Early data to answer one of your research questions to be presented at world’s biggest CF research conference

How CF STORM was shaped by the priorities of the CF community

People with CF have a huge treatment burden. On average, people with CF take 34 tablets a day and can spend hours doing physiotherapy. In both 2017, and in 2022, through the Trust’s James Lind Alliance priority setting partnership. “What are the effective ways of simplifying the treatment burden of people with CF?” was identified by the CF community as a top research priority.

From 2019, members of our Involvement group took part in a series of focus group discussions about which part of their treatment burden they would most like to stop. Nebulisers came out as the most time-consuming and burdensome treatment, as a lot of time is spent prepping, carrying out and cleaning nebulisers. However, people were less keen to stop nebulised antibiotics.

The design focus for the trial was then established and the name CF STORM (Streaming Treatments Or Reducing Medication) was developed by the Involvement team.

As the trial progressed, community representatives stayed involved in the Trial Management Group to help make sure the focus of the study remained on providing answers for the community in the new era of modulator therapy.

Funding for the preliminary work, to prepare for the trial and appoint a trial manager, was provided by a grant from our Venture and Innovation Award (VIA) scheme which helped them to start the trial as quickly as they did. 

What did the study do?

With the introduction of CFTR modulators, many people on Kaftrio have found that their lung function and overall health has improved, and treatments and physio that may have been essential before, may feel less necessary than before.

The study asked for people with CF, aged 12 years or older who were taking Kaftrio and taking either nebulised hypertonic saline, DNase, or both. Participants were then asked to either continue these nebulised therapies as usual, or stop taking them for 12 months.

Almost 600 people with CF took part in the trial, with 40 hospitals recruiting to the study. After each person had taken part for 12 months, they compared those who had stopped taking their nebulised therapy with those continuing. The main thing the study team looked at was if there was any change in lung function between the ‘stop’ and ‘continue’ groups. They are also looking at factors like: 

• change in respiratory function 

• number of new chest infections

• quality of life 

• need for extra treatments

How was the Registry involved?

All these outcomes were collected at standard clinic visits and recorded on the national UK CF Registry, meaning participants didn’t necessarily need to have extra visits to the hospital.

Dr Gwyneth Davies, co-chief investigator of the CF STORM study, said: “With data from the UK CF Registry, we can design clinical trials to fit into routine CF care, minimising the extra time it can take to participate in a clinical trial, whilst still getting answers to important questions from the community about the effects of treatment or the safety of reducing medications.”

Thank you

We, and the entire CF STORM study team, would like to thank each member of the CF community who helped define, design and oversee the trial, and extend thanks to every one of the participants for their part in this important research.

What’s next?

Dr Davies will be presenting the early data from the study at the North American CF Conference tomorrow, look out for a summary on our website soon.

Full results will be published next year, which we will share with the community.

Is it safe for me to stop taking my nebuliser?

No, although the main study results relating to lung function are being presented at the North American CF Conference, results for important other factors are not yet available. Please talk to your CF Team if you have any questions about your treatments. Watch this space, as we are keen to keep the community updated!