Abstract
BACKGROUND: Acquired or immune-mediated TTP (iTTP) is a life-threatening thrombotic microangiopathy, characterized by the presence of microangiopathic hemolytic anemia and severe thrombocytopenia, and a variable degree of ischemic organ damage, related to a severe deficiency of ADAMTS13, which is a serine metalloprotease necessary for cleavage of large vWF multimers. There has been a dramatic decrease in mortality rates with the recognition of the pathophysiology of iTTP over the years. Although therapeutic plasma exchange (TPE) together with corticosteroids are the backbone of the upfront treatment of patients with iTTP with successful outcomes, patients may remain refractory and/or relapse following an initial response to this treatment. METHODS: We performed a review regarding the pathogenesis, diagnosis, treatment strategies, monitoring, and prognosis of iTTP. RESULTS: There are several new treatment strategies, which can be used among these patients, helping in improving outcomes of iTTP. Rituximab has been shown to be a safe and effective adjunct to TPE, especially in patients with refractory and/or relapse as well as it is increasingly used preemptively to prevent exacerbation or recurrence. Recently, caplacizumab, a nanobody targeting vWF, was approved as an addition to the current regimen of TPE and immunomodulation for patients of iTTP. CONCLUSION: Specific predictors of relapse in patients in remission can be relevant for an optimal patient management. Different models including ADAMTS13 biomarkers can provide a new screening strategy to identify patients who may predict outcomes and the risk of relapse, benefit from preemptive therapy prior to relapse.