Eculizumab: A Review of Its Use in Atypical Haemolytic Uraemic Syndrome

Gillian M. Keating

The recombinant humanized monoclonal antibody eculizumab (Soliris(®)) is a complement inhibitor that is indicated for use in the treatment of atypical haemolytic uraemic syndrome (aHUS). This article reviews the clinical efficacy and tolerability of eculizumab in the treatment of patients with aHUS, as well as summarizing its pharmacological properties. Intravenous eculizumab inhibited complement-mediated thrombotic microangiopathy in patients aged ≥12 years with aHUS, according to the results of two noncomparative, multinational, 26-week, phase II trials. At 26 weeks, the platelet count was significantly increased in patients with progressing thrombotic microangiopathy despite plasma exchange/infusion, and thrombotic microangiopathic event-free status was achieved in 80 % of patients with a long disease duration and chronic kidney disease who received long-term plasma exchange/infusion. Renal function and health-related quality of life also improved with eculizumab therapy in both studies. Outcomes were maintained or further improved throughout 2 years of follow-up. Eculizumab was also effective in adult and paediatric patients with aHUS, according to the results of additional prospective or retrospective trials. Intravenous eculizumab was generally well tolerated in patients with aHUS. Eculizumab is associated with an increased susceptibility to meningococcal infection, so patients should be immunized with meningococcal vaccine. In conclusion, eculizumab is a valuable new agent for use in the treatment of aHUS.