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Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Middleton, Peter G
Autor Middleton, Peter G ORCID From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Mall, Marcus A
Autor Mall, Marcus A From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Dřevínek, Pavel
Autor Dřevínek, Pavel From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Lands, Larry C
Autor Lands, Larry C From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
McKone, Edward F
Autor McKone, Edward F From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Polineni, Deepika
Autor Polineni, Deepika From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Ramsey, Bonnie W
Autor Ramsey, Bonnie W From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Taylor-Cousar, Jennifer L
Autor Taylor-Cousar, Jennifer L From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Tullis, Elizabeth
Autor Tullis, Elizabeth From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Vermeulen, François
Autor Vermeulen, François From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Marigowda, Gautham
Autor Marigowda, Gautham From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
McKee, Charlotte M
Autor McKee, Charlotte M From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Moskowitz, Samuel M
Autor Moskowitz, Samuel M From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Nair, Nitin
Autor Nair, Nitin From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Savage, Jessica
Autor Savage, Jessica From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Simard, Christopher
Autor Simard, Christopher From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Tian, Simon
Autor Tian, Simon From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Waltz, David
Autor Waltz, David From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Xuan, Fengjuan
Autor Xuan, Fengjuan From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Rowe, Steven M
Autor Rowe, Steven M From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
Jain, Raksha
Autor Jain, Raksha From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of Pediatrics, 2nd Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic (P.D.); the Pediatric Respiratory Medicine and Pediatric Cystic Fibrosis Clinic, McGill University Health Centre, Montreal (L.C.L.); St. Vincent's University Hospital and University College Dublin School of Medicine, Dublin (E.F.M.); the Department of Internal Medicine, University of Kansas Medical Center, Kansas City (D.P.); the Department of Pediatrics, University of Washington School of Medicine, and Seattle Children's Research Institute, Seattle (B.W.R.); the Departments of Medicine and Pediatrics, National Jewish Health, Denver (J.L.T.-C.); the Division of Respirology, St. Michael's Hospital, University of Toronto, Toronto (E.T.); the Cystic Fibrosis Reference Center, Department of Pediatrics, Catholic University of Leuven, Leuven, Belgium (F.V.); Vertex Pharmaceuticals, Boston (G.M., C.M.M., S.M.M., N.N., J.S., C.S., S.T., D.W., F.X.); the Departments of Medicine, Pediatrics, and Cell, Developmental, and Integrative Biology, University of Alabama at Birmingham, Birmingham (S.M.R.); and the Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas (R.J.)
VX17-445-102 Study Group
Autor VX17-445-102 Study Group

The New England journal of medicine. 2019 Nov 07 ; 381 (19) : 1809-1819. [epub] 20191031

N Engl J Med
ISSN 1533-4406 | 0028-4793
Zdroj

Jazyk angličtina Země Spojené státy americké Médium print-electronic

Typ dokumentu klinické zkoušky, fáze III, časopisecké články, multicentrická studie, randomizované kontrolované studie, Research Support, N.I.H., Extramural, práce podpořená grantem

Perzistentní odkaz https://www.medvik.cz/link/pmid31697873

Grantová podpora
U54 TR001368 NCATS NIH HHS - United States
P30 DK089507 NIDDK NIH HHS - United States
R35 HL135816 NHLBI NIH HHS - United States
P20 GM130423 NIGMS NIH HHS - United States
UL1 TR002319 NCATS NIH HHS - United States
U01 TR002487 NCATS NIH HHS - United States
P30 DK072482 NIDDK NIH HHS - United States

Online Plný text

PubMed 31697873
PubMed Central PMC7282384
DOI 10.1056/nejmoa1908639
Knihovny.cz E-zdroje

BACKGROUND: Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation. In a phase 2 trial involving patients who were heterozygous for the Phe508del CFTR mutation and a minimal-function mutation (Phe508del-minimal function genotype), the next-generation CFTR corrector elexacaftor, in combination with tezacaftor and ivacaftor, improved Phe508del CFTR function and clinical outcomes. METHODS: We conducted a phase 3, randomized, double-blind, placebo-controlled trial to confirm the efficacy and safety of elexacaftor-tezacaftor-ivacaftor in patients 12 years of age or older with cystic fibrosis with Phe508del-minimal function genotypes. Patients were randomly assigned to receive elexacaftor-tezacaftor-ivacaftor or placebo for 24 weeks. The primary end point was absolute change from baseline in percentage of predicted forced expiratory volume in 1 second (FEV1) at week 4. RESULTS: A total of 403 patients underwent randomization and received at least one dose of active treatment or placebo. Elexacaftor-tezacaftor-ivacaftor, relative to placebo, resulted in a percentage of predicted FEV1 that was 13.8 points higher at 4 weeks and 14.3 points higher through 24 weeks, a rate of pulmonary exacerbations that was 63% lower, a respiratory domain score on the Cystic Fibrosis Questionnaire-Revised (range, 0 to 100, with higher scores indicating a higher patient-reported quality of life with regard to respiratory symptoms; minimum clinically important difference, 4 points) that was 20.2 points higher, and a sweat chloride concentration that was 41.8 mmol per liter lower (P<0.001 for all comparisons). Elexacaftor-tezacaftor-ivacaftor was generally safe and had an acceptable side-effect profile. Most patients had adverse events that were mild or moderate. Adverse events leading to discontinuation of the trial regimen occurred in 1% of the patients in the elexacaftor-tezacaftor-ivacaftor group. CONCLUSIONS: Elexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective. (Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.gov number, NCT03525444.).

From the Department of Respiratory and Sleep Medicine Westmead Hospital and CF Research Group Ludwig Engel Centre for Respiratory Research Westmead Institute for Medical Research University of Sydney Westmead NSW Australia

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