Prophylactic Eculizumab after Renal Transplantation in Atypical Hemolytic–Uremic Syndrome

Abstract

Atypical hemolytic-uremic syndrome is a rare microangiopathic hemolytic condition characterized by thrombocytopenia and acute renal failure.1 The prognosis for patients with atypical hemolytic-uremic syndrome with a factor H mutation is poor; 60% have end-stage renal disease or die within a year.2 The expected rate of graft failure due to recurrent atypical hemolytic-uremic syndrome among patients with a factor H or factor I mutation is 80% within 1 to 2 years.3 Here, we describe a child with atypical hemolytic-uremic syndrome and a known factor H mutation who had normal renal function 1 year after high-risk kidney transplantation with prophylactic dosing of the terminal complement inhibitor eculizumab. Owing to the mechanism of action of eculizumab, our patient was vaccinated against meningococcal infection before the initiation of eculizumab treatment.5 Unabated complement activation due to the production of mutant factor H may explain the high rate of recurrence of atypical hemolytic-uremic syndrome, and overall lack of success of renal transplantation, in patients with factor H mutation.