View clinical trials related to Thalassemia.
Filter by:Bone denisty changes in children with beta thalassemia major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-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.
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
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.
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.
This retrospective study aimed to evaluate the demographic characteristics, clinical conditions in term of physical examination findings), functional status, and laboratory results of patients with thalassemia minor (TM) and breast cancer (BC) in order to identify any differences between the group with BC only. Available data as anticancer treatment, comorbidities, weight and height will be combined to report body mass index (BMI) in kg/m2, systolic and diastolic blood pressure, heart rate, ECG, transthoracic echocardiography, blood count, lipid panels, glucose, kidney function tests, (N terminal) NT-proBNP, troponins, handgrip assessments, functional status were extracted from patients files and hospital electronic archives.
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.
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India