The risk of post-transplantation recurrence of aHUS depends on the genetic abnormality involved. Patients with complement factor H (CFH) mutations have an estimated recurrence rate of 74%, leading to graft loss in 93%. Consequently, isolated kidney transplantation has been contraindicated in these patients and combined liver-kidney transplantation and pre-emptive plasma exchange (PE) have been used as alternatives. However, both strategies have major limitations. Eculizumab, a humanized monoclonal antibody against terminal complement component 5 (C5), has recently been approved for the treatment of aHUS. Recent successful experiences of its prophylactic use in renal transplantation have been reported (one living nonrelated donor and eight deceased donor transplantations). We report an adult patient with aHUS-ESRD with a CFH mutation who received prophylactic eculizumab therapy prior to living-related kidney transplantation

A typical hemolytic uremic syndrome (aHUS) is a rare disease characterized by nonimmune hemolytic anemia, thrombocytopenia and renal impairment. In the last few decades, a series of studies has established that dysregulation of the alternative pathway of complement plays a fundamental role in the pathogenesis of this disease, leading to endothelial damage and systemic thrombotic microangiopathy (TMA). The prognosis of aHUS is poor, with progression to end-stage renal disease (ESRD) or death in half of patients during the first clinical manifestation.

Santiago Rodríguez de Córdoba is supported by the Spanish Ministry of Economy and Competetiveness (SAF2011-26583), the Comunidad de Madrid (S2010/BMD-2316) and the Fundación Renal Iñigo Alvarez de Toledo.

Peer reviewed

14 p-.1 tab.