Ravulizumab in Atypical Hemolytic Uremic Syndrome: An Analysis of 2-Year Efficacy and Safety Outcomes in 2 Phase 3 Trials.

ABSTRACT:

Rationale & objective

Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) caused by complement dysregulation. Ravulizumab is a C5i approved for the treatment of aHUS. This analysis assessed long-term outcomes of ravulizumab in adults and pediatric patients with aHUS.

Study design

This analysis reports 2-year data from 2 phase 3, single-arm studies.

Setting & participants

One study included C5i-naïve adults (NCT02949128), and the other included 2 cohorts of pediatric patients (C5i-naïve and those who switched to ravulizumab from eculizumab [pediatric switch patients]; NCT03131219).

Exposure

Patients received intravenous ravulizumab every 4-8 weeks, with the dose depending on body weight.

Outcomes

The primary endpoi

SUBMITTER: Dixon BP 

PROVIDER: S-EPMC11298908 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature