Transfusion medicine reviews
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Immune thrombotic thrombocytopenic purpura (iTTP) is a chronically relapsing, humorally-mediated autoimmune disorder characterized by unpredictable episodes of microangiopathic hemolytic anemia and thrombocytopenia, commonly associated with neurologic dysfunction, kidney injury, and fever. Episodes are caused by immune destruction or inhibition of the von Willebrand Factor (vWF) cleaving protease ADAMTS13. Currently, the standard of care is therapeutic plasma exchange (TPE), and most add immunosuppression with corticosteroids - a standard that is unchanged for nearly 30 years. ⋯ Finally, progress has long been hampered in iTTP due to difficulty with accrual and disagreement about trial design. A good surrogate endpoint for relapse-free survival is also needed. Despite these challenges, a new era of precision medicine is likely soon emerging for treatment of iTTP, and with it comes the opportunity to further improve outcomes in this rare and deadly disease.