Long-Term Results of Pediatric Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

Abstract

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.

Outcomes of pediatric liver transplantation for progressive familial intrahepatic cholestasis

“…This study presents the short‐ and long‐term outcomes of the largest single‐center cohort (60) of children who underwent LT by diving into each group depending on the types of PFIC. Similar large studies include those by Hang et al from Shanghai, China (41 patients) and Englert et al from Germany (33 children who underwent LT) 6,8 …”

“…More recently, ileal bile acid transporter (IBAT) inhibitors such as Odevixibat and Maralixibat interrupt enterohepatic circulation and have been shown to effectively reduce pruritus and possibly disease progression in PFIC. Refractoriness to the above‐mentioned therapies with a poor quality of life, and/or progression to liver failure have been considered indications for liver transplantation (LT) 6–8 …”

“…Refractoriness to the abovementioned therapies with a poor quality of life, and/or progression to liver failure have been considered indications for liver transplantation (LT). [6][7][8] Although the outcomes of pediatric LT (PLT) have been suboptimal for patients with Familial intrahepatic cholestasis 1 (FIC1) deficiency also known as PFIC1, the results of LT for other PFICs have been on par with those for other metabolic indications. 7,[9][10][11][12] While the short-term outcomes of LT for PFIC are well described, there remains a paucity of data on the long-term outcomes of these children.…”

Internal Ileal Diversion as Treatment for Progressive Familial Intrahepatic Cholestasis Type 1-Associated Graft Inflammation and Steatosis after Liver Transplantation

“…Some patients suffer from aggravated diarrhea, increasing liver graft steatosis and inflammation with progressive fibrosis, which can even lead to graft loss [ 18 , 19 , 20 , 21 , 22 , 23 ]. Oral cholestyramine as a bile acid sequestrant drug is reported