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

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.

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Journal
European Journal of Pediatrics
Published
2026-06-27
DOI
https://doi.org/10.1007/s00431-026-07170-4
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
Field-Weighted Citation Impact
0.00

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article

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

Engy A. Mogahed, Noha Adel Yassin, Mona Kaddah, Marwa Abd Elhady et al.
European Journal of Pediatrics
Hemoglobinopathies and Related Disorders
article

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

Engy A. Mogahed, Noha Adel Yassin, Mona Kaddah, Marwa Abd Elhady, Ahmed Marzouk
article en

Abstract

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.

European Journal of PediatricsVol. 185(7)
Cairo University (EG)
Cairo University
No poverty
Openalex Percentile: Top 7%
Hemoglobinopathies and Related Disorders
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