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The pathophysiology of the disease is based on a severe functional deficiency of <jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 (activity &lt;10%), the specific von Willebrand factor (<jats:styled-content style=\"fixed-case\">VWF<\/jats:styled-content>)\u2010cleavage protease. This deficiency may be either acquired (associated anti\u2010<jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 autoantibodies) or congenital (resulting from biallelic mutations of <jats:italic><jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13<\/jats:italic> gene). <jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 deficiency is responsible for the accumulation of high molecular weight multimers of <jats:styled-content style=\"fixed-case\">VWF<\/jats:styled-content> and the formation of platelet thrombi in the microcirculation. Consequently, microangiopathic hemolytic anemia and consumption thrombocytopenia are associated with organ ischemia. The differential diagnosis with other <jats:styled-content style=\"fixed-case\">TMA<\/jats:styled-content>s, autoimmune cytopenias or hematological malignancies may be challenging. The exploration of <jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 (activity, antibodies, antigen, <jats:italic><jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13<\/jats:italic> gene) supports the diagnosis of <jats:styled-content style=\"fixed-case\">TTP<\/jats:styled-content>. The first\u2010line treatment of the acute phase of <jats:styled-content style=\"fixed-case\">TTP<\/jats:styled-content> is based on plasmatherapy. In congenital <jats:styled-content style=\"fixed-case\">TTP<\/jats:styled-content>, patients with a chronic disease benefit from a prophylactic plasmatherapy. In autoimmune <jats:styled-content style=\"fixed-case\">TTP<\/jats:styled-content>, steroids and B\u2010cells depleting therapies increasingly are used together with plasma exchange. Long\u2010term follow\u2010up including the monitoring of <jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 activity is mandatory. A severe decrease in <jats:styled-content style=\"fixed-case\">ADAMTS<\/jats:styled-content>13 activity (&lt;10%) may predict relapses and preemptive B\u2010cell depletion with rituximab can be used to prevent relapses.<\/jats:p>","DOI":"10.1111\/ejh.13107","type":"journal-article","created":{"date-parts":[[2018,6,11]],"date-time":"2018-06-11T14:56:08Z","timestamp":1528728968000},"page":"425-434","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":57,"title":["Pediatric thrombotic thrombocytopenic purpura"],"prefix":"10.1111","volume":"101","author":[{"given":"B\u00e9rang\u00e8re S.","family":"Joly","sequence":"first","affiliation":[{"name":"Service d'h\u00e9matologie biologique, groupe hospitalier Saint\u2010Louis\u2010Lariboisi\u00e8re Assistance Publique\u2010H\u00f4pitaux de Paris Universit\u00e9 Paris\u2010Diderot  Paris France"},{"name":"EA3518 Recherche clinique en h\u00e9matologie, immunologie et transplantation, 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