小儿重型β地中海贫血肝活检63例临床分析

Clinical analysis of 63 liver biopsies in children with β-thalassemia major

目的分析小儿重型β地中海贫血(β-thalassemia,β地贫)的肝脏病理特点,探讨肝活检的方法与安全性。方法对1998年10月—2006年10月住院的63例重型β地贫患儿行肝活检,观察肝脏病理改变,并结合临床资料进行分析。结果共作66例次肝活检,均1次成功,成功率100%。肝活检显示肝组织内有不同程度铁沉积,中重度占42例(66.7%),铁沉积程度与血清铁蛋白(SF)水平呈明显正相关(r=0.70,P<0.01)。检出肝纤维化54例(85.7%),年龄(7.3±3.2)岁,SF在纤维化患儿中明显升高(P<0.01),但纤维化分期与SF、肝脏铁沉积程度无相关性(r=0.12、0.21,P均>0.05)。结论我国小儿重型β地贫存在肝脏铁含量重、肝纤维化普遍且发生年龄早的特点,与不规范治疗有关。小儿地贫肝活检安全、可行,能为治疗提供重要指导。

Objectives To study the pathological features of hepatic tissue in children with β-thalassemia major and evaluate the method and safety of liver biopsy. Methods Sixty-three thalassemic patients （median age 6.5 years, range 2.0-15.0 years） admitted from 1998 to 2006 underwent percutaneous liver biopsies. Histological changes including degree of iron deposition and hepatic fibrosis were studied and analyzed for clinical correlation. Results A total of 66 liver biopsies were successfully performed in 66 patients. Moderate and severe liver iron storage （Grade Ⅱ-Ⅲ） were observed in 42 patients （66.7%）. Liver iron storage was significantly related to serum ferritin level （r = 0.70, P 〈 0.01 ）. Fifty-four cases （85.7%） presented with fibrosis at median age of 7.3 years with significantly high level of serum ferritin （P 〈 0.01） . However, there were no correlations between the stages of liver fibrosis and ferritin iron （r = 0.12, P 〉 0.05） or liver iron deposition （r = 0.21, P 〉 0.05） . Conclusions Liver iron overloading and liver fibrosis were common in children with β-thalassemia major in China with liver fibrosis related to irregular treatment. Liver biopsy is a safe and feasible method for investigating β-thalassemia, the result can provide the important information for the treatment, assessment of iron storage and fibrosis.