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ivacaftor response - Efficacy

MedGen UID:
1002553
Concept ID:
CN322735
Sign or Symptom

Recent clinical studies

Etiology

McNally P, Singh A, McColley SA, Davies JC, Higgins M, Liu M, Lu J, Rodriguez-Romero V, Shih JL, Rosenfeld M; VX15-770-124 Study Group
J Cyst Fibros 2024 May;23(3):429-435. Epub 2024 Apr 4 doi: 10.1016/j.jcf.2024.03.012. PMID: 38580563
Dave K, Dobra R, Scott S, Saunders C, Matthews J, Simmonds NJ, Davies JC
Pediatr Pulmonol 2021 Feb;56 Suppl 1:S79-S89. doi: 10.1002/ppul.24968. PMID: 33434412
Schechter MS
Paediatr Respir Rev 2017 Sep;24:24-28. Epub 2017 Jun 12 doi: 10.1016/j.prrv.2017.06.002. PMID: 28712576
Konstan MW, Plant BJ, Elborn JS, Rodriguez S, Munck A, Ahrens R, Johnson C
Pediatr Pulmonol 2015 May;50(5):447-55. Epub 2015 Mar 9 doi: 10.1002/ppul.23173. PMID: 25755212
Quintana-Gallego E, Delgado-Pecellín I, Calero Acuña C
Arch Bronconeumol 2014 Apr;50(4):146-50. Epub 2013 Oct 2 doi: 10.1016/j.arbres.2013.07.013. PMID: 24095197

Diagnosis

McNally P, Singh A, McColley SA, Davies JC, Higgins M, Liu M, Lu J, Rodriguez-Romero V, Shih JL, Rosenfeld M; VX15-770-124 Study Group
J Cyst Fibros 2024 May;23(3):429-435. Epub 2024 Apr 4 doi: 10.1016/j.jcf.2024.03.012. PMID: 38580563
Dave K, Dobra R, Scott S, Saunders C, Matthews J, Simmonds NJ, Davies JC
Pediatr Pulmonol 2021 Feb;56 Suppl 1:S79-S89. doi: 10.1002/ppul.24968. PMID: 33434412
Donaldson SH, Pilewski JM, Griese M, Cooke J, Viswanathan L, Tullis E, Davies JC, Lekstrom-Himes JA, Wang LT; VX11-661-101 Study Group
Am J Respir Crit Care Med 2018 Jan 15;197(2):214-224. doi: 10.1164/rccm.201704-0717OC. PMID: 28930490Free PMC Article
Quintana-Gallego E, Delgado-Pecellín I, Calero Acuña C
Arch Bronconeumol 2014 Apr;50(4):146-50. Epub 2013 Oct 2 doi: 10.1016/j.arbres.2013.07.013. PMID: 24095197
De Boeck K, Kent L, Davies J, Derichs N, Amaral M, Rowe SM, Middleton P, de Jonge H, Bronsveld I, Wilschanski M, Melotti P, Danner-Boucher I, Boerner S, Fajac I, Southern K, de Nooijer RA, Bot A, de Rijke Y, de Wachter E, Leal T, Vermeulen F, Hug MJ, Rault G, Nguyen-Khoa T, Barreto C, Proesmans M, Sermet-Gaudelus I; European Cystic Fibrosis Society Clinical Trial Network Standardisation Committee
Eur Respir J 2013 Jan;41(1):203-16. Epub 2012 Aug 9 doi: 10.1183/09031936.00057512. PMID: 22878883

Therapy

McNally P, Singh A, McColley SA, Davies JC, Higgins M, Liu M, Lu J, Rodriguez-Romero V, Shih JL, Rosenfeld M; VX15-770-124 Study Group
J Cyst Fibros 2024 May;23(3):429-435. Epub 2024 Apr 4 doi: 10.1016/j.jcf.2024.03.012. PMID: 38580563
Sadras I, Kerem E, Livnat G, Sarouk I, Breuer O, Reiter J, Gileles-Hillel A, Inbar O, Cohen M, Gamliel A, Stanleigh N, Gunawardena T, Bartlett C, Gonska T, Moraes T, Eckford PDW, Bear CE, Ratjen F, Kerem B, Wilschanski M, Shteinberg M, Cohen-Cymberknoh M
J Cyst Fibros 2023 Nov;22(6):1062-1069. Epub 2023 Jun 16 doi: 10.1016/j.jcf.2023.06.001. PMID: 37331863
Guerra L, Favia M, Di Gioia S, Laselva O, Bisogno A, Casavola V, Colombo C, Conese M
Expert Opin Drug Discov 2020 Aug;15(8):873-891. Epub 2020 Apr 15 doi: 10.1080/17460441.2020.1750592. PMID: 32290721
Schechter MS
Paediatr Respir Rev 2017 Sep;24:24-28. Epub 2017 Jun 12 doi: 10.1016/j.prrv.2017.06.002. PMID: 28712576
Quintana-Gallego E, Delgado-Pecellín I, Calero Acuña C
Arch Bronconeumol 2014 Apr;50(4):146-50. Epub 2013 Oct 2 doi: 10.1016/j.arbres.2013.07.013. PMID: 24095197

