Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

Not Applicable

Recruiting

• Conditions: Thalassemia Intermedia; Thalassemia Major; Growth Delay; Puberty, Delayed

• Registration Number: NCT06931912
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief Summary

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.

1. 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.

2. 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.

3. Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions.

4. Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-12-01
• Study First Submitted: 2025-01-07
• Status Verified: 2025-04
• Study First Submitted QC: 2025-04-09
• Last Update Submitted: 2025-04-09
• Study First Posted: 2025-04-17
• Last Update Posted: 2025-04-17
• Primary Completion: 2026-10-31
• Study Completion: 2026-11-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 369

Inclusion Criteria

• Subjects diagnosed with transfusion-dependent thalassemia (TDT)
• Male or female age ≤18 years
• Subjects who are willing and able to provide written informed consent

Exclusion Criteria

• Not applicable

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Height-for-age (m)	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)	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'.
Endocrine function	Up to 1 year	Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination
Nutritional status	Up to 1 year	Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc
Intelligence quotient (IQ)	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	Up to 1 year	Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations
Quality of life	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.
Puberty status	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).

Trial Locations

Locations (1)

Regenerative Medicine Center and Red Blood Cell Disorders Center; 🇨🇳 Tianjin, Tianjin, China