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The long-acting C5 inhibitor, Ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment

Lookup NU author(s): Professor David KavanaghORCiD



This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).


Ravulizumab is a long-acting C5 inhibitor engineered from eculizumab with increased elimination half-life, allowing an extended dosing interval from 2 to 8 weeks. Here we evaluate the efficacy and safety of ravulizumab in adults with atypical hemolytic uremic syndrome (aHUS) presenting with thrombotic microangiopathy (TMA). In this global, phase 3, single arm study in complement inhibitor-naïve adults (≥18 years) who fulfilled diagnostic criteria for aHUS, enrolled patients received ravulizumab through a 26-week initial evaluation period. The primary endpoint was complete TMA response defined as normalization of platelet count and lactate dehydrogenase (LDH) and ≥25% improvement in serum creatinine (SCr). Secondary endpoints included changes in hematologic variables and renal function. Safety was also evaluated. Ravulizumab treatment resulted in an immediate, complete, and sustained C5 inhibition in all patients. Complete TMA response was achieved in 30 of 56 (53.6%) patients. Normalization of platelet count, LDH and ≥25% improvement in SCr was achieved in 83.9%, 76.8% and 58.9% of patients, respectively. Median improvement in platelet count at Day 8 was 70.5 × 109/L. Improvement in estimated glomerular filtration rate category of ≥1 stage was achieved in 68.1% of patients by Day 183. No unexpected adverse events were reported across the safety analysis set (n=58). Four deaths occurred (three within 1 month of study initiation, including one in a patient excluded based on eligibility criteria after the first dose) with none considered treatment-related by the study investigator. Treatment with ravulizumab once every 8 weeks resulted in rapidly improved hematologic and renal endpoints with no unexpected adverse events in adults with aHUS.

Publication metadata

Author(s): Rondeau E, Scully M, Ariceta G, Barbour T, Cataland S, Heyne N, Miyakawa Y, Ortiz S, Swenson E, Vallee M, Yoon S, Kavanagh D, Haller H

Publication type: Article

Publication status: Published

Journal: Kidney International

Year: 2020

Volume: 97

Issue: 6

Pages: 1287-1296

Print publication date: 01/06/2020

Online publication date: 06/03/2020

Acceptance date: 24/01/2020

Date deposited: 04/07/2020

ISSN (print): 0085-2538

ISSN (electronic): 1523-1755

Publisher: Elsevier


DOI: 10.1016/j.kint.2020.01.035


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