Haemolytic uraemic syndrome

Ravulizumab

Ravulizumab, a long-acting monoclonal antibody complement inhibitor, has been approved for the treatment of adults and children with atypical HUS (aHUS) who are complement inhibitor treatment-naive or have received eculizumab for at least 3 months and have evidence of response to eculizumab.[77]Barbour T, Scully M, Ariceta G, et al; 311 Study Group Members. Long-term efficacy and safety of the long-acting complement C5 inhibitor ravulizumab for the treatment of atypical hemolytic uremic syndrome in adults. Kidney Int Rep. 2021 Jun;6(6):1603-13. https://www.kireports.org/article/S2468-0249(21)01032-9/fulltext http://www.ncbi.nlm.nih.gov/pubmed/34169200?tool=bestpractice.com [78]Tanaka K, Adams B, Aris AM, et al. The long-acting C5 inhibitor, ravulizumab, is efficacious and safe in pediatric patients with atypical hemolytic uremic syndrome previously treated with eculizumab. Pediatr Nephrol. 2021 Apr;36(4):889-98. https://link.springer.com/article/10.1007/s00467-020-04774-2 http://www.ncbi.nlm.nih.gov/pubmed/33048203?tool=bestpractice.com Small, single-arm, phase 3 open-label studies to assess the efficacy of ravulizumab in children and adolescents with aHUS are ongoing.[79]ClinicalTrials.gov. Study of ravulizumab in children and adolescents with atypical hemolytic uremic syndrome (aHUS). NCT03131219. Apr 2022 [internet publication]. https://clinicaltrials.gov/ct2/show/NCT03131219 [80]ClinicalTrials.gov. Study of ALXN1210 in complement inhibitor treatment-naïve adult and adolescent participants with atypical hemolytic uremic syndrome (aHUS). NCT02949128. Mar 2022 [internet publication]. https://clinicaltrials.gov/ct2/show/NCT02949128 Ravulizumab is contraindicated in patients with unresolved Neisseria meningitidis infection, and in those who are not currently vaccinated against N meningitidis unless the risks of delaying treatment outweigh the risks of developing an infection. Patients who have not been previously vaccinated should be vaccinated against N meningitidis at least 2 weeks before starting treatment with ravulizumab.