End organ damage reversed with complement inhibitor treatment

Abstract

The thrombotic microangiopathy is a group of diseases with the pathognomonic triad of thrombocytopenia, microangiopathic hemolysis and ischemic end organ damage (hemodialysis requiring acute kidney failure is common). One of its subtypes is atypical hemolytic uremic syndrome. As in this case report, in case of suspicion of thrombotic microangiopathy, the evaluation of ADAMTS-13 metalloprotease activity, the complex complement diagnostics, the microbiological testing and the exploration of the secondary etiological factors are important. The most common cause of atypical hemolytic uremic syndrome is the overamplification of the alternative complement pathway, which is most often caused by genetic mutation. Genetic diagnostics are important. The complement C5 inhibitor eculizumab and ravulizumab play an increasingly important role in the therapy of atypical hemolytic uremic syndrome. With treatment started in time the target organ damage is largely reversible.