Clinical Analysis Of Han Ethnic Children With Atypical Hemolytic Uremic Syndrome In Single Center

Background Hemolytic uremic syndrome(HUS)is a disease characterized by the triad of microangiopathic hemolytic anemia,thrombocytopenia and acute renal failure.According to the diarrheal prodrome,HUS was divided into typical HUS and atypical HUS(a HUS).The causes of a HUS are various,while at least50% of patients with a HUS have an underlying inherited and/or acquired complement abnormality,which leads to dysregulated activity of the complement alternative pathway at the endothelial cell surface.The main treatment for a HUS is plasma therapy,including plasma exchange and plasma infusion.Atypical HUS is associated with a poorer prognosis,and 25-50% of patients may progress to end-stage renal disease or death.Here we analyzed the etiology,treatment and prognosis of Han Ethnic children with a HUS in single center,and investigated the effect of the etiology as well as the treatment on the prognosis of a HUS,so as to improve the diagnosis and treatment for a HUS.Methods Clinical data were collected for retrospectively analysis from 24 Han Ethnic children with a HUS in single center,Department of Pediatrics,Fuzhou General Hospital,from November 1993 to November 2016.All 24 cases of children with a HUS received etiological analysis,including mutation analysis in complement genes in 11 cases of them.All patients were treated with comprehensive therapy including plasma therapy,dialysis therapy,glucocorticoids,immunosuppressants and symptomatic treatment.Short-term outcomes are classified as complete remission,partial remission,and ineffective treatment.Long-term outcomes are classified as complete renal recovery,moderate renal impairment,chronic renal failure and end-stage renal disease(ESRD).Data processing in two groups used two independent sample t-test or Wilcoxon Mann-Whitney test for non-categorical variables as well as the Fisher exact test for categorical variables.And a two-tailed P value < 0.05 was considered as statistically significant.Results Twenty-four children with aHUS included two cases with CFB-mutated a HUS,one case with CFHR5-mutated a HUS,one case with streptococcus pneumoniae(SP)associated HUS and the remaining 20 cases whose causes were unknown.Three cases of children with alternative complement pathway dysregulation-HUS(two cases with CFB-mutated a HUS,one case with CFHR5-mutated a HUS)received plasma therapy;the case with SP associated HUS received albumin replacement therapy as well as symptomatic and supportive treatment;the other 20 cases with a HUS were treated with comprehensive treatment,including plasma therapy which 16 cases received.Short-term outcomes were: 3 cases(12.5%)achieved complete remission,15cases(62.5%)achieved partial remission and 6 cases(25%)ended in ineffective therapy.19 children with a HUS were treated with plasma therapy and the remission rate was 73.7%(14/19).Long-term outcomes were : of 14 children with a HUS,9 cases(64.3%)achieved complete renal recovery,5 cases(35.7%)achieved moderate impairment of renal function and none progressed to chronic renal insufficiency or ESRD,after a follow-up period of 0.02 to 10.96(median,1.75)years.The recurrence rate of a HUS was 21.4%(3/14).A recurrence was observed in one of two cases with CFB-mutated a HUS after a follow-up period of 1.00 to 2.92 years.Conclusions Our results support that the necessity of etiological diagnosis in all children with a HUS should be made,and plasma therapy should be performed as early as possible,except for the children with SP associated HUS in which plasma therapy was forbidden,and the children with a HUS should be received long-term follow-up.