Role of Vitamin C to Augment Iron Chelation With DFP or DFX
Vitamin cThalassemia Major1 morerole of Vit C to Augment iron chelation with DFP or DFX in thalassemic patients.
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-ThalassemiaThis is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
the Efficacy and Safety of Sugammadex in Children 0-2 Years Old
AnesthesiaPediatric Cancer4 moreSugammadex is a selective antidote to muscle relaxants rocuronium bromide and vecuronium bromide. Sugammadex is a modified gamma-cyclodextrin, a compound that selectively binds rocuronium bromide and vecuronium bromide. It forms a complex with them in the blood plasma, which leads to the decrease in the concentration of muscle relaxant binding to nicotinic receptors in the neuromuscular synapse. The result is the the elimination of neuromuscular blockade caused by rocuronium bromide or vecuronium bromide. Sugammadex is used to eliminate neuromuscular blockade caused by rocuronium bromide in children aged 2 years and adolescents in standard clinical situations. The aim of the study is to prove the efficacy and safety of sugammadex in children under 2 years
Sperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants?
Endocrine DiseaseFertility Decreased Male1 moreAccumulation of iron in patients with beta thalassemia major causes free radical formation which leads to damage of biological membranes. Sperm DNA damage may result from these generated antioxidants. We aimed at investigating the current DNA damage in the sperms of adult patients with beta thalassemia major and the effect generated by giving antioxidant treatment for 6 months.
Thalassemia Treatment Based on the Stem Cell Technology
Beta-ThalassemiaIn order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.
Gene Therapy for Transfusion Dependent Beta-thalassemia
Beta-ThalassemiaThis is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and...
Beta-thalassemia MajorSickle Cell Disease1 moreInterventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Thalassemia IntermediaThe study hypothesis that treatment with Erythropoietin (EPO) combined with Human Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia intermedia patients. Second is to determine whether any of the following correlate with improved hematologic response: A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin levels and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs). Goal: The aim is to assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation,quality of life (QoL) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding.
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective...
Thalassemia MajorThe only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation...
Thalassemia MajorAllo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool,we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.