Eculizumab is efficacious and safe in pediatric patients with various forms of hemolytic uremic syndrome: a retrospective clinical experience of a tertiary center.

ABSTRACT:

Background

Eculizumab, a terminal complement inhibitor, prevents thrombotic microangiopathy (TMA) and multiorgan damage in hemolytic uremic syndrome (HUS). We evaluated its efficacy and safety in pediatric patients with TMA sub-types: atypical HUS (aHUS), Shiga toxin-producing Escherichia coli (STEC)-HUS, and transplant-associated TMA (TA-TMA).

Methods

This retrospective study included all pediatric patients treated with eculizumab for HUS at Schneider Children's Medical Center (2011-2020), including those with pre-existing end-stage kidney disease. Clinical and laboratory parameters were analyzed over 28 weeks. The primary endpoint was achievement of complete TMA response, defined by sustained normalization of hematologic parameters and renal function. Secondary endp

SUBMITTER: Lax N 

PROVIDER: S-EPMC12006164 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature