Pediatric Nephrology

, Volume 30, Issue 5, pp 775–781 | Cite as

Eculizumab hepatotoxicity in pediatric aHUS

  • Wesley Hayes
  • Sibylle Tschumi
  • Simon C. Ling
  • Janusz Feber
  • Michael Kirschfink
  • Christoph LichtEmail author
Original Article



Eculizumab is a humanized anti-C5 antibody approved for the treatment of atypical hemolytic uremic syndrome (aHUS). Its use is increasing in children following reports of its safety and efficacy.


We reviewed biochemical and clinical data related to possible drug-induced liver injury in 11 children treated with eculizumab for aHUS in a single center.


Elevated aminotransferases were observed in 7 children aged 6 to 11 years following eculizumab treatment for aHUS. Internationally accepted liver enzyme thresholds for drug-induced liver injury were exceeded in 5 cases. In all cases, liver injury was classified as mixed hepatocellular and cholestatic. Infectious and other causes were excluded in each case. One patient with no pre-existing liver disease developed tender hepatomegaly and liver enzyme derangement exceeding 20 times the upper limit of normal following initiation of eculizumab. Recurrent liver injury following re-challenge with eculizumab necessitated its discontinuation and transition to plasma therapy.


Hepatotoxicity in association with eculizumab is a potentially important yet previously unreported adverse event. We recommend monitoring liver enzymes in all patients receiving eculizumab. Further research is required to clarify the impact of this adverse event, to characterize the mechanism of potential hepatotoxicity, and to identify which patients are most at risk.


Atypical hemolytic uremic syndrome Thrombotic microangiopathy Complement Eculizumab Transaminitis Hepatotoxicity 



The authors wish to thank Camille Bedrosian, MD, CMO, Alexion Pharmaceuticals, for helpful discussion of this manuscript.


CL is advisory board member of Alexion Pharmaceuticals and Achillon Pharmaceuticals. He has received travel and speaker stipends as well as unrestricted research funds from Alexion Pharmaceuticals.

Conflict of interest

The other authors declare no conflict of interest.


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Copyright information

© IPNA 2014

Authors and Affiliations

  • Wesley Hayes
    • 1
  • Sibylle Tschumi
    • 1
  • Simon C. Ling
    • 2
    • 3
  • Janusz Feber
    • 4
  • Michael Kirschfink
    • 5
  • Christoph Licht
    • 1
    • 3
    Email author
  1. 1.Division of NephrologyThe Hospital for Sick ChildrenTorontoCanada
  2. 2.Division of Gastroenterology, Hepatology and NutritionThe Hospital for Sick ChildrenTorontoCanada
  3. 3.Department of PaediatricsUniversity of TorontoTorontoCanada
  4. 4.Division of NephrologyChildren’s Hospital of Eastern OntarioOttawaCanada
  5. 5.Division of ImmunologyUniversity of HeidelbergHeidelbergGermany

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