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Trials and tribulations of highly effective modulator therapies in cystic fibrosis.
- Source :
-
Paediatric respiratory reviews [Paediatr Respir Rev] 2023 Dec; Vol. 48, pp. 10-19. Date of Electronic Publication: 2023 Sep 15. - Publication Year :
- 2023
-
Abstract
- Highly effective modulator therapies (HEMTs) have revolutionised the management approach of most patients living with cystic fibrosis (CF) who have access to these therapies. Clinical trials have reported significant improvements across multiorgan systems, with patients surviving longer. However, there are accumulating case reports and observational data describing various adverse events following initiation of HEMTs including drug-to-drug interactions, drug induced liver injury, Stevens-Johnson syndrome, and neurocognitive symptoms including psychosis and depression, which have required discontinuation of therapy. Current clinical trials are assessing efficacy in younger patients with CF, yet long-term studies are also required to better understand the safety profile in the real-world setting across all ages and the impact of HEMT dose alteration or discontinuation.<br />Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.<br /> (Crown Copyright © 2023. Published by Elsevier Ltd. All rights reserved.)
Details
- Language :
- English
- ISSN :
- 1526-0550
- Volume :
- 48
- Database :
- MEDLINE
- Journal :
- Paediatric respiratory reviews
- Publication Type :
- Academic Journal
- Accession number :
- 37914566
- Full Text :
- https://doi.org/10.1016/j.prrv.2023.09.001