Longitudinal Improvements in Clinical Outcomes for Children with Cystic Fibrosis Aged Six Years

Abstract

Background
Cystic Fibrosis (CF) is the most common life-shortening inherited lung disorder of Caucasian children. Advances in early diagnosis and management in the last twenty years have considerably improved the outcome of patients with CF. We aimed to assess whether outcomes for cystic fibrosis patients aged 6 years have improved in our centre since the introduction of these key measures over the past 20 years.

Methods
A retrospective observational study utilising a database of CF patients at Cork University Hospital (CUH).

Results
Over the 20-year period, age at CF diagnosis shifted markedly, with all patients in 2019–2023 diagnosed by newborn screening (20/20, 100%). Since 2014, mortality at age six has been eliminated (0/50, 0%) compared with 2/29 (6.9%) in 2002–2007 and 2/30 (6.7%) in 2008–2013, and chronic Pseudomonas aeruginosa colonisation fell to 0% from 3/29 (10.3%) in 2002–2007. CFTR modulator use rose from 0/29 (0%) to 10/20 (50.0%), though most recent patients had short treatment durations. Higher BMI was a statistically significant factor associated with better lung function (FEV₁ p=0.008; FVC p=0.006).

Discussion
Improvements in outcomes for children aged 6 years are yet to be fully realised with the commencement of CFTR modulator therapy in the first year of life; however, more sensitive diagnostics will be needed to monitor clinical progression.