Active clinical trials for "Purpura, Thrombocytopenic, Idiopathic"

A randomized, open-label, multicenter study to compare the efficacy and safety of terbutaline plus danazol compared to danazol monotherapy for the second-line treatment of adults with corticosteroid-resistant or relapsed primary immune thrombocytopenia (ITP).

Autoimmune diseases are characterized by various factors that contribute to a breakdown in self-tolerance, that is, the ability of the immune system to effectively distinguish self from non-self and to refrain from attacking self. Autoimmune diseases include a broad spectrum of disorders, such as idiopathic thrombocytopenic purpura, systemic lupus erythematosus, rheumatoid arthritis, systemic sclerosis, and inflammatory bowel disease. Although significant progress has been achieved in the development of approaches to the treatment of autoimmune diseases, the etiologies, and pathogenesis of autoimmune diseases remain obscure (Tao et al., 2016) Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder characterized by bleeding due to isolated thrombocytopenia with platelet count less than 100 × 109/L (Neunert et al., 2019). ITP is classified based on course of disease into acute (3- <12 months), and chronic (≥12 months) (Provan et al., 2019). ITP usually has a chronic course in adults (Moulis et al., 2017) whereas approximately 8090% of children undergo spontaneous remission within weeks to months of disease onset (Heitink et al., 2018). The main pathogenesis of ITP is the loss of immune tolerance to platelet auto-antigens, which results in increased platelet destruction and impaired thrombopoiesis by autoantibodies and cytotoxic T lymphocytes (CTLs) (Adiua et al., 2017). Among these abnormalities include the increased number of the T helper 1 (Th1) cells (Panitsas et al.,2004). the decreased number or defective suppressive function of regulatory T cells (Tregs) (Yu et al., 2008) , and the

Primary immune thrombocytopenia (ITP), one of the most common bleeding disorders, is characterized by reduced platelet count and an increased risk of bleeding ITP is an acquired autoimmune disease, in which platelets are opsonized by auto-antibodies and destroyed by phagocytic cells ITP pathogenesis involves a hyper-activated T cell response, which is important for cell-mediated cytotoxicity and IgG production Therefore, investigating T cell abnormalities in ITP patients may reveal the mechanism of pathogenesis and development of ITP. The costimulatory molecules of T cells consist of CD28, inducible costimulatory (ICOS), TNF superfamily member 4 (TNFSF4), and DNAM1 (CD226), and the co-inhibitory molecules contain TIM3, cytotoxic T-lymphocyte associated protein 4 (CTLA4), programmed death-1 (PD1), and lymphocyte activating 3 (LAG3) Among these, CD28 and CTLA4 represent the best-studied costimulatory pathways. CD28 and CTLA4 interact with two ligands (CD80 and CD86) on the surface of antigen-presenting cells (APCs), introducing a positive stimulatory and a negative inhibitory signal into T cells, respectively

Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.

Multicenter retrospective and prospective observational study based on the collection of sequential anonymized data from principal Italian ITP centers, to describe the clinical course of ITP patients becoming infected by SARS-CoV-2 and of COVID-19 patients developing de novo ITP.

Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the differential diagnostic work-up, and document the clinical course under various treatments. Time points: at inclusion, after 6 months, after 12 months, then yearly up to 4 years after inclusion. No intervention, mere observation and documentation. Guided pre-inclusion (differential) diagnostic work-up.

From 2004, OBS'CEREVANCE is a national real-world prospective clinical cohort of patients with auto-immune cytopenia of pediatric-onset : Immune thrombocytopenia (ITP), Autoimmune Hemolytic anemia (AIHA), or Evans syndrome (all bi or tri cytopenias). Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.

Immune thrombocytopenia (ITP) is an autoimmune disease characterized by low platelet counts with or without mucocutaneous bleeding (McMillan, 2007). Like the majority of autoimmune diseases, ITP is an organ-specific disease, and abnormalities in the regulation of the immune system have been shown to play an important role in the initiation and/or perpetuation of the disease (McKenzie et al.,2013). Still, immune thrombocytopenia (ITP) is a significant clinical problem due to chronicity, treatment cost, occurrence mainly in, young, and relatively poorer quality of life

Immune thrombocytopenia (ITP) is an autoimmune disease characterized by a peripheral destruction of platelets responsible for bleedings. Monocytes/macrophages play a double role by phagocyting platelets recognized by autoantibodies and by maintaining the autoimmune response via their antigen-presenting cell functions. Fcgamma receptors (FcγR), that are represented by activating receptors (FcγRI, FcγRIIa, FcγRIII) and an inhibiting one (FcγRIIb), are involved in the regulation of macrophages and have been reported to be dysregulated in autoimmune diseases such as rheumatoid arthritis and systemic lupus erythematous. The aim of this study is to compare the expression of FcγR in patients with ITP on circulating monocytes and on splenic macrophages.

The Internal Medicine Department of Haut-Lévêque Hospital (Pr E LAZARO, Pr JL PELLEGRIN, Pr JF VIALLARD) was accredited in 2017 by the Ministry of Health as a Constitutive Reference Center for Autoimmune Cytopenia. The investigators wish to launch new research projects in autoimmune cytopenia and propose a translational and fundamental research based on collaboration between the clinical department, the biological resource center and the CNRS and INSERM research units ("Bedside to the Bench Strategy"). Thus, in the perspective of future research work, it seems imperative to set up a biological bank for the patients followed in our Reference Center.