View clinical trials related to Thalassemia.
Filter by:This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
Bone denisty changes in children with beta thalassemia major
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world . Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients. Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family. A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes. Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.
Objective: To longitudinally track the dynamic changes in the survival quality of pediatric patients after hematopoietic stem cell transplantation at different time points within 1 year post-transplantation, analyze the influencing factors of survival quality at each time point, identify independent risk factors that can be intervened, provide reference for medical staff to recognize survival quality problems early, guide the dynamic management of clinical survival quality, and formulate continuation care management plans. Methods: This study adopted a repeated measurement study design. A total of 250 pediatric patients who underwent hematopoietic stem cell transplantation in three tertiary hospitals in Guangdong Province from August 2023 to December 2025 and met the research standards were selected as the research subjects. The "Childhood Health Assessment Questionnaire Transplant Module 3.0 Chinese Version" was used to evaluate the survival quality of the patients at six time points: 1 week before pre-treatment (T0), the day of stem cell infusion (T1), 1 month (T2), 3 months (T3), 6 months (T4), and 1 year (T5) after transplantation. Statistical methods for repeated measures were used to analyze the relevant information, and mixed-effect linear models were used to analyze the influencing factors of survival quality at the six time points, and to identify independent risk factors.
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in people with SCD. Better treatments are needed. Objective: To test a drug (fostamatinib) in people with SCD. Eligibility: People aged 18 to 65 with SCD. Design: Participants will have 6 clinic visits over 12 weeks. Each visit will be 2 to 3 hours. Participants will be screened. They will have a physical exam with blood tests. They will tell the researchers about the medications they take. Fostamatinib is a tablet taken by mouth. Participants will take the drug at home, twice a day, for up to 6 weeks. Participants will have a clinic visit every 2 weeks while they are taking the drug. At each visit they will have a physical exam with blood tests. They will talk about any side effects the drug may be causing. If they are tolerating the drug well after the first 2 weeks, they may begin taking a higher dose. Participants will have a final visit 4 weeks after they stop taking the drug. They will have a physical exam and blood tests; they will be checked for any side effects of the drug.
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3. To correlate PUM1 gene expression pattern and protein levels with HbF levels in sickle cell anemia and β-thalassemia intermedia patients.
The thalassemias are a group of inherited hematologic disorders caused by defects in the synthesis of one or more of the hemoglobin chains [1]. Thalassemia are classified into the alpha (α) and (β) thalassemia, which contain deficits in (α) and (β) globin production respectively (α)thalassemia are caused by decreased production of alpha-globin chains from chromosome 16. There are 4 types of (α) thalassemia: thalassemia silent carrier thalassemia carrier . Hemoglobin H disease thalassemia major Beta-thalassemia are caused by point mutations or more rarely deletions in the β-globin gene on chromosome 11, leading to reduced (β+) or absent (β0) synthesis of the β chains of hemoglobin. Imbalances of globin chains cause hemolysis and impair erythropoiesis [4-7]. β-thalassemia can be classified into: Beta Thalassemia major, Beta Thalassemia intermedia, Beta Thalassemia minor Thalassemia is a chronic disease that presents a range of serious clinical and psychological challenges. The effects of thalassemia on physical health can lead to physical deformity, growth retardation, and delayed puberty [9, 10]. Its impact on physical appearance, e.g., bone deformities and short stature, also contributes to a poor self-image [10, 11]. Severe complications such as heart failure, cardiac arrhythmia, liver disease, endocrine complications, and infections are common among thalassemia patients [8, 12].
The effect of N_acetylcystein as an antioxidant on iron overload and frequency of blood transfusion in β-thalassemia major patients at Assiut Childern Hospital University And its cosubmitted for partial fulfillment of master degree in Pediatrics
This study will be intented to evaluate the safety and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.
This study will be intented to evaluate the safety and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.