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CF STORM early results
Background to the study
The CF STORM study, designed in partnership with people with CF, wanted to address the treatment burden of living with cystic fibrosis. With over 8,000 people with CF in the UK now taking the modulator Kaftrio, and the improvement in health seen, there was an opportunity to find out if daily nebulised treatments still have the same role to play, or if they can be stopped without affecting lung health.
How was the study designed?
People with CF have been involved in the design of CF STORM right from the very start. This has included creating the name CF STORM, which stands for ‘Streamlining Treatments Or Reducing Medications’ and suggesting which nebulised treatments (e.g. mucoactive or antibiotics) should be in a trial of ‘stopping’ treatments for people with stable health established on Kaftrio.
Professor Kevin Southern, from the University of Liverpool and Alder Hey Children’s Hospital, and I led the study. We were able to design a study which could easily fit in with usual CF care, by using and developing new features within the UK CF Registry. This meant we could use the information already being collected by the Registry as part of usual care, but also some other outcomes that we needed just for the trial - without the study itself being too much extra trouble and adding burden back into the lives of people taking part.
Who took part?
CF STORM enrolled 591 people with CF in the UK, who had been taking Kaftrio for at least three months, with a minimum lung function (FEV1) of 40% predicted and using daily nebulised mucoactive therapies (dornase alfa and/or hypertonic saline). FEV1 is a measure of lung function from spirometry tests performed in CF clinics or at home. The first people with CF were recruited in 2021, initially for those aged 12 years and above, but as the licensing age for Kaftrio was lowered, CF STORM was able to expand to include children from 6 years of age from 2023.
Each person enrolled had an equal chance of being asked to either continue or stop their daily mucoactive nebulisers. They were then followed up over the next 12 months, and progress was recorded on the UK CF Registry using a combination of data collected during routine clinic visits and other study-specific information. CF STORM was supported by the Trust’s Clinical Trials Accelerator Platform (CTAP) and was open in 40 CF centres across the UK.
What do we know about the results so far?
It’s still early days, but in response to encouragement from community members and the study committee, the main ‘primary outcome’ results have been released ahead of the full study results next year. This reflects their potential impact for people with CF.
The study found that, on average, stopping nebulised mucoactive therapies did not negatively impact lung function (measured by ‘FEV1’, from the spirometry test) over a period of 12 months compared with continuing nebulised mucoactive therapies. This was true for children and adults, and across different lung disease severities included in the study. CF STORM also found that FEV1 was on average slightly better in those stopping rather than continuing nebulised mucoactive treatments, particularly for children. This unexpected finding has generated a lot of additional discussion to try and understand why this may be and will need further investigation.
What do these results mean?
Only the results on lung function have been studied so far. The CF STORM team are still looking at other results on the effects of stopping nebulised mucoactives, including the impact on number of hospital stays, and the need for antibiotic use.
Once the full results are available in Summer 2026, these will provide critical additional information to the lung function results already known, and help with personalised treatment planning.
These data will help people with CF and their care teams make decisions about treatments together, but it is important to note that everyone with CF is different, and what works for one person might not work for another.
My lived experience of CF gave me really useful expertise that no-one else on the committee had. Every single person with CF is different, and the evidence from this study can really help people make decisions. Making a difference to the CF community by being part of this study is just the best thing.
Maria, Trial Steering Group member and whose son took part in CF STORM
We’d like to say a big thank you to everyone in the CF community who took part in this important study. We’re so grateful for your support.
You should not stop any CF treatments without talking to your CF team first.
How do these results compare to other studies?
The lung function results are consistent with a study called SIMPLIFY undertaken in the United States which looked at discontinuation of either daily dornase alfa or hypertonic saline for people aged 12 years and above on Trikafta (Kaftrio), over a six-week period.
SIMPLIFY was too short to consider the frequency of chest exacerbations, but because CF STORM followed everyone for a year, this will be possible to find out.
How was the study funded?
The CF STORM clinical trial was funded by the National Institute for Health and Care Research (NIHR). A Venture and Innovation grant from Cystic Fibrosis Trust was critical in supporting the early set up of the study. Dr Gwyneth Davies is funded by a Future Leaders Fellowship from UK Research and Innovation.
Frequently Asked Questions
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What was the aim of the study?
The CF STORM study wanted to see if people with cystic fibrosis (CF), aged 6 and older, who were taking Kaftrio for longer than three months, could safely stop some of their mucoactive nebuliser medicines.
Mucoactive medicines are drugs that help the body clear mucus from the lungs, such as dornase alfa and/or hypertonic saline.
The study wanted to know if these treatments could be safely stopped without it impacting on lung function.
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What did the study find?
The early study results showed that, on average, people who stopped their nebulised mucoactive therapies, did not see a reduction in their lung function (measured by ‘FEV1’, from the spirometry test) over 12 months. This was true for both children and adults, and for people with different levels of lung function (everyone in the study had to have a minimum lung function score of 40% predicted FEV1).
More of the study results will now be looked at. The researchers will look at factors that were measured other than lung function, such as:
- How people felt about their quality of life
- How well they stuck to their treatment plan
- How often they had chest flare ups (where lung symptoms become more severe)
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What do the results mean for people with CF?
Many people with CF have already chosen to stop their nebulised mucoactive medicines, even outside of this study. These results will help guide conversations in clinic about what is safe when it comes to treatment options
However, only the results on lung function have been studied so far. The team are still looking at other results on the effects of stopping nebulised mucoactives, including the impact on number of hospital stays, and the need for antibiotic use. All of this is really needed to guide discussions.
The results will help people with CF and their care teams make decisions about treatments together. It is important to note that everyone with CF is different, and what works for one person might not work for another.
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Should I stop taking mucoactive nebuliser treatments?
No, but the study team hope that the results of CF STORM will help support discussions between people with CF and their clinical teams.
Even though the study suggests lung function may not be affected for most people on Kaftrio, there’s still more to learn about the impact on other areas of health. Updates will be shared on the study website (www.cfstorm.org.uk).
You / your child should not stop any CF treatments without talking to your CF team first.
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Who should I speak to if I have more questions?
Talk to your CF team if you have questions about your treatments. They know your situation best and can guide you. Cystic Fibrosis Trust will also keep sharing updates with the community.