Journal article

Atypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: the experience of the Australian compassionate access cohort

A Mallett, P Hughes, J Szer, A Tuckfield, C Van Eps, SB Cambell, C Hawley, J Burke, J Kausman, I Hewitt, A Parnham, S Ford, N Isbel

INTERNAL MEDICINE JOURNAL | WILEY | Published : 2015

Abstract

BACKGROUND/AIM: This study aimed to report the clinical characteristics and outcomes of Australian patients treated with eculizumab for atypical haemolytic uraemic syndrome (aHUS). METHODS: A retrospective cohort study was undertaken of all patients in Australia treated with eculizumab provided in a compassionate access programme for a clinical diagnosis of aHUS using prospectively collected clinical data. RESULTS: A total of 10 patients with a median age of 23.5 years (interquartile range (IQR) 24.83 years) received compassionate access eculizumab for aHUS in Australia. Eight patients were female, and three had a family history of aHUS. Three received eculizumab for an initial acute aHUS pr..

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Grants

Funding Acknowledgements

Conflict of interest: A. Mallett is a current recipient of a RACP Foundation Jacquot Research Entry Scholarship and past recipient of a RBWH Foundation Research Postgraduate Scholarship and Churchill Fellowship. He has received a travel grant from Amgen. N. Isbel is the Chair of the Australian aHUS registry (sponsored by Alexion), received honorarium for advisory board membership from Alexion Pharmaceuticals Australasia Pty Ltd and Novartis and has received travel grant funding from Alexion and Novartis. J. Szer has been an advisor, speaker and advisory board member for Alexion Pharmaceuticals Australasia Pty Ltd and has received honorarium in this context. P. Hughes has received honorarium for advisory board membership from Alexion Pharmaceuticals Australasia Pty Ltd and Novartis and has received travel grant funding Novartis. J. Kausman has received honorarium for advisory board membership from Alexion Pharmaceuticals Australasia Pty Ltd and has received travel grant funding. Alexion provided all original, unaltered and de-identified patient request and data collection documents to the authors upon their request. All case description and analysis was subsequently undertaken by the authors independently of and without condition of approval or review by Alexion. All data extraction for and authorship of this scientific article has been clinician-initiated and independent of Alexion.