Immune thrombotic thrombocytopenic purpura: Spotlight on long-term outcomes and survivorship

Advances in diagnosis and treatment have dramatically improved survival of acute immune thrombotic thrombocytopenic purpura (iTTP) and iTTP has evolved from an acute fatal condition to a chronic relapsing disorder. In addition to the risk of iTTP relapse, iTTP survivors are at risk of multiple adverse health outcomes including higher than expected rates of all-cause mortality, increased rates of stroke and other cardiovascular disease, and higher rates of morbidities such as obesity, hypertension, and autoimmune disorders. iTTP survivors also report neurocognitive impairment, depression, and reduced quality of life. Women with iTTP are at risk for recurrent iTTP, preeclampsia, and other maternal and fetal complications in subsequent pregnancies. ADAMTS13 activity during clinical remission has emerged as an important targetable risk factor for iTTP relapse and other outcomes including stroke and all-cause mortality. This review summarizes current literature regarding the epidemiology and potential mechanisms for adverse long-term sequelae of iTTP, outlines current best practices in iTTP survivorship care, and highlights a research agenda to improve long-term iTTP outcomes.

Automated summary

Advances in diagnosis and treatment have significantly improved survival rates of acute immune thrombotic thrombocytopenic purpura (iTTP) and changed it from an acute fatal condition to a chronic relapsing disorder. iTTP survivors are at risk of various adverse outcomes, including higher rates of all-cause mortality, cardiovascular diseases, and comorbidities like obesity and autoimmune disorders. Women with iTTP face risks of recurrence, preeclampsia, and other complications during subsequent pregnancies. ADAMTS13 activity during remission is an important factor for predicting relapse and other outcomes. This review summarizes the epidemiology and mechanisms of adverse long-term sequelae of iTTP, current best practices in survivorship care, and research agenda for improving long-term outcomes.