EMERGING THERAPEUTIC AND DIGITAL INNOVATIONS IN CYSTIC FIBROSIS: A COMPREHENSIVE REVIEW

Gabriela Mroczka; Paulina Łobaza; Idalia Myszor; Michał Madej; Maria Kubacka; Lucjan Kocemba; Sylwia Nycz-Jucha; Brygida Pinkowicz; Piotr Rachwał; Barbara Nowak-Misiąg

doi:10.31435/ijitss.1(49).2026.4912

Authors

Gabriela Mroczka National Medical Institute of the Ministry of the Interior and Administration in Rzeszów, Rzeszów, Poland https://orcid.org/0009-0008-6382-3123
Paulina Łobaza Medical Center in Łańcut Sp. z o.o., Łańcut, Poland https://orcid.org/0009-0003-3566-005X
Idalia Myszor University Clinical Hospital F. Chopin in Rzeszów, Rzeszów, Poland https://orcid.org/0009-0008-5365-7037
Michał Madej University Clinical Hospital F. Chopin in Rzeszów, Rzeszów, Poland https://orcid.org/0009-0009-3411-2178
Maria Kubacka Saint Queen Jadwiga Clinical Regional Hospital No. 2 in Rzeszów, Rzeszów, Poland https://orcid.org/0000-0002-0789-5201
Lucjan Kocemba University Clinical Hospital F. Chopin in Rzeszów, Rzeszów, Poland https://orcid.org/0009-0006-0441-9927
Sylwia Nycz-Jucha Medical Center in Łańcut Sp. z o.o., Łańcut, Poland https://orcid.org/0009-0004-9042-2449
Brygida Pinkowicz Medical Center in Łańcut Sp. z o.o., Łańcut, Poland https://orcid.org/0009-0003-3290-7109
Piotr Rachwał University Clinical Hospital F. Chopin in Rzeszów, Rzeszów, Poland https://orcid.org/0009-0005-1434-1433
Barbara Nowak-Misiąg Medical Center in Łańcut Sp. z o.o., Łańcut, Poland https://orcid.org/0009-0000-9466-3443

DOI:

https://doi.org/10.31435/ijitss.1(49).2026.4912

Keywords:

Cystic Fibrosis, CFTR Modulators, Gene Therapy, Digital Health, Quality of Life

Abstract

Cystic fibrosis (CF) is an autosomal recessive, multisystem genetic disorder caused by mutations in the CFTR gene, leading to defective chloride transport across epithelial surfaces. Historically associated with early mortality, CF care has transformed over the last decade due to CFTR modulator therapies that target the underlying protein defect, significantly improving pulmonary function, reducing exacerbations, and enhancing quality of life. Concurrently, digital health technologies such as telemedicine, remote monitoring, artificial intelligence (AI) facilitated predictive tools, and wearable sensors are reshaping care delivery. Additionally, advances in gene therapy and personalized medicine hold potential for future curative approaches. This review synthesizes current evidence on CFTR modulators, emerging therapeutic strategies, the integration of digital health in CF care, and the psychosocial and economic challenges that persist. We provide a holistic perspective on how these developments collectively advance CF management, and we highlight ongoing research directions to further improve outcomes.

References

Winiarska, H. M., Springer, D., Wojtaś, F., Wysocka, E., & Cofta, S. (2024). CFTR modulators therapy efficacy in reducing cystic fibrosis (CF) exacerbation and improving selected spirometry parameters: A real-life study in a single-centre Polish population. Journal of Clinical Medicine, 13(15), Article 4491. https://doi.org/10.3390/jcm13154491

Regard, L., Martin, C., Burnet, E., Da Silva, J., & Burgel, P.-R. (2022). CFTR modulators in people with cystic fibrosis: Real-world evidence in France. Cells, 11(11), Article 1769. https://doi.org/10.3390/cells11111769

Fainardi, V., Capoferri, G., Tornesello, M., Pisi, G., & Esposito, S. (2023). Telemedicine and its application in cystic fibrosis. Journal of Personalized Medicine, 13(7), Article 1041. https://doi.org/10.3390/jpm13071041

Gramegna, A., Contarini, M., Aliberti, S., Casciaro, R., Blasi, F., & Castellani, C. (2020). From ivacaftor to triple combination: A systematic review of efficacy and safety of CFTR modulators in people with cystic fibrosis. International Journal of Molecular Sciences, 21(16), Article 5882. https://doi.org/10.3390/ijms21165882

Pastor-Vivero, M. D., Costa i Colomer, J., Martín de Vicente, C., Vicente-Santamaría, S., García Romero, R., González Jiménez, D., & Luna Paredes, C. (2025). Advances in the treatment of cystic fibrosis: CFTR modulators. Anales de Pediatría (English Edition), 102(5), Article 503857. https://doi.org/10.1016/j.anpede.2025.503857

Tümmler, B., Pallenberg, S. T., Dittrich, A. M., Graeber, S. Y., Naehrlich, L., Sommerburg, O., & Mall, M. A. (2025). Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators. Molecular and Cellular Pediatrics, 12(1), Article 6. https://doi.org/10.1186/s40348-025-00194-0

Vagg, T., Shanthikumar, S., Morrissy, D., Chapman, W. W., Plant, B. J., & Ranganathan, S. (2021). Telehealth and virtual health monitoring in cystic fibrosis. Current Opinion in Pulmonary Medicine, 27(6), 544–553. https://doi.org/10.1097/MCP.0000000000000821

Lomunova, M. A., & Gershovich, P. M. (2023). Gene therapy for cystic fibrosis: Recent advances and future prospects. Acta Naturae, 15(2), 20–31. https://doi.org/10.32607/actanaturae.11708

Mazurek, H., Emeryk, A., Janeczek, K., Derom, E., Kuźnar-Kamińska, B., Grzywalski, T., Biniakowski, A., Szarzyński, K., Pastusiak, A., Kaminiarczyk-Pyzałka, D., Botteldooren, D., Hafke-Dys, H., & Kociński, J. (2025). AI-facilitated home monitoring for cystic fibrosis exacerbations across pediatric and adult populations. Journal of Cystic Fibrosis, 24(2), 390–397. https://doi.org/10.1016/j.jcf.2025.02.011