European journal of haematology
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Child-onset thrombotic thrombocytopenic purpura (TTP) is a rare entity of thrombotic microangiopathy (TMA). The pathophysiology of the disease is based on a severe functional deficiency of ADAMTS13 (activity <10%), the specific von Willebrand factor (VWF)-cleavage protease. This deficiency may be either acquired (associated anti-ADAMTS13 autoantibodies) or congenital (resulting from biallelic mutations of ADAMTS13 gene). ⋯ In autoimmune TTP, steroids and B-cells depleting therapies increasingly are used together with plasma exchange. Long-term follow-up including the monitoring of ADAMTS13 activity is mandatory. A severe decrease in ADAMTS13 activity (<10%) may predict relapses and preemptive B-cell depletion with rituximab can be used to prevent relapses.