Validation study of the open ADAMTS13 conformation assay as a novel biomarker for correct diagnosis and predicting relapse in immune mediated thrombotic thrombocytopenic purpura

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare autoimmune disorder caused by neutralizing antibodies against the blood protease ADAMTS13. In the current guidelines, the diagnosis of iTTP is established by an ADAMTS13 activity below 10% and presence of anti-ADAMTS13 autoantibodies. While both two biomarkers have proven very useful in TTP diagnosis, there are still seronegative gaps in TTP diagnosis. We recently discovered that ADAMTS13 with an open conformation, open ADAMTS13, is a novel biomarker for iTTP. However, it has not been well elucidated if open ADAMTS13 analysis would (1) improve iTTP diagnosis correctly and (2) be an integrated tool for predicting relapse in iTTP patients. In this project, we are planning to validate the efficacy of open ADAMTS13 analysis as a novel biomarker for correct diagnosis and predicting relapse in iTTP. Sufficient iTTP plasma samples will be available via international collaborations and via my unique Japanese TTP cohort.

Trefwoorden:Thrombotic thrombocytopenic purpura, ADAMTS13, Open conformation, Novel biomarker, Validation study

Disciplines:Toegepaste immunologie, Hematologie