Diagnosis and treatment of severe thrombotic microangiopathy in children / 中华实用儿科临床杂志

ABSTRACT

Thrombotic microangiopathy(TMA) is a group of clinical and pathological syndromes, characterized mainly by hemolytic anemia, thrombocytopenia, and multiple organ dysfunctions caused by a variety of factors.Classic types of TMA include hemolytic uremic syndrome (HUS) and thrombotic thrombocytopenic purpura(TTP). HUS is a critically ill glomerular disease.Depending on the pathogenesis, HUS is currently divided into 2 categories, namely typical and atypical HUS.Atypical HUS is at a rapid onset and is a type of dangerous disease, which tends to recur easily, and has high mortality in its acute phase.TTP is a rare but fatal entity of TMA.The pathophysiology of the disease is based on a severe functional deficiency of a disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13(ADAMTS-13), the specific von Willebrand factor(VWF)-cleavage protease.This deficiency may be either acquired or congenital.The activity of plasma ADAMTS-13<10%is the only definitive method for diagnosing TTP.This article reports the recent advances on the diagnosis and treatment of severe TMA in children.