Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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.
⁃ Gene therapy group-inclusion
• Male or female age of 3-14 years
• TDT Children who have received gene therapy.
• Subjects who are willing and able to provide written informed consent.
⁃ Supportive therapy group-inclusion
• Gender same as the matched case
• Age similar to the matched case
• Children with β-TDT
• No history of gene therapy or allogeneic hematopoietic stem cell transplantation
• Subjects who are willing and able to provide written informed consent.
⁃ Healthy children group-inclusion
• Gender same as the matched case
• Age similar to the matched case
• Subjects who are willing and able to provide written informed consent.