Methods of testing effectiveness of a treatment for thromobotic thrombocytopenic purpura by measuring ADAMTS13-mediated in vivo cleavage of von Willebrand factor

摘要

The invention generally relates to methods of measuring cleaved von Willebrand factor (VWF) fragments. More specifically, the invention relates to methods of measuring the ability of a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) to cleave VWF in vivo. The invention also relates to methods of using various animal models which demonstrate ADAMTS13 activity similar to that of a human. The invention further relates to methods of measuring the cleavage products of rVWF in mammals, particularly in humans and in human plasma.

主权项

1. An in vivo method for testing effectiveness of a treatment for thrombotic thrombocytopenic purpura (TTP) in a subject comprising measuring a level of von Willebrand Factor (VWF) or recombinant VWF cleavage fragments in a blood sample from the subject before and after the treatment, wherein a decrease in the level of ultra-large multimer forms of VWF with a reduced triplet structure or an increase in the level of recombinant VWF fragment with satellite bands after the treatment indicates that the treatment is effective in increasing a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) activity or concentration in vivo in the subject thereby indicating effectiveness of the treatment for TTP.