Lumacaftor-ivacaftor-associated health stabilisation in adults with severe cystic fibrosis
- Susannah J. King1,2,3⇑,
- Dominic Keating2,4,
- Elyssa Williams2,
- Eldho Paul5,
- Brigitte M. Borg6,
- Felicity Finlayson2,
- Brenda M. Button2,4,7,
- John W. Wilson2,4 and
- Tom Kotsimbos2,4
- 1Nutrition Department, Alfred Hospital, Melbourne, Victoria, Australia
- 2Cystic Fibrosis Service, Department of Respiratory Medicine, Alfred Hospital, Melbourne, Victoria, Australia
- 3Department of Dietetics, Nutrition and Sport, LaTrobe University, Bundoora, Victoria, Australia
- 4Department of Medicine, Monash University, Melbourne, Victoria, Australia
- 5School of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia
- 6Physiology Service, Department of Respiratory Medicine, Alfred Hospital, Melbourne, Victoria, Australia
- 7Physiotherapy Department, Alfred Hospital, Melbourne, Victoria, Australia
- Dr Susannah King, Nutrition Department, Alfred Hospital, Commercial Road, Melbourne Victoria 3004, Australia. E-mail: s.king{at}alfred.org.au
Abstract
Introduction Lumacaftor-ivacaftor (LUM-IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with ppFEV1>40%. We assessed the clinical utility of LUM-IVA in all eligible adult CF patients with ppFEV1<40% treated for at least 1 year under a single centre managed access program.
Methods Following clinical optimisation eligible patients (n=40) with ppFEV1<40% were commenced on LUM-IVA and monitored for tolerance and clinical outcomes including health service utilisation, pulmonary function, weight and body composition. Twenty-four patients reached 1 year of treatment by the time of evaluation. Six discontinued due to adverse events (five for increased airways reactivity) and 3 underwent lung transplantation.
Results In comparison to the year prior to LUM-IVA commencement, significant reductions (median/year) were observed in the treatment year in the number of pulmonary exacerbations requiring hospitalisation (3 to 1.5, p=0.002); hospitalisation days (27 to 17, p=0.0002) and intravenous antibiotic days (45 to 27, p=0.0007). Mean change in ppFEV1 was −2.10(se 1.18)% per year in the year prior, with the decline reversed in the year following (+1.45(se 1.13)% per year, p=0.035) although there was significant heterogeneity in individual responses. Mean weight gain at 1 year was 2.5±4.1 kg; p=0.0007), comprising mainly fat mass (mean 2.2 kg). The proportion of patients with severe underweight (BMI<18.5 kg·m−2) decreased from 33% at baseline to 13% at 1 year (p=0.003).
Conclusion This real-world evaluation study demonstrated benefits over several clinical domains (infective exacerbations requiring hospitalisation, intravenous antibiotics, pulmonary function decline and nutritional parameters) in CF patients with severe lung disease.
Footnotes
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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 Inc. 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: Dr. Paul has nothing to disclose.
Conflict of interest: Dr. Borg has nothing to disclose.
Conflict of interest: Dr. Finlayson has nothing to disclose.
Conflict of interest: B.M. Button reports lecture and consultancy fees from Vertex Pharmaceutical Inc. outside the submitted work.
Conflict of interest: J.W. Wilson reports consultancy and lecture fees from Vertex Pharmaceuticals Inc. outside the submitted work.
Conflict of interest: T. Kotsimbos reports lecture fees from Vertex Pharmaceuticals outside the submitted work.
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- Received April 21, 2020.
- Accepted October 27, 2020.
- Copyright ©ERS 2020
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