About Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura is a life-threatening thrombotic microangiopathy caused by severe deficiency of the metalloprotease ADAMTS13, which normally cleaves ultra-large von Willebrand factor multimers; in acquired TTP this deficiency results from autoantibodies against ADAMTS13, while congenital TTP (Upshaw-Schulman syndrome) is caused by biallelic ADAMTS13 mutations. Unopposed ultra-large VWF multimers promote widespread platelet microthrombi in the microcirculation, causing thrombocytopenia, microangiopathic hemolytic anemia, and ischemic organ injury. Without prompt treatment with therapeutic plasma exchange, mortality approaches 90%.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Thrombotic Thrombocytopenic Purpura trials.
ADAMTS13 activity level below 10% with a detectable inhibitor or anti-ADAMTS13 IgG confirms acquired TTP and is required for most immunotherapy and novel agent trials; bring your acute episode laboratory results.
Caplacizumab use during acute episode and number of prior acute TTP episodes (relapses) are important eligibility variables for trials of rituximab combinations and novel immunosuppressants.
Congenital TTP (Upshaw-Schulman syndrome) patients with confirmed ADAMTS13 mutations may qualify for separate recombinant ADAMTS13 replacement trials distinct from acquired TTP studies.
Patient Resources
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