Volume 19 Supplement 1

35th International Symposium on Intensive Care and Emergency Medicine

Open Access

Early initiation of eculizumab treatment in patients with atypical haemolytic uraemic syndrome improves long-term outcomes: a pooled analysis of clinical trials

  • J Vande Walle1,
  • Y Delmas2,
  • G Ardissino3,
  • J Wang4,
  • J Kincaid4 and
  • H Haller5
Critical Care201519(Suppl 1):P330

https://doi.org/10.1186/cc14410

Published: 16 March 2015

Introduction

Atypical haemolytic uraemic syndrome (aHUS) is a severe, life-threatening disease requiring rapid treatment to inhibit complement-mediated thrombotic microangiopathy (TMA) and avoid irreversible organ damage. Four prospective clinical trials have reported the safety and efficacy of eculizumab (Ecu) in the treatment of aHUS [1, 2]. We report data from a pooled analysis of these trials on renal function in patients starting Ecu within ≤7 days or >7 days after the current aHUS manifestation.

Methods

Data from four phase 2, open-label, single-arm trials including both paediatric and adult patients with aHUS were pooled. Patients with a documented date of onset of current TMA manifestation and a baseline estimated glomerular filtration rate (eGFR) of <90 ml/ minute/1.73 m2 were included. Changes from baseline in eGFR were analysed at study visits using a one-sample t test.

Results

Data from 97 patients were analysed: median (range) age at enrolment was 29 (0 to 80) years; 62% of patients were females; median (range) duration of current manifestation to start of Ecu treatment was 23 (1 to 1,447) days; median (range) baseline eGFR was 15.9 (5.6 to 76.1) ml/minute/1.73 m2. Ecu treatment was started in 21 patients in ≤7 days and 76 patients in >7 days after presentation with TMA. Median eGFR was 11 ml/minute/1.73 m2 for the patients started within 7 days and 16 ml/minute/1.73 m2 for those initiating >7 days. The mean change from baseline in eGFR for patients starting Ecu in ≤7 days and in >7 days after presentation with TMA were 57 and 23 ml/minute/1.73 m2 at 1 year, respectively (Figure 1).
Figure 1

Mean change in eGFR from baseline over 1 year (standard error).

Conclusion

This pooled analysis indicates that patients treated with Ecu within 7 days of a TMA manifestation had a greater improvement in eGFR over time than patients in whom treatment was delayed. These data show the importance of rapid diagnosis and treatment of aHUS for recovery of renal function.

Authors’ Affiliations

(1)
University Hospital Ghent
(2)
Centre Hospitalier Universitaire de Bordeaux
(3)
Ospedale Maggiore Policlinico
(4)
Alexion Pharmaceuticals
(5)
Medical School Hannover

References

  1. Legendre C, et al: N Engl J Med. 2013, 368: 2169-81. 10.1056/NEJMoa1208981.View ArticlePubMedGoogle Scholar
  2. Keating GM: Drugs. 2013, 73: 2053-66. 10.1007/s40265-013-0147-7.View ArticlePubMedGoogle Scholar

Copyright

© Walle et al.; licensee BioMed Central Ltd. 2015

This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

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