Discontinuation of dialysis with eculizumab therapy in a pediatric XXXX with @entity3630 .

@entity1 @entity644 @entity8 @entity4417 @entity786 @entity288 @entity3630 @entity4050 @entity454
BACKGROUND: @entity3630 ( @entity3630 ) is a rare @entity8 caused by an uncontrolled activation of the alternative complement pathway leading to @entity454 in 50  % of @entity1 . As such, @entity3630 has been classified within the spectrum of complement component 3 ( @entity4417 ) glomerulopathies due to its pathogenesis from alternative pathway dysregulation. Conventional immunosuppressive therapies have no proven effectiveness. Eculizumab, a terminal complement inhibitor, has been reported to mitigate disease in some cases. CASE-DIAGNOSIS/TREATMENT: We report on the efficacy of eculizumab in a pediatric @entity1 who failed to respond to @entity786 , @entity288 , and plasma exchange. Complement biomarker profiling was remarkable for low serum @entity4417 , low properdin, and elevated soluble C5b-9. Consistent with these findings, the alternative pathway functional assay was abnormally low, indicative of alternative pathway activity, although neither @entity4417 -nephritic factors nor @entity4050 autoantibodies were detected. Eculizumab therapy was associated with significant improvement in @entity644 and renal function allowing discontinuation of hemodialysis (HD). Repeat @entity4417 and soluble C5b-9 levels normalized, showing that terminal complement pathway activity was successfully blocked while the @entity1 was receiving eculizumab therapy. Repeat testing for alternative pathway activation allowed for a successful decrease in eculizumab dosing. CONCLUSIONS: The case reported here demonstrates the successful recovery of renal function in a pediatric @entity1 on HD following the use of eculizumab.
