Optimal management of atypical hemolytic uremic disease: challenges and solutions.

ABSTRACT

Atypical hemolytic uremic syndrome (aHUS) is a chronic life threatening condition that arises from genetic abnormalities resulting in uncontrolled complement amplifying activity. The introduction of eculizumab, the humanized monoclonal antibody, has brought about a paradigm shift in the management of aHUS. However, there are many knowledge gaps, diagnostic issues, access and cost issues, and patient or physician challenges associated with the use of this agent. Limited data on the natural history of aHUS along with the underlying genetic mutations make it difficult to predict the relapses and thereby raising concerns about the appropriate duration and monitoring of treatment. In this review, we discuss the safety and efficacy of eculizumab in patients with aHUS and its associated challenges.