Drug combination for cystic fibrosis looks promising

Conclusion This trial has demonstrated that this new treatment combination could be effective in improving lung function for people with cystic fibrosis who have two copies of the common Phe508del CFTR mutation. The trial has many strengths, including its large sample size and the fact it captured outcomes at six months for almost all participants. The improvements in lung function were seen while the participants continued to use their standard cystic fibrosis treatments. As the researchers suggest, this indicates the treatment could be a beneficial add-on to normal care to further improve symptoms. The results seem very promising, but there are limitations that should be addressed. Though lung function improvements were said to be clinically meaningful, improvements in quality of life relating to lung function fell short of what is considered to be meaningful clinically (four points and above on the CFQ-R scale). The trial only included people with well-controlled cystic fibrosis, and effects of the treatment might not be as good for people with poorer disease control. The treatment combination would also only be suitable for people with the Phe508del CFTR mutation. This trial only included people with two copies of this mutation, which is only the case in around 45% of people with the condition. Whether the treatment would benefit people who carry one copy of the Phe508del mutation and a different second CFTR mutation is not yet clear, and people with two non-Phe508del ...
Source: NHS News Feed - Category: Consumer Health News Tags: Heart/lungs Medication Source Type: news