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Войти в исследования The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant.
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Burgel Pierre-Régis, Sermet-Gaudelus Isabelle, Durieu Isabelle, Kanaan Reem, Macey Julie, Grenet Dominique, Porzio Michele, Coolen-Allou Nathalie, Chiron Raphael, Marguet Christophe, Douvry Benoit, Dufeu Nadine, Danner-Boucher Isabelle, Foucaud Pierre, Lemonnier Lydie, Girodon Emmanuelle, Da Silva Jennifer, Martin Clémence, French CF Reference Network study group, Andrejak Claire, Priou Pascaline, Richaud-Thiriez Bénédicte, Macey Julie, Montcouquiol Sylvie, Douvry Benoit, Remus Natacha, Fanton Annlyse, Coolen-Allou Nathalie, Gachelin Elsa, Vuillard Constance, Audousset Camille, Duthoit Louise, Bellet-Fraysse Elisabeth, Languepin Jeanne, Durieu Isabelle, Nove-Josserand Raphaele, Ohlmann Camille, Raynaud Quitterie, Dufeu Nadine, Chiron Raphael, Billon Yves, Danner-Boucher Isabelle, Tissot Adrien, Leroy Sylvie, Aubourg Frédérique, Burnet Espérie, Burgel Pierre-Régis, Carlier Nicolas, Da Silva Jennifer, Fajac Isabelle, Girodon Emmanuelle, Kanaan Reem, Honoré Isabelle, Martin Clémence, Le Bourgeois Muriel, Sermet-Gaudelus Isabelle, Le Clainche-Viala Laurence, Corvol Harriet, Dehillotte Clémence, Foucaud Pierre, Lemonnier Lydie, Brinchault Graziella, Ravoninjatovo Bruno, Le Bihan Jean, Ramel Sophie, Marguet Christophe, Porzio Michele, Weiss Laurence, Grenet Dominique, de Miranda Sandra, Cosson Laure, Mankikian Julie, Pouradier Delphine
Аннотация
METHODS: An observational study was conducted to evaluate the effectiveness of ETI in people with CF with advanced lung disease who were not eligible for ETI in Europe. All patients with no F508del variant and advanced lung disease (defined as having a percent predicted forced expiratory volume (ppFEV1) <40% and/or being under evaluation for lung transplantation) and enrolled in the French compassionate programme initiated ETI at recommended doses. Effectiveness was evaluated by a centralised adjudication committee at 4-6 weeks in terms of clinical manifestations, sweat chloride concentration and ppFEV1.
RESULTS: Among the first 84 people with CF included in the programme, ETI was effective in 45 (54%), and 39 (46%) were considered to be nonresponders. Among the responders, 22 (49%) out of 45 carried a CFTR variant that is not currently approved by the FDA for ETI eligibility. Important clinical benefits, including suspending the indication for lung transplantation, a significant decrease in sweat chloride concentration by a median (interquartile range (IQR)) -30 (-14--43) mmol·L-1 (n=42; p<0.0001) and an improvement in ppFEV1 by +10.0 (6.0-20.5) percentage points (n=44; p<0.0001), were observed in those for whom treatment was effective.
CONCLUSION: Clinical benefits were observed in a large subset of people with CF with advanced lung disease and CFTR variants not currently approved for ETI.