Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study
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Background Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele. To assess long-term effects of ELX/TEZ/IVA under real-world conditions of use, a 5-year observational registry-based study is being conducted. We report interim results from the first 2 years of follow-up. Methods The study included people with CF in the US Cystic Fibrosis Foundation Patient Registry (CFFPR) who initiated ELX/TEZ/IVA between October 2019 and December 2020. Pulmonary exacerbations (PEx), percent predicted forced expiratory volume in 1 second (ppFEV 1 ), hospitalizations, bacterial pathogens, body mass index (BMI), CF complications and comorbidities, and liver function tests (LFTs) after treatment initiation were compared with the 5-year pre-treatment period. Death and lung transplantation were assessed relative to 2019 CFFPR data. Results 16,116 people with CF were included (mean treatment duration 20.4 months). Among those with 5 years of pre-treatment data, mean PEx/patient/year declined to 0.18 (95% CI: 0.17, 0.19) in Years 1 and 2 post-treatment from 0.86 (95% CI: 0.83, 0.88) in the baseline year (79% reduction), after a continued increase observed pre-treatment. Similarly, a decline in mean hospitalizations/patient/year was observed in Year 1 that was sustained in Year 2 (74% reduction from baseline year). The mean absolute change in ppFEV 1 from baseline was +8.2 percentage points (95% CI: 8.0, 8.4) in Year 1 and +8.9 percentage points (95% CI: 8.7, 9.1) in Year 2, after a continued decline observed pre-treatment. Positive bacterial cultures decreased for all evaluated pathogens, and mean BMI increased by 1.6 kg/m 2 (95% CI: 1.5, 1.6) by Year 2. No new safety concerns were identified based on evaluation of CF complications, comorbidities, and LFTs. The annualized rates of death (0.47% [95% CI: 0.39, 0.55]) and lung transplantation (0.16% [95% CI: 0.12, 0.22]) were considerably lower than reported in 2019 (1.65% and 1.08%, respectively). Conclusions ELX/TEZ/IVA treatment was associated with sustained improvements in lung function, reduced frequency of PEx and all-cause hospitalization, increased BMI, and lower prevalence of positive bacterial cultures. Additionally, there was a 72% lower rate of death and 85% lower rate of lung transplantation relative to the year before ELX/TEZ/IVA availability. These results, from the largest cohort of ELX/TEZ/IVA-treated people to date, extend our understanding of the broad clinical benefits of ELX/TEZ/IVA.
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Results from studies on the safety and efficacy of real-world use of ELX/TEZ/IVA have been consistent with the results reported here.
“However, results from studies on the safety and efficacy of real-world use of ELX/TEZ/IVA have been consistent with the results reported here [25].”
Cites
Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA
Riordan, J. R., Rommens, J. M., Kerem, B.-S., Alon, N., Rozmahel, R., Grzelczak, Z., Zielenski, J., Lok, S., Plavsic, N., Chou, J.-L., et al. (1989) · Science · doi:10.1126/science.2475911 · Cited by 7,418 · see citing works
Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Middleton, P. G., Mall, M. A., Dřevínek, P., Lands, L. C., McKone, E. F., Polineni, D., Ramsey, B. W., Taylor-Cousar, J. L., Tullis, E., Vermeulen, F., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1908639 · Cited by 2,198 · see citing works
Self-citationElborn, J. S. (2016) · The Lancet · doi:10.1016/s0140-6736(16)00576-6 · Cited by 1,710 · see citing works
Heijerman, H. G. M., McKone, E. F., Downey, D. G., Van Braeckel, E., Rowe, S. M., Tullis, E., Mall, M. A., Welter, J. J., Ramsey, B. W., McKee, C. M., et al. (2019) · The Lancet · doi:10.1016/s0140-6736(19)32597-8 · Cited by 1,414 · see citing works
