Eculizumab treatment of atypical hemolytic uremic syndrome

Bernard S. Kaplan, Rebecca L. Ruebner, Lawrence Copelovitch

Research output: Contribution to journalArticlepeer-review


Introduction: This review focuses on the use of an orphan drug eculizumab (Soliris®; Alexion Pharmaceuticals Inc.), for the treatment of an extremely rare disease, atypical hemolytic uremic syndrome (aHUS). aHUS is associated with very high mortality and morbidity rates. Current therapies (plasma therapy, liver transplant) are imperfect and fraught with complications. The authors briefly describe the clinical features and diagnosis of aHUS. The pathogenesis is the result of mutations in genes encoding alternative complement regulators. This results in unregulated activity of the alternate complement pathway, endothelial injury, and thrombotic microangiopathy (TMA). Eculizumab is a humanized monoclonal antibody that inhibits the production of the terminal complement components C5a and the membrane attack complex (C5b-9) by binding to complement protein C5a. This blocks the proinflammatory and cytolytic effects of terminal complement activation. Areas covered: Eculizumab use is described in many case reports and in two prospective Phase II open-label clinical trials in adolescent and adult patients with aHUS. There is also a retrospective study in children, adolescents and adults with aHUS. There are few serious side-effects and no reports of tachphylaxis or drug resistance. Expert opinion: The results are very encouraging and more information is being obtained regarding the optimal timing of initiation, doses and maintenance protocols. Cost and availability may be important limitations to its widespread use. In summary, eculizumab therapy is now the standard of care in the prevention and treatment of episodes of aHUS. Prophylactic eculizumab should be used for renal transplant candidates with a genetically determined risk of post-transplantation aHUS recurrence.

Original languageEnglish (US)
Pages (from-to)167-176
Number of pages10
JournalExpert Opinion on Orphan Drugs
Issue number2
StatePublished - 2013
Externally publishedYes


  • Alternate pathway of complement
  • Atypical hemolytic uremic syndrome
  • Eculizumab
  • Hemolytic uremic syndrome
  • Soliris®
  • Thrombotic microangiopathy

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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