The long-acting C5 inhibitor, ravulizumab, is efficacious and safe in pediatric patients with atypical hemolytic uremic syndrome previously treated with eculizumab

• DOI: 10.1007/s00467-020-04774-2
• Publication Date: 2020-10-13T13:04:20Z
• Authors: Kazuki Tanaka; Brigitte Adams; Alvaro Madrid Aris; Naoya Fujita; Masayo Ogawa; Stephan Ortiz; Marc Vallee; Larry A. Greenbaum
• Keywords: Eculizumab; Nephrology

Abstract Background Atypical hemolytic uremic syndrome (aHUS) is a rare, complement-mediated disease associated with poor outcomes if untreated. Ravulizumab, long-acting C5 inhibitor developed through minimal, targeted modifications to eculizumab was recently approved for the treatment of aHUS. Here, we report from pediatric patient cohort ravulizumab clinical trial (NCT03131219) who were switched chronic treatment. Methods Ten patients received loading dose on Day 1, followed by maintenance doses administered initially 15, and then, every 4–8 weeks thereafter, depending body weight. All completed initial evaluation period 26 entered extension period. Results No required dialysis at any point throughout study. The median estimated glomerular filtration rate values remained stable during trial: 99.8 mL/min/1.73m 2 baseline, 93.5 weeks, 104 52 weeks. At last available follow-up, all in same kidney stage as recorded baseline. Hematologic variables (platelets, lactate dehydrogenase, hemoglobin) also up follow-up. experienced adverse events; most common upper respiratory tract infection (40%) oropharyngeal pain (30%). There no meningococcal infections reported, deaths occurred, discontinued Conclusions Overall, aHUS previously treated resulted hematologic parameters, unexpected safety concerns when Trial registration identifiers: ID: ALXN1210-aHUS-312 Clinical trials.gov : NCT03131219 EudraCT number: 2016-002499-29