Self-citationO'Sullivan, B. P., & Freedman, S. D. (2009) · The Lancet · doi:10.1016/s0140-6736(09)60327-5 · Cited by 1,320 · see citing works
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
Goor, F. V., Hadidaꝉ, S., Grootenhuis, P. D. J., Burton, B., Cao, D., Neuberger, T., Turnbull, A., Singh, A. K., Joubran, J., Hazlewood, A., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0904709106 · Cited by 1,225 · see citing works
The future of cystic fibrosis care: a global perspective
Bell, S. C., Mall, M. A., Gutierrez, H., Macek, M., Madge, S. L., Davies, J. C., Burgel, P., Tullis, E., Castaños, C., Castellani, C., et al. (2019) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(19)30337-6 · Cited by 1,042 · see citing works
VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
Keating, D., Marigowda, G., Burr, L., Daines, C., Mall, M. A., McKone, E. F., Ramsey, B. W., Rowe, S. M., Sass, L. A., Tullis, E., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1807120 · Cited by 726 · see citing works
Self-citationCFTR Function and Prospects for Therapy
Riordan, J. R. (2008) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.75.103004.142532 · Cited by 610 · see citing works
Quittner, A. L., Goldbeck, L., Abbott, J., Duff, A., Lambrecht, P., Solé, A., Tibosch, M. M., Bergsten Brucefors, A., Yüksel, H., Catastini, P., et al. (2014) · Thorax · doi:10.1136/thoraxjnl-2014-205983 · Cited by 524 · see citing works
Knapp, E. A., Fink, A. K., Goss, C. H., Sewall, A., Ostrenga, J., Dowd, C., Elbert, A., Petren, K., & Marshall, B. C. (2016) · Annals of the American Thoracic Society · doi:10.1513/annalsats.201511-781oc · Cited by 467 · see citing works
Self-citationZemanick, E. T., Taylor‐Cousar, J. L., Davies, J. C., Gibson, R. L., Mall, M. A., McKone, E. F., McNally, P., Ramsey, B. W., Rayment, J. H., Rowe, S. M., et al. (2021) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.202102-0509oc · Cited by 315 · see citing works
Self-citationBessonova, L., Volkova, N., Higgins, M., Bengtsson, L., Tian, S., Simard, C., Konstan, M. W., Sawicki, G. S., Sewall, A., Nyangoma, S., et al. (2018) · Thorax · doi:10.1136/thoraxjnl-2017-210394 · Cited by 214 · see citing works
Self-citationGriese, M., Costa, S., Linnemann, R. W., Mall, M. A., McKone, E. F., Polineni, D., Quon, B. S., Ringshausen, F. C., Taylor-Cousar, J. L., Withers, N. J., et al. (2021) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.202008-3176le · Cited by 163 · see citing works
Pulmonary Exacerbations in Adults With Cystic Fibrosis
Stanford, G. E., Dave, K., & Simmonds, N. J. (2020) · CHEST Journal · doi:10.1016/j.chest.2020.09.084 · Cited by 58 · see citing works
The impact of CFTR modulator therapies on CF airway microbiology
Rogers, G. B., Taylor, S., Hoffman, L. R., & Burr, L. D. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.07.008 · Cited by 56 · see citing works
Patel, S., Thompson, M., Slaven, J. E., Sanders, D. B., & Ren, C. L. (2021) · Pediatric Pulmonology · doi:10.1002/ppul.25250 · Cited by 41 · see citing works
Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations
Munck, À., Kerem, E., Ellemunter, H., Campbell, D., Wang, L. T., Ahluwalia, N., Owen, C. A., & Wainwright, C. E. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.04.015 · Cited by 36 · see citing works
Self-citationCFTR modulators: transformative therapies for cystic fibrosis
Dwight, M., & Marshall, B. C. (2021) · Journal of Managed Care & Specialty Pharmacy · doi:10.18553/jmcp.2021.27.2.281 · Cited by 32 · see citing works
Triple Therapy for Cystic Fibrosis Phe508del –Gating and –Residual Function Genotypes
Barry, P. J., Mall, M. A., & Polineni, D. (2021) · New England Journal of Medicine · doi:10.1056/nejmc2115966 · Cited by 19 · see citing works
Griese, M., Tullis, E. D., Chilvers, M. A., Fabrizzi, B., Jain, R., Legg, J., Mall, M. A., McKone, E. F., Polineni, D., Poplawska, K., et al. (2022) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(22)00861-x · Cited by 12 · see citing works
