Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor

Balmpouzis, Zisis; Rossum, Annabelle Faure van; Baud, David; Panchaud, Alice; Mitropoulou, Georgia; Stalder, Jesica Mazza; Koutsokera, Angela

doi:10.1016/j.rmcr.2022.101776

Cited by 5 publications

(10 citation statements)

References 10 publications

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“…Thirteen case reports included pregnant pwCF who remained on IVA (four), LUM/IVA (three), or ELE/TEZ/IVA (six) throughout all three trimesters 12,18–27 . One pregnant person on IVA and another pregnant person on LUM/IVA remained on modulator therapy throughout all three trimesters for two separate pregnancies 22,23 .…”

Section: Resultsmentioning

confidence: 99%

“…One case report described a pregnant person with borderline severe lung disease (ppFEV 1 = 40%) who remained on ELE/TEZ/IVA throughout all three trimesters. In this case, the person gave birth to a healthy infant at 36 weeks as well as experienced no postpartum complications 26 . In a cohort study evaluating the outcomes of pwCF who continued, discontinued, or never started ELE/TEZ/IVA, among those that continued therapy did not require intravenous antibiotics.…”

Section: Resultsmentioning

confidence: 99%

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Use of CFTR modulators in special populations, part 1: Pregnancy and lactation

Elijah,

Fitzgerald,

Phan

2023

Pediatric Pulmonology

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Safety and efficacy data regarding cystic fibrosis transmembrane conductance regulator (CFTR) modulator use in the setting of pregnancy or breastfeeding remains lacking due to exclusion from key trials and lack of multicenter prospective and retrospective studies in the post‐CFTR modulator era. A scoping review of English articles from the period of January 1, 2012, to July 31, 2023, was conducted utilizing PubMed and EmBase databases with the following terms: “special population (pregnancy, lactation, breastfeeding)” AND “ivacaftor OR lumacaftor OR tezacaftor OR elexacaftor”; “cystic fibrosis transmembrane conductance regulator” AND “off label drug use.” Search results were reviewed by title and abstract for duplications and relevance. Relative to pregnancy or breastfeeding, a total of 18 publications were included for review. Majority of case reports and surveys concluded maternal and infant health were preserved throughout gestation. Likewise, breastfeeding infant case reports show possible changes in liver function and lens opacities, though risk may be increased with both in‐utero and breastfeeding exposure. Ivacaftor (IVA) and lumacaftor (LUM) concentrations in fetal cord blood and maternal blood were found to be equivalent. Yet, low concentrations of IVA and LUM were detectable in breastmilk and infant plasma. Current safety data surrounding CFTR modulator use in the setting of pregnancy and lactation is relatively reassuring; however, long‐term safety remains unclear, necessitating ongoing observation, and reporting by care teams. As such, treatment decisions should be individualized and coproduced.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Use of CFTR modulators in special populations, part 1: Pregnancy and lactation

Elijah,

Fitzgerald,

Phan

2023

Pediatric Pulmonology

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“…Over the follow-up of this study, high SCC was the only clinical feature observed in this infant with CF [ 114 ]. Another woman with CF (30 years old, F508del/Y1092X) got pregnant four months after she started ETI therapy [ 115 ]. Her FEV 1 improved one month after the start of ETI therapy and remained between 35–40% throughout the pregnancy.…”

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

confidence: 99%

“…After childbirth, oxygen therapy was stopped and her FEV 1 improved again to 40% of the predicted value. Over the follow-up of one year, no further complications were reported [ 115 ]. In another case report performed by Szentpetery and collaborators, the effects of ETI therapy were assessed on a woman (F508del-CFTR carrier) who was pregnant with a fetus with CF (F508del/F508del genotype) [ 116 ].…”

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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“…Most women who discontinued ELX/TEZ/IVA during pregnancy out of concern for unknown fetal risk restarted because of clinical deterioration (Taylor-Cousar and Jain, 2021). Even a case of successful pregnancy and uncomplicated delivery has been reported for a woman with CF with very poor lung function (ppFEV1 23%) prior to conception (Balmpouzis et al, 2022). ELX/TEZ/IVA pass the placental barrier (Collins et al, 2022).…”

Section: Reproductive Tract and Pregnancymentioning

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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Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor

Cited by 5 publications

References 10 publications

Use of CFTR modulators in special populations, part 1: Pregnancy and lactation

Use of CFTR modulators in special populations, part 1: Pregnancy and lactation

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

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