Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings

摘要: Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy associated with defective regulation of the alternative complement pathway. The prognosis for patients aHUS poor, and plasma exchange represents first-line therapy. Eculizumab humanized monoclonal anti-C5 antibody that prevents activation terminal Here, we report case 9-year-old girl frequent relapsing due to heterozygous factor H mutation who was initially treated three times per week 150% volume. This treatment frequently caused allergic reactions school absences. Because any reduction in frequency immediately induced relapses aHUS, eculizumab, 600 mg every 2 weeks, started completely stopped. On this drug regimen patient showed no evidence disease activity during period more than 24 months. Renal function improved, proteinuria disappeared, number antihypertensive medications could be decreased, quality life increased substantially. inhibition pathway by eculizumab also confirmed renal biopsy, which absence months after initiation illustrates long-term favorable outcome treatment.