Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura (ITP) of adults and children: report from an International Working Group

Department of Hematology, San Bortolo Hospital, Vicenza, Italy.
Blood (Impact Factor: 10.45). 12/2008; 113(11):2386-93. DOI: 10.1182/blood-2008-07-162503
Source: PubMed


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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    • "Clinical definition, terminology in evaluating patient characteristics, treatment responses, and outcome in both adults and children with ITP show great variety from one study to another. Therefore, a new revision in children and adults has been made for standardization of terminology, definition, and outcome [4]. "
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    ABSTRACT: Objective: Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood, characterized by isolated thrombocytopenia. The International Working Group (IWG) on ITP recently published a consensus report about the standardization of terminology, definitions, and outcome criteria in ITP to overcome the difficulties in these areas. Materials and Methods: The records of patients were retrospectively collected from January 2000 to December 2009 to evaluate the data of children with ITP by using the new definitions of the IWG. Results: The data of 201 children were included in the study. The median follow-up period was 22 months (range: 12-131 months). The median age and platelet count at presentation were 69 months (range: 7-208 months) and 19x109/L (range: 1x109/L to 93x109/L), respectively. We found 2 risk factors for chronic course of ITP: female sex (OR=2.55, CI=1.31-4.95) and age being more than 10 years (OR=3.0, CI=1.5-5.98). Life-threatening bleeding occurred in 5% (n=9) of the patients. Splenectomy was required in 7 (3%) cases. When we excluded 2 splenectomized cases, complete remission at 1 year was achieved in 70% (n=139/199). The disease was resolved in 9 more children between 12 and 90 months. Conclusion: Female sex and age above 10 years old significantly influenced chronicity. Therefore, long-term follow-up is necessary in these children.
    Full-text · Article · Mar 2014 · Turkish Journal of Haematology
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    • "Primary autoimmune thrombocytopenia (AITP) is an acquired autoimmune disorder defined by isolated thrombocytopenia and exclusion of other causes [1, 2]. The triggering event for AITP remains unknown [3]. "
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    ABSTRACT: As the involvement of oxidative stress (OS) in autoimmune thrombocytopenia (AITP) has been reported, a fast and rapid test for the reliable measurement of OS and antioxidant capacities (AOCs) might be a useful tool in extending current diagnostic possibilities. The free oxygen radical test (FORT) and free oxygen radical defence (FORD) assay (Callegari, Italy) are easy to perform and reliable, with results available within 15 minutes. Thirty-seven AITP patients and 37 matched healthy individuals were included in this study. All participants responded to a standard questionnaire provided by these assays. Female patients with AITP were observed to demonstrate significantly higher OS in comparison to female controls (P = 0.0027) and male AITP patients (P = 0.0018). The AOCs were not reduced in patients with AITP (P = 0.7648). Correlation of OS with platelet count identified a weak positive correlation (P = 0.0327, Spearman R = 0.4672). The questionnaire revealed that ITP patients in comparison to healthy controls are more stressed, feel exhausted and fatigued, and eat a healthier diet. In conclusion, OS is predominant in female but not in male patients with AITP suggesting gender-specific differences in the pathomechanisms of AITP. Identification of patients with high levels of OS might be beneficial in the management of AITP.
    Full-text · Article · Jan 2014 · Oxidative Medicine and Cellular Longevity
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    • "Primary immune thrombocytopenia (ITP) is an acquired immune-mediated disorder characterized by isolated thrombocytopenia, which is defined as a peripheral blood platelet count of <100×109/l and the absence of any clear initiating and/or underlying cause (1). Antibodies that are autoreactive to platelet antigens, mainly the platelet glycoprotein IIb/IIIa complex, are considered responsible for the reduced platelet production and accelerated destruction of platelets by the reticuloendothelial system (2,3). "
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    ABSTRACT: The aim of this study was to investigate the frequency of circulating natural killer T (NKT) cells and regulatory T cells (Tregs), as well as serum cytokine profiles, in adult chronic primary immune thrombocytopenia (ITP). The frequency of circulating T cell receptor (TCR) Vα24(+)Vβ11(+)CD3(+) NKT cells and CD4(+)CD25(+)CD127(-/low) Tregs was measured using multi-color flow cytometry. The serum concentrations of 11 cytokines were determined with a cytometric bead assay. The frequency of circulating NKT cells in patients with ITP was 0.13±0.03%, whereas the frequency in healthy controls was 0.07±0.01% of CD3(+) (P>0.05). However, the frequency of NKT cells in patients with ITP with platelet counts ≤20×10(9)/l (0.22±0.05%) was significantly higher than that in patients with platelet counts >20×10(9)/l (0.05±0.01%; P<0.05) and that in healthy controls (0.07±0.01%; P<0.05). The frequency of peripheral Tregs was comparable between patients with ITP (3.97±0.44% of CD4(+)) and healthy controls (3.69±0.31%; P>0.05). No significant differences were observed in the serum concentrations of 11 cytokines between patients with ITP and healthy controls, despite the fact that the serum levels of interleukin (IL)-12p70, IL-8, IL-4, interferon (IFN)-γ and tumor necrosis factor (TNF)-α in patients with ITP were higher than those in the healthy controls. The platelet count was negatively correlated with the frequency of circulating NKT cells in chronic ITP. These results indicate that NKT cells may be involved in ITP with severe thrombocytopenia, and NKT and Tregs may be important in cytokine deregulation in chronic ITP.
    Full-text · Article · Jan 2014 · Experimental and therapeutic medicine
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