Self-citationMenten, E. R., Saroya, T. K., DiMango, E., & Keating, C. (2021) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(21)01602-7 · Cited by 3 · see citing works
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Mall, M. A., Burgel, P., Castellani, C., Davies, J. C., Salathe, M. A., & Taylor‐Cousar, J. L. (2024) · Nature Reviews Disease Primers · doi:10.1038/s41572-024-00538-6 · Cited by 223 · see citing works
CFTR modulator therapy: Transforming the landscape of clinical care in cystic fibrosis
Taylor-Cousar, J. L., Robinson, P. D., Shteinberg, M., & Downey, D. G. (2023) · The Lancet · doi:10.1016/s0140-6736(23)01609-4 · Cited by 173 · see citing works
Keating, C. L., Yonker, L. M., Vermeulen, F. L., Prais, D., Linnemann, R. W., Trimble, A. T., Kotsimbos, T. C., Mermis, J. D., Braun, A. T., O’Carroll, M., et al. (2025) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(24)00411-9 · Cited by 132 · see citing works
Self-citationDaines, C. L., Tullis, E., Costa, S., Linnemann, R. W., Mall, M. A., McKone, E. F., Polineni, D., Quon, B. S., Ringshausen, F. C., Rowe, S. M., et al. (2023) · European Respiratory Journal · doi:10.1183/13993003.02029-2022 · Cited by 100 · see citing works
Self-citationBurgel, P., Southern, K. W., Addy, C., Battezzati, A., Berry, C. A., Bouchara, J., Brokaar, E. J., Brown, A. W., Azevedo, P., Durieu, I., et al. (2024) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2024.01.005 · Cited by 94 · see citing works
Hoppe, J. E., Kasi, A. S., Pittman, J. E., Jensen, R., Thia, L. P., Robinson, P. J., Tirakitsoontorn, P., Ramsey, B. W., Mall, M. A., Taylor‐Cousar, J. L., et al. (2025) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(24)00407-7 · Cited by 69 · see citing works
Burgel, P., Sermet‐Gaudelus, I., Girodon, E., Durieu, I., Houdouin, V., Audousset, C., Macey, J., Grenet, D., Porzio, M., Murris‐Espin, M., et al. (2024) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(24)00208-x · Cited by 63 · see citing works
Hisert, K. B., Birket, S. E., Clancy, J. P., Downey, D. G., Engelhardt, J. F., Fajac, I., Gray, R. D., Lachowicz-Scroggins, M. E., Mayer-Hamblett, N., Thibodeau, P. H., et al. (2023) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(23)00324-7 · Cited by 61 · see citing works
Elexacaftor/Tezacaftor/Ivacaftor Treatment and Depression-related Events
Ramsey, B. W., Correll, C. U., DeMaso, D. R., McKone, E. F., Tullis, E. D., Taylor‐Cousar, J. L., Chu, C., Volkova, N. V., Ahluwalia, N., Waltz, D., et al. (2023) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.202308-1525oc · Cited by 57 · see citing works
Self-citationDittrich, A., Sieber, S., Naehrlich, L., Burkhart, M., Hafkemeyer, S., & Tümmler, B. (2023) · International Journal of Infectious Diseases · doi:10.1016/j.ijid.2023.11.013 · Cited by 46 · see citing works
Kerem, E., Orenti, A., Adamoli, A., Hatziagorou, E., Naehrlich, L., & Sermet‐Gaudelus, I. (2024) · European Respiratory Journal · doi:10.1183/13993003.01241-2023 · Cited by 44 · see citing works
Burgel, P., Sermet‐Gaudelus, I., Girodon, E., Kanaan, R., Bihan, J. L., Remus, N., Ravoninjatovo, B., Grenet, D., Porzio, M., Houdouin, V., et al. (2024) · European Respiratory Journal · doi:10.1183/13993003.01959-2023 · Cited by 38 · see citing works
Current landscape of cystic fibrosis gene therapy
Plasschaert, L. W., MacDonald, K. D., & Moffit, J. S. (2024) · Frontiers in Pharmacology · doi:10.3389/fphar.2024.1476331 · Cited by 27 · see citing works
Milczewska, J., Syunyaeva, Z., Żabińska-Jaroń, A., Sands, D., & Thee, S. A. (2024) · European Respiratory Review · doi:10.1183/16000617.0068-2024 · Cited by 26 · see citing works
Terlizzi, V., Fevola, C., Presti, S., Castaldo, A., Daccò, V., Claut, L. E., Sepe, A. O., Majo, F., Casciaro, R., Esposito, I., et al. (2024) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2024.114176 · Cited by 25 · see citing works
