To celebrate British Science Week at Royal Brompton and Harefield hospitals we’re highlighting some of the important work our researchers are tackling.
Research to improve the quality-of-life of cystic fibrosis patients
Cystic fibrosis is an inherited disease where thick sticky mucus is produced in the lungs making it difficult to breathe. Symptoms start in childhood and slowly get worse over time, and while there is no cure for the disease, treatments are available to help manage it. These can include multiple oral and inhaled medications that often take an hour or two to complete every day. Patients also need to adjust their lifestyle to account for the disease; this includes being very careful to avoid infections, eating more food as mucus can also clog the pancreas making it harder to digest food and get enough nutrients from it, and physiotherapy to help clear mucus from the lungs.
We spoke to Dr Siobhán Carr, a consultant in paediatric respiratory medicine, about new research at Royal Brompton Hospital that aims to reduce the amount of daily medication that patients with cystic fibrosis need to take.
Dr Carr said: “British Science Week is all about celebrating science and highlighting its diversity and what better time to shine a focus on the rare disease research we are doing here at Royal Brompton and Harefield hospitals.”
A brand-new treatment for cystic fibrosis called Kaftrio® was approved for use by the NHS in August 2020. This new drug is an oral tablet that targets the underlying genetic defect that causes cystic fibrosis and should improve the lung health and life expectancy of many patients. Now researchers at Royal Brompton Hospital are teaming up with researchers from UCL and Liverpool University to investigate whether Kaftrio® could reduce the number of medications patients need to take.
Dr Carr said: “This national study is called CF STORM, and we will be studying around 700 people in the UK for one year. We’re really hoping that Kaftrio will be a game changer for patients with cystic fibrosis, not only improving symptoms of the disease but also reducing the number of other medications patients need to take, particularly the inhaled medicines. If we can achieve this, it will give patients back valuable time every day. Given that cystic fibrosis is a chronic disease with no cure that shortens life expectancy, being able to safely reduce the time burden of treatments for patients is really important.”
In a separate study she is leading, Dr Carr is looking at the amount of time that patients with cystic fibrosis spend taking all their different treatments, known as ‘burden of care’. For the study, called VALU-CF, patients are involved in evaluating what elements of their cystic fibrosis care are most important to them, such as reducing this burden of care or avoiding infections and hospital stays, and how they associated these with their quality-of-life.
Dr Carr added: “We are a leading centre for cardiac and respiratory disease, and we believe that by involving patients in design and delivery of research it will allow us to continue to provide the best care for all patients and to constantly improve that care.”
The cystic fibrosis centre at Royal Brompton Hospital is one of the largest centres in Europe and treats almost 1,000 people with cystic fibrosis each year.
Read the other posts in our British Science Week 2021 series: