Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials

Autor: Ahmet Z Uluer, Gordon MacGregor, Pilar Azevedo, Veronica Indihar, Claire Keating, Marcus A Mall, Edward F McKone, Bonnie W Ramsey, Steven M Rowe, Ronald C Rubenstein, Jennifer L Taylor-Cousar, Elizabeth Tullis, Lael M Yonker, Chenghao Chu, Anna P Lam, Nitin Nair, Patrick R Sosnay, Simon Tian, Fredrick Van Goor, Lakshmi Viswanathan, David Waltz, Linda T Wang, Yingmei Xi, Joanne Billings, Alexander Horsley, Edward F. Nash, Marleen Bakker, Renske van der Meer, Petrus Merkus, Christof Majoor, Karen McCoy, Krishna Pancham, James Tolle, Bryon Quick, Ahmet Uluer, Emily DiMango, Adupa Rao, Santiago Reyes, Ross Klingsberg, Celeste Barreto, Victor Ortega, Donna Willey-Courand, Carsten Schwarz, Sivagurunathan Sutharsan, Rainald Fischer, Jane Davies, Jamie Duckers, Simon Doe, Harry Heijerman, George M. Solomon, Christian Merlo, Jennifer Griffonnet, Joseph Pilewski, Jordan Dunitz, Saba Sheikh, Ronald C. Rubenstein, Daniel B. Rosenbluth, Theodore Liou, Maria Indihar, Lael Yonker, Samya Nasr, Cynthia D. Brown, Gregory S. Sawicki, Jennifer Ruddy, Bryan Garcia, Andrew Braun, Alex H. Gifford, Nighat Mehdi, Maria Tupayachi Ortiz, Raksha Jain, Francisco J. Calimano, Jimmy Johannes, Cori L. Daines, Jason Fullmer, Joel Mermis, Christopher Barrios, Ngoc Ly, Brian P. Casserly, Stephan Eisenmann, Helge Hebestreit, Alexander Kiefer, Daniel Peckham, Martin Ledson, Eva Van Braeckel, Noel Gerard McElvaney, Edward McKone, Barry Plant, Lucy Burr, Daniel J. Smith, Peter Middleton, John Wilson

Publikováno v: The Lancet Respiratory Medicine, 11(6), 550-562. Elsevier Limited
VX18-121-101 & VX18-561-101 Study Groups 2023, ' Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis : randomised, double-blind, controlled, phase 2 trials ', The Lancet Respiratory Medicine, vol. 11, no. 6, pp. 550-562 . https://doi.org/10.1016/S2213-2600(22)00504-5
LANCET RESPIRATORY MEDICINE

Background: Elexacaftor–tezacaftor–ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modu

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::72b19fd4ef9823a3f92122d18b15dec4
https://research.vumc.nl/en/publications/0a8b171c-28b7-4da9-8e4a-5749733cacf9

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Autor: Gitte Berkers, Peter van Mourik, Annelotte M. Vonk, Evelien Kruisselbrink, Johanna F. Dekkers, Karin M. de Winter-de Groot, Hubertus G.M. Arets, Rozemarijn E.P. Marck-van der Wilt, Jasper S. Dijkema, Maaike M. Vanderschuren, Roderick H.J. Houwen, Harry G.M. Heijerman, Eduard A. van de Graaf, Sjoerd G. Elias, Christof J. Majoor, Gerard H. Koppelman, Jolt Roukema, Marleen Bakker, Hettie M. Janssens, Renske van der Meer, Robert G.J. Vries, Hans C. Clevers, Hugo R. de Jonge, Jeffrey M. Beekman, Cornelis K. van der Ent

Publikováno v: Cell Reports, Vol 26, Iss 7, Pp 1701-1708.e3 (2019)

Summary: In vitro drug tests using patient-derived stem cell cultures offer opportunities to individually select efficacious treatments. Here, we provide a study that demonstrates that in vitro drug responses in rectal organoids from individual patie

Externí odkaz: https://doaj.org/article/dc8d3a11cbb948418c73a2e8f5b11051

Type 1 interferon-inducible gene expression in QuantiFERON Gold TB-positive uveitis: A tool to stratify a high versus low risk of active tuberculosis?

Autor: Rina La Distia Nora, Ratna Sitompul, Marleen Bakker, Marjan A Versnel, Sigrid M A Swagemakers, Peter J van der Spek, Made Susiyanti, Lukman Edwar, Soedarman Sjamsoe, Gurmeet Singh, Rr Diah Handayani, Aniki Rothova, P Martin van Hagen, Willem A Dik

Publikováno v: PLoS ONE, Vol 13, Iss 10, p e0206073 (2018)

QuantiFERON-Gold TB (QFT)-positive patients with undetermined cause of uveitis are problematic in terms of whether to diagnose and treat them for tuberculosis (TB). Here, we investigated whether peripheral blood expression of type 1 interferon (IFN)-

Externí odkaz: https://doaj.org/article/8298dbc53c5046e4bf7d049e35dac3a0

Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study

Autor: Patrick A Flume, Reta Fischer Biner, Damian G Downey, Cynthia Brown, Manu Jain, Rainald Fischer, Kris De Boeck, Gregory S Sawicki, Philip Chang, Hildegarde Paz-Diaz, Jaime L Rubin, Yoojung Yang, Xingdi Hu, David J Pasta, Stefanie J Millar, Daniel Campbell, Xin Wang, Neil Ahluwalia, Caroline A Owen, Claire E Wainwright, Ronald L. Gibson, Steven M. Rowe, Noah Lechtzin, Richard C. Ahrens, Karen S. McCoy, Moira Aitken, Scott H. Donaldson, Kimberly Ann McBennett, Joseph M. Pilewski, Joanne Billings, Carlos Milla, Ronald Rubenstein, Daniel Brian Rosenbluth, Rachel Linnemann, Michael R. Powers, Christopher Fortner, Carla Anne Frederick, Theodore G. Liou, Philip Black, Janice Wang, John L. Colombo, Maria Berdella, Maria Veronica Indihar, Cynthia D. Brown, Michael Anstead, Lara Bilodeau, Leonard Sicilian, James Jerome Tolle, Kathryn Moffett, Samya Nasr, Jennifer Taylor-Cousar, Tara Lynn Barto, Nicholas Antos, John S. Rogers, Bryon Quick, Henry R. Thompson, Gregory Sawicki, Bruce Barnett, Robert L. Zanni, Thomas C. Smith, Karen D. Schultz, Claire Keating, Patrick Flume, Gregory J. Omlor, Alix Ashare, Karen Voter, Nighat Mehdi, Maria Gabriela Tupayachi Ortiz, Tonia E. Gardner, Steven R. Boas, Barbara Messore, Edith Zemanick, Raksha Jain, Michael McCarthy, Dana G. Kissner, Kapilkumar Patel, John McNamara, Julie Philley, Ariel Berlinski, Francisco J. Calimano, Terry Chin, Douglas Conrad, Cori Daines, Hengameh H. Raissy, Thomas G. Keens, Jorge E. Lascano, Bennie McWilliams, Brian Morrissey, Santiago Reyes, Subramanyam Chittivelu, Sabiha Hussain, Arvey Stone, James Wallace, Ross Klingsberg, Julie A. Biller, Stephanie Bui, Olaf Sommerburg, Elisabetta Bignamini, Mirella Collura, Alexander Moller, Donatello Salvatore, Chantal Belleguic, Lea Bentur, Ori Efrati, Eitan Kerem, Dario Prais, Esther Quintana Gallego, Peter Barry, Galit Livnat-Levanon, Jose Ramon Villa Asensi, David Stuart Armstrong, Oscar Asensio de la Cruz, Francis Gilchrist, Diana Elizabeth Tullis, Bradley Quon, Larry C. Lands, Nancy Morrison, Annick Lavoie, Barry Linnane, Okan Elidemir, Felix Ringshausen, Matthias Kappler, Helge Hebestreit, Jochen Mainz, Alexander Kiefer, Cordula Koerner-Rettberg, Doris Staab, Wolfgang Gleiber, Tacjana Pressler, Florian Stehling, Andreas Hector, Sivagurunathan Sutharsan, Lutz Naehrlich, Damian Downey, Jane Carolyn Davies, Robert Ian Ketchell, Mary Patricia Carroll, Simon Doe, Gordon MacGregor, Edward Fairbairn Nash, Nicholas Withers, Daniel Gavin Peckham, Martin James Ledson, Sonal Kansra, Timothy William Rayner Lee, Bertrand Delaisi, Gilles Rault, Jean Le Bihan, Dominique Hubert, Isabelle Fajac, Isabelle Sermet-Gaudelus, Marleen Bakker, Bert Arets, Christiane De Boeck, Raphael Chiron, Philippe Reix, Catherine Mainguy, Eva van Braeckel, Anne Malfroot, Isabelle Durieu, Nadine Desmazes Dufeu, Anne Prevotat, Renske van der Meer, Petrus Merkus, E.J.M. Weersink, Isabel Barrio Gomez-Aguero, Silvia Gartner, Amparo Sole Jover, Antonio Alvarez Fernandez, Desmond William Cox, Edward F. McKone, Barry James Plant, Hiranjan Selvadurai, Simon David Bowler, Claire Elizabeth Wainwright, Daniel Smith, Peter Gordon Middleton, John William Wilson, Sonia Volpi, Carla Colombo, Benedetta Fabrizzi, Vincenzina Lucidi, Federico Cresta, Salvatore Cucchiara, Ernst Eber, Helmut Ellemunter, Isidor Huttegger, Lena Hjelte, Christina Krantz, Marita Gilljam

Publikováno v: lancet. Respiratory medicine, 9(7), 733-746. Elsevier Limited
VX14-661-110 study group 2021, ' Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study ', The Lancet Respiratory Medicine . https://doi.org/10.1016/S2213-2600(20)30510-5

Summary Background Tezacaftor–ivacaftor is an approved cystic fibrosis transmembrane conductance regulator (CFTR) modulator shown to be efficacious and generally safe and well tolerated over 8–24 weeks in phase 3 clinical studies in participants

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bf49af3de3f90b3a65bf102dbba3756b
https://pure.amc.nl/en/publications/longterm-safety-and-efficacy-of-tezacaftorivacaftor-in-individuals-with-cystic-fibrosis-aged-12-years-or-older-who-are-homozygous-or-heterozygous-for-phe508del-cftr-extend-an-openlabel-extension-study(cc1b629c-44f6-4a77-aff2-ea80f4598819).html

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation

Autor: Harry G M Heijerman, Edward F McKone, Damian G Downey, Eva Van Braeckel, Steven M Rowe, Elizabeth Tullis, Marcus A Mall, John J Welter, Bonnie W Ramsey, Charlotte M McKee, Gautham Marigowda, Samuel M Moskowitz, David Waltz, Patrick R Sosnay, Christopher Simard, Neil Ahluwalia, Fengjuan Xuan, Yaohua Zhang, Jennifer L Taylor-Cousar, Karen S McCoy, Karen McCoy, Scott Donaldson, Seth Walker, James Chmiel, Ronald Rubenstein, Deborah K. Froh, Isabel Neuringer, Manu Jain, Kathryn Moffett, Jennifer L. Taylor-Cousar, Bruce Barnett, Gary Mueller, Patrick Flume, Floyd Livingston, Nighat Mehdi, Charlotte Teneback, John Welter, Raksha Jain, Dana Kissner, Kapilkumar Patel, Francisco J. Calimano, Jimmy Johannes, Cori Daines, Thomas Keens, Herschel Scher, Subramanyam Chittivelu, Sudhakar Reddivalam, Ross Carl Klingsberg, Larry G. Johnson, Stijn Verhulst, Patricia Macedo, Damien Downey, Gary Connett, Edward Nash, Nicholas Withers, Timothy Lee, Marleen Bakker, Harry Heijerman, Francois Vermeulen, Christiane Knoop, Elke De Wachter, Renske van der Meer, Petrus Merkus, Christof Majoor

Publikováno v: Lancet, 394(10212), 1940-1948. Elsevier Limited
VX17-445-103 Trial Group 2019, ' Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial ', Lancet, vol. 394, no. 10212, pp. 1940 . https://doi.org/10.1016/S0140-6736(19)32597-8
Lancet
The Lancet, 394(10212), 1940. Elsevier Limited

BACKGROUND: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CFTR corrector and potentiator in people

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::221e25362cf07bd88d1d3728962d9a50
http://www.scopus.com/inward/record.url?scp=85074786708&partnerID=8YFLogxK