CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation...
Bone Marrow Failure SyndromeSevere Aplastic Anemia14 moreThis study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease.
ASPirin in Immune thRombocytopenia Patients With Cardiovascular disEase
PurpuraThrombocytopenicThe incidence of immune thrombocytopenia increases with older age. This population is at risk for arterial thrombosis. Due to an increased turn-over of platelets, low-dose aspirin once daily may be insufficient in this population to protect against arterial thrombosis. This study is aimed at assessing the pharmacodynamics of aspirin once daily on platelet function in these patients.
Contribution of Anti-platelet Antibodies Identified With MAIPA Assay in the Demonstration of the...
ThrombocytopeniaImmune Thrombocytopenia1 moreImmune thrombocytopenia (ITP) is an autoimmune disease but, paradoxically, and unlike other autoimmune diseases, antiplatelet antibodies are not used either for the diagnosis of the disease or for its prognosis. ITP is a diagnosis of exclusion retained after elimination of other pathologies leading to a thrombocytopenia. No major study has prospectively evaluated the diagnostic value of the presence of anti-platelet antibodies in the etiological investigation of a thrombocytopenia, nor the impact of platelet antibodies on the course of ITP. The gold standard analysis for the determination of platelet antibodies, is the "monoclonal antibody immobilization of platelet antigens" assay (MAIPA), either direct to detect autoantibodies attached to platelets, or indirect to detect circulating antiplatelet antibodies. Therefore, this work aims to study the contribution of the presence of anti-platelet antibodies detected in MAIPA to determine the autoimmune nature of a thrombocytopenia at diagnosis.
Gene Sequencing as a Strategy for Identifying Genetic Factors Associated With Serious Adverse Events...
Vaccine-induced Thrombotic Thrombocytopenia SyndromeGuillain-Barre Syndrome2 moreThis protocol proposes to investigate genetic factors that may be involved in the pathogenesis of adverse events of interest with selected covid-19 vaccines: vaccine-induced immune thrombotic thrombocytopenia, and neurological adverse events, such as Guillain-Barré syndrome, acute disseminated encephalomyelitis and transverse myelitis, with the intention of identifying useful biomarkers in identifying people at higher risk, thus reducing the occurrence of these serious adverse events (SAE).
PRedicting OutcomeS in Preterm nEonates With thromboCyTopenia (PROSPECT)
Neonatal ThrombocytopeniaIntraventricular Hemorrhage1 moreRationale: Preterm neonates with low platelet counts receive prophylactic platelet transfusions with the aim to prevent bleeding. However, it is not clear in which cases platelet transfusions reduce the risk of bleeding or whether they do more harm than good. A large, randomized trial showed that the higher platelet count threshold for transfusion was associated with a higher rate of death and major bleeding, which suggests that platelet transfusions caused harm in neonates. To gain insight into the risk/benefits of platelet transfusions, the investigators will validate a recently developed dynamic prediction model for major bleeding in multiple NICUs in Europe and investigate the effects of prophylactic platelet transfusions on the risks of bleeding and potential transfusion-associated adverse events. This model could then be used in future studies to define enhanced indications for transfusion, with the ultimate goal to prevent transfusion-associated harm in this vulnerable population. Objectives: Validation of the existing dynamic prediction model in an international cohort of preterm neonates with severe thrombocytopenia (platelet count <50x10^9/L) admitted to a NICU. Model amendment to enable prediction of bleeding risks under various hypothetical platelet transfusion strategies in preterm neonates with severe thrombocytopenia. To examine whether prophylactic platelet transfusions are causally associated with the occurrence of bronchopulmonary dysplasia (BPD), necrotizing enterocolitis (NEC), proven sepsis, retinopathy of prematurity (ROP), major bleeding, and mortality. Study design: Multicenter international retrospective cohort study. Study population: Neonates with a gestational age <34 weeks and a platelet count <50x10^9/L, admitted to a NICU between January 1st 2017 and January 1st 2022. Main study endpoints: Major bleeding, BPD, NEC, proven sepsis, ROP and mortality. Nature and extent of the burden and risks associated with participation, benefit, and group relatedness: Not applicable, as this is a retrospective study.
Hetrombopag for the Treatment of Chemotherapy-Induced Thrombocytopenia in Adults With Solid Tumors....
Chemotherapy-Induced ThrombocytopeniaThe purpose of current study is to evaluate the efficacy and safety of hetrombopag for the treatment of chemotherapy-induced thrombocytopenia in patients with solid tumors.
Efficacy and Safety of Iguratimod in the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia...
Immune ThrombocytopeniaA phase 2, open-label, single-arm study to evaluate the efficacy and safety of iguratimod for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP)
A Prospective, One-arm and Open Clinical Study of Obinutuzumab in the Treatment of Pediatric Primary...
Immune ThrombocytopeniaTreatmentTo evaluate the safety and efficacy of Obinutuzumab in the treatment of pediatric primary immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line glucocorticoid treatment.
The Combination of Zanubrutinib and High-dose Dexamethasone as First-line Treatment in Adult Immune...
Immune ThrombocytopeniaRandomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Assessment of FOXP3 Gene Polymorphisms and Serum Interleukin 10 in Patients With ITP
ThrombocytopeniaImmune thrombocytopenia (ITP) is an autoimmune condition characterized by increased platelet destruction and suppression of production resulting in isolated thrombocytopenia. The exact etiology of ITP is unknown; however, multiple disease mechanisms exist and are mostly related to immune dysregulation [1]. Many studies in recent years have indicated that regulatory T cells (Tregs) play a critical role in the maintenance of immunological tolerance, and they have been reported to be defective in ITP patients, either numerically or functionally. [2-6]. They inhibit the activation and proliferation of effector T cells by the secretion of cytokines such as interleukin-10 (IL-10) and tumor growth factor-β (TGF-β) and by cell-to-cell interaction [7, 8].