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.<Table Float="No" ID="Taba"> <tgroup align="left" cols="2"> <colspec align="left" colname="c1" colnum="1" /> <colspec align="left" colname="c2" colnum="2" /> <tbody> <row> <entry nameend="c2" namest="c1"> 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. </entry> </row> <row> <entry nameend="c2" namest="c1"> 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. </entry> </row> </tbody> </tgroup> </Table>

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

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2026年6月27日

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Marwa Abd Elhady,
Mona Kaddah,
Ahmed Marzouk,
Engy Adel Mogahed,
Noha Adel Yassin

摘要

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.