Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 385
- 试验地点
- 1
- 主要终点
- Height-for-age (m)
研究概览
简要总结
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.
- 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.
- 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.
- Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions.
- Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects diagnosed with transfusion-dependent thalassemia (TDT)
- •Male or female age ≤19 years
- •Subjects who are willing and able to provide written informed consent
排除标准
- •Subjects with clinically significant thyroid dysfunction
- •Subjects received allo-HSCT or gene therapy
研究组 & 干预措施
Pediatric patients with growth problems
We identify pediatric patients with growth problems by conducting growth and development assessments. Implement the MENBS clinical interventions for pediatric patients with growth problems.
干预措施: MENBS clinical interventions (Behavioral)
结局指标
主要结局
Height-for-age (m)
时间窗: Up to 1 year
It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Standard for Height Level Classification among Children and Adolescents Aged 7-18 Years' issued by the National Health Commission
Body mass index-for-age (BMI-for-age, kg/m^2)
时间窗: Up to 1 year
It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Dietary Guidelines for Chinese Residents'.
Puberty status
时间窗: Up to 1 year
Puberty status will be measured using by Tanner scale (TS). It is a five-stage system to assess breast development (in girls), genital development (in boys), pubic hair growth (in both sexes).
Endocrine function
时间窗: Up to 1 year
Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination
Nutritional status
时间窗: Up to 1 year
Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc
Intelligence quotient (IQ)
时间窗: Up to 1 year
IQ will be measured by scores obtained from Raven's Progressive Matrices. Raw scores are converted into percentile ranks or IQ scores based on age-group norms: * ≥95th percentile (IQ \~125+): Very high intelligence. * 75th-94th percentile (IQ \~110-124): Above average. * 25th-74th percentile (IQ \~90-109): Average range (most common). * 5th-24th percentile (IQ \~80-89): Below average. * \<5th percentile (IQ \<80): Potential intellectual disability.
Brain function
时间窗: Up to 1 year
Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations
Quality of life
时间窗: Up to 1 year
Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL), with higher scores indicating better quality of life. The score range is 0-100.
Bone age
时间窗: Up to 1 year
one age was assessed from left hand-wrist radiographs according to the Chinese Hand-Wrist Bone Development Standard (China 05).
Height-for-age (m)
时间窗: Up to 1 yearUp to 1 year
It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Standard for Height Level Classification among Children and Adolescents Aged 7-18 Years' issued by the National Health Commission
Body mass index-for-age (BMI-for-age, kg/m^2)
时间窗: Up to 1 yearUp to 1 year
It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Dietary Guidelines for Chinese Residents'.
次要结局
未报告次要终点
研究者
Kuang Zhexiang
Head nurse of Regenerative Medical Center
Institute of Hematology & Blood Diseases Hospital, China