Cystic fibrosis: deaths on the decline – now the focus is on ‘quality of life’

For healthcare professionals and patients alike, part of this positive outcome is down to modulatory drugs. This is because, in the treatment of cystic fibrosis, the picture over the last five years shows improving figures, with a reduction in crude mortality and in the median age at death. The data were presented during the National Cystic Fibrosis Forum in Alghero. As highlighted by the organisers, this is the “most important annual event” for the Italian cystic fibrosis community. According to the latest report from 2024, crude mortality (including transplant recipients) fell from 6.3 per cent to 2.7 per cent between 2019 and 2024. The median age at death rose from 35.7 to 57.2 between 2019 and 2024.

The focus of researchers, patients and their families is on modulator drugs, “which have profoundly changed the lives of many people with cystic fibrosis, opening up prospects that were unthinkable just a few years ago”. “Access to modulators has represented a genuine revolution in the lives of a great many patients, a historic achievement secured thanks to the tenacity and constant commitment of our association, working alongside the institutions,” says Antonio Guarini, president of the LIFC (Italian Cystic Fibrosis League). “It is a development which, alongside the clinical results, also brings with it new organisational and care-related challenges.”

Not only that: ‘Precisely in light of this change,’ emphasises Guarini, ‘it is essential to continue discussing adherence to conventional treatments, psychological wellbeing, independence, multidisciplinary care pathways and quality of life’. Equally important is the dialogue between associations, specialists and healthcare professionals, which highlights how therapeutic advances are making care for adult patients increasingly central, requiring specialised centres, multidisciplinary teams and integrated care pathways linking hospitals and the local community, with particular attention to the transition from paediatric to adult care.

One of the key issues highlighted during the Forum was that of social and labour market inclusion. It is no coincidence that the association has signed agreements with various Italian companies to facilitate the integration of people with cystic fibrosis into the labour market. At the heart of the League’s work are also what have been termed ‘unresolved issues’, such as access to treatment ‘for those who are still excluded from it, and the management of treatment centres; these will certainly not remain mere words on paper’. ‘ “We will continue to fight for the things the cystic fibrosis community needs and we will leave no one behind,” Guarini emphasised once again, “even if there were just one patient without treatment, we would fight for them, because this is our aim and the mission that sets us apart.”