Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis.

Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV Following clinical optimisation, eligible patients (n=40) with FEV In comparison with the year prior to LUM/IVA commencement, significant reductions (median per year) were observed in the treatment year in the number of pulmonary exacerbations requiring hospitalisation (from 3 to 1.5; p=0.0002), hospitalisation days (from 27 to 17; p=0.0002) and intravenous antibiotic (IVAB) usage days (from 45 to 27; p=0.0007). Mean±sd change in FEV This real-world evaluation study demonstrated benefits over several clinical domains (infective exacerbations requiring hospitalisation, IVABs, pulmonary function decline and nutritional parameters) in CF patients with severe lung disease.

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Conflict of interest: S.J. King reports a lecture fee from Vertex Pharmaceuticals in November 2017, outside the submitted work. Conflict of interest: D. Keating reports consultation fees from Vertex Pharmaceuticals, outside the submitted work. Conflict of interest: E. Williams reports a consultancy fee from Vertex Pharmaceuticals for a paid, 1-day workshop, outside the submitted work. Conflict of interest: E. Paul has nothing to disclose. Conflict of interest: B.M. Borg has nothing to disclose. Conflict of interest: F. Finlayson has nothing to disclose. Conflict of interest: B.M. Button reports lecture and consultancy fees from Vertex Pharmaceuticals, outside the submitted work. Conflict of interest: J.W. Wilson reports consultancy and lecture fees from Vertex Pharmaceuticals, outside the submitted work. Conflict of interest: T. Kotsimbos reports lecture fees from Vertex Pharmaceuticals, outside the submitted work.