ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

European Cystic Fibrosis Society   213  OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS CFTR MODULATORS, CFTR DIRECTED THERAPIES AND PRECISION MEDICINE CHAPTER 11 References [1] De Boeck K, Amaral MD. Progress in therapies for cystic fibrosis. Lancet Respir Med. 2016;4(8):662-74. [2] Cuyx S, De Boeck K. Treating the Underlying Cystic Fibrosis Trans- membrane Conductance Regulator Defect in Patients with Cystic Fibrosis. Semin Respir Crit Care Med. 2019;40(06):762-74. [3] Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, et al. A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation. N Engl J Med. 2011;365(18):1663-72. [4] Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, et al . Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219-25. [5] Davies J, Sheridan H, Bell N, Cunningham S, Davis SD, Elborn JS, et al . Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D- CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013;1(8):630-8. [6] De Boeck K, Munck A, Walker S, Faro A, Hiatt P, Gilmartin G, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13(6):674-80. [7] Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4(2):107-15. [8] Rosenfeld M, Wainwright CE, Higgins M, Wang LT, McKee C, Campbell D, et al . Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-53. [9] Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His- CFTR muta- tion: a double-blind, randomised controlled trial. Lancet Respir Med. 2015;3(7):524-33. [10] Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, et al. Tezacaftor–Ivacaftor in Resid- ual-Function Heterozygotes with Cystic Fibrosis. N Engl J Med. 2017;377(21):2024-35. [11] De Boeck K, Zolin A, Cuppens H, Olesen HV, Viviani L. The rela- tive frequency of CFTR mutation classes in European patients with cystic fibrosis. J Cyst Fibros. 2014;13(4):403-9.