Major Decrease in Lung Transplantation for Patients with Cystic Fibrosis in France

Martin, Clémence; Legeai, Camille; Regard, Lucile; Cantrelle, C.; Dorent, Richard; Carlier, Nicolas; Kerbaul, François; Burgel, Pierre‐Régis

doi:10.1164/rccm.202109-2121le

Cited by 37 publications

(16 citation statements)

References 12 publications

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“…A prospective, observational study found a twofold decrease in the number of LTxs performed in PwCF over 12 years of age in 2020 compared to the 2 previous years 5 . Further work by others has confirmed that the need for LTx is dropping in PwCF 6,7 . Notably, none of this work has addressed whether these changes in LTx in PwCF differ by race and/or ethnicity.…”

Section: Figurementioning

confidence: 89%

Changing racial and ethnic differences for lung transplantation in cystic fibrosis

Hayes

Dani

Guzman-Gomez

et al. 2022

Pediatric Transplantation

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Section: Figurementioning

confidence: 89%

Changing racial and ethnic differences for lung transplantation in cystic fibrosis

Hayes

Dani

Guzman-Gomez

et al. 2022

Pediatric Transplantation

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“…These findings are the first published evidence of prolonged disease modification in patients with advanced pulmonary disease who met the eligibility criteria for lung transplantation [ 46 ]. Interestingly, recent data from the French Agence de la Biomédecine, which monitors organ transplantation at the national level, showed a major decrease in lung transplantation for pwCF since the release of ELX/TEZ/IVA [ 71 ]. The French ELX/TEZ/IVA real-word studies are ongoing and include all children and adults with CF receiving this modulator combination ELX/TEZ/IVA nationally.…”

Section: Elexacaftor/tezacaftor/ivacaftormentioning

confidence: 99%

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

Regard

Martin

Burnet

et al. 2022

Cells

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Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death. CFTR modulators, which were developed in the past decade, partially restore CFTR protein function. Their clinical efficacy has been demonstrated in phase 3 clinical trials, particularly in terms of lung function and pulmonary exacerbations, nutritional status, and quality of life in people with gating mutations (ivacaftor), homozygous for the F508del mutation (lumacaftor/ivacaftor and tezacaftor/ivacaftor), and in those with at least one F508del mutation (elexacaftor/tezacaftor/ivacaftor). However, many questions remain regarding their long-term safety and effectiveness, particularly in patients with advanced lung disease, liver disease, renal insufficiency, or problematic bacterial colonization. The impact of CFTR modulators on other important outcomes such as concurrent treatments, lung transplantation, chest imaging, or pregnancies also warrants further investigation. The French CF Reference Network includes 47 CF centers that contribute patient data to the comprehensive French CF Registry and have conducted nationwide real-world studies on CFTR modulators. This review seeks to summarize the results of these real-world studies and examine their findings against those of randomized control trials.

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“…Overall, lung transplants decreased by 56.4% in France, attributed to ETI, as the COVID related decrease in non -CF indications only dropped 26.4% 31 . When data from prior to 2002 was comparted to late 2020/early 2021, France saw a tenfold reduction in transplantation rate 35 . Similarly, according to an analysis of the International Society of Health Lung Transplantation, between 1990-2017, there was a decrease in CF as an indication for lung transplantation 36 .…”

Section: Elexacaftor/tezacaftor/ivacaftormentioning

confidence: 99%

Cystic fibrosis year in review 2021

Savant

2022

Preprint

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People with cystic fibrosis (CF) have an amazing outlook with the treatment availability of highly effective modulators. Unfortunately, not all PwCF are eligible for modulators leading to continued pulmonary exacerbations and advanced lung disease. Additionally, optimizing diagnosis and evaluation for CF in the newborn period continues to be an area of focus for research. This review article will work to cover articles published in 2021 with high clinical relevance related to the above topics, however due to the extensive body of research published, this review will not be comprehensive.

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Major Decrease in Lung Transplantation for Patients with Cystic Fibrosis in France

Cited by 37 publications

References 12 publications

Changing racial and ethnic differences for lung transplantation in cystic fibrosis

Changing racial and ethnic differences for lung transplantation in cystic fibrosis

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

Cystic fibrosis year in review 2021

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