View clinical trials related to Purpura, Thrombocytopenic.
Filter by:The association between hematologic malignancies and ITP is well described, but this link is much less clear with solid cancers. In cases of ITP associated with cancers, specific cancer treatment can lead to remission or even cure of ITP. Thus, our hypothesis was that chronic expression of GPIIB by tumor cells could have initiated an autoimmune loop against GPIIB, leading to the onset and perpetuation of ITP.
This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP. During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.
ItaliTTP is an observational, prospective, single-arm, national, multicenter, non-pharmacological cohort study aimed at better defining and understanding the natural history, disease severity, and clinical outcomes of patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in Italy. A minimum of 132 consecutive patients with acute iTTP (first event or relapse) will be enrolled for 3 years, with the possibility of extension, with a follow-up period of 3 years.
Immune thrombotic thrombocytopenic purpura (iTTP) is caused by a severe, autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi in blood vessels. The current standard of care of iTTP consists in the triple association of daily plasma exchange (PEX, 60 ml/kg/day), immunosuppressive agents and anti-adhesive treatment (Caplacizumab). Our group recently reported the outcome of 90 patients with iTTP treated with this triple association and when compared to historical patients, the triplet regimen prevented death, refractoriness and exacerbations. Likewise, plasma volumes were reduced by 2 to 3-fold and we could reduce the median number of PEX sessions from 13 to 6. PEX is an invasive and time-consuming procedure, associated with catheter and plasma-related complications ranging from 22% to 30%. Consequently, to alleviate the burden of care in iTTP, using a regimen without PEX would represent a major and topical goal. Attempts to treat patients with plasma infusion (PI) without PEX were previously reported and provided evidence that large volumes of PI (20-30 ml/kg/day) improved the initial outcome of iTTP. However, fluid overload occurred in most cases after 5-7 days, limiting the feasibility of this strategy. Nevertheless, the recent availability of caplacizumab opens the perspective of treating patients with plasma for a shorter period. Recently, strategies without PEX have been carried out in Jehovah's Witnesses with iTTP [5]. Impressively, improvement was rapid and comparable to those provided with a standard PEX-based treatment. Additionally, a treatment combining caplacizumab and immunosuppression only was successfully performed in six iTTP patients with severe neurologic and/or cardiac involvement. The rapid and durable improvement provides evidence that a regimen without plasma seems feasible. However, we consider that robust data are still lacking to completely remove plasmatherapy from iTTP management. Based on these statements, we wish here to address the efficacy and safety of a PEX-free regimen, combining PI only (15 ml/kg/day), corticosteroids/rituximab, and caplacizumab.
This exploratory study is to investigate the efficacy, safety and tolerability of Lusutrombopag in the treatment of primary immune thrombocytopenia in Chinese patients who have failed first-line therapy
1. Assesement of response to Thrombopoietin receptor agonists (TPO-RAs) as treatment in Idiopathic thrombocytopenia purpura patients in Assiut University hospital. 2. Explore side effects of Thrombopoietin receptor agonists in Idiopathic thrombocytopenia purpura . 3. To study effect of thrombopoietin receptor agonists and Quality life in Idiopathic thrombocytopenia purpura patients.
Immune thrombocytopenia is an autoimmune disease in which platelets are targeted by the host immune system. Platelet depletion occurs when autoantibodies targeting glycoproteins (αIIb-β3 and GPIb) found on the surface of platelets opsonize the cells, resulting in destruction by macrophages. These antibodies can also bind to megakaryocytes and prevent complete maturation, which results in lowered levels of platelet production Vitamin D (VD) or 1,25-dihydroxyvitamin D, may be a potent immunomodulator, and it may have potential therapeutic use in many autoimmune diseases In 1991, researchers found that the production of interleukin (IL)-6 and IL-2 in cell cultures were reduced by 1,25(OH)2D3 VD deficiency is very common in children with either newly diagnosed or chronic ITP form.Correlation between hypovitaminosis D and a higher incidence of infections and autoimmune diseases was reported as well Case-Control interventional study (over time each participant will be randomized to be enrolled in one of either study arms in a random sequence). To evaluate the efficacy of VD supplementation as an adjuvant therapy in chronic ITP children and its effect on platelet count and bleeding score. The participants will be - Children from 1 year to 18 years diagnosed with Chronic ITP with platelet count <100,000/microL. Patients groups will be: Patients of Chronic ITP aged from 1year till 18 years old . in this study will be divided into 2 groups ● Group A : All patients with VIT D insufficiency(The preferred level for 25(OH)D is now recommended by many experts to be >30 ng/ml) will receive supplementary Vitamin D3(NIVAGEN Vitamin D3 Cholecalciferol 50000 IU) oral once weekly for 3 months as adjuvant therapy in combination with their treatment for ITP. ● Group B : patients in this group have sufficient VIT D level and will be divided into 2 groups : - Group 1 B :will receive their standard treatment for ITP with VIT D. - Group 2 B :will receive their standard treatment for ITP without VIT D. Complete blood picture will be made at the beginning of study then monthly to evaluate the number of platelets that expected to increase after VD supplementation
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
Immune thrombocytopenia is an acquired autoimmune disease, in which platelets are opsonized by auto-antibodies and destroyed by phagocytic cells .Genetic polymorphisms in the immune mediators have been suggested to play a pivotal role in the pathogenesis of many autoimmune disorders . Several genetic polymorphisms of the immune system genes have been described in ITP such as interleukins, tumor necrosis factors (TNF) alpha and beta, and interferon-gamma., These polymorphisms were found to be associated with an increased risk of ITP progression or exacerbation .CD40 is a co-stimulatory 4348 kDa glycoprotein molecule composed of 277 amino acid residues which belongs to the tumor necrosis family. It is encoded by a gene which is located at chromosome 20q11-13, expressed mainly on antigen presenting cells (APCs), some non-immune cells and tumors.Antinuclear antibodies (ANA) is a collective term for a large and heterogeneous group of circulating autoantibody. Reflecting their clinical importance, ANA are diagnostic, prognostic or classification criteria for many autoimmune diseases.
Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by thrombocytopenia, microangiopathic hemolytic anemia, and microvascular thrombosis causing neurological and renal abnormalities; it is associated with massive depletion of platelets in the microvasculature to form microthrombi1 . Long-term follow-up of patients with congenital TTP (cTTP) revealed frequent strokes and renal injury. Of 217 surviving patients, 62 (29%) had a stroke; the median age was 21 years. iTTP patients also require long-term follow-up. iTTP patients with low ADAMTS13 activity (<70%) in remission have a 28% risk of stroke. Survival rates of iTTP patients in remission were lower than those of age-, race-, and sex-matched populations. In terms of stable treatment, maintenance therapy is not recommended for patients with iTTP. Previous studies have shown that aspirin may be able to prevent stroke complications in patients with cTTP and iTTP. In addition to its potential efficacy, the risks of aspirin are small and inexpensive. Aspirin is very effective in secondary prevention of stroke 6. However, the therapeutic value of aspirin in TTP has not been studied previously. To improve the prognosis and survival of patients with cTTP and iTTP, we propose to conduct a prospective study to observe the efficacy and safety of aspirin in patients with cTTP and iTTP in remission.