Peripheral lung effect of elexacaftor/tezacaftor/ivacaftor in adult cystic fibrosis

Stylemans, Dimitri; Darquenne, Chantal; Schuermans, Daniël; Verbanck, Sylvia; Vanderhelst, Eef

doi:10.1016/j.jcf.2021.03.016

Cited by 15 publications

(13 citation statements)

References 18 publications

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“…Taking into consideration the significant amelioration of other markers, such as acinar ventilation heterogeneity, RV/TLC ratio, FVC, and V A , they concluded that there was a considerable ventilation distribution improvement. In addition, the exacerbation rate diminished from 0.33 to 0.07/month after 3 months of treatment [ 27 ].…”

Section: Resultsmentioning

confidence: 99%

“…Stylemans et al, during the 3-month therapy, showed that it was well-tolerated and only one patient had a drug-induced liver injury leading to treatment discontinuation. No rash, dyspnea, or cough was observed [ 27 ]. A serious adverse event of pancreatitis and distal intestinal obstructive syndrome occurred in one patient who participated in the Bermingham et al study, without interruption of the treatment; mild side effects, such as rashes, constipation, and hypoglycemia were seen in 10 participants [ 28 ].…”

Section: Resultsmentioning

confidence: 99%

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Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

et al. 2023

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Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic fibrosis (CF). The objective of this study was a systematic research of the literature on the efficacy and safety of this CFTR modulator on patients with CF. A search of Pubmed was conducted for randomized clinical trials and observational studies published from 2012 to September 2022. The included full manuscripts comprised nine clinical trials and 16 observational studies, whose participants were aged ≥12 years or were children 6–11 years old with at least one Phe508del mutation and/or advanced lung disease (ALD). These studies reported that ELX/TEZ/IVA has a significant positive effect on the lung function of patients with CF, by ameliorating parameters such as FEV1, LCI, pulmonary exacerbations or sweat chloride concentration, increasing BMI and improving quality of their life. Its role in cystic fibrosis-related diabetes (CFRD) is not yet clear. It was found that this new CFTR modulator has an overall favorable safety profile, with mild to moderate adverse events. Further studies are needed for a deeper understanding of the impact of CFTR modulators on other CF manifestations, or the possibility of treating with ELX/TEZ/IVA CF patients with rare CFTR mutations.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

et al. 2023

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“…MRI and MBW again have been shown to be more sensitive than FEV 1 , especially when the treatment effect is of modest size [ 74 , 76 ]. In a study of MBNW in ALD during treatment with ETI, MBW outcome parameters showed larger improvements than ppFEV 1 , suggesting the main treatment effect takes place in the peripheral airways [77] . Longitudinal studies confirm that MBNW, CT, and hyperpolarized (129) xenon MRI are all able to detect disease progression in preschool children and children and adolescents with CF [78] , [79] , [80] .…”

Section: Early Lung Diseasementioning

confidence: 94%

A year in review: Real world evidence, functional monitoring and emerging therapeutics in 2021

Barr

Bihouée²,

Zwitserloot

2022

Journal of Cystic Fibrosis

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“…Stylemans and collaborators performed a real-life, three-months follow-up study and investigated the effects of ETI therapy on LCI and the proximal and peripheral components of the ventilation distribution in 14 adults with CF and advanced lung disease (average age of 36 years, median ppFEV 1 of 34%) [ 52 ]. All these individuals had pancreatic insufficiency, 50% had CFRD, and 64% were homozygous for F508del-CFTR.…”

Section: Clinical Outcomes Of Eti Therapy In Case Reports Observation...mentioning

confidence: 99%

“…The LCI (by 31% predicted, below baseline 247% predicted) and ventilation heterogeneity in the acinar compartment (by 411% predicted, below baseline 798% predicted) were measured. Overall, most therapeutic benefits were observed within 2–4 weeks after the start of ETI therapy and particularly beneficial effects were observed in peripheral zones of the lungs [ 52 ]. Another prospective, real-life study was performed by Kos and collaborators in a follow-up of 12 months [ 53 ].…”

Section: Clinical Outcomes Of Eti Therapy In Case Reports Observation...mentioning

confidence: 99%

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

et al. 2023

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Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly modified the lives of many people with CF (PwCF) by targeting the fundamental cause of the disease. These drugs consist of the potentiator ivacaftor (VX-770) and the correctors lumacaftor (VX-809), tezacaftor (VX-661), and elexacaftor (VX-445). In particular, the triple combination of CFTR modulators composed of elexacaftor, tezacaftor, and ivacaftor (ETI) represents a life-changing therapy for the majority of PwCF worldwide. A growing number of clinical studies have demonstrated the safety and efficacy of ETI therapy in both short- and long-term (up to two years of follow-up to date) and its ability to significantly reduce pulmonary and gastrointestinal manifestations, sweat chloride concentration, exocrine pancreatic dysfunction, and infertility/subfertility, among other disease signs and symptoms. Nevertheless, ETI therapy-related adverse effects have also been reported, and close monitoring by a multidisciplinary healthcare team remains vital. This review aims to address and discuss the major therapeutic benefits and adverse effects reported by the clinical use of ETI therapy for PwCF.

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Peripheral lung effect of elexacaftor/tezacaftor/ivacaftor in adult cystic fibrosis

Cited by 15 publications

References 18 publications

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

A year in review: Real world evidence, functional monitoring and emerging therapeutics in 2021

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

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