Two cases of anti-GBM disease treated with eculizumab

Anti-glomerular basement membrane (anti-GBM) is a rare autoimmune but life-threatening disease cause by pathogenic autoantibodies targeting a well characterized autoantigen (a-3 chain of type IV collagen) expressed in the basement membranes of the kidney and lung. Standard treatment normally includes the combination of plasma ex- change, oral cyclophosphamide and corticosteroids. Despite all therapeutic efforts, renal prognosis remains very poor in many cases. Recent studies demonstrated the pathogenic role of complement activation in renal damage of this disease. Eculizumab is a terminal complement inhibitor that binds to the human C5 complement protein, thus blocking the generation of proinflammatory C5a and C5b-9. This provides an immediate inhibition of the proinflammatory and cytotoxic sequelae of the complement system. Here, we report the successfully use of eculizumab in 2 patients with progressive anti-GBM disease.