About this trial
Thalassemia major is a hereditary hemoglobinopathy characterized by ineffective erythropoiesis and severe anemia, necessitating lifelong blood transfusions(1,2). Regular transfusions lead to iron overload, a primary driver of growth retardation in affected children. Iron accumulation in tissues like the pituitary and liver disrupts growth hormone secretion and insulin-like growth factor-1 production.
Eligibility criteria
Qualifiers
Children aged 1 to 18 years diagnosed with β-thalassemia major based on hemoglobin electrophoresis or high-performance liquid chromatography (HPLC).
Receiving regular blood transfusions as part of standard management at AUCH.
Attending the hematology unit for at least one year prior to enrollment.
Disqualifiers
• Children with other types of thalassemia.
Presence of congenital diseases, chronic illnesses other than thalassemia (e.g., malignancy, tuberculosis, chronic hepatitis, congenital heart disease, chronic renal failure, epilepsy, diabetes mellitus), or primary endocrinopathies.
Patients with other causes of short stature, such as hereditary bone dysplasia or systemic disorders.
Trial design
Treatments tested in this trial
- Non-interventional assessment of growth and risk factors in thalassemic children