Hang Chenyue, Jin Yijie, Luo Yi, Feng Mingxuan, Zhou Tao, Zhu Jianjun, Zhang Jianjun, Liu Yuan, Xia Qiang
Department of Liver Surgery, Ren Ji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200127, China.
Shanghai Engineering Research Center of Transplantation and Immunology, Shanghai 200127, China.
J Clin Med. 2022 Aug 11;11(16):4684. doi: 10.3390/jcm11164684.
We analyzed the long-term survival rate and development of progressive familial intrahepatic cholestasis (PFIC) patients after liver transplantation (LT). From October 2007 to May 2019, 41 patients were diagnosed as PFIC (type I-III) and received LT in Ren Ji Hospital due to end-stage liver diseases. The median age at LT was 2.93 years, with 75.6% of patients receiving living donor liver transplantation (LDLT). The 5- and 10-year patient survival rates after LT were 92.7% and 92.7%, respectively, and no difference was found among the three subtypes of PFIC. Two PFIC type II patients received re-transplantation due to vascular complications. Liver function and bile acid metabolism returned to normal levels in all living recipients. Catch-up growth was recorded as the height and weight Z scores increased from -2.53 and -1.54 to -0.55 and -0.27 with a median follow-up time of 5.55 years. Improved psychomotor ability and age-appropriate study ability was also observed. A total of 72.4% of school-aged recipients exhibited average academic performance. Diarrhea was reported in all PFIC type I recipients but resolved after resin absorptive treatment. However, allograft steatosis occurred in one PFIC type I patient and exhibited a "remission-relapse circle" under the treatment of cholestyramine. In conclusion, LT is an effective treatment for end-stage PFIC patients with encouraging long-term survival rate and development. However, allograft steatosis should be closely monitored in PFIC type I patients even if diarrhea has been well treated.
我们分析了肝移植（LT）后进行性家族性肝内胆汁淤积症（PFIC）患者的长期生存率和病情发展情况。2007年10月至2019年5月，41例患者被诊断为PFIC（I - III型），因终末期肝病在仁济医院接受了肝移植。肝移植时的中位年龄为2.93岁，75.6%的患者接受了活体供肝肝移植（LDLT）。肝移植后患者的5年和10年生存率分别为92.7%，PFIC的三种亚型之间未发现差异。两名PFIC II型患者因血管并发症接受了再次移植。所有存活受者的肝功能和胆汁酸代谢均恢复至正常水平。随着中位随访时间5.55年，身高和体重Z评分从 - 2.53和 - 1.54增加到 - 0.55和 - 0.27，实现了追赶生长。还观察到精神运动能力和适龄学习能力有所改善。72.4%的学龄受者学业成绩表现为中等。所有PFIC I型受者均出现腹泻，但经树脂吸附治疗后缓解。然而，一名PFIC I型患者发生了移植肝脂肪变性，在考来烯胺治疗下呈现“缓解 - 复发循环”。总之，肝移植是治疗终末期PFIC患者的有效方法，长期生存率和病情发展情况令人鼓舞。然而，即使腹泻已得到良好治疗，PFIC I型患者仍应密切监测移植肝脂肪变性情况。
