Clinical Effects of Eculizumab in PNH

Abstract

Eculizumab is a humanized monoclonal antibody which binds with high affinity to the fifth component of complement preventing its cleavage into C5a and C5b. Given that the clinical manifestations of paroxysmal nocturnal haemoglobinuria (PNH) are based on the activity of C5b-9, (the membrane attack complex) on unprotected PNH erythrocytes, eculizumab was tested as a therapeutic in this disease in four clinical trials beginning in 2002, including a placebo-controlled double-blind study, and was found to have efficacy in reducing markers of intravascular haemolysis, improving haemoglobin levels and reducing transfusion requirements as well as improving renal impairment, decreasing the rate and r..