ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

214    www.ecfs.eu OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS CFTR MODULATORS, CFTR DIRECTED THERAPIES AND PRECISION MEDICINE CHAPTER 11 [12] Bessonova L, Volkova N, Higgins M, Bengtsson L, Tian S, Simard C, et al. Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor. Thorax. 2018;73(8):731-40. [13] Sawicki GS, McKone EF, Pasta DJ, Millar SJ, Wagener JS, Johnson CA, et al. Sustained Benefit from Ivacaftor Demonstrated by Combining Clinical Trial and Cystic Fibrosis Patient Registry Data. Am J Respir Crit Care Med. 2015;192(7):836-42. [14] Barry PJ, Plant BJ, Nair A, Bicknell S, Simmonds NJ, Bell NJ, et al . Effects of Ivacaftor in Patients With Cystic Fibrosis Who Carry the G551D Muta- tion and Have Severe Lung Disease. Chest. 2014;146(1):152-8. [15] Hisert KB, Heltshe SL, Pope C, Jorth P, Wu X, Edwards RM, et al . Restoring Cystic Fibrosis Transmembrane Conductance Regulator Function Reduces Airway Bacteria and Inflammation in People with Cystic Fibrosis and Chronic Lung Infec- tions. Am J Respir Crit Care Med. 2017;195(12):1617-28. [16] Ronan NJ, Einarsson GG, Twomey M, Mooney D, Mullane D, NiChroinin M, et al. CORK Study in Cystic Fibrosis. Chest. 2018;153(2):395-403. [17] Harris JK, Wagner BD, Zemanick ET, Robertson CE, Stevens MJ, Heltshe SL, et al . Changes in Airway Microbiome and Inflammation with Ivacaftor Treatment in Patients with Cystic Fibrosis and the G551D Mutation. Ann Am Thorac Soc. 2020;17(2):212-20. [18] McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, et al . 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. 2014;2(11):902-10. [19] Dilokthornsakul P, Hansen RN, Campbell JD. Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation. Eur Respir J. 2016;47(6):1697-705. [20] Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, et al . A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014;2(7):527-38. [21] Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, et al . Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homo- zygous for Phe508del CFTR . N Engl J Med. 2015;373(3):220-31. [22] Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, et al . Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del- CFTR : a randomised, placebo-con- trolled phase 3 trial. Lancet Respir Med. 2017;5(7):557-67.