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

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
figure1

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.

References

  1. 1.

    Legendre C, et al: N Engl J Med. 2013, 368: 2169-81. 10.1056/NEJMoa1208981.

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    Keating GM: Drugs. 2013, 73: 2053-66. 10.1007/s40265-013-0147-7.

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Walle, J.V., Delmas, Y., Ardissino, G. et al. Early initiation of eculizumab treatment in patients with atypical haemolytic uraemic syndrome improves long-term outcomes: a pooled analysis of clinical trials. Crit Care 19, P330 (2015). https://doi.org/10.1186/cc14410

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Keywords

  • Glomerular Filtration Rate
  • Glomerular Filtration
  • Pool Analysis
  • Organ Damage
  • Early Initiation
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