Objective To explore the therapeutic benefit of liver transplantation （LT）on progressive familial intrahepatic cholestasis （PFIC）. Methods The clinical data of 5 cases with PFIC who underwent liver transplantation at Huashan Hospital affiliated to Fudan University from October 2015 to April 2018，were analyzed retrospectively. There are PFIC Ⅱ patients in 3 cases and 2 PFIC Ⅲ patients in 2 cases ；there were 3 cases male and 2 cases female with a median age of 34 months（9 ～ 102 months）；living donor liver transplantation with left lateral segment patient in 4 cases and classical orthotopic liver transplantation in 1 case. The clinical data， including pre-operative basic features， surgical situation， postoperative management and outcomes were analyzed to evaluate the curative effect of LT. Results All the surgeries achieved success and the liver function recovered smoothly. After the operation，1 case was complicated with lymphatic leakage and 1 case with intrahepatic bile duct dilation. One case was infected by the hepatitis B virus， cytomegalovirus and EB virus. All the children and grafts were alive after median follow up duration of 23 months（4 ～ 34 months）. The clinical manifestations of jaundice and pruritus were gradually vanished. No patient suffered from apparent acute or chronic rejection and vascular complications Conclusions LT is the definitive treatment for PFIC as it could correct the genetic defect. 

目的 探讨肝移植术对进行性家族性肝内胆汁淤积症（processive familial intrahepatic cholestasis，PFIC）的临床疗效。方法 回顾性分析 2015 年 10 月— 2018 年 4 月在复旦大学附属华山医院接 受肝移植术的 5 例 PFIC 患者的临床资料。其中 PFIC Ⅱ型患者 3 例，Ⅲ型患者 2 例 ；男性 3 例，女性 2 例， 中位年龄为 34 个月（9 ～ 102 个月）；左外叶活体肝移植术 4 例，经典原位肝移植术 1 例。分析患者术前基 本特征、术中情况、术后管理及预后资料，评估肝移植疗效。结果 所有手术顺利完成，患者肝功能恢复正常。 术后 1 例患者发生淋巴瘘，1 例患者出现肝内胆管扩张，1 例患者感染乙型肝炎病毒、巨细胞病毒及 EB 病 毒。经中位时间为 23 个月（4 ～ 34 个月）的随访，所有患者及移植肝正常存活，黄疸、瘙痒等症状消退。 无患者发生明显急、慢性排斥反应及血管并发症。结论 肝移植能改变 PFIC 基因缺陷，是彻底治愈疾病的 有效方法。