Eldredge, J. A., Oliver, M. R., & Ooi, C. Y. (2023) · Paediatric Respiratory Reviews · doi:10.1016/j.prrv.2023.12.005 · Cited by 24 · see citing works
Burgel, P., Orenti, A., Cromwell, E. A., Maçek, M., Gutierrez, H. H., Karadag, B. T., Faro, A., van Rens, J. G., Naehrlich, L., Bakkeheim, E., et al. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.10.007 · Cited by 22 · see citing works
Self-citationSomerville, L., Borish, L., Imre, N., & Albon, D. P. (2024) · Therapeutic Advances in Respiratory Disease · doi:10.1177/17534666241297877 · Cited by 21 · see citing works
Zampoli, M., Verstraete, J., Baird, C., Calligaro, G., & Morrow, B. M. (2023) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.202307-1200vp · Cited by 21 · see citing works
Castaldo, A., Gelzo, M., Iacotucci, P., Longobardi, A., Taccetti, G., Terlizzi, V., & Carnovale, V. (2024) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2023.1327958 · Cited by 21 · see citing works
Castaldo, A., Iacotucci, P., Bagnasco, S., Fevola, C., Carnovale, V., Antonelli, F., Cernera, G., Gelzo, M., & Terlizzi, V. (2024) · Scientific Reports · doi:10.1038/s41598-024-68511-7 · Cited by 21 · see citing works
Cromwell, E. A., Ostrenga, J. S., Sanders, D. B., Morgan, W. J., Castellani, C., Szczesniak, R. D., & Burgel, P. (2024) · European Respiratory Journal · doi:10.1183/13993003.01146-2024 · Cited by 20 · see citing works
Self-citationRubin, J. L., McKinnon, C. J., Pedra, G., Morgan, D. A., Zweig, K., & Liou, T. G. (2025) · Pulmonary Therapy · doi:10.1007/s41030-025-00303-4 · Cited by 19 · see citing works
Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators
Tümmler, B., Pallenberg, S. T., Dittrich, A., Graeber, S. Y., Naehrlich, L., Sommerburg, O., & Mall, M. A. (2025) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-025-00194-0 · Cited by 18 · see citing works
Salvatore, D., & Pepe, A. (2025) · Journal of Clinical Medicine · doi:10.3390/jcm14113978 · Cited by 18 · see citing works
Robertson, J. K., & Goldberg, J. B. (2025) · Microbiology · doi:10.1099/mic.0.001553 · Cited by 15 · see citing works
Schechter, M. S., Anaya, N. S., Oster, G., Weycker, D. A., Wu, H., Arteaga‐Solis, E., Bagal, S., McGarry, L. J., Brunt, K. V., & Geiger, J. M. (2023) · Pulmonary Therapy · doi:10.1007/s41030-023-00241-z · Cited by 15 · see citing works
Beswick, D. M., Khatiwada, A., Miller, J. E., Humphries, S. M., Wilson, A. W. M., Vladar, E. K., Lynch, D. A., & Taylor‐Cousar, J. L. (2023) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2023.09.013 · Cited by 14 · see citing works
Filho, L. V. R. F. D. S., Athanazio, R. A., Tonon, C. R., Ferreira, J. C., & Tanni, S. É. (2023) · Jornal Brasileiro de Pneumologia · doi:10.36416/1806-3756/e20230187 · Cited by 14 · see citing works
Vega-Hernandez, G., MacGregor, G., Wilfin, A., Adams, F., Haugh, C., Baxter, C. A., Wöhling, H., Clarke, S. L., Charman, S. C., & Carr, S. B. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.04.012 · Cited by 13 · see citing works
Lucca, F., Meneghelli, I., Tridello, G., Buniotto, F., Cucchetto, G., Volpi, S., Pintani, E., Bezzerri, V., & Cipolli, M. (2025) · Journal of Clinical Medicine · doi:10.3390/jcm14124335 · Cited by 13 · see citing works
Keens, T. G., Hoffman, V., Topuria, I., Elder, K., Cerf, S., Mulder, K., Roberts, J., Lysinger, J., Reyes, M. D. C., Berdella, M. N., et al. (2024) · Heliyon · doi:10.1016/j.heliyon.2024.e28508 · Cited by 12 · see citing works
Changing Epidemiology of Pediatric Pulmonary Exacerbations in Cystic Fibrosis
Fireizen, Y., Ahmed, M., Vigers, T., Akong, K., Ryu, J., Hahn, A., Fanous, H., Koumbourlis, A. C., Tirakitsoontorn, P., Arrieta, A. C., et al. (2025) · Pediatric Pulmonology · doi:10.1002/ppul.71019 · Cited by 11 · see citing works
Naehrig, S., Shad, C., Breuling, M., Goetschke, M., Habler, K., Sieber, S., Kastenberger, J., Kunzelmann, A. K., Sommerburg, O., Liebchen, U., et al. (2024) · Journal of Personalized Medicine · doi:10.3390/jpm14101065 · Cited by 11 · see citing works
