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      Assessment and Monitoring of Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP): Strategies to Improve Outcomes

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          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.

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          Most cited references49

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          Guidelines on the Use of Therapeutic Apheresis in Clinical Practice - Evidence-Based Approach from the Writing Committee of the American Society for Apheresis: The Eighth Special Issue.

          The American Society for Apheresis (ASFA) Journal of Clinical Apheresis (JCA) Special Issue Writing Committee is charged with reviewing, updating and categorizing indications for the evidence-based use of therapeutic apheresis (TA) in human disease. Since the 2007 JCA Special Issue (Fourth Edition), the committee has incorporated systematic review and evidence-based approaches in the grading and categorization of apheresis indications. This Eighth Edition of the JCA Special Issue continues to maintain this methodology and rigor in order to make recommendations on the use of apheresis in a wide variety of diseases/conditions. The JCA Eighth Edition, like its predecessor, continues to apply the category and grading system definitions in fact sheets. The general layout and concept of a fact sheet that was introduced in the Fourth Edition, has largely been maintained in this edition. Each fact sheet succinctly summarizes the evidence for the use of TA in a specific disease entity or medical condition. The Eighth Edition comprises 84 fact sheets for relevant diseases and medical conditions, with 157 graded and categorized indications and/or TA modalities. The Eighth Edition of the JCA Special Issue seeks to continue to serve as a key resource that guides the utilization of TA in the treatment of human disease.
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            Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies

            Essentials An international collaboration provides a consensus for clinical definitions. This concerns thrombotic microangiopathies and thrombotic thrombocytopenic purpura (TTP). The consensus defines diagnosis, disease monitoring and response to treatment. Requirements for ADAMTS-13 are given.
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              Survival and relapse in patients with thrombotic thrombocytopenic purpura.

              Survival of patients with thrombotic thrombocytopenic purpura (TTP) improved dramatically with plasma exchange treatment, revealing risk for relapse. The Oklahoma TTP Registry is a population-based inception cohort of all 376 consecutive patients with an initial episode of clinically diagnosed TTP (defined as microangiopathic hemolytic anemia and thrombocytopenia with or without signs and symptoms of ischemic organ dysfunctions) for whom plasma exchange was requested, 1989 to 2008. Survival was not different between the first and second 10-year periods for all patients (68% and 69%, P = .83) and for patients with idiopathic TTP (83% and 77%, P = .33). ADAMTS13 activity was measured in 261 (93%) of 282 patients since 1995. Survival was not different between patients with ADAMTS13 activity < 10% (47 of 60, 78%) and patients with 10% or more (136 of 201, 68%, P = .11). Among patients with ADAMTS13 activity < 10%, an inhibitor titer of 2 or more Bethesda units/mL was associated with lower survival (P = .05). Relapse rate was greater among survivors with ADAMTS13 activity < 10% (16 of 47, 34%; estimated risk for relapse at 7.5 years, 41%) than among survivors with ADAMTS13 activity of 10% or more (5 of 136, 4%; P < .001). In 41 (93%) of 44 survivors, ADAMTS13 deficiency during remission was not clearly related to subsequent relapse.
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                Author and article information

                Journal
                J Blood Med
                jbm
                jbm
                Journal of Blood Medicine
                Dove
                1179-2736
                28 September 2020
                2020
                : 11
                : 319-326
                Affiliations
                [1 ]Division of Hematology, Department of Internal Medicine, Cerrahpasa Faculty of Medicine, Istanbul University-Cerrahpasa , Istanbul, Turkey
                Author notes
                Correspondence: Ahmet Emre Eskazan Istanbul University-Cerrahpasa , Cerrahpasa Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Fatih, Istanbul, TurkeyTel +90 533 722 73 76Fax +90 212 589 79 34 Email emre.eskazan@istanbul.edu.tr
                Author information
                http://orcid.org/0000-0001-9568-0894
                Article
                205630
                10.2147/JBM.S205630
                7532071
                33061729
                193f92be-8c8c-4609-98d0-b08f55b24e07
                © 2020 Kucukyurt and Eskazan.

                This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License ( http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms ( https://www.dovepress.com/terms.php).

                History
                : 29 February 2020
                : 11 September 2020
                Page count
                Figures: 0, Tables: 1, References: 60, Pages: 8
                Categories
                Review

                Hematology
                immune-mediated thrombotic thrombocytopenic purpura,adamts13,caplacizumab,monitorization,outcome,ttp

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