View clinical trials related to Thalassemia.
Filter by:Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease. Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister. Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
The primary purpose of this study is to evaluate the effect of deferasirox on renal hemodynamics by determining glomerular filtration rate (GFR), renal plasma flow (RPF) and filtration fraction (FF).
The purpose of this study is to provide accurate prognostic data linking cardiac complications to myocardial T2* values (A measure of iron levels in the heart using MRI)in patients predisposed to heart iron overload.
Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't developed significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpose of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and laboratory studies including Non Transferrin Binding Iron, Labile Iron and Hepcidin, in order to determine the cardiac and liver iron.
Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't develope significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpouse of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and cardiac T2*MRI in order to determine the cardiac and liver iron.
Hepatitis C is one of the most common causes of long-term liver disease in the United States. Ribavirin and peginterferon alfa-2a are two medications that are used to treat hepatitis C infection. The purpose of this study is to evaluate the safety of these two medications in adults with hepatitis C and thalassemia, a type of blood disorder.
β thalassemia is an autosomal recessive hemoglobinopathy and considered as the most widespread genetic mutation. According to the World Health Organization (WHO) between 1.5-7% of the world population are carriers for this disease, and every year 60,000-400,000 birth of new patients are reported. In Israel, the incidence of carriers for β thalassemia is around 20% among the Jewish from Kurdish origin and around 5-10% among the Arab population. β thalassemia is a severe disease which requires many resources, both medical and financial. The disease is expressed by chronic hemolytic anemia which requires regular blood transfusions every 3 weeks. As a result of the blood transfusions and the iron absorption by the digestive tract, those patients suffer from severe hemosiderosis which is the main mortality cause in the disease, mainly in the second decade for life. Daily treatment with iron chelator is required. Moreover, despite the actual treatment, the quality of life of those patients is still low. Therefore the implementation of a prevention program which includes finding an effective and inexpensive way for identifying the β thalassemia carriers is a humanitary and publicly important goal. In β thalassemia carriers, laboratory tests will show hypochromic microcytic anemia. Those findings are similar in iron deficiency anemia, but the RBC number and the RDW are normal in thalassemia carriers. Few researchers tried in the past to determine cutoff point for diagnosis of β thalassemia carriers by different formulas. We used the algorithm SVM (support vector machine) to find a reliable formula that can separate patients with Iron deficiency anemia/ healthy from patients with β thalassemia minor (carriers). This formula can be inserted to any automatic blood counter and search for suspected carriers without deliberately intention and without any further blood test.
Hydroxyurea was found to be a good treatment in adult patients with sickle cell anemia with significant decrease in the frequency of vaso-occlusive crises and other crises related to SCA. Several studies were published with relative short term follow up in pediatric and young adult age. The purpose of this study is to assess the long term follow up in a group of patients that initiated Hydroxyurea treatment in childhood.
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to patients with thalassemia. Development of such an assay would be useful both for identification of the exact mutation causing the disease, as well as for providing a direct method to measure and monitor the kinetics of donor erythropoiesis in this patient population following transplant.