Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare autosomal recessive condition that causes deficiency of the von Willebrand factor (vWF)-cleaving metalloprotease, ADAMTS13. Recombinant ADAMTS13 (rADAMTS13) is the first FDA-approved product for management of cTTP. This article reviews the pharmacology, pharmacokinetics, efficacy, safety, dosing, administration, and implications for advanced practitioners of rADAMTS13.
This twice-monthly newsletter highlights recently published research where Dana-Farber faculty are listed as first or senior authors. The information is pulled from PubMed and this issue notes papers published from March 16 - 31.