The long-acting C5 inhibitor, ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment
Kidney International Apr 08, 2020
Rondeau E, Scully M, Ariceta G, et al. - Researchers performed this global, phase 3, single arm study, to test ravulizumab (a long-acting C5 inhibitor engineered from eculizumab) in terms of its efficacy and safety in adults with atypical hemolytic uremic syndrome presenting with thrombotic microangiopathy. Participants were complement inhibitor-naïve adults (18 years and older) who met diagnostic criteria for atypical hemolytic uremic syndrome. The included patients were administered ravulizumab through a 26-week initial assessment span. Complete thrombotic microangiopathy response, described as normalization of platelet count and lactate dehydrogenase and 25% or more betterment in serum creatinine, was considered as the primary endpoint. Alterations in hematologic variables as well as renal function were regarded as secondary endpoints. In adults with atypical hemolytic uremic syndrome, a rapid improvement of hematologic and renal endpoints was evident, without unexpected adverse events, as a consequence of treatment with ravulizumab once every eight weeks.
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