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Updated: May 11, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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[Monitoring and interventions of growth disorders and endocrine function in children with transfusion-dependent

Xin Fan1, Yi-Yun Huang1

  • 1Department of Pediatrics, Second Affiliated Hospital of Guangxi Medical University, Nanning 530007, China.

Zhongguo Dang Dai Er Ke Za Zhi = Chinese Journal of Contemporary Pediatrics
|April 17, 2025
PubMed

Insights

Transfusion-dependent thalassemia (TDT) causes growth issues and endocrine problems due to iron overload from blood transfusions. This review covers monitoring and treatment for these complications in TDT patients.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Hematology

Background:

  • Transfusion-dependent thalassemia (TDT) is a severe inherited hemolytic anemia.
  • Growth retardation is a frequent complication in TDT patients.
  • Chronic blood transfusions in TDT lead to iron overload, impacting endocrine function.

Purpose of the Study:

  • To review clinical monitoring strategies for growth disorders in TDT.
  • To outline intervention measures for endocrine dysfunctions associated with TDT.
  • To provide clinicians with references for managing TDT-related growth and endocrine issues.

Main Methods:

  • Literature review of clinical studies and guidelines.
  • Analysis of endocrine abnormalities in TDT patients.
  • Synthesis of monitoring and treatment recommendations.

Main Results:

  • Common endocrine dysfunctions include growth hormone/IGF axis abnormalities, hypothyroidism, hypoparathyroidism, hypogonadism, and adrenal insufficiency.
  • Iron overload is the primary driver of these endocrine complications.
  • Decreased bone density is also a significant concern.

Conclusions:

  • Comprehensive monitoring of growth and endocrine function is crucial for TDT patients.
  • Timely intervention can mitigate the impact of endocrine dysfunctions.
  • This review offers a clinical framework for managing TDT-related growth and endocrine challenges.

Keywords:
Endocrine dysfunctionGrowth and developmentIron overloadRecombinant human growth hormoneThalassemia

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