In most countries the elexacaftor / tezacaftor / ivacaftor compound (ETI) is approved
for pwCF aged 6 and older and at least one F508del mutation. ETI is widely considered
a life changer by most who use or prescribe it, to the point that there is now some
discussion in the CF community about changes in the strategy of care and consideration
on how the future framework of CF centers may be affected by long term ETI usage [
[1]
]. The achievements of ETI have indeed been so patent that CF drug development is now
largely focused towards treatments for pwCF ineligible for CFTR modulator therapies
[
[2]
].To read this article in full you will need to make a payment
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References
Southern K.W., Castellani C., Lammertyn E., Smyth A., VanDevanter D., van Koningsbruggen-Rietschelet S., et al. Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis. Published online: 2022. DOI:10.1016/j.jcf.2022.10.002.
- Cystic fibrosis in the year 2020: a disease with a new face.Acta Paediatr. 2020; 109: 893-899https://doi.org/10.1111/apa.15155
- The L467F-F508del complex allele hampers pharmacological rescue of mutant CFTR by elexacaftor/tezacaftor/ivacaftor in cystic fibrosis patients: the value of the ex vivo nasal epithelial model to address non-responders to CFTR-modulating drugs.Int J Mol Sci. 2022; 23: 3175https://doi.org/10.3390/ijms23063175
- Personalized medicine with drugs targeting the underlying protein defect in cystic fibrosis: is monitoring of treatment response necessary?.Ther Adv Chronic Dis. 2022; 1320406223221108627https://doi.org/10.1177/20406223221108627
- Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele.N Engl J Med. 2019; 381: 1809-1819https://doi.org/10.1056/NEJMoa1908639
- Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.Lancet. 2019; 394: 1940-1948https://doi.org/10.1016/S0140-6736(19)32597-8
- Elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis homozygous for the F508del mutation and advanced lung disease: a 48-week observational study.J Clin Med. 2022; 11: 1021https://doi.org/10.3390/jcm11041021
- Clinical effectiveness of elexacaftor/tezacftor/ivacaftor in people with cystic fibrosis.Am J Respir Crit Care Med. 2021; https://doi.org/10.1164/rccm.202108-1986OC
- Efficacy of elexacaftor/tezacaftor/ivacaftor in advanced cystic fibrosis lung disease.Ann Am Thorac Soc. 2021; 18: 1924-1927https://doi.org/10.1513/AnnalsATS.202102-220RL
- Electronic monitoring reveals highly variable adherence patterns in patients prescribed ivacaftor.J Cyst Fibros. 2015; 14: 621-626https://doi.org/10.1016/j.jcf.2015.05.009
- Cis variants identified in F508del complex alleles modulate CFTR channel rescue by small molecules.Hum Mutat. 2018; 39: 506-514
- Evaluation of the complex p.[Leu467Phe;Phe508del] CFTR allele in the intestinal organoids model: implications for therapy.Int J Mol Sci. 2022; 23: 10377https://doi.org/10.3390/ijms231810377
- High frequency of complex CFTR alleles associated with c.1521_1523delCTT (F508del) in Russian cystic fibrosis patients.BMC Genomics. 2022; 23: 252https://doi.org/10.1186/s12864-022-08466-z
- Modifier factors of cystic fibrosis phenotypes: a focus on modifier genes.Int J Mol Sci. 2022; 23: 14205https://doi.org/10.3390/ijms232214205
- Synergy in cystic fibrosis therapies: targeting SLC26A9.Int J Mol Sci. 2021; 22: 13064
- Extracellular phosphate enhances the function of F508del-CFTR rescued by CFTR correctors.J Cyst Fibros. 2021; 20: 843-850
- Biological variability of the sweat chloride in diagnostic sweat tests: a retrospective analysis.J Cyst Fibros. 2017; 16: 30-35https://doi.org/10.1016/j.jcf.2016.11.008
- Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection.Chest. 2009; 135: 1610-1618https://doi.org/10.1378/chest.08-1190
Article info
Publication history
Published online: March 05, 2023
Accepted:
February 20,
2023
Received in revised form:
February 15,
2023
Received:
February 2,
2023
Publication stage
In Press Corrected ProofIdentification
Copyright
© 2023 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
