Cystic fibrosis: novel therapies, remaining challenges

Abstract

Developments in the treatment of cystic fibrosis (CF) over the past 50 years have seen survival extend from death in the first decade of life to an expected median survival now approaching 50 years of age. Adults with CF now outnumber children, a dramatic shift if one considers that many adult CF clinics were only established in the 1980s. Together with speciality multidisciplinary clinics, new medicines and their aggressive application have played a pivotal role in this achievement. Although there is still no clinically available cure, novel therapies targeting the underlying basic defects have resulted in major beneficial clinical outcomes and quality of life for those living with CF, albeit at significant cost. Although this review concentrates on currently available novel pharmacological therapies, the important role of potential ‘cures’, such as gene therapy, must not be overlooked. The complex drug interactions of the new agents, the need for detailed patient education and research opportunities in CF in general support the role of the specialist pharmacist in CF clinics.

Thriving alongside cystic fibrosis: Developing a grounded theory of empowerment in children and young people with cystic fibrosis during key life transitions

“…Approximately one in every 2,500 babies in the UK is born with CF (Cystic Fibrosis Trust, 2020a). Fifty years ago it was unlikely for a child with CF to live to their 10th birthday (Coulthard, 2018). With universal screening, earlier diagnosis, new understandings and treatments, life expectancy has increased dramatically, with over half of people with CF living beyond 47 years (Cystic Fibrosis Trust, 2020a).…”