Authors: Esra Baskin Kaan Gulleroglu Asli Kantar Umut Bayrakci Ozan Ozkaya
Publish Date: 2014/11/11
Volume: 30, Issue: 5, Pages: 783-789
Abstract
Disorders of complement regulation are the most important etiology of atypical hemolytic uremic syndrome aHUS Recent studies demonstrate that eculizumab is beneficial in longterm aHUS treatment We present a series of children with aHUS resistant to/dependent on plasma exchange PE who were treated with eculizumabThis was a retrospective study in which data were retrieved from the medical files of children who had received PE as treatment for aHUS The data retrieved included age sex presenting symptoms presence of diarrhea/vomiting hospitalization duration laboratory data on admission and followup need for transfusion or dialysis response to PE response to eculizumab and outcomeOf the 15 children diagnosed with aHUS in 2011 and 2012 in our departments ten were resistant to or dependent on plasma therapy and treated with eculizumab these children were enrolled in the study Three patients had relapses and seven had a new diagnosis Nine children had oliguria or anuria and eight required dialysis Hypertension was observed in six patients Neurologic involvement developed in six patients with the symptoms including seizures loss of balance vision loss and severe confusion Five and five patients were resistant to and dependent on plasma therapy respectively Following the start of eculizumab treatment all patients achieved full recovery of renal function and hematologic parametersIn our ten pediatric patients with aHUS who did not respond to PE eculizumab was a lifesaving therapy and improved their quality of life Early eculizumab use was a rescue therapy for renal function Our results show that eculizumab is an effective treatment for aHUS However more studies are needed on the longterm efficacy and safety of eculizumab in children with aHUS and to determine the optimal duration of treatment
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