Prognosis

McNally P, Singh A, McColley SA, Davies JC, Higgins M, Liu M, Lu J, Rodriguez-Romero V, Shih JL, Rosenfeld M; VX15-770-124 Study Group
J Cyst Fibros 2024 May;23(3):429-435. Epub 2024 Apr 4 doi: 10.1016/j.jcf.2024.03.012. PMID: 38580563
Durfey SL, Pipavath S, Li A, Vo AT, Ratjen A, Carter S, Morgan SJ, Radey MC, Grogan B, Salipante SJ, Welsh MJ, Stoltz DA, Goss CH, McKone EF, Singh PK
mBio 2021 Dec 21;12(6):e0314821. Epub 2021 Dec 14 doi: 10.1128/mbio.03148-21. PMID: 34903059Free PMC Article
Donaldson SH, Pilewski JM, Griese M, Cooke J, Viswanathan L, Tullis E, Davies JC, Lekstrom-Himes JA, Wang LT; VX11-661-101 Study Group
Am J Respir Crit Care Med 2018 Jan 15;197(2):214-224. doi: 10.1164/rccm.201704-0717OC. PMID: 28930490Free PMC Article
Guimbellot JS, Leach JM, Chaudhry IG, Quinney NL, Boyles SE, Chua M, Aban I, Jaspers I, Gentzsch M
JCI Insight 2017 Nov 16;2(22) doi: 10.1172/jci.insight.95734. PMID: 29202459Free PMC Article
Konstan MW, Plant BJ, Elborn JS, Rodriguez S, Munck A, Ahrens R, Johnson C
Pediatr Pulmonol 2015 May;50(5):447-55. Epub 2015 Mar 9 doi: 10.1002/ppul.23173. PMID: 25755212

Clinical prediction guides

McNally P, Singh A, McColley SA, Davies JC, Higgins M, Liu M, Lu J, Rodriguez-Romero V, Shih JL, Rosenfeld M; VX15-770-124 Study Group
J Cyst Fibros 2024 May;23(3):429-435. Epub 2024 Apr 4 doi: 10.1016/j.jcf.2024.03.012. PMID: 38580563
Bouazza N, Urien S, Foissac F, Choupeaux L, Lui G, Froelicher Bournaud L, Rouillon S, Zheng Y, Bardin E, Stremler N, Bessaci K, Bihouee T, Coirier-Duet E, Marguet C, Deneuville E, Laurans M, Reix P, Gerardin M, Mittaine M, Epaud R, Thumerelle C, Weiss L, Berthaud R, Semeraro M, Treluyer JM, Benaboud S, Sermet-Gaudelus I
Clin Pharmacokinet 2024 Mar;63(3):333-342. Epub 2024 Feb 4 doi: 10.1007/s40262-023-01342-3. PMID: 38310629
Xia X
Cell Biol Toxicol 2020 Dec;36(6):591-602. Epub 2020 Aug 11 doi: 10.1007/s10565-020-09552-2. PMID: 32780246Free PMC Article
Schechter MS
Paediatr Respir Rev 2017 Sep;24:24-28. Epub 2017 Jun 12 doi: 10.1016/j.prrv.2017.06.002. PMID: 28712576
Konstan MW, Plant BJ, Elborn JS, Rodriguez S, Munck A, Ahrens R, Johnson C
Pediatr Pulmonol 2015 May;50(5):447-55. Epub 2015 Mar 9 doi: 10.1002/ppul.23173. PMID: 25755212

Recent systematic reviews

Lupas D, Chou FY, Hakani MAA, Kuthiala I, Srikrishnaraj A, Li X, Potter N, Quon BS
J Cyst Fibros 2024 Sep;23(5):950-958. Epub 2024 Jul 23 doi: 10.1016/j.jcf.2024.07.012. PMID: 39048464
Wu HX, Zhu M, Xiong XF, Wei J, Zhuo KQ, Cheng DY
Adv Ther 2019 Feb;36(2):451-461. Epub 2018 Dec 15 doi: 10.1007/s12325-018-0860-4. PMID: 30554331

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