Article

Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group

Department of Hematology, San Bortolo Hospital, Vicenza, Italy.
Blood (Impact Factor: 10.43). 12/2008; 113(11):2386-93. DOI: 10.1182/blood-2008-07-162503
Source: PubMed

ABSTRACT 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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    • "Patients aged 1–18 years, who received romiplostim for non-responsive or refractory chronic ITP or ES, were enrolled. Non-responsive chronic ITP and refractory disease were defined according to the International Working Group crtieria (Rodeghiero et al, 2009). Exclusion criteria were secondary ITP, infection-related thrombocytopenia, inherited thrombocytopenia, systemic lupus erythematous or known genetic primary immune deficiency disorders. "
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    ABSTRACT: A minority of children with chronic immune thrombocytopenia (ITP) require therapeutic intervention to prevent haemorrhagic risk. This retrospective national study evaluated romiplostim in childhood non-responsive or refractory chronic ITP. Between 2009 and 2012, 10 patients whose Buchanan score was 3–4 were treated with romiplostim. The median duration of thrombocytopenia was 1·9 years (0·8–15). The median duration of romiplostim treatment was 9 months (3–36). A response was observed in 5/10 patients (one complete, four partial). No serious adverse effect was noticed. The long-term benefit/risk balance of this innovative treatment is currently recorded by Centre de Référence National des Cytopénies Auto-immunes de l'Enfant.
    British Journal of Haematology 01/2014; 164(2). DOI:10.1111/bjh.12609 · 4.96 Impact Factor
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    • "Primary ITP is an autoimmune disorder characterized by isolated thrombocytopenia (peripheral blood platelet count < 100 × 10 9 /L) in the absence of other causes or disorders. Chronic ITP was defined as thrombocytopenia lasting for more than 6 months [8]. Careful physical and laboratory evaluation including antinuclear antibody (ANA), direct antiglobulin test, double-stranded DNA, serum complement (C3 and C4), and antineutrophil cytoplasmic antibodies (ANCA) were performed at the time of diagnosis. "
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    ABSTRACT: Background and objective: To determine the prevalence and the clinical significance of thyroid autoantibodies and their influence on treatment response in children with idiopathic thrombocytopenic purpura (ITP). Patient and Method: We retrospectively analyzed the antithyroglobulin (anti-TG) and antithyroid peroxidase (anti-TPO) antibodies from the records of 151 ITP patients who were admitted to the Pediatric Hematology Department of Gaziantep University between 2009 and 2012. Results: Anti-TPO and/or anti-TG was found positive in 38 (36.8%) of 103 patients whose thyroid autoantibody levels were measured. The comparison of positivity ratios of autoantibodies between acute and chronic ITP patients showed no significant difference. However, the separate comparison of each group of ITP patients with control group showed significantly high positivity ratios of autoantibodies in ITP patients. The initial mean platelet count of anti-TPO positive patients at diagnosis was significantly less than that of the negative patients (P = .008). One month after treatment, platelet count of anti-TPO positive patients was significantly less than that of the negative patients (P = .01). Moreover, the mean platelet counts of anti-TPO positive patients were significantly less than those of the negative patients after intravenous immunoglobulin treatment (P < .001). Conclusion: We demonstrated that the thyroid-autoimmune-diseases-related autoantibodies are frequently found in childhood ITP. Although no recommendation is found in international guidelines regarding screening for thyroid autoantibodies in patients with ITP, in view of the high incidence of antithyroid antibodies and their potential negative effect on treatment response, screening these patients for such antibodies would be recommended.
    Pediatric Hematology and Oncology 01/2013; DOI:10.3109/08880018.2012.756564 · 0.96 Impact Factor
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    • "Tabela II – Oporność na leczenie [3] [6] Table II – Therapy resistance [3] [6] Konsensu IWG 2009 r. Modyfikacja Grace i wsp. "
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    ABSTRACT: Background Primary immune thrombocytopenia (ITP) is one of the most common hematologic disorders in pediatric population. In 2009 the new unified terminology regarding: definition, clinical classification of the disease and response to treatment was proposed. The main study objective was the comparative analysis of clinical aspects of primary ITP in children regarding the contemporary definitions and historical criteria. Methods Data were collected through medical chart review of subjects identified from hospitalization records (Pediatrics, Hematology and Oncology Department) from the period of 2002–2011. Results Data of 209 subjects were analyzed. According to recent definitions 206/209 patients (98.6%) could be defined. Using the historical criteria 86.12% were classified as acute and 13.88% as chronic ITP. Newly diagnosed primary immune thrombocytopenia was confirmed in 166/206 cases, persistent ITP in 20/206, and chronic ITP in 20/206 of subjects. Depending on applied criteria we noticed significant differences in acute ITP patient number. Regardless of adjusted definitions, the response rates were higher among treated patients (p < 0.0001). Historical criteria allowed to recognize lower response rate in patients treated with intravenous immunoglobulins (p = 0.009) or steroids (p = 0.033). Conclusions Contemporary definitions allow for more adequate categorization on most of the patients with primary immune thrombocytopenia considering the specific clinical aspects and different natural history of primary ITP in children.
    Acta haematologica Polonica 01/2013; DOI:10.1016/j.achaem.2013.12.001
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