Growth Profile and Endocrine Dysfunction in Children Aged 2-10 Years With Transfusion-Dependent β-Thalassemia Major

Background Advances in regular blood transfusion and iron chelation therapy have markedly improved survival in children with transfusion-dependent β-thalassemia major (TDT). As survival has increased, growth impairment and endocrine disorders related to chronic iron overload have become increasingly important causes of long-term morbidity. This study examined the prevalence of endocrine abnormalities and their relationship with growth parameters and iron overload in children with TDT. Methods A hospital-based cross-sectional study was conducted among 150 children aged 2-10 years with TDT attending a tertiary care center in northern India. Clinical details, anthropometric measurements, serum ferritin concentrations, and endocrine investigations, including thyroid function, glycemic profile, gonadal hormones, growth hormone, cortisol, calcium, parathyroid hormone, and vitamin D levels, were recorded. Correlation analyses and multivariable logistic regression were used to explore factors associated with endocrine dysfunction. Results Overall, 78.7% of children had at least one endocrine abnormality. Thyroid dysfunction (63.3%) and vitamin D deficiency (62.0%) were the most frequent disorders, followed by gonadal dysfunction (46.7%), serum cortisol abnormalities (41.3%), and low random growth hormone levels (38.7%). Older children had a greater burden of endocrine dysfunction than younger children (all p<0.001). Serum ferritin showed positive correlations with thyroid-stimulating hormone, follicle-stimulating hormone, luteinizing hormone, and glycated hemoglobin, while negative correlations were observed with height-for-age Z-score, weight-for-age Z-score, free thyroxine, and serum cortisol (all p<0.001). Increasing age (adjusted OR: 1.32), higher serum ferritin levels (adjusted OR: 1.12 per 100 ng/mL increase), longer duration of transfusion therapy, and poor adherence to chelation therapy independently predicted endocrine dysfunction. Conclusions Endocrine abnormalities are common even in young children with TDT and are closely associated with increasing iron burden. Early endocrine evaluation, effective transfusion practices, good adherence to iron chelation therapy, and regular monitoring of iron overload may help reduce endocrine complications and support better growth outcomes.