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Volume 12
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10.3390/children12020157
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This is an early access version, the complete PDF, HTML, and XML versions will be available soon.
Article

Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation

by
Eman A. Toraih
1,2,3,*,
Hassan A. Malik
4,
Rahib K. Islam
4,
Humza A. Pirzadah
4,
Ahmed Abdelmaksoud
1,
Rami M. Elshazli
1,5,6,
Paul Antwi Boasiako
7,
Shehab Ahmed Alenazi
8,
Angelique Dabel
7,
Jessan A. Jishu
9,
Bandar T. Alenezi
10,
Hani Aiash
11 and
Manal S. Fawzy
12
1
Department of Surgery, School of Medicine, Tulane University, New Orleans, LA 70112, USA
2
Department of Cardiovascular Perfusion, Interprofessional Research, College of Health Professions, SUNY Upstate Medical University, New York, NY 13210, USA
3
Medical Genetics Unit, Department of Histology and Cell Biology, Suez Canal University, Ismailia 41522, Egypt
4
Louisiana State University Health Sciences Center, New Orleans School of Medicine, New Orleans, LA 70112, USA
5
Department of Biochemistry and Molecular Genetics, Faculty of Physical Therapy, Horus University, New Damietta 34517, Egypt
6
Department of Biological Sciences, Faculty of Science, New Mansoura University, New Mansoura City 35742, Egypt
7
College of Health Professions, SUNY Upstate Medical University, New York, NY 13210, USA
8
Department of Pediatrics, Faculty of Medicine, Northern Border University, Arar 91431, Saudi Arabia
9
School of Medicine, Tulane University, New Orleans, LA 70112, USA
10
Department of Pharmacology, Faculty of Medicine, Northern Border University, Arar 91431, Saudi Arabia
11
SUNY Upstate Medical University, New York, NY 13210, USA
12
Center for Health Research, Northern Border University, Arar 91431, Saudi Arabia
 
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Author to whom correspondence should be addressed.
Children 2025, 12(2), 157; https://doi.org/10.3390/children12020157
Submission received: 18 December 2024 / Revised: 13 January 2025 / Accepted: 22 January 2025 / Published: 28 January 2025
(This article belongs to the Section Pediatric Pulmonary and Sleep Medicine)
Versions Notes

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.
Keywords: genetic inheritance; targeted therapy; global research network; multicenter; healthcare burden genetic inheritance; targeted therapy; global research network; multicenter; healthcare burden

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MDPI and ACS Style

Toraih, E.A.; Malik, H.A.; Islam, R.K.; Pirzadah, H.A.; Abdelmaksoud, A.; Elshazli, R.M.; Boasiako, P.A.; Alenazi, S.A.; Dabel, A.; Jishu, J.A.; et al. Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation. Children 2025, 12, 157. https://doi.org/10.3390/children12020157

AMA Style

Toraih EA, Malik HA, Islam RK, Pirzadah HA, Abdelmaksoud A, Elshazli RM, Boasiako PA, Alenazi SA, Dabel A, Jishu JA, et al. Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation. Children. 2025; 12(2):157. https://doi.org/10.3390/children12020157

Chicago/Turabian Style

Toraih, Eman A., Hassan A. Malik, Rahib K. Islam, Humza A. Pirzadah, Ahmed Abdelmaksoud, Rami M. Elshazli, Paul Antwi Boasiako, Shehab Ahmed Alenazi, Angelique Dabel, Jessan A. Jishu, and et al. 2025. "Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation" Children 12, no. 2: 157. https://doi.org/10.3390/children12020157

APA Style

Toraih, E. A., Malik, H. A., Islam, R. K., Pirzadah, H. A., Abdelmaksoud, A., Elshazli, R. M., Boasiako, P. A., Alenazi, S. A., Dabel, A., Jishu, J. A., Alenezi, B. T., Aiash, H., & Fawzy, M. S. (2025). Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation. Children, 12(2), 157. https://doi.org/10.3390/children12020157

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