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.
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.
Gender same as the matched case
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
Participants are divided into three groups: TDT children who have received gene therapy, TDT children who have received lifelong supportive therapy, and healthy children.
Cohort
Prospective
Gene therapy
GeneticAutologous edited hematopoietic stem cell transplantation
Growth in pediatric subjects will be assessed by height.
Height in meters.
Growth in pediatric subjects will be assessed by weight.
Weight in kilograms.
Growth in pediatric subjects will be assessed by the head and chest circumference.
Head and chest circumference in centimeters.
Growth in pediatric subjects will be assessed by interocular distance.
Interocular distance in centimeters.
Click on the lead sponsor to view all of their trials.
