Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry.

ABSTRACT:

Introduction

Eculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an observational registry setting.

Methods

The Global aHUS Registry recruits patients with clinical diagnoses of aHUS. This analysis includes baseline characteristics and targeted safety events from adult and pediatric patients who were "ever treated" versus "never treated" with eculizumab in the first 5 years of the registry, through January 26, 2017.

Results

Overall, 1321 patients (adult, n = 842; pediatric, n = 479; ever treated, n = 865; never treated, n = 456) were enrolle

SUBMITTER: Rondeau E 

PROVIDER: S-EPMC6933459 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature