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      Management of Adult Patients with Immune Thrombocytopenia (ITP): A Review on Current Guidance and Experience from Clinical Practice

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          Abstract

          Immune thrombocytopenia (ITP) is an autoimmune process resulting in increased destruction and inadequate production of platelets that can result in bleeding, fatigue, and reduced health-related quality of life. While treatment is not required for many patients with ITP, the occurrence of bleeding manifestations, severe thrombocytopenia, and requirement for invasive procedures are among the reasons necessitating initiation of therapy. Corticosteroids, intravenous immunoglobulin, and anti-RhD immune globulin are typical first-line and rescue treatments, but these agents typically do not result in a durable remission in adult patients. Most patients requiring treatment therefore require subsequent line therapies, such as thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, splenectomy, or a number of other immunosuppressive agents. In this focused review, we discuss management of adult ITP in the acute and chronic settings.

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          Most cited references128

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          International consensus report on the investigation and management of primary immune thrombocytopenia.

          Previously published guidelines for the diagnosis and management of primary immune thrombocytopenia (ITP) require updating largely due to the introduction of new classes of therapeutic agents, and a greater understanding of the disease pathophysiology. However, treatment-related decisions still remain principally dependent on clinical expertise or patient preference rather than high-quality clinical trial evidence. This consensus document aims to report on new data and provide consensus-based recommendations relating to diagnosis and treatment of ITP in adults, in children, and during pregnancy. The inclusion of summary tables within this document, supported by information tables in the online appendices, is intended to aid in clinical decision making.
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            Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group.

            Diagnosis and management of immune thrombocytopenic purpura (ITP) remain largely dependent on clinical expertise and observations more than on evidence derived from clinical trials of high scientific quality. One major obstacle to the implementation of such studies and in producing reliable meta-analyses of existing data is a lack of consensus on standardized critical definitions, outcome criteria, and terminology. Moreover, the demand for comparative clinical trials has dramatically increased since the introduction of new classes of therapeutic agents, such as thrombopoietin receptor agonists, and innovative treatment modalities, such as anti-CD 20 antibodies. To overcome the present heterogeneity, an International Working Group of recognized expert clinicians convened a 2-day structured meeting (the Vicenza Consensus Conference) to define standard terminology and definitions for primary ITP and its different phases and criteria for the grading of severity, and clinically meaningful outcomes and response. These consensus criteria and definitions could be used by investigational clinical trials or cohort studies. Adoption of these recommendations would serve to improve communication among investigators, to enhance comparability among clinical trials, to facilitate meta-analyses and development of therapeutic guidelines, and to provide a standardized framework for regulatory agencies.
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              American Society of Hematology 2019 guidelines for immune thrombocytopenia

              Despite an increase in the number of therapies available to treat patients with immune thrombocytopenia (ITP), there are minimal data from randomized trials to assist physicians with the management of patients. These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and other health care professionals in their decisions about the management of ITP. In 2015, ASH formed a multidisciplinary guideline panel that included 8 adult clinical experts, 5 pediatric clinical experts, 2 methodologists with expertise in ITP, and 2 patient representatives. The panel was balanced to minimize potential bias from conflicts of interest. The panel reviewed the ASH 2011 guideline recommendations and prioritized questions. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including evidence-to-decision frameworks, to appraise evidence (up to May 2017) and formulate recommendations. The panel agreed on 21 recommendations covering management of ITP in adults and children with newly diagnosed, persistent, and chronic disease refractory to first-line therapy who have non–life-threatening bleeding. Management approaches included: observation, corticosteroids, IV immunoglobulin, anti-D immunoglobulin, rituximab, splenectomy, and thrombopoietin receptor agonists. There was a lack of evidence to support strong recommendations for various management approaches. In general, strategies that avoided medication side effects were favored. A large focus was placed on shared decision-making, especially with regard to second-line therapy. Future research should apply standard corticosteroid-dosing regimens, report patient-reported outcomes, and include cost-analysis evaluations.
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                Author and article information

                Journal
                J Blood Med
                J Blood Med
                jbm
                jbm
                Journal of Blood Medicine
                Dove
                1179-2736
                26 July 2021
                2021
                : 12
                : 653-664
                Affiliations
                [1 ]Department of Medicine, Massachusetts General Hospital , Boston, MA, USA
                [2 ]Harvard Medical School , Boston, MA, USA
                [3 ]Division of Hematology, Massachusetts General Hospital , Boston, MA, USA
                Author notes
                Correspondence: Hanny Al-Samkari Division of Hematology, Massachusetts General Hospital , Suite 118, Room 112, Zero Emerson Place, Boston, MA, 02114, USATel +617-643-6214Fax +617-643-8840 Email hal-samkari@mgh.harvard.edu
                Author information
                http://orcid.org/0000-0001-6175-1383
                Article
                259101
                10.2147/JBM.S259101
                8323851
                34345191
                39628fe3-3b84-42ea-adcf-e064094771a7
                © 2021 Song and Al-Samkari.

                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
                : 18 March 2021
                : 12 July 2021
                Page count
                Figures: 0, Tables: 6, References: 128, Pages: 12
                Categories
                Review

                Hematology
                platelets,immune thrombocytopenia,itp,treatment,corticosteroids,ivig,splenectomy,thrombopoietin receptor agonist,rituximab,fostamatinib

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