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Thrombotic microangiopathy

Efficacy of eculizumab in paediatric aHUS

Nature Reviews Nephrology volume 12, page 198 (2016)Cite this article

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Eculizumab is currently the only approved treatment for atypical haemolytic uraemic syndrome (aHUS), but data in paediatric patients are limited. Recent findings from a prospective phase II study in 22 children with aHUS suggest that eculizumab is safe and efficacious in this setting. After 26 weeks of treatment, 14 patients had a complete thrombotic microangiopathy response, 18 patients showed normalization of haematological parameters and 16 patients had a ≥25% decrease in serum creatinine levels from baseline. No serious adverse events related to eculizumab therapy were reported.

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References

  1. Greenbaum, L. A. et al. Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome. Kidney Int. http://dx.doi.org/10.1016/j.kint.2015.11.026

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  1. Andrea Aguilar
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Aguilar, A. Efficacy of eculizumab in paediatric aHUS. Nat Rev Nephrol 12, 198 (2016). https://doi.org/10.1038/nrneph.2016.24

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