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New study on impact of Kaftrio in preschool-age children
The study looked at the outcomes and side effects for 197 pre-school aged children (2–5 years) after starting Kaftrio. As part of this, parents completed a questionnaire about behavioural effects and sleep disturbances they’d seen in their children after one month and again at 3 months. It found that changes were seen for just under half of the children in the study (93 children). The most commonly reported symptom was sleep difficulties, though ADHD, irritability and sadness were also reported.
Previous studies in people with CF over 6 years old have reported potential side effects of taking Kaftrio such as low mood, anxiety, depression, disturbed sleep, and brain fog.
Last year the European Medicines Agency, the regulator for medicines in the European Union, requested a changed to the information included with Kaftrio to reflect this, which we commented on here.
There is also more information about this in our factsheet Kaftrio – complex and individual experiences.
CFTR modulators, including Kaftrio, have helped transform the lives of many people with cystic fibrosis by treating the underlying cause of the condition. For most, the effect on their physical, social, and emotional wellbeing has been positive with no reported side effects. But, as we've noted in our factsheet, we know this has not been the case for everyone.
David Ramsden, Chief Executive of Cystic Fibrosis Trust
This latest Lancet study – along with other ongoing research – plays a vital role in furthering our understanding of the effects of the cystic fibrosis treatment, Kaftrio. Emerging knowledge is critical to improving care and treatment which is why Cystic Fibrosis Trust is co-funding the RECOVER study, to understand the real-world effects of Kaftrio, as well as co-funding and recruiting to the ENHANCE study, looking at how CF impacts young children.
Dr Keith Brownlee, Director of Medical Affairs at Cystic Fibrosis Trust: “It’s important to speak to your CF team if you are concerned about negative changes to you or your child’s health, even if you’re unsure whether Kaftrio is causing them or not. You should also always speak with your CF team before you change, start, or stop any part of your treatment regimen.
“It’s also important that any possible side effects of Kaftrio are recorded so improvements can be made, for yourself and everyone else in the CF community. You can also report suspected side effects to medicines using the Yellow Card reporting scheme.”
You can read Jon’s story and Alexandra’s story if you’d like to read more about people who have had mixed experiences on Kaftrio.
Please speak to your CF team if you have any questions about Kaftrio or you or your child experience any negative changes to mental or physical health while taking the treatment. They are there to help you and support you. You should always speak to your CF team before you change, start, or stop any part of your treatment regimen.
Further information can be found in our factsheet Kaftrio: Complex and Individual Experiences. We will continue to update our information and guidance if and when new information becomes available.
If you’re struggling with any aspect of cystic fibrosis, we’re here to help. Contact our Helpline for support, a listening ear or just someone to talk to.
You can also report suspected side effects to medicines using the Yellow Card reporting scheme.
Here to help
If you’re struggling with any aspect of cystic fibrosis, we’re here to help. Contact our Helpline for support, a listening ear or just someone to talk to.