Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2 years of treatment with ivacaftor in a real-world setting

Journal of Cystic Fibrosis - Tập 17 - Trang 89-95 - 2018
Dominique Hubert1,2, Clémence Dehillotte3, Anne Munck4, Valérie David5, Jinmi Baek6, Laurent Mely7, Stéphane Dominique8, Sophie Ramel9, Isabelle Danner Boucher10, Sylvaine Lefeuvre11, Quitterie Reynaud12, Virginie Colomb-Jung3, Prissile Bakouboula6, Lydie Lemonnier3
1Pulmonary Department, Adult CF Centre, Cochin Hospital, AP-HP, Paris, France
2Université Paris Descartes, Sorbonne Paris Cité, Paris, France
3Vaincre La Mucoviscidose, Paris, France
4Pediatric CF Centre, Robert Debré Hospital, AP-HP, Paris, France
5Pediatric CF Centre, Hôpital Mère-Enfant, Nantes, France
6Clinical Research Unit, Cochin Hospital, AP-HP, Paris, France
7CF Centre, Renée Sabran Hospital, Giens, France
8Pulmonary Department, Adult CF Centre, Charles Nicolle Hospital, Rouen University Hospital, Rouen, France
9CF Centre, Centre héliomarin de Perharidy, Roscoff, France
10Pulmonary Department, Adult CF Centre, Laennec Hospital, Nantes, France
11Paediatric CF Centre, Hôpital Sud, Rennes, France
12Adult CF Centre Lyon Sud, Hospices Civils de Lyon, Lyon, France

Tài liệu tham khảo

Ramsey, 2011, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation, N Engl J Med, 365, 1663, 10.1056/NEJMoa1105185

Davies, 2013, Efficacy and safety of ivacaftor in patients aged 6 to 11years with cystic fibrosis with a G551D mutation, Am J Respir Crit Care Med, 187, 1219, 10.1164/rccm.201301-0153OC

McKone, 2014, Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST), Lancet Respir Med, 2, 902, 10.1016/S2213-2600(14)70218-8

Davies, 2016, Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study, Lancet Respir Med, 4, 107, 10.1016/S2213-2600(15)00545-7

Sawicki, 2015, Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data, Am J Respir Crit Care Med, 192, 836, 10.1164/rccm.201503-0578OC

Rowe, 2014, Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis, Am J Respir Crit Care Med, 190, 175, 10.1164/rccm.201404-0703OC

Barry, 2014, Effects of ivacaftor in patients with cystic fibrosis who carry the G551D mutation and have severe lung disease, Chest, 146, 152, 10.1378/chest.13-2397

Borowitz, 2016, Nutritional status improved in cystic fibrosis patients with the G551D mutation after treatment with ivacaftor, Dig Dis Sci, 61, 198, 10.1007/s10620-015-3834-2

Castro, 2002, Resting energy expenditure in young patients with cystic fibrosis receiving antibiotic therapy for acute respiratory exacerbations, Clin Nutr, 2, 141, 10.1054/clnu.2001.0522

Heltshe, 2015, Pseudomonas aeruginosa in cystic fibrosis patients with G551D-CFTR treated with ivacaftor, Clin Infect Dis, 60, 703, 10.1093/cid/ciu944

Hisert, 2017, Restoring CFTR function reduces airway bacteria and inflammation in people with cystic fibrosis and chronic lung infections, Am J Respir Crit Care Med, 195, 1617, 10.1164/rccm.201609-1954OC

Bellin, 2013, Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study, Pediatr Diabetes, 14, 417, 10.1111/pedi.12026

Hayes, 2014, Resolution of cystic fibrosis–related diabetes with ivacaftor therapy, Am J Respir Crit Care Med, 190, 590, 10.1164/rccm.201405-0882LE

Sermet-Gaudelus, 2016, Bone demineralization is improved by ivacaftor in patients with cystic fibrosis carrying the p.Gly551Asp mutation, J Cyst Fibros, 15, e67, 10.1016/j.jcf.2016.09.003