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Atypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: the experience of the A ustralian compassionate access cohort
Author(s) -
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.,
Isbel N.
Publication year - 2015
Publication title -
internal medicine journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.596
H-Index - 70
eISSN - 1445-5994
pISSN - 1444-0903
DOI - 10.1111/imj.12864
Subject(s) - eculizumab , medicine , transplantation , cohort , retrospective cohort study , interquartile range , atypical hemolytic uremic syndrome , surgery , pediatrics , immunology , complement system , antibody
Background/Aim This study aimed to report the clinical characteristics and outcomes of A ustralian patients treated with eculizumab for atypical haemolytic uraemic syndrome (a HUS ). Methods A retrospective cohort study was undertaken of all patients in A ustralia treated with eculizumab provided in a compassionate access programme for a clinical diagnosis of a HUS 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 a HUS in A ustralia. Eight patients were female, and three had a family history of a HUS . Three received eculizumab for an initial acute a HUS presentation, three for relapsing and refractory acute a HUS , two for de novo a HUS post‐renal transplantation, and one each for a HUS recurrence post‐transplantation and facilitation of transplantation with a history of a HUS . 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 a HUS involvement, with up to three non‐renal organs affected. All but one patient, who died from uncontrollable gastrointestinal a HUS manifestations, have continued. The nine continuing patients achieved remission of a HUS . 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 a HUS in this cohort, including when other therapies have failed.