Treatment of progressive familial intrahepatic cholestasis: liver transplantation or partial external biliary diversion.
Treatment of progressive familial intrahepatic cholestasis: liver transplantation or partial external biliary diversion.
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DOI：
10.1034/j.1399-3046.1999.00046.x
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发表时间：
1999-08-01
影响因子：
1.3
通讯作者：
Revillon, Y
中科院分区：
文献类型：
--
作者：
Ismail, H;Kalicinski, P;Revillon, Y
进行性肝内家族性胆汁淤积 (PFIC)，以前称为拜勒氏病，是一种儿童出现严重胆汁淤积并进展为胆汁性肝硬化和慢性肝功能衰竭的综合征，通常发生在生命的最初十年。临床特征包括黄疸、肝肿大、脾肿大、生长迟缓和严重瘙痒。实验室检查显示胆红素和胆汁酸升高，但血清γ-谷氨酰转肽酶或胆固醇未升高。这项研究的目的是评估我们在 PFIC 儿童中使用的药物治疗以及两种手术治疗的经验，特别是部分胆道外流改道 (PEBD) 作为肝移植 (OLTx) 的替代治疗方法。 1979 年至 1998 年间，我们治疗了 46 名 PFIC 儿童（27 名男孩和 19 名女孩），年龄为 10 个月至 19 岁（在本研究时）。 39 名患者接受了熊去氧胆酸 (UDCA) 治疗，治疗时间为 6 至 82 个月。 16 名患者接受了 PEBD（胆囊空肠皮肤造口术），8 名儿童接受了 OLTx（包括 1 名 PEBD 失败后的儿童）。对临床病程和选定的实验室检查（胆红素、ASPAT、ALAT、胆汁酸）和组织病理学检查进行回顾性分析。通过治疗类型对临床症状、实验室检查、肝硬化和肝衰竭进展以及身体发育和生存的影响来评估治疗结果。从长远来看，药物治疗对四名患者（10%）有效，导致临床和生化正常化。 PFIC、PEBD 和 OLTx 的两种手术治疗方法的成功率均为 80%，因此应作为补充疗法。对于肝硬化之前的患者，PEBD应该是首选治疗。患有肝硬化或 PEBD 无效的患者应有资格接受 OLTx。通过这种策略，大多数 PIFC 儿童都可以治愈。
Progressive intrahepatic familial cholestasis (PFIC), previously called Byler's disease, is a syndrome in which children develop severe cholestasis progressing to biliary cirrhosis and chronic liver failure, usually during the first decade of life. Clinical features include jaundice, hepatomegaly, splenomegaly, growth retardation and severe pruritus. Laboratory tests demonstrate elevated bilirubin and bile acids, without an increase in serum gamma-glutamyl-transpeptidase or cholesterol. This study was performed to evaluate our experience with medical therapy as well as two types of surgical treatment used in children with PFIC, particularly partial external biliary diversion (PEBD) as an alternative method of therapy to liver transplantation (OLTx). Between 1979 and 1998 we have treated 46 children with PFIC (27 boys and 19 girls), aged 10 months to 19 yr (at the time of this study). Medical treatment with ursodeoxycholic (UDCA) was used in 39 patients for the period between 6 and 82 months. PEBD (cholecysto-jejuno-cutaneostomy) was performed in 16 patients, OLTx in eight children (including one after unsuccessful PEBD). Retrospective analysis of the clinical course and selected laboratory tests (bilirubin, ASPAT, ALAT, bile acids), and histopathological examinations were performed. Results of treatment were assessed by means of influence of the type of treatment on clinical symptoms, laboratory tests, progress of liver cirrhosis and hepatic failure, as well as physical development and survival. Medical therapy was effective in the long term in four (10%) of the patients resulting in clinical and biochemical normalization. Both surgical methods of therapy of PFIC, PEBD and OLTx, resulted in an 80% success rate and therefore should be used as complementary therapies. In patients before established liver cirrhosis, PEBD should be the first choice of treatment. Patients presenting with cirrhosis or after ineffective PEBD should qualify for OLTx. With this strategy most children with PIFC can be cured.