Related Experiment Video
Updated: May 24, 2026
Detection of Antibodies That Neutralize the Cellular Uptake of Enzyme Replacement Therapies with a Cell-based Assay
Published on: September 10, 2018
Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet count
Eiske M Dorresteijn1, Nicole C A J van de Kar, Karlien Cransberg
1Pediatric Nephrology, Erasmus MC - Sophia Childrens Hospital, Dr Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands. e.dorresteijn@erasmusmc.nl
Background:
Atypical hemolytic uremic syndrome (aHUS) in childhood is a rare disease with frequent progression to end-stage renal disease and a high recurrence after kidney transplantation. Eculizumab, a humanized monoclonal antibody that binds to complement protein C5, may be beneficial in the treatment of aHUS.
Case-Diagnosis/Treatment:
A 6-year-old girl developed aHUS with only slightly elevated C3d (4.4%), no mutations in complement factors, and no antibodies against factor H. Plasma exchange treatment was successful initially, until aHUS recurred. After reinitiating plasma exchange, normalization of the platelet count and improvement of hemolysis occurred, but renal function worsened. Renal function then improved dramatically promptly after the switch to eculizumab.
Conclusions:
This case demonstrates that platelet count is not always a reliable marker for improvement of aHUS and that eculizumab can prevent dialysis in plasma-resistant aHUS patients.
