Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Trial Parameters
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: * Diagnosis of compound α-thalassemia * Uncontrolled systemic fungal, bacterial, or viral infection * History of malignant solid tumors, myeloproliferative or immunodeficiency diseases * Diagnosed with mental illness * Patients considered to be ineligible for the study by the investigator for reasons other than the abov