Clinical trial NCT06931912: Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
Official title: Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Thalassemia Intermedia
Thalassemia Major
Growth Delay
Puberty, Delayed
Keywords: Thalassemia
Children and adolescents
Growth development
Health Promotion
Clinical Management
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2025-04-17
Enrollment: 369
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Height-for-age (m)
timeFrame: Up to 1 year
measure: Body mass index-for-age (BMI-for-age, kg/m^2)
timeFrame: Up to 1 year
measure: Puberty status
timeFrame: Up to 1 year
measure: Endocrine function
timeFrame: Up to 1 year
measure: Nutritional status
timeFrame: Up to 1 year
measure: Intelligence quotient (IQ)
timeFrame: Up to 1 year
measure: Brain function
timeFrame: Up to 1 year
measure: Quality of life
timeFrame: Up to 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Regenerative Medicine Center and Red Blood Cell Disorders Center
state: Tianjin
Study officials:
References: