Poor tolerability of cystic fibrosis transmembrane conductance regulator modulator therapy in lung transplant recipients

Doligalski, Christina T.; McKinzie, Cameron J; Yang, Anita; Lobo, Leonard J.; Coakley, Raymond D.

doi:10.1002/phar.2710

Cited by 18 publications

(32 citation statements)

References 11 publications

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“…In lung-transplanted recipients, the potential beneficial effects of ETI have been assessed for other CF-related implications, such as sinus disease, gastrointestinal symptoms, and CFRD [ 89 , 90 , 91 ]. Benniger and collaborators followed nine adults with CF during one year of ETI therapy and observed an improvement in BMI, gastrointestinal and sinus symptoms, as well as a decrease in glucose levels.…”

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

confidence: 99%

“…The remaining eight participants who continued ETI therapy demonstrated no differences in lung function and BMI, although improvements in sinus and gastrointestinal symptoms were reported by six and four individuals, respectively. Only one participant demonstrated an improved glycemic index [ 90 ]. In the study performed by Ramos and collaborators, only 8 out of 94 lung transplant recipients eligible for ETI therapy received the prescription in the first year after the transplant.…”

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

confidence: 99%

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

confidence: 99%

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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“…Therapy with ELX/TEZ/IVA was safe and efficacious post liver transplant ( McKinzie et al, 2022 ; Ragan et al, 2022 ). Conversely, when ELX/TEZ/IVA was prescribed to lung transplant recipients for extrapulmonary complications of CF, triple therapy was poorly tolerated with modest perceived extrapulmonary benefit so that about 40% of patients discontinued the medication ( Doligalski et al, 2022 ; Ramos et al, 2022 ). In summary, with the exception of lung transplant recipients, treatment with ELX/TEZ/IVA led to a strong improvement of the quality of life.…”

Section: Quality Of Life During Elx/tez/iva Therapy In Patient Groups...mentioning

confidence: 99%

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor

Tümmler

2023

Front. Pharmacol.

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Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF). We provide an overview of the body of literature on ELX/TEZ/IVA published between November 2019 and February 2023 after approval by the regulators. Recombinant ELX/TEZ/IVA-bound Phe508del CFTR exhibits a wild type conformation in vitro, but in patient’s tissue a CFTR glyoisoform is synthesized that is distinct from the wild type and Phe508del isoforms. ELX/TEZ/IVA therapy improved the quality of life of people with CF in the real-life setting irrespective of their anthropometry and lung function at baseline. ELX/TEZ/IVA improved sinonasal and abdominal disease, lung function and morphology, airway microbiology and the basic defect of impaired epithelial chloride and bicarbonate transport. Pregnancy rates were increasing in women with CF. Side effects of mental status changes deserve particular attention in the future.

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“…For PwCF who have received a lung transplant, use of CFTR modulators is an unstudied area. When used, however, most are initiated on ETI for nonpulmonary reasons 48,49 . Interactions with immunosuppressant medications were seen, with one single‐center study of 14 PwCF reporting a median dose reduction of 50% 48 while a combined report from 14 lung transplant centers of 94 PwCF, showed only about one‐third required dose adjustments 49 .…”

Section: Cf Modulatorsmentioning

confidence: 99%

Cystic fibrosis year in review 2022

Kim,

Lyman,

Savant

2023

Pediatric Pulmonology

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Remarkable medical advancements have been made for people with cystic fibrosis (CF) in recent years, with an abundance of research continuing to be conducted worldwide. With concern for limitations in access to highly effective CFTR modulators, as well as the recent Coronavirus Disease‐19 pandemic, there has been a consistent effort to understand and improve CF screening, disease burden, diagnosis, and management. Our aim in this review is to present articles from 2022 with an emphasis on clinically relevant studies. We hope this will serve as a broad overview of the research published in the past year.

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Poor tolerability of cystic fibrosis transmembrane conductance regulator modulator therapy in lung transplant recipients

Cited by 18 publications

References 11 publications

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

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

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor

Cystic fibrosis year in review 2022

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