Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP...
Thrombotic Thrombocytopenic PurpuraAcquiredThe aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.
Honey in Chronic Immune Thrombocytopenia
Chronic Thrombocytopenic PurpuraBased on the assumption that honey may have an immunomodulatory effect, the aim of our present study will be to verify whether the intake of honey, as a natural substance, in patients with chronic Immune Thrombocytopenia (ITP) could affect the platelet number and hence could affect the bleeding severity. Thirty patients suffering from chronic ITP will be recruited from the Hematology Clinic of the Pediatric Hospital of Ain Shams University, Cairo, Egypt. The diagnosis of ITP will be based on the presence of isolated thrombocytopenia with a peripheral blood platelet count less than 100 x 109/L, and absence of any obvious initiating and/or underlying cause of the thrombocytopenia. Chronic ITP is defined as ITP for at least 12 months. The age of the patients will range from 5 to 15 years, and they will be of both sexes. This study was approved by the local Ethics Committee of the Pediatric Department of Ain Shams University, and an informed consent to participate in this study will be obtained from at least one parent of each patient. Furthermore, an ascent form will be obtained from each patient above 7 years of age.
Efficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With...
ThrombocytopeniaImmune Thrombocytopenia1 moreThe purpose of this study is to evaluate the efficacy and safety of rhTPO in the maintenance treatment of ITP, to explore the appropriate dosing interval of the maintenance treatment of rhTPO .
A Study of Hetrombopag Olamine in Chronic Idiopathic Thrombocytopenic Purpura (ITP) Patients
PurpuraThrombocytopenic1 moreThe purpose of this study is to obtain information on efficacy, safety and Pharmacokinetics (PK)/Pharmacodynamics (PD) of Hetrombopag over 14 days in Chinese patients with chronic ITP.
Association of FcγRIIIA Polymorphism and THPO Expression With Response to Eltrombopag in Refractory...
Immune Thrombocytopenic Purpura (ITP)Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease characterized by antibody-mediated platelet destruction. The complex pathogenesis of ITP with multiple challenges to immune system in terms of genetic predisposition, infection, responsiveness to immunosuppressive therapy (IST) and inhibition of platelet production has proven the diversity of constraints in diagnosing and treating ITP. Thrombopoietin receptor agonist (Eltrombopag) is specifically indicated for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This clinical trial aims to investigate the association of Fc gammaRIIIA gene (V158F) genetic predisposition with treatment outcome of Immune Thrombocytopenia (ITP) in refractory ITP patients and especially with Eltrombopag.
Early Predictive Factors of Cardiac and Cerebral Involvement in TMA
Thrombotic MicroangiopathiesThrombotic Thrombocytopenic PurpuraThe aim of this study is to determine the frequency of cardiac and cerebral involvements in patients with idiopathic thrombotic microangiopathies on diagnosis. Patients will be assessed for cardiac involvement (troponin Ic level and cardiac ultrasonography) and cerebral involvement (cerebral MRI). The investigators will assess whether serum troponin Ic on diagnosis can predict morbidity and mortality of patients with a thrombotic microangiopathy at the acute phase. The primary outcome measurement is the event free survival at day 30, as defined by death, myocardial ischemia, arrhythmia, severe cerebral injury and disease exacerbation. An increase in troponin Ic on diagnosis is defined as at least one result above 0.2 ng/ml among the three daily analyses performed after TMA diagnosis.
Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune...
PurpuraSchoenlein-Henoch15 moreOBJECTIVES: I. Determine whether there is prompt engraftment after autologous peripheral blood stem cell transplantation using filgrastim (G-CSF) mobilization in patients with life threatening autoimmune diseases. II. Determine the kinetics of T- and B-cell immune reconstitution after a combination of timed plasmapheresis, high dose cyclophosphamide and total lymphoid irradiation, and posttransplant immunosuppression with cyclosporine in these patients. III. Determine whether this treatment regimen beneficially influences the clinical course of these patients.
A Study to Investigate Belimumab for the Treatment of Chronic Immune Thrombocytopenia.
PurpuraThrombocytopaenic1 moreChronic immune thrombocytopenia (ITP) is a longterm disease in which the blood does not clot normally. This is due to a low number of blood cell fragments called platelets. Platelets clot to seal small cuts or breaks on blood vessel walls and stop bleeding. Normally the immune system makes proteins called antibodies to fight off harmful substances that enter the body. In ITP, the immune system produces antibodies that attack and destroy the body's platelets by mistake. Patients can suffer from bleeding under the skin, nosebleeds, blood in urine or stools and in very severe cases bleeding in the brain. Patients have an increased frequency of death from bleeding complications compared to normal. Chronic ITP is fairly rare , with an incidence of 32 new cases/million people each year. Existing treatments work by lowering the activity of the immune system or directly increasing platelet count. These treatments do not work effectively in all patients and can have side effects. We hope that understanding how belimumab works in ITP will help in the development of future treatments for ITP and other autoimmune diseases. We will test the safety, blood levels and effects of the study medication in people with chronic ITP. Patients will receive the study medication intravenously (through a needle inserted into a vein) and blood samples will be taken before and on several occasions afterwards. Up to 40 patients with chronic ITP, aged 18 to 75 will participate. Approximately 11 patients will take dummy medicine instead of the study medicine neither they or their study doctor will know which one they are given. Participants will take up to 57 weeks to finish the study. They'll make 12 outpatient visits. The study will take place in hospitals in the UK. Other sites in mainland Europe may also be initiated. A pharmaceutical company, GlaxoSmithKline, is funding the study.
Efficacy of Immunomudulatory Therapy With All-trans Retinoid Acid for Adults With Chronic Immune...
Immune Thrombocytopenic PurpuraThe purpose of this study is to determine whether All-trans retinoic acid (ATRA) are effective in the treatment of refractory idiopathic thrombocytopenic purpura (RITP).
Vaccine Response After Rituximab for Chronic, Severe Idiopathic Thrombocytopenic Purpura
Idiopathic Thrombocytopenic PurpuraImmune Thrombocytopenic PurpuraThe purpose of this study is to determine how children with a history of severe, chronic Idiopathic Thrombocytopenic Purpura (ITP) who were treated with rituximab might respond to vaccines. Eligible patients are previously or currently enrolled in a study entitled "Open Label, Phase I/II Trial of Rituximab for Chronic, Severe Idiopathic Thrombocytopenic Purpura in Children and Adolescents" and have decided to obtain an inactivated influenza vaccination. These patients will be invited to provide one blood sample prior to vaccination and a second sample following vaccination to quantify immune response to vaccination.