Sunday, March 6, 2016

Eculizumab is safe in children with aHUS

Current issue of Kidney International has an excellent original article on Eculizumab in aHUS in children, with a commentary on the paper as well.

Atypical hemolytic uremic syndrome (aHUS) is caused by alternative complement pathway dysregulation, leading to systemic thrombotic microangiopathy (TMA) and severe end-organ damage. Based on 2 prospective studies in mostly adults and retrospective data in children, eculizumab, a terminal complement inhibitor, is approved for aHUS treatment.

Greenbaum et al. report the first prospective trial of eculizumab in pediatric atypical hemolytic uremic syndrome. As in adult trials, eculizumab appears effective and no serious safety signals were reported.


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In summary, Eculizumab appears effective in treating both adult and pediatric aHUS. It remains to define the optimal treatment regimen to minimize the infectious complications of terminal pathway complement blockade.

Soliris is currently not available in India, and is not marketed.
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