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

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108 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)633-637
Number of pages5
JournalAmerican Journal of Kidney Diseases
Volume64
Issue number4
DOIs
Publication statusPublished - Oct 1 2014

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Therapeutics
Thrombotic Microangiopathies
Antibodies, Monoclonal, Humanized
Recurrence
Meningitis
Health Care Costs
Atypical Hemolytic Uremic Syndrome
eculizumab
Complement System Proteins
Hemoglobins
Quality of Life
Observation
Urine
Safety
Mutation
Genes

Keywords

  • Atypical hemolytic uremic syndrome
  • discontinuation
  • eculizumab

ASJC Scopus subject areas

  • Nephrology
  • Medicine(all)

Cite this

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abstract = "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.",
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AU - Salardi, Stefania

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AU - Belingheri, Mirco

AU - Cugno, Massimo

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