The NEEMO Study: Investigating inhaled therapy adherence and outcomes in people with CF taking Kaftrio

A study to look at adherence to preventative inhaled therapy and outcomes for people with cystic fibrosis who take Ivacaftor/Tezacaftor/Elexacaftor (Kaftrio)

There is a difference between the improvements seen in clinical trials (known as 'efficacy’) and improvements in everyday use (known as ‘effectiveness’) for Ivacaftor, the first modulator that became available for people with CF. A 10.6% improvement in lung function was seen in clinical trials, whereas only a 6.0% improvement was seen in the real world after Ivacaftor became more widely available to the CF community.

Other data showed that the prescription of inhaled therapies (e.g. inhaled mucolytics and antibiotics) dropped following the availability of Ivacaftor in the UK. There is a possibility that this lower use of inhaled therapies may explain why a lesser improvement in lung function after taking Ivacaftor was seen in the wider CF community compared to people who received Ivacaftor in clinical trials. However, this has not yet been explored.

Kaftrio is a combination modulator treatment made up of Elexacaftor, Tezacaftor and Ivacaftor. Clinical trials of Kaftrio showed an increase of over 10% in lung function. The aim of this study is to understand whether changes in the preventative inhaled treatments that people take before and after starting Kaftrio affect their lung function improvement.  

This study is important because lung function is key to length of life and how well people with CF are. Making a choice about taking burdensome inhaled treatments is difficult because there isn’t enough information about how taking less inhaled treatment when on Kaftrio might affect lung function. This study uses people’s own choice about continuing or stopping inhaled therapy since starting Kaftrio, to understand whether this may affect lung function improvement.

To be eligible to take part, you must be an adult with CF in the UK attending one of the CF centres who has participated or is participating in the CFHealthHub learning health system. You will need to consent for your anonymised data to be added to the UK CF registry where it can be used for future research. You may also be asked to consent for CFHealthHub to collect anonymised adherence data to be used for future research. Routinely collected clinical and adherence data will be analysed from twelve months before starting Kaftrio and for up to five years after.