Thrombotic thrombocytopenic purpura as a rare cause of anemia with thrombocytopenia in childhood: report of 2 cases

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare multisystem disorder characterized by single or recurrent episodes of thrombocytopenia, microangiopathic hemolytic anemia and widespread microvascular thrombosis, which causes significant morbidity and mortality unless promptly recognized and treated. The underlying pathogenesis is a defect in von Willebrand factor (vWF) cleaving protease, called "A Disintegrin and Metalloproteinase with Thrombospondin Type 1 Repeats 13 (ADAMTS-13)". There are 2 forms: congenital TTP (ADAMTS-13 gene mutations) and acquired TTP (autoantibodies and ADAMTS-13 deficiency). We presented two patients who initially presented with thrombotic microangiopathy and were later diagnosed with TTP upon demonstration of the deficiency in ADAMTS-13 activity.

Recognizing and managing hereditary and acquired thrombotic thrombocytopenic purpura in infants and children

“…Four (6%) of the 69 children died with their initial episode (Table 2). Two patients died from cerebral hemorrhage: a 12‐month‐old girl who died 20 hours after admission before treatment was begun 76 and a 12‐year‐old boy who was unresponsive to 5 weeks of plasma exchange and corticosteroids 77 . A 12‐year‐old girl who presented with kidney failure responded to initial treatment then died suddenly from acute kidney tubular necrosis 76 .…”

“…19,72,74,75 The outcomes of response, exacerbation, remission, and relapse were defined in 2003 13 corticosteroids. 77 A 12-year-old girl who presented with kidney failure responded to initial treatment then died suddenly from acute kidney tubular necrosis. 76 A 2-year-old girl was unresponsive to treatment and died from tracheal hemorrhage.…”