Tuesday, October 9, 2012
Eculizumab in Atypical Hemolytic Uremic Syndrome
Hemolytic Uremic Syndrome (HUS) has stubbornly resisted specific therapy despite growing understanding of its pathophysiology. Treatment has been expectant and supportive including dialysis when necessary for renal failure (more recently use of early dialysis) and management of the complications of renal failure (hypertension, hyperkalemia) in the non-dialysis patient. Atypical HUS is a rare form of this disease sparked by infection or by other yet to be recognized trigger(s) that activated the alternative complement pathway. This uncontrolled activation of the alternative complement pathway leads to endothelial damage that manifests itself most significantly in the renal vascular bed. Giordano et al. (doi: 10.1542/peds.2011-1685), working at Bari University in Italy, chose to treat an infant with atypical HUS and a defined genetic complement defect with Eclizamab, a monoclonal antibody against the C5 component of complement, in an attempt to quiet this runaway alternative pathway. The child, who had failed to respond to plasma infusions, responded shortly after the Eclizamab was provided. Perhaps a specific therapy for this problematic condition has reached the horizon.