AI Article Synopsis
- The study focused on Australian patients with atypical haemolytic uraemic syndrome (aHUS) who were treated with eculizumab, a medication provided through a compassionate access program.
- A total of 10 patients participated, mostly younger adults, and various forms of aHUS were observed, including acute presentations and cases post-transplantation, with the majority achieving remission after treatment.
- Despite some complications reported during eculizumab therapy, the overall outcome showed that eculizumab is a viable treatment option for aHUS, particularly for those when other treatments were ineffective.
Article 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.
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| http://dx.doi.org/10.1111/imj.12864 | DOI Listing |
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