Treatment of Hemolytic Uremic Syndrome With Eculizumab

Marcia L. Buck, Pharm.D., FCCP, FPPAG


Pediatr Pharm. 2012;18(2) 

In This Article

Mechanism of Action

Hemolytic uremic syndrome is one of the thrombotic microangiopathies (TMA). Thrombi formation in arterioles and capillaries produce the classic triad of HUS symptoms: microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney failure. In children, HUS is often associated with a prodromal infection. The most common form, STEC-HUS, is estimated to cause more than 90% of cases. Approximately 5-10% of HUS cases are considered atypical. Unlike STEC-HUS, these cases are not typically accompanied by diarrhea and may have a recurrent course. Patients with aHUS may present at a younger age and are more likely to experience severe hypertension and develop chronic kidney failure. Current research suggests that many patients with aHUS have genetic defects resulting in faulty regulation of the alternative complement pathway.[3,4]

Eculizumab is a monoclonal antibody that binds to the complement protein C5, inhibiting its cleavage to C5a and C5b. This prevents circulation of the pro-inflammatory C5a peptide and generation of the cytotoxic terminal complement complex C5b-9 (referred to as the membrane attack complex or MAC). In patients with HUS, eculizumab inhibits complement-mediated TMA.[3–6]


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