Mallett, A., Hughes, P., Szer, J., Tuckfield, A., Van Eps, C., Cambell, S.B., Hawley, C., Burke, J., Kausman, J., Hewitt, I., Parnham, A., Ford, S., and Isbel, N. (2015) Atypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: the experience of the Australian compassionate access cohort. Internal Medicine Journal, 45. pp. 1054-1065.

PDF (Published Version) - Published Version Restricted to Repository staff only

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 presentation, three for relapsing and refractory acute aHUS, two for de novo aHUS post-renal transplantation, and one each for aHUS recurrence post-transplantation and facilitation of transplantation with a history of aHUS. The median duration of eculizumab therapy has been 911.5 days (IQR 569 days) with a cumulative exposure of 9184 days. At baseline all patients had renal and extra-renal aHUS involvement, with up to three non-renal organs affected. All but one patient, who died from uncontrollable gastrointestinal aHUS manifestations, have continued. The nine continuing patients achieved remission of aHUS. Two of the four patients requiring renal replacement therapy (RRT) at eculizumab commencement subsequently ceased RRT. Clinical events occurring in this cohort while on eculizumab treatment included neutropenia (two), posterior reversible encephalopathy syndrome (one), cardiomyopathy (one), pulmonary embolus (one), antibody-mediated rejection resulting in renal graft failure (one), iron deficiency (one), gastrointestinal haemorrhage (one) and death (one).

Conclusion: Eculizumab has been an effective therapy for aHUS in this cohort, including when other therapies have failed.

Keywords: atypical haemolytic uraemic syndrome; eculizumab; thrombotic microangiopathy; transplantation.

Item ID:	67893
Item Type:	Article (Research - C1)
ISSN:	1445-5994
Copyright Information:	© 2015 Royal Australasian College of Physicians.
Date Deposited:	18 Dec 2024 00:57
FoR Codes:	32 BIOMEDICAL AND CLINICAL SCIENCES > 3202 Clinical sciences > 320214 Nephrology and urology @ 100%
SEO Codes:	20 HEALTH > 2001 Clinical health > 200105 Treatment of human diseases and conditions @ 100%
Downloads:	Total: 2 Last 12 Months: 2
	More Statistics