Cystic fibrosis liver disease in the post-modulator era
Narkewicz, M. R. (2023) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000001017 · Cited by 10 · see citing works
Vonk, S. E. M., van der Meer‐Vos, M., Kos, R., Neerincx, A. H., Terheggen‐Lagro, S. W. J., Altenburg, J., der Zee, A. H. M., Mathôt, R. A. A., & Kemper, E. M. (2024) · Therapeutic Drug Monitoring · doi:10.1097/ftd.0000000000001231 · Cited by 10 · see citing works
Vonk, S. E. M., Terheggen‐Lagro, S. W. J., Haarman, E. G., Hashimoto, S., der Zee, A. H. M., Mathôt, R. A. A., & Kemper, E. M. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.03.008 · Cited by 10 · see citing works
Managing an ageing cystic fibrosis population: challenges and priorities
Frost, F. J., Peckham, D. G., Felton, I. C., Snowball, J., Gray, R. D., Jones, A. M., Simmonds, N. J., Lord, R. W., Lip, G. Y. H., Chandler, H., et al. (2025) · European Respiratory Review · doi:10.1183/16000617.0261-2024 · Cited by 10 · see citing works
Daccò, V., Alicandro, G., Trespìdi, L., Gramegna, A., & Blasi, F. (2023) · Archives of Gynecology and Obstetrics · doi:10.1007/s00404-023-07153-y · Cited by 10 · see citing works
Bader-Larsen, K. S., Nielsen, B. U., Henriksen, E., Bryrup, T., Leo-Hansen, C., Mathiesen, I. H. M., Faurholt-Jepsen, D., Qvist, T., Oturai, P., Langdahl, B. L., et al. (2025) · Clinical Nutrition · doi:10.1016/j.clnu.2025.09.008 · Cited by 9 · see citing works
Castellani, C., Mondéjar-López, P., Goor, F. V., Quon, B., Alghisi, F., Fabrizzi, B., Ramsey, B. W., Taylor-Cousar, J. L., McKone, E. F., Tullis, E. W., et al. (2026) · American Journal of Respiratory and Critical Care Medicine · doi:10.1093/ajrccm/aamaf001 · Cited by 9 · see citing works
Self-citationAspergillus in Children and Young People with Cystic Fibrosis: A Narrative Review
Chesshyre, E. L. D., Wooding, E. L., Sey, E. A., & Warris, A. (2025) · Journal of Fungi · doi:10.3390/jof11030210 · Cited by 8 · see citing works
Williams, C. L., Billings, J., McGowan, H., McDevitt, R., Esther, C. R., McKinzie, C. J., Wilson, W. S., & Kam, C. W. (2025) · Pediatric Pulmonology · doi:10.1002/ppul.71038 · Cited by 8 · see citing works
Leo‐Hansen, C., Faurholt‐Jepsen, D., Qvist, T., Højte, C., Nielsen, B. U., Bryrup, T., Henriksen, E. H., Katzenstein, T. L., Skov, M., Mathiesen, I. H. M., et al. (2024) · ERJ Open Research · doi:10.1183/23120541.00339-2024 · Cited by 8 · see citing works
Management of liver disease and portal hypertension in cystic fibrosis: a review
Colombo, C. M., Lanfranchi, C., Tosetti, G., Corti, F., & Primignani, M. (2024) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2024.2365842 · Cited by 8 · see citing works
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Yue, M., Weiner, D. J., Gaietto, K. M., Rosser, F. J., Qoyawayma, C., Manni, M. L., Myerburg, M. M., Pilewski, J. M., Celedon, J. C., Chen, W., et al. (2024) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2024-0103oc · Cited by 8 · see citing works
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Gold, L. S., Hansen, R. N., Mayer-Hamblett, N., Nichols, D. P., Gifford, A. H., Kloster, M., Goss, C. H., & Kessler, L. G. (2023) · Journal of Managed Care & Specialty Pharmacy · doi:10.18553/jmcp.2024.30.1.26 · Cited by 8 · see citing works
Durieu, I., Clements, B. S., Fabrizzi, B., Mall, M. A., McKone, E. F., Ramsey, B. W., Tullis, E. D., Taylor‐Cousar, J. L., van der Meer, R., Bachman, E. S., et al. (2025) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.202411-2312oc · Cited by 7 · see citing works
Self-citationHøjte, C., Olsen, M. F., Faurholt‐Jepsen, D., Jørgensen, M. H., Pressler, T., Leo‐Hansen, C., Bryrup, T., Jeppesen, M., Olesen, H. V., Petersen, J., et al. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.08.008 · Cited by 6 · see citing works
Volpi, S., Ambroni, M., Buzzetti, R., Cimino, G. G., Gramegna, A., Lucanto, M. C., Ripani, P., Ros, M., Salvatore, D., Spada, E., et al. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms262110513 · Cited by 6 · see citing works
Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis
Merlo, C. A., McGarry, L. J., Thorat, T., Nguyen, C., DerSarkissian, M., Muthukumar, A., Healy, J., Brookhart, M. A., & Rubin, J. L. (2024) · Thorax · doi:10.1136/thorax-2023-220559 · Cited by 6 · see citing works
Yaacoby‐Bianu, K., Dagan, A., Cohen‐Cymberknoh, M., Tsviban, L., Gur, M., Golan‐Tripto, I., Aviram, M., Stein, N., Shteinberg, M., Kerem, E., et al. (2024) · Annals of the American Thoracic Society · doi:10.1513/annalsats.202401-030rl · Cited by 6 · see citing works
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Wu, M., & Chen, J.-H. (2024) · Frontiers in Physiology · doi:10.3389/fphys.2024.1385661 · Cited by 5 · see citing works
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IFTIKHAR, I. H., Rao, S. T., Nadama, R., Janahi, I. A., & BaHammam, A. S. (2025) · Lung · doi:10.1007/s00408-025-00802-w · Cited by 5 · see citing works
Merlo, C. A., Geiger, J. M., Wang, Z., Lyden, G. R., Schladt, D. P., & McGarry, L. J. (2025) · Respiratory Medicine · doi:10.1016/j.rmed.2025.108171 · Cited by 5 · see citing works
Salvatore, D., Iacotucci, P., Pepe, A., Terminiello, A., Majo, F., Carnovale, V., Vitullo, P., Messore, B., Parisi, G. F., Calderazzo, M. A., et al. (2025) · Respiratory Medicine · doi:10.1016/j.rmed.2025.108221 · Cited by 5 · see citing works
Elexacaftor–tezacaftor–ivacaftor use after solid organ transplant
Kapnadak, S. G., & Ramos, K. J. (2024) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000001110 · Cited by 5 · see citing works
Chesshyre, E. L. D., Enderby, B., Shore, A. C., Warren, F. C., & Warris, A. (2025) · Journal of Fungi · doi:10.3390/jof11020116 · Cited by 5 · see citing works
Walicka‐Serzysko, K., Postek, M., Mielus, M., Borawska-Kowalczyk, U., Milczewska, J., Zybert, K., Woźniacki, Ł., Wołkowicz, A., & Sands, D. (2025) · Journal of Clinical Medicine · doi:10.3390/jcm14227969 · Cited by 4 · see citing works
Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience
FRAGOSO, E. C., Boaventura, R. D. P., Almeida, L., Amorim, A. A., Gamboa, F., Santos, A. S., Gonçalves, F., Cruz, C. M., Carreiro, A., Gonçalves, A. S., et al. (2024) · Pulmonary Pharmacology & Therapeutics · doi:10.1016/j.pupt.2024.102328 · Cited by 4 · see citing works
Exploring reduced pulmonary exacerbation rates in cystic fibrosis: a pandemic or modulator effect?
Stephenson, A. L., Sykes, J. R., Stanojevic, S., Ma, X., Ostrenga, J., Todd, J., Faro, A., Elbert, A., Marshall, B. C., Ramos, K. J., et al. (2025) · ERJ Open Research · doi:10.1183/23120541.00111-2025 · Cited by 4 · see citing works
Self-citationCastaldo, A., Cimbalo, C., Fevola, C., Carnovale, V., Iacotucci, P., Raia, V., Terlizzi, V., Gelzo, M., Sepe, A. O., & Tosco, A. (2025) · Scientific Reports · doi:10.1038/s41598-025-12333-8 · Cited by 4 · see citing works
Epidemiology of advanced cystic fibrosis lung disease in the modulator era
Westhoff, J., & Naehrlich, L. (2024) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000001115 · Cited by 4 · see citing works
Hadhud, M., Halevy, D., Reiter, J. M., Ben-Meir, E., Gileles‐Hillel, A., Sadras, I., Kerem, E., Cohen‐Cymberknoh, M., & Breuer, O. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.05.005 · Cited by 3 · see citing works
Castaldo, A., Cimbalo, C., Fevola, C., Raia, V., Terlizzi, V., Gelzo, M., Sepe, A. O., & Tosco, A. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.10.001 · Cited by 3 · see citing works
Cipolli, M., Thorat, T., Pafundi, P. C., Roggeri, A., Roggeri, D. P., Rossi, C., Pintani, E., Meneghelli, I., Lucca, F., Fratoni, A., et al. (2025) · Pulmonary Therapy · doi:10.1007/s41030-025-00334-x · Cited by 3 · see citing works
Salvatore, D., Campagna, G., Padoan, R., Pepe, A., Amato, A., & Salvatore, M. (2026) · Journal of Clinical Medicine · doi:10.3390/jcm15072699 · Cited by 3 · see citing works
