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American Journal of Kidney Diseases

Discontinuation of Eculizumab Maintenance Treatment for Atypical Hemolytic Uremic Syndrome: A Report of 10 Cases

Published:March 21, 2014DOI:https://doi.org/10.1053/j.ajkd.2014.01.434
      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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      Linked Article

      • Discontinuation of Eculizumab Maintenance Treatment for Atypical Hemolytic Uremic Syndrome
        American Journal of Kidney DiseasesVol. 65Issue 2
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          Dr Ardissino and colleagues1 report successful discontinuation of eculizumab treatment in 7 of 10 patients with atypical hemolytic uremic syndrome (aHUS). This important report provides evidence that continued lifelong treatment may not be necessary in patients with aHUS, thus reducing side effects and costs. To ascertain generalizability and exclude selection bias, we should note the clinical characteristics of 12 patients who continued eculizumab treatment. Data presented in the first table of Ardissino et al1 suggest that discontinuing eculizumab treatment may be impossible in patients with a CFH mutation; in all 3 of these patients, treatment discontinuation was followed by recurrent disease that was evident within 6 weeks.
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        • PDF
      • Discontinuation of Eculizumab Treatment in Atypical Hemolytic Uremic Syndrome: An Update
        American Journal of Kidney DiseasesVol. 66Issue 1
        • Preview
          We write to update our previously published report of discontinuing eculizumab therapy after successful treatment of atypical hemolytic uremic syndrome in 10 patients,1 with longer-term follow-up of the original cases and a report of 6 additional cases. When stable clinical remission had been obtained, patients were offered the choice of continuing or discontinuing eculizumab treatment with the rationale and procedure previously described.1 Patients had received eculizumab for a median of 4.3 (range, 0.5-14.4) months (Table 1).
        • Full-Text
        • PDF