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Editorial
. 2021 May 6;137(18):2419-2420.
doi: 10.1182/blood.2020010234.

Eculizumab and aHUS: to stop or not

Robert A Brodsky  1
Affiliations
Editorial

Eculizumab and aHUS: to stop or not

Robert A Brodsky. Blood. .

Abstract

In this issue of Blood, Fakhouri et al provide evidence in a prospective phase 4, multicentric, noncontrolled study that discontinuing eculizumab is safe in most patients with atypical hemolytic uremic syndrome (aHUS) once they achieve complete remission. Risk of relapse was <25% overall, but as high as 50% in patients with a rare variant in at least 1 complement gene.

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Conflict of interest statement

Conflict-of-interest disclosure: R.A.B. reports grants and other from Alexion, outside the submitted work.

Conflict-of-interest disclosure: R.A.B. reports grants and other from Alexion, outside the submitted work.

Figures

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Potential algorithm for discontinuing eculizumab in patients who achieve complete remission.
See this image and copyright information in PMC

Comment on

References

    1. Fakhouri F, Fila M, Hummel A, et al. Eculizumab discontinuation in children and adults with atypical hemolytic-uremic syndrome: a prospective multicentric study. Blood. 2021;137(18):2438-2449. - PubMed
    1. Fakhouri F, Zuber J, Frémeaux-Bacchi V, Loirat C. Haemolytic uraemic syndrome. Lancet. 2017;390(10095):681-696. - PubMed
    1. Gavriilaki E, Brodsky RA. Complementopathies and precision medicine. J Clin Invest. 2020;130(5):2152-2163. - PMC - PubMed
    1. Legendre CM, Licht C, Muus P, et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med. 2013;368(23):2169-2181. - PubMed
    1. Ariceta G, Dixon BP, Kim SH, et al. The long-acting C5 inhibitor, ravulizumab, is effective and safe in pediatric patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment [published online ahead of print 8 December 2020]. Kidney Int. doi:10.1016/j.kint.2020.10.046. - PubMed

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