Thrombotic thrombocytopenic purpura: New insights in disease pathogenesis and therapy

ADAMTS13 activity in thrombotic thrombocytopenic purpura–hemolytic uremic syndrome: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients

“…14 Therefore, it has been postulated that plasma exchange may not be effective in patients without ADAMTS13 deficiency and that measurements of ADAMTS13 activity may be able to guide treatment decisions. 14,[16][17][18][19] However, in our cohort, patients at all levels of ADAMTS13 activity apparently responded to plasma exchange treatment. In all ADAMTS13 activity categories, some patients responded with few Acute renal failure (%) 1 (6) 11 (34) .040…”

“…14,15 Because ADAMTS13 deficiency caused by an autoantibody provides a possible explanation for the effectiveness of plasma exchange (removal of the autoantibody by apheresis; supply of ADAMTS13 by plasma replacement), a role for ADAMTS13 activity measurements to guide treatment decisions has been suggested. 14,[16][17][18][19] However, the sensitivity of severe ADAMTS13 deficiency for identifying all patients who have an appropriate clinical diagnosis of TTP and who respond to plasma exchange has not been evaluated. In 7 reports, 45% to 100% of patients described as having TTP were reported to be severely deficient in ADAMTS13 activity.…”

Cloning, expression and functional characterization of the full‐length murine ADAMTS13

“…The disease associated with the formation of platelet and VWF‐rich thrombi is life threatening and has been termed thrombotic thrombocytopenic purpura (TTP). Clinical symptoms of TTP include microangiopathic hemolytic anemia, thrombocytopenia, fluctuating neurological impairment, renal dysfunction and fever [8–10].…”

Cloning, expression, and functional characterization of the von Willebrand factor–cleaving protease (ADAMTS13)

“…6 This disease, originally described by Moschcowitz,7 is characterized by microangiopathic hemolytic anemia, thrombocytopenia, fluctuating neurologic impairment, renal dysfunction, and fever. [8][9][10][11] There are 3 clinical subcategories of TTP: an acute idiopathic or sporadic form, an intermittent form with eventual relapse, and a chronic relapsing form, often but not always occurring in siblings. In the familial or hereditary form of TTP, VWF-cleaving protease (VWF-cp) is not produced in sufficient quantity, whereas in acute idiopathic TTP, an autoantibodyinhibiting VWF-cp activity is found transiently in most patients.…”

“…For unknown reasons, but probably not simply because of improved recognition, the incidence of the disease seems to have increased in recent years. 9,10 A treatment regimen of fresh-frozen plasma (FFP) or cryosupernatant plasma results in the disappearance of ULvWF multimers in patients with chronic relapsing TTP. 16 From normal human plasma, 5 a specific VWF-cp of high molecular weight has been partially purified.…”