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Congenital thrombotic thrombocytopenic purpura (cTTP) is characterized by periodic episodes of tissue ischemia due to extensive microvascular thrombosis. Patients with cTTP lack ADAMTS13, a protease that cleaves the ultra-large multimers of von Willebrand factor (UL-VWF) that contribute to the formation of these thrombi. Current therapy consists of regular plasma infusions to replace the deficient or defective ADAMTS13.
To examine the pharmacokinetics, safety, and tolerability of a recombinant human (rh) ADAMTS13, investigators conducted an industry-sponsored, prospective, open-label, multicenter, dose-escalation study in 15 patients with severe cTTP (ADAMTS13 activity <6%). Intravenous rhADAMTS13 was administered in doses ranging from 5 to …