Ruck, J. M., Feng, S. N., Toporek, A. H., Shah, P. D., Tallarico, E., Lechtzin, N., Massie, A. B., Segev, D. L., Bush, E. L., & Merlo, C. A. (2025) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2025.01.006 · Cited by 3 · see citing works
Qualitative Analysis of Perspectives on Lung Transplant among People with Cystic Fibrosis
Milinic, T., Hobler, M. R., Bartlett, L., Gill, E. R., Burdis, N., Engelberg, R. A., Curtis, J. R., Hartzler, A. L., Aitken, M. L., Kapnadak, S. G., et al. (2024) · Annals of the American Thoracic Society · doi:10.1513/annalsats.202307-625oc · Cited by 3 · see citing works
Wajda, K. E., Roesch, E. A., & Gifford, A. H. (2023) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000001006 · Cited by 3 · see citing works
Cystic fibrosis year in review 2023
Swetland, D. V., & Savant, A. P. (2024) · Pediatric Pulmonology · doi:10.1002/ppul.27190 · Cited by 3 · see citing works
Letter to the editor: A contemporary assessment of CFTR modulator use and eligibility
Rubin, J. L., & Kreindler, J. L. (2024) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2024.02.014 · Cited by 3 · see citing works
Outcomes of Modulator Therapy Discontinued After Short‐Term Use in Adult Cystic Fibrosis
Karcıoğlu, O., İdikut, A., Öztürk, E., & Damadoğlu, E. (2024) · Pediatric Pulmonology · doi:10.1002/ppul.27416 · Cited by 2 · see citing works
Long-term exercise capacity in paediatric cystic fibrosis: a modern era perspective
Gaupmann, R., Stöger, L., Schmidthaler, K., Pauger, B., Mersi, B., Graf, A. C., Szépfalusi, Z., Renner, S., Dehlink, E., & Gruber, S. (2025) · ERJ Open Research · doi:10.1183/23120541.00971-2024 · Cited by 2 · see citing works
The Changing Transplant Landscape in the Era of Elexacaftor/Tezacaftor/Ivacaftor: A Word of Caution
Semenchuk, J. A., Tuff-Gordon, E., Ma, X., Sykes, J. R., Cheng, S. Y., Aversa, M. M., Chaparro, C., Tullis, E. D., & Stephenson, A. L. (2025) · Clinical Transplantation · doi:10.1111/ctr.70317 · Cited by 2 · see citing works
Dillenhöfer, S., Schütz, K., Burkhart, M., Ellemunter, H., Kappler, M., Sieber, S., Naehrlich, L., Brinkmann, F., Sutharsan, S., & German CF Registry of the Mukoviszidose e.V. (2025) · ERJ Open Research · doi:10.1183/23120541.00813-2025 · Cited by 2 · see citing works
Johnston, J. R., Donnelley, M., Smith, R., Louise, J., Grunert, J., Vanda, C. L., & Tai, A. (2025) · Respiratory Medicine · doi:10.1016/j.rmed.2025.108410 · Cited by 2 · see citing works
Ergenekon, P., Selçuk, M., Ünal, G., Bayramoğlu, G. G., Altuntaş, C., Köse, M., Başaran, A. E., Oksay, S. C., Uytun, S., Öktem, S., et al. (2025) · Frontiers in Pediatrics · doi:10.3389/fped.2025.1659633 · Cited by 2 · see citing works
Manioudaki, S., Vasilieva, L., Geladari, E. V., Mani, I., Athanassa, Z., Elefsiniotis, I. S., Hadziyannis, E. S., Sevastianos, V., Oikonomou, A., Theophilou, A., et al. (2025) · Life · doi:10.3390/life15121794 · Cited by 2 · see citing works
Canan, M. G. M., Sokoloski, C. S., Severo, C. R., Rached, S. Z., & Athanazio, R. A. (2024) · Archivos de Bronconeumología · doi:10.1016/j.arbres.2024.04.007 · Cited by 2 · see citing works
Nicholson, J. M., Goldstein, R. S., Nourouzpour, S., Elangeswaran, B., Munawar, M., Wickerson, L. M., Keshavjee, S. H., Tullis, E. D., Chaparro, C., & Rozenberg, D. (2025) · Respiratory Medicine · doi:10.1016/j.rmed.2025.107982 · Cited by 2 · see citing works
García, M. S., Peláez, A., Gómez-Punter, R. M., López, M. C., Carbajal, C. M., Ancochea, J., Bachiller, J. M. E., Hernández, A. S. M., Rodrigo-García, M., Clemente, M. M. G., et al. (2025) · BMC Pulmonary Medicine · doi:10.1186/s12890-024-03455-2 · Cited by 2 · see citing works
Cystic fibrosis and other ion channel-related diseases
Graeber, S. Y., & Mall, M. A. (2023) · European Respiratory Society eBooks · doi:10.1183/2312508x.10018022 · Cited by 2 · see citing works
Median age of survival in the 80s! Is there sufficient evidence to believe it?
Stanojevic, S., Mayer-Hamblett, N., Szczesniak, R. D., Cromwell, E. A., & Keogh, R. H. (2023) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2023.06.004 · Cited by 2 · see citing works
Cast no shadow: assessing the disease-modifying effects of elexacaftor/tezacaftor/ivacaftor
Barry, P. J., & Burgel, P. (2023) · European Respiratory Journal · doi:10.1183/13993003.02026-2023 · Cited by 2 · see citing works
The course of cystic fibrosis in adult patients with Burkholderia cepacia complex infection
Afanaseva, M. V., Krasovskiy, S., & Amelina, E. (2025) · PULMONOLOGIYA · doi:10.18093/0869-0189-2025-35-2-241-253 · Cited by 1 · see citing works
Isse, S., Wahla, A. S., Uzbeck, M. H., Zoumot, Z., Abuzakouk, M., & Shafiq, I. (2025) · Pulmonary Medicine · doi:10.1155/pm/3622052 · Cited by 1 · see citing works
An update on cystic fibrosis Hepato-biliary involvement
Freeman, A. J., & Bodewes, F. A. J. A. (2026) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2026.03.022 · Cited by 1 · see citing works
Валеева, Д. С., & Trofimova, V. G. (2026) · Meditsinskiy sovet = Medical Council · doi:10.21518/ms2026-289 · Cited by 1 · see citing works
Hussain, K., Karhana, S., Garg, A., & Khan, M. A. (2025) · Thoracic research and practice · doi:10.4274/thoracrespract.2025.2025-1-1 · Cited by 1 · see citing works
Afonso, P. M., Zhou, G. C., Su, W., Burgel, P., Cromwell, E. A., Goss, C. H., Keogh, R. H., Liou, T. G., Marshall, B. C., Mayer-Hamblett, N., et al. (2025) · Annals of the American Thoracic Society · doi:10.1513/annalsats.202503-267oc · Cited by 1 · see citing works
Self-citationTosto, M., Parisi, G. F., Presti, S., Papale, M. R., Pecora, G., Mulè, E., Ornato, V., Aloisio, D., Manti, S., & Leonardi, S. (2025) · Life · doi:10.3390/life15121942 · Cited by 1 · see citing works
The impact of elexacaftor–tezacaftor–ivacaftor on cardiometabolic risk factors: a systematic review
Girouard, H., Jaber, F., Gharib, J., Boudreau, V., Alexandre‐Heymann, L., & Coriati, A. (2026) · European Respiratory Review · doi:10.1183/16000617.0197-2025 · Cited by 1 · see citing works
Aridgides, D. S., Mellinger, D., Gwilt, L. L., Correia, A. R., Finger, C. E., Goddard, J., Hampton, T. H., Mould, D. L., Hogan, D. A., & Ashare, A. (2026) · mSphere · doi:10.1128/msphere.00702-25 · Cited by 1 · see citing works
Zamarrón, E., Cuadrado, A. P., Baloira, L., Carpio, C., García, I. A., Milla, S. P., de Villar, N. G. P., Mangas, A., Aguilar, P. M., Walther, R. Á.-S., et al. (2026) · Clinical Nutrition ESPEN · doi:10.1016/j.clnesp.2026.103275 · Cited by 1 · see citing works
Årman, F., Diemer, S., Happonen, L., & Påhlman, L. I. (2026) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2026.05.002 · Cited by 1 · see citing works
Råket, H. K., Ankarfeldt, M. Z., Wang, J. N., Pressler, T., Jensen‐Fangel, S., Qvist, T., Faurholt‐Jepsen, D., Jimenez‐Solem, E., Petersen, J., Jensen, C. B., et al. (2025) · Pulmonary Therapy · doi:10.1007/s41030-025-00287-1 · Cited by 1 · see citing works
Terlizzi, V., Benedetto, M. D. D., Renzo, S., Galici, V., Scarallo, L., Mainz, J. G., & Lionetti, P. (2025) · Clinical Therapeutics · doi:10.1016/j.clinthera.2025.04.002 · Cited by 1 · see citing works
Somayaji, R., & Quon, B. S. (2023) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000001010 · Cited by 1 · see citing works
Reducing treatment burden in the era of CFTR modulators – Authors' reply
Gifford, A. H., Mayer-Hamblett, N., & Nichols, D. P. (2023) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(23)00224-2 · Cited by 1 · see citing works
- Authors
- Bower, J. K., Volkova, N., Ahluwalia, N., Sahota, G., Xuan, F., Chin, A., Weinstock, T. G., Ostrenga, J., & Elbert, A.
- Affiliations
- Vertex Pharmaceuticals (United States) (US)
- Cystic Fibrosis Foundation (US)
- Corresponding author
- Bower, Julie K.
- Venue
- Journal of Cystic Fibrosis
- Publisher
- Elsevier BV
- Published
- 2023-07
- Volume
- 22
- Issue
- 4
- Pages
- 730-737
- ISSN
- 1569-1993 (ISSN-L)
- OpenAlex
- W4353046996
- Cited by
- 140
- References
- 22
- Citations by year
- 2026: 35 · 2025: 51 · 2024: 33 · 2023: 21
- FWCI
- 25.67
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Journal of Cystic Fibrosis (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Funders
- Vertex Pharmaceuticals Incorporated
- Topics
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmacogenetics and Drug Metabolism
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Keywords
- elexacaftor/tezacaftor/ivacaftor, CFTR modulator therapy, F508del-CFTR, pulmonary exacerbations, hospitalization reduction, FEV1 decline, pathogenic bacteria, body mass index, liver function tests, lung transplantation, long-term safety, real-world effectiveness
- MeSH terms
- Aminophenols, Cystic Fibrosis, Humans, Indoles, Mutation, Pyrazoles, Pyridines, Pyrrolidines, Registries, Quinolones, Cystic Fibrosis Transmembrane Conductance Regulator, Benzodioxoles, Chloride Channel Agonists
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