Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study.

ABSTRACT:

Background

There are limited long-term outcome data in eculizumab-treated patients with atypical hemolytic uremic syndrome (aHUS). We report final results from the largest prospective, observational, multicenter study of patients with aHUS treated with eculizumab.

Methods

Patients with aHUS who participated in any of five parent eculizumab trials and received at least one eculizumab infusion were eligible for enrollment in a long-term follow-up study. Rates of thrombotic microangiopathy (TMA) manifestations off versus on eculizumab were evaluated. Additional endpoints included change from baseline estimated glomerular filtration rate (eGFR), long-term renal outcomes, and serious targeted treatment-emergent adverse events.

Results

Among 93 patients (0-80 years of age),

SUBMITTER: Menne J 

PROVIDER: S-EPMC6456946 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature