciliary dyskinesia, both conditions you are born with due to a faulty gene. The gene affected by cystic fibrosis controls the movement of salt and water in and out of cells. People with the condition experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body.

There are around 4,400 children with cystic fibrosis across the UK. Each year at Royal Brompton, our teams care for between 300-330 of them, making our service one of the largest and leading specialist services in the UK for this complex condition. 

What does your job involve?

I work in a large multidisciplinary team with colleagues from a range of different disciplines, including nurses, dietitians, physiotherapists, psychologists, pharmacists and physiologists, to help children and their families manage the condition.

In the UK, most cases of cystic fibrosis are picked up at birth using the newborn screening heel prick test. We tend to see a baby within their first 3 weeks of life and see them every 2-4 weeks during their first year of life. We continue to see them regularly on an ongoing basis for the rest of their lives.

Symptoms usually start in early childhood and vary from child to child, but these can get slowly worse over time. By providing comprehensive care to the child, careful monitoring, encouraging good nutrition, and using good and aggressive treatments to treat any infections, we can reduce the problems caused by the condition and make it easier to live with.

Our care starts very early in a baby’s life and we continue to support our patients until they are ready to transfer to our adult service, the largest in Europe. We work hard to ensure a smooth transition.

What has changed in the treatment of cystic fibrosis?

The life expectancy and life span for people with cystic fibrosis has been going up steadily over many years. Key to this has been good and aggressive management of infections and good nutrition.

When I became a consultant in 1998, I talked about what we expected to happen in the management and treatment of CF with the colleague I was taking over from. We talked about the CF modulator drugs, a hope for the future, that were being designed to correct the malfunctioning protein made by the CFTR gene but were still only in the very early stages of the drug discovery process. While they sounded promising, we didn’t really know whether or when they might become a reality.

It has been fantastic to see the difference these drugs have made in recent years. The first of these drugs introduced into clinical practice around a decade ago could only be used by 5% of patients with the condition. Now, we have a number of CF modulator drugs available, we can treat over 90% of people with the condition, now the work is on to find treatment for the remainder.

We were already seeing a gradual decrease in the use of antibiotics and better survival thanks to improvements in the management of cystic fibrosis before the introduction of these drugs. There has been a step change since 2020 with the introduction of the new modulators with a dramatic decline in the proportion of patients needing antibiotics in any given year.

You have been chairing the UK Cystic Fibrosis Registry too. What’s that?

I’ve been chair of the UK Cystic Fibrosis Registry for the last 7 years and will be handing over the role to my successor this year.

The UK Cystic Fibrosis Registry is a secure centralised database, sponsored and managed by Cystic Fibrosis Trust. It records health data on consenting people with cystic fibrosis in England, Wales, Scotland and Northern Ireland.

Across the UK the Cystic fibrosis care teams enter clinical data to the UK CF Registry, with about 99% of people with CF consenting for their data to be submitted.

The registry plays a really important role in furthering understanding of the CF, the annual report is freely available and gives a summary of the health of people living in the UK with CF. It helps us drive quality improvement and is a well-respected research database, with many high-class research papers coming from the anonymised data. It has been an honour to be the principal investigator for the research side of the registry and to have been chair of the steering committee.

Why is research important?

Research into cystic fibrosis is important, not just for discovering and testing new treatments, but also to understand other ways we can improve management of the condition. An important strength of our service at Royal Brompton Hospital is the amount and range of research we carry out and the opportunities for involvement this opens up to patients.

I’ve played a role in research across several areas. This has ranged from a randomised controlled trial to understand the impact of people with cystic fibrosis doing Tai Chi to co-leading the global epidemiological work studying the impact of COVID-19 on people with cystic fibrosis. This was important in showing firstly that people with CF, despite their young age were disproportionately affected, with more mortality and morbidity, especially after transplant. It then went on to show the protective effect of starting modulator drugs, which were introduced in late 2020.

Why do you love your role?

Every day, I feel lucky to be part of a well-respected, cohesive, specialist multidisciplinary team that is internationally renowned and that makes a real difference to the lives of children and families with CF.

Useful links

Find out more about the Royal Brompton paediatric service.

Read more about the CF Trust.

Thank you to the children and young people who have so brilliantly illustrated our blog pages.