Assessment of hepatic fibrosis in Egyptian children and adolescents with beta thalassemia major: a single center study.

UNLABELLED: Liver fibrosis is a major complication in patients with transfusion-dependent beta-thalassemia, primarily driven by iron overload and suboptimal chelation therapy. This study aimed to assess the prevalence and severity of hepatic fibrosis in children with beta-thalassemia major using non-invasive modalities and to identify associated clinical and laboratory risk factors. This cross-sectional analytical study included 82 transfusion-dependent thalassemia patients. All participants underwent clinical evaluation, hematological and biochemical investigations, and liver fibrosis assessment using transient elastography (TE) (by FibroScan) and calculation of aspartate aminotransferase (AST)/platelet ratio index (APRI) and Fibrosis 4 score (FIB-4). Based on TE, 18 patients (22%) exhibited significant hepatic fibrosis (F2-F4). Both APRI and FIB-4 scores were significantly elevated in patients with significant fibrosis. Significant fibrosis was associated with older age (p = 0.019), longer disease duration (p = 0.015), heterogeneous liver echotexture on ultrasound (p < 0.001), poor adherence to chelation therapy (p < 0.001), and elevated transaminases and serum ferritin levels (p < 0.05).

CONCLUSION: Significant hepatic fibrosis was observed in approximately one-fifth of the studied cohort of Egyptian children with beta-thalassemia major. It is strongly associated with older age, longer disease duration, iron overload, and inadequate chelation. TE, APRI, and FIB-4 are effective for early detection of hepatic fibrosis in these patients.

WHAT IS KNOWN: • Hepatic fibrosis is a well-recognized complication of transfusion-dependent beta-thalassemia major, primarily resulting from chronic iron overload. • Liver biopsy remains the gold standard for fibrosis assessment; however, it is invasive. Non-invasive tools such as transient elastography, APRI, and FIB-4 have been increasingly used in adults, but limited data are available in pediatric populations, particularly in low- and middle-income countries.

WHAT IS NEW: • This study shows that significant hepatic fibrosis affects approximately one in five Egyptian children with beta-thalassemia major. • It supports the clinical utility of FibroScan, APRI, and FIB-4 for fibrosis assessment in pediatric patients and highlights poor chelation adherence, iron overload, and longer disease duration as key modifiable risk factors.