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Eculizumab, a monoclonal antibody directed against the terminal complement component C5, is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria and has activity in other complement-mediated disorders, such as cold agglutinin disease and atypical hemolytic uremic syndrome (aHUS). Clinical trials and case reports have provided strong evidence that eculizumab is effective for aHUS (e.g., J Blood Med 2016; 7:39). But it is unclear when, or even if, the agent can be safely discontinued.
To address this issue, investigators examined outcomes of 15 aHUS patients for whom eculizumab therapy was discontinued. The patients had a variety of triggering conditions and several had mutations in complement components.
The mean dur…