Document Type
Article
Publication Date
1-28-2025
Publication Title
Children
Abstract
BACKGROUND/OBJECTIVES: Cystic fibrosis (CF) is a life-limiting genetic disorder affecting multiple organ systems. This study compared clinical outcomes, hospitalization rates, and survival between children and adolescents with CF who received CFTR modulator therapies (ivacaftor, lumacaftor, tezacaftor, and elexacaftor). METHODS: A retrospective cohort study was conducted using data from the TriNetX global collaborative network. Patients with CF aged 2-12 years (children) and 13-18 years (adolescents) who received CFTR modulator therapies were included. The propensity score matching balanced baseline characteristics between the two age groups. RESULTS: After propensity score matching, 946 patients per group were analyzed. The incidence of respiratory failure (3.81% vs. 1.06%, p < 0.001) and respiratory infections (62.7% vs. 57.5%, p = 0.021) were significantly higher in adolescents compared to children. Adolescents had a higher risk of respiratory failure (HR = 3.6, 95% CI = 1.79-7.21, p < 0.001) and respiratory infections (HR = 1.09, 95% CI = 1.01-1.17, p < 0.001). Adolescents also had a higher hospitalization rate (29.6% vs. 20.3%, p < 0.001), with a 47% higher risk (HR = 1.47, 95% CI = 1.22-1.77, p = 0.001), more hospital visits per person (8.8 vs. 3.7, p = 0.004), and longer hospital stays (32.7 vs. 20.4 days, p = 0.006). Mortality rates were similar between the groups (1.58% vs. 1.26%, p = 0.56). CONCLUSIONS: CF patients who initiated CFTR modulator therapies during adolescence had a higher incidence of respiratory failure, respiratory infections, hospitalization rates, and healthcare resource utilization compared to those who started therapy in childhood, despite similar mortality rates. These findings highlight the importance of the early initiation of CFTR modulator therapies.
PubMed ID
40003259
Volume
12
Issue
2
Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 International License.
Recommended Citation
Toraih, Eman A.; Malik, Hassan A.; Islam, Rahib K.; Pirzadah, Humza A.; Abdelmaksoud, Ahmed; Elshazli, Rami M.; Boasiako, Paul Antwi; Alenazi, Shehab Ahmed; Dabel, Angelique; Jishu, Jessan A.; Alenezi, Bandar T.; Aiash, Hani; and Fawzy, Manal S., "Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation" (2025). School of Medicine Faculty Publications. 3528.
https://digitalscholar.lsuhsc.edu/som_facpubs/3528
10.3390/children12020157
Included in
Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Genetic Phenomena Commons, Investigative Techniques Commons