Rapid Improvement after Starting Elexacaftor-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease
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.
Errataetall: |
CommentIn: Am J Respir Crit Care Med. 2021 Aug 1;204(3):372-374. - PMID 33951399 |
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Medienart: |
E-Artikel |
Erscheinungsjahr: |
2021 |
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Erschienen: |
2021 |
Enthalten in: |
Zur Gesamtaufnahme - volume:204 |
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Enthalten in: |
American journal of respiratory and critical care medicine - 204(2021), 1 vom: 01. Juli, Seite 64-73 |
Sprache: |
Englisch |
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Beteiligte Personen: |
Burgel, Pierre-Régis [VerfasserIn] |
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Links: |
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Anmerkungen: |
Date Completed 13.09.2021 Date Revised 13.09.2021 published: Print CommentIn: Am J Respir Crit Care Med. 2021 Aug 1;204(3):372-374. - PMID 33951399 Citation Status MEDLINE |
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doi: |
10.1164/rccm.202011-4153OC |
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funding: |
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Förderinstitution / Projekttitel: |
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PPN (Katalog-ID): |
NLM321595416 |
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500 | |a CommentIn: Am J Respir Crit Care Med. 2021 Aug 1;204(3):372-374. - PMID 33951399 | ||
500 | |a Citation Status MEDLINE | ||
520 | |a 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 | ||
650 | 4 | |a Journal Article | |
650 | 4 | |a Observational Study | |
650 | 4 | |a Research Support, Non-U.S. Gov't | |
650 | 4 | |a cystic fibrosis transmembrane conductance regulator modulators | |
650 | 4 | |a elexacaftor | |
650 | 4 | |a lung transplantation | |
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650 | 7 | |a Pyridines |2 NLM | |
650 | 7 | |a Quinolines |2 NLM | |
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700 | 1 | |a Prevotat, Anne |e verfasserin |4 aut | |
700 | 1 | |a Grenet, Dominique |e verfasserin |4 aut | |
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700 | 1 | |a Fanton, Annlyse |e verfasserin |4 aut | |
700 | 1 | |a Quetant, Sébastien |e verfasserin |4 aut | |
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700 | 1 | |a Da Silva, Jennifer |e verfasserin |4 aut | |
700 | 1 | |a Martin, Clémence |e verfasserin |4 aut | |
700 | 0 | |a French Cystic Fibrosis Reference Network Study Group |e verfasserin |4 aut | |
700 | 1 | |a Andrejak, Claire |e investigator |4 oth | |
700 | 1 | |a Becourt, Arnaud |e investigator |4 oth | |
700 | 1 | |a Mounard, Julie |e investigator |4 oth | |
700 | 1 | |a Poulet, Claire |e investigator |4 oth | |
700 | 1 | |a Rames, Cinthia |e investigator |4 oth | |
700 | 1 | |a Talleux, Marie |e investigator |4 oth | |
700 | 1 | |a Chevalier, Marie-Chantal |e investigator |4 oth | |
700 | 1 | |a Darviot, Estelle |e investigator |4 oth | |
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700 | 1 | |a Person, Christine |e investigator |4 oth | |
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700 | 1 | |a Troussier, Françoise |e investigator |4 oth | |
700 | 1 | |a Urban, Thierry |e investigator |4 oth | |
700 | 1 | |a Dalphin, Marie-Laure |e investigator |4 oth | |
700 | 1 | |a Dalphin, Jean-Charles |e investigator |4 oth | |
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