Clinical Research Directory

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a substantial economic burden on their families and a serious social strain on the labor force. Investigating the growth and development of these children and adolescents, and establishing targeted intervention plans, holds significant social value for public health practice. 1. To screen and identify pediatric patients with growth problems by conducting growth and development assessments in high-incidence areas of China, including physical development, endocrine function, nutritional status, brain function and lifestyle behaviors. 2. Implement the MENBS clinical interventions for pediatric patients with growth problems, concentrating on the following areas: * Monitor: Continuously monitor health-related indicators through regular follow-up. * Education: Provide health education to improve the cognition of patients and their families. * Nutrition: Assess patients' nutritional risks and develop personalized diet plans. * Behavior: Recommend appropriate exercise plans to promote physical development. * Support: Conduct home visits, offer free clinics and establish a support network. 3. Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions. 4. Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia