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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gene therapy group | TDT Children who have received gene therapy between 3~14 years old. |
| |
| Supportive therapy group | TDT Children who received lifelong treatment with blood transfusions and iron chelation. They were selected based on the age and sex of the gene therapy group. | ||
| Healthy children group | Healthy children were selected based on the age and sex of the gene therapy group. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Gene therapy | Genetic | Autologous edited hematopoietic stem cell transplantation |
|
| Measure | Description | Time Frame |
|---|---|---|
| Growth in pediatric subjects will be assessed by height. | Height in meters. | Up to 5 years. |
| Growth in pediatric subjects will be assessed by weight. | Weight in kilograms. | Up to 5 years. |
| Growth in pediatric subjects will be assessed by the head and chest circumference. | Head and chest circumference in centimeters. | Up to 5 years. |
| Growth in pediatric subjects will be assessed by interocular distance. | Interocular distance in centimeters. | Up to 5 years. |
| Growth in pediatric subjects will be assessed by bone density. | Bone density measurement is measured by a procedure called densitometry, performed in the radiology medicine departments of hospitals or clinics. The result is the bone mineral density in grams per unit area (g/cm2). | Up to 5 years. |
| The pubertal status will be measured using by Tanner scale. | The Tanner scale, a well-known technique used to define pubertal stage, is based on physical measurements of development based on external primary and secondary sex characteristics (from Tanner 1: pre-pubertal to Tanner 5: mature adolescent).It was developed by Marshall and Tanner while conducting a longitudinal study during the 1940s to the 1960s in England. Based on observational data, they developed separate scales for the development of external genitalia: phallus, scrotum, and testes volume in males; breasts in females; and pubic hair in both males and females. | Up to 5 years. |
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Inclusion Criteria:
Gene therapy group-inclusion
Supportive therapy group-inclusion
Healthy children group-inclusion
Exclusion Criteria:
Discontinuation of Study :
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Participants are divided into three groups: TDT children who have received gene therapy, TDT children who have received lifelong supportive therapy, and healthy children.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jun Shi, PhD | Contact | 2223900913 | shijun@ihcams.ac.cn | |
| Jingyu Zhao, MPH | Contact | 13752253515 | zhaojingyu@ihcams.ac.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Regenerative Medicine Center | Recruiting | Tianjin | Tianjin Municipality | China |
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| ID | Term |
|---|---|
| D015316 | Genetic Therapy |
| ID | Term |
|---|---|
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D005818 | Genetic Engineering |
| D005821 | Genetic Techniques |
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The peripheral blood sample was collected and stored in the IHBDH biobank.
| Hormonal levels will be measured by clinical examination. | Hormonal levels included thyroid-stimulating hormone (TSH), serum triiodothyronine (T3), serum-free triiodothyronine (T4), growth hormone (GH, including IGF-1 and IGF-2), and sex hormone examination. Sex hormone examination is only for the following subjects: estradiol, FSH and LH for girls aged 9-18; testosterone, FSH and LH for boys aged 12-18. | Up to 5 years. |
| Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study. | The Pediatric Quality of Life Inventory (PedsQL) is a valid, standardized, generic, and self-reporting assessment tool to measure health-related quality of life HRQOL for pediatrics and adolescents. PedsQL contain 23-items, and consists of the following: physical functioning, emotional functioning, social functioning, and school functioning. Each item is scored on a 5-point scale, scores are linearly transformed to a 0-100 scale in which high score means better condition. | Up to 5 years. |
| Lifestyle behaviors will be assessed by general questioning. | Up to 5 years. |
| D008919 |
| Investigative Techniques |