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Rapid Improvement after Starting Elexacaftor–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease

2021; American Thoracic Society; Volume: 204; Issue: 1 Linguagem: Inglês

10.1164/rccm.202011-4153oc

1535-4970

Pierre‐Régis Burgel, I. Durieu, R. Chiron, Sophie Ramel, Isabelle Danner‐Boucher, Anne Prévötat, Dominique Grenet, Christophe Marguet, Martine Reynaud‐Gaubert, Julie Macey, Laurent Mély, Annlyse Fanton, S. Quétant, Lydie Lemonnier, Jean-Louis Paillasseur, Jennifer Da Silva, C. Martín, Claire Andréjak, Arnaud Bécourt, J. Mounard, Claire Poulet, Cinthia Rames, Marie Talleux, Marie-Chantal Chevalier, E. Darviot, Marie Jouvenot, Caroline Marien, Audrey Paris, C. Pélatan, Christine Person, Pascaline Priou, Françoise Troussier, T. Urban, Marie‐Laure Dalphin, Jean‐Charles Dalphin, Alice Ladaurade, D. Pernet, Bénédicte Richaud-Thiriez, Pauline Roux-Claude, Nathalia Blanc, V. Boisserie-Lacroix, S. Bui, Cyrielle Collet, Stéphane Debelleix, Julie Macey, Emmanuel Bergot, Jacques Brouard, Karine Campbell, Muriel Laurans, Virginie Ribault, Corinne Borderon, M Héraud, Guillaume Labbé, Sylvie Montcouquiol, Isabelle Petit, M. Ruivard, Céline Delestrain, Benoît Douvry, Ralph Epaud, Bernard Maître, Natascha Remus, Guillaume Beltramo, Annlyse Fanton, Anne Houzel, Frédéric Huet, Stéphanie Perez, Amale Boldron-Ghaddar, Manuëla Scalbert, Rabah Bouzioukh, Laurent Mély, Charles E. Simon, B. Camara, Rébecca Hamidfar, C. Llerena, Isabelle Pin, S. Quétant, A. Deschildre, Alice Gicquello, Olivier Le Rouzic, Clara Leroy, Nicolas Paris, T. Pérez, Anne Prévötat, C. Thumerelle, Dominique Turck, Nathalie Wizla, Magali Dupuy-Grasset, J. Languepin, Alexandra Masson-Rouchaud, Céline Ménétrey, I. Durieu, S. Durupt, S. L’Excellent, R. Nové-Josserand, Camille Ohlmann, Philippe Reix, Quitterie Reynaud, Marie‐Christine Werck‐Gallois, Mélissandre Baravalle, Bérangère Coltey, N. Desmazes-Dufeu, J.‐C. Dubus, Clarisse Gautier, Jean‐Baptiste Rey, Martine Reynaud‐Gaubert, Nathalie Stremler, Davide Caimmi, R. Chiron, Margot Devrait, Johan Moreau, Yves Billon, Aurore Blondé, Anne Guillaumot, Sébastien Kiefer, Laura Peretti, Aurélie Tatopoulos, Angélica Tiotiu, Myriam Benhamida, Tiphaine Bihouée, Isabelle Danner‐Boucher, E. Eschapasse, Adrien Tissot, Marie Giannantonio, Sylvie Leroy, C. Piccini-Bailly, J. Pradelli, Jonathan Messika, Véronique Boussaud, Pierre‐Régis Burgel, Nicolas Carlier, Isabelle Honoré, D. Hubert, Reem Kanaan, C. Martín, Céline Bailly-Botuha, Frédérique Chedevergne, J. de Blic, Christophe Delacourt, David Drummond, Brigitte Fauroux, Chantal Karila, Muriel Le Bourgeois, Isabelle Sermet‐Gaudelus, Bertrand Delaisi, M. Gérardin, Véronique Houdouin, Laurence Le-Clainche, Guillaume Aubertin, Laura Berdah, Annick Clément, Harriet Corvol, Nadia Nathan, Blandine Prévost, N. Richard, Aline Tamalet, Jessica Taytard, Guillaume Thouvenin, B. Tourniaire, M. Abély, K. Bessaci-Kabouya, Sandra Dury, Bruno Ravoninjatovo, A. Dabadie, M. Dagorne, Eric Deneuville, Marie Jamin, Mélanie Ribault, C. Vigier, Chantal Belleguic, Graziella Brinchault, B. Desrues, Audrey Barzic, Anne Dirou-Prigent, Jean Le Bihan, Sophie Ramel, Krista Revert, Thomas Ropars, L. Couderc, S. Dominique, Christophe Marguet, Hélène Morisse‐Pradier, S. Pramil, Luc Thiberville, Nathalie Allou, Laurent Enaud, Elsa Gachelin, Eric Huchot, Annabelle Payet, Caroline Périsson, S. Piyaraly, Sophie Valois, Audrey Herzog, Romain Kessler, Michele Porzio, Laurence Weiss, L. Beaumont, Olivier Brugière, Sylvie Colin, de Verdiere, É. Cuquemelle, Sandra de Miranda, Dominique Grenet, Adbdul Monem Hamid, F Parquin, C. Picard, A. Roux, Charlotte Roy, François Brémont, A. Didier, Marion Dupuis, G. Faviez, Géraldine Labouret, M. Mittaine, M. Murris‐Espin, Léa Roditis, Laure Cosson, P. Diot, Thomas Flament, C. Giraut, Julie Mankikian, Baptiste Arnouat, Gaétane Mousset, V. Storni, Philippe Vigneron, Emmanuelle Coirier-Duet, Asma Gabsi,

Neonatal Respiratory Health Research

Rationale: Elexacaftor-tezacaftor-ivacaftor is a CFTR (cystic fibrosis [CF] transmembrane conductance regulator) modulator combination, developed for patients with CF with at least one Phe508del mutation. Objectives: To evaluate the effects of elexacaftor-tezacaftor- ivacaftor in patients with CF and advanced respiratory disease. Methods: A prospective observational study, including all patients aged ⩾12 years and with a percent-predicted FEV1 (ppFEV1) <40 who initiated elexacaftor-tezacaftor-ivacaftor from December 2019 to August 2020 in France was conducted. Clinical characteristics were collected at initiation and at 1 and 3 months. Safety and effectiveness were evaluated by September 2020. National-level transplantation and mortality figures for 2020 were obtained from the French CF and transplant centers and registries. Measurements and Main Results: Elexacaftor-tezacaftor- ivacaftor was initiated in 245 patients with a median (interquartile range) ppFEV1 = 29 (24-34). The mean (95% confidence interval) absolute increase in the ppFEV1 was +15.1 (+13.8 to +16.4; P < 0.0001), and the mean (95% confidence interval) in weight was +4.2 kg (+3.9 to +4.6; P < 0.0001). The number of patients requiring long-term oxygen, noninvasive ventilation, and/or enteral tube feeding decreased by 50%, 30%, and 50%, respectively (P < 0.01). Although 16 patients were on the transplant waiting list and 37 were undergoing transplantation evaluation at treatment initiation, only 2 received a transplant, and 1 died. By September 2020, only five patients were still on the transplantation path. Compared with the previous 2 years, a twofold decrease in the number of lung transplantations in patients with CF was observed in 2020, whereas the number of deaths without transplantation remained stable. Conclusions: In patients with advanced disease, elexacaftor-tezacaftor-ivacaftor is associated with rapid clinical improvement, often leading to the indication for lung transplantation being suspended.