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

NCT06931912 | Recruiting

• 1 other identifier: CAMSCRF2024041
• Study Type: interventional
• Target: 369
• Locations: 1 country
• Sites: 1

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.

• 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.

Conditions

Thalassemia Major; Thalassemia Intermedia; Growth Delay; Puberty, Delayed

Keywords

Thalassemia; Children and adolescents; Growth development; Health Promotion; Clinical Management

Outcome Measures

Primary Outcomes (8)

• Height-for-age (m)

  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

  Up to 1 year

• Body mass index-for-age (BMI-for-age, kg/m^2)

  It is assessed using the 'Growth Standard for Children under 7 Years of Age' and the 'Dietary Guidelines for Chinese Residents'.

  Up to 1 year

• Puberty status

  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).

  Up to 1 year

• Endocrine function

  Endocrine function such as hormonal levels (IGF-1, GH, etc.) will be measured by clinical examination

  Up to 1 year

• Nutritional status

  Nutritional status will be measured by clinical examination, such as concentration of Vitamin D and Zinc

  Up to 1 year

• Intelligence quotient (IQ)

  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.

  Up to 1 year

• Brain function

  Brain function will be measured by Functional Near-infrared Spectroscopy (fNIRS) to record brain activations

  Up to 1 year

• Quality of life

  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.

  Up to 1 year

Study Arms (1)

Pediatric patients with growth problems

EXPERIMENTAL

We identify pediatric patients with growth problems by conducting growth and development assessments. Implement the MENBS clinical interventions for pediatric patients with growth problems.

Behavioral: MENBS clinical interventions

Interventions

MENBS clinical interventions BEHAVIORAL

* Monitor: Continuously monitor health-related indicators through regular follow-up. * Education: Provide health education to improve 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.

Pediatric patients with growth problems

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• Not applicable

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Regenerative Medicine Center and Red Blood Cell Disorders Center

Tianjin, Tianjin Municipality, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia; Puberty, Delayed; Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Gonadal Disorders; Endocrine System Diseases

Central Study Contacts

Jingyu Zhao, MPH

CONTACT

13752253515; zhaojingyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2025
• First Posted: April 17, 2025
• Study Start: December 1, 2024
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: February 11, 2026

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)