Discontinuation of eculizumab maintenance treatment for atypical hemolytic uremic syndrome: a report of 10 cases

Am J Kidney Dis. 2014 Oct;64(4):633-7. doi: 10.1053/j.ajkd.2014.01.434. Epub 2014 Mar 19.


Atypical hemolytic uremic syndrome (aHUS) is a life-threatening thrombotic microangiopathy, and as many as 70% of patients with aHUS have mutations in the genes encoding complement regulatory proteins. Eculizumab, a humanized recombinant monoclonal antibody targeting C5, has been used successfully in patients with aHUS since 2009. The standard maintenance treatment requires life-long eculizumab therapy, but the possibility of discontinuation has not yet been tested systematically. We report the safety of discontinuing eculizumab treatment in 10 patients who stopped treatment with the aim of minimizing the risk of adverse reactions, reducing the risk of meningitis, and improving quality of life while also reducing the considerable treatment costs. Disease activity was monitored closely at home by means of urine dipstick testing for hemoglobin. During the cumulative observation period of 95 months, 3 of the 10 patients experienced relapse within 6 weeks of discontinuation, but then immediately resumed treatment and completely recovered. Our experience supports the possibility of discontinuing eculizumab therapy with strict home monitoring for early signs of relapse in patients with aHUS who achieve stable remission.

Keywords: Atypical hemolytic uremic syndrome; discontinuation; eculizumab.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Antibodies, Monoclonal, Humanized* / administration & dosage
  • Antibodies, Monoclonal, Humanized* / adverse effects
  • Antibodies, Monoclonal, Humanized* / economics
  • Atypical Hemolytic Uremic Syndrome* / diagnosis
  • Atypical Hemolytic Uremic Syndrome* / drug therapy
  • Atypical Hemolytic Uremic Syndrome* / economics
  • Atypical Hemolytic Uremic Syndrome* / physiopathology
  • Atypical Hemolytic Uremic Syndrome* / psychology
  • Child
  • Child, Preschool
  • Complement Inactivating Agents / administration & dosage
  • Complement Inactivating Agents / adverse effects
  • Complement Inactivating Agents / economics
  • Cost of Illness*
  • Drug-Related Side Effects and Adverse Reactions / classification
  • Drug-Related Side Effects and Adverse Reactions / etiology
  • Drug-Related Side Effects and Adverse Reactions / prevention & control*
  • Female
  • Hemoglobinuria* / diagnosis
  • Hemoglobinuria* / etiology
  • Humans
  • Infant
  • Male
  • Middle Aged
  • Monitoring, Physiologic / methods
  • Quality of Life*
  • Recurrence
  • Remission Induction
  • Treatment Outcome
  • Urinalysis / methods
  • Withholding Treatment*


  • Antibodies, Monoclonal, Humanized
  • Complement Inactivating Agents
  • eculizumab