The effectiveness of CFTR modulators in people with CF and rare mutations: A real-world study

被引:1
|
作者
Hanger, Sofia [1 ]
Felton, Imogen [1 ]
Ukor, Emem-Fong [1 ]
Bowman, Elaine [1 ]
Caldwell, Clare [1 ]
Banya, Winston [2 ]
Madge, Susan [1 ]
Jones, Andrew L. [1 ]
Simmonds, Nicholas J. [1 ,3 ]
机构
[1] Royal Brompton Hosp, Adult Cyst Fibrosis Ctr, London, England
[2] Royal Brompton Hosp, Res Off, Med Stat, London, England
[3] Imperial Coll London, Natl Heart & Lung Inst, London, England
关键词
TEZACAFTOR-IVACAFTOR; CYSTIC-FIBROSIS;
D O I
10.1002/ppul.26713
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
引用
收藏
页码:221 / 224
页数:4
相关论文
共 50 条
  • [41] CFTR FUNCTION IN CF PATIENT-DERIVED AIRWAY CELLS WITH COMMON AND RARE MUTATIONS
    Illek, B.
    Zlock, L.
    Boecking, C.
    Green, B.
    Lefebvre, E.
    Bates, M.
    McGarry, M. E.
    Nielson, D. W.
    Finkbeiner, W. E.
    PEDIATRIC PULMONOLOGY, 2016, 51 : 198 - 199
  • [42] Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
    Amaral, Margarida
    Davies, Jane C.
    de Boeck, Kris
    Drevinek, Pavel
    Elborn, Stuart
    Kerem, Eitan
    Lee, Tim
    JOURNAL OF CYSTIC FIBROSIS, 2019, 18 (05) : 685 - 692
  • [43] Real-World Study Real-World Study of the Use of Azacitidine in Myelodysplasia in Australia
    Enjeti, Anoop
    Ashraf, Asma
    Caillet, Vincent
    Alam, Arif
    Sillar, Jonathan Robert
    Castaldi, Francesco
    Paine, Taliesha
    Keer, Harold N.
    BLOOD, 2022, 140 : 12341 - 12342
  • [44] The Real-World Effectiveness of Antifungals in People with Cystic Fibrosis and Aspergillus-Positive Cultures
    Faerber, Jennifer A.
    Kawut, Steven M.
    Hadjiliadis, Denis
    Hong, Gina
    ANNALS OF THE AMERICAN THORACIC SOCIETY, 2025, 22 (02)
  • [45] AN OVERVIEW OF REAL-WORLD DATA SOURCES FOR REAL-WORLD STUDIES IN ONCOLOGY AND RARE DISEASES IN CHINA
    Jia, Y.
    Zhan, S.
    Guo, J. J.
    Cao, X.
    Zhang, Y. J.
    VALUE IN HEALTH, 2023, 26 (06) : S392 - S392
  • [46] REAL-WORLD EFFECTIVENESS AND SAFETY OF OZANIMOD IN ULCERATIVE COLITIS: A MULTICENTER STUDY
    Lieto, Stephen
    Scalzo, Nicholas
    Huang, Katherine
    Khan, Abdul
    Xu, Anthony
    Pekow, Joel
    Rajauria, Palak
    Ayoub, Malek
    Johnson, Amanda M.
    Yarur, Andres
    Patel, Anish
    Bishu, Shrinivas
    Shukla, Richa
    Osterman, Mark T.
    Paul, Damemarie
    Sokol, Uyen
    Tharmarajah, Soba
    Lawlor, Garrett
    Ahmad, Harris
    Rubin, David T.
    Sands, Bruce E.
    Deepak, Parakkal
    Ungaro, Ryan C.
    GASTROENTEROLOGY, 2024, 166 (05) : S1455 - S1456
  • [47] Effectiveness of ustekinumab in patients with psoriatic arthritis in a real-world, multicenter study
    A. B. Azuaga
    B. Frade-Sosa
    A. Laiz
    P. Estrada
    A. Prior-Español
    L. Horcada
    L. Polino
    M. Moreno
    C. Moragues
    A. Urruticoechea-Arana
    A. Sellas
    J. L. Tandaipan
    V. Torrente-Segarra
    J. Garcia-Miguel
    I. Ros
    S. Ordoñez
    P. Moya
    D. Reina
    L. Mateo-Soria
    C. Fito
    E. Beltrán
    M. Pujol
    A. M. Cuervo
    J. D. Cañete
    Julio Ramírez
    Clinical Rheumatology, 2020, 39 : 2963 - 2971
  • [48] Study demonstrates disappointing real-world effectiveness of heart failure drug
    Askeland, Georgina
    EXPERT REVIEW OF CARDIOVASCULAR THERAPY, 2013, 11 (02) : 127 - 127
  • [49] Real-World Use, Effectiveness, and Safety of Intravenous Fosfomycin: The FORTRESS Study
    Bodmann, Klaus-Friedrich
    Hagel, Stefan
    Oliva, Alessandra
    Kluge, Stefan
    Mularoni, Alessandra
    Galfo, Valentina
    Falcone, Marco
    Pletz, Mathias W.
    Lindau, Simone
    Kaeding, Nadja
    Kielstein, Jan T.
    Zoller, Michael
    Tascini, Carlo
    Kintrup, Sebastian
    Schaedler, Dirk
    Spies, Claudia
    De Rosa, Francesco G.
    Radnoti, Szilvia
    Bandera, Alessandra
    Luzzati, Roberto
    Allen, Sam
    Sarmati, Loredana
    Cascio, Antonio
    Kapravelos, Nikolaos
    Subudhi, Chinari P. K.
    Dimopoulos, George
    Vossen, Matthias G.
    Bal, Abhijit M.
    Venditti, Mario
    Mastroianni, Claudio M.
    Borrmann, Thomas
    Mayer, Christian
    INFECTIOUS DISEASES AND THERAPY, 2025, : 765 - 791
  • [50] Real-World Effectiveness of Omalizumab in Chronic Urticaria: A Clinical Observational Study
    Zhang, Fan
    Liao, Jiashun
    Qian, Yuan
    Niu, Xinyu
    Wei, Jia
    Li, Man
    Guo, Duyi
    Zhu, Peiqiu
    INTERNATIONAL ARCHIVES OF ALLERGY AND IMMUNOLOGY, 2024, 185 (07) : 718 - 727