Atypical Hemolytic-Uremic Syndrome: From Pathogenesis to Therapy

Abstract

Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome are part of a spectrum of thrombotic microangiopathies. In the last years enormous progress has been made in understanding the pathogenesis of these entities. The fact that complement dysregulation is considered central in the development of atypical hemolytic uremic syndrome has led to the introduction of Eculizumab, a monoclonal anti-C5 inhibitor, in its treatment. These new advances have improved the outcomes in patients with atypical hemolytic uremic syndrome. Many unsolved issues remains, the most pressing ones being identifying the optimal treatment schedules and the underlying cause of the disease in patients without detectable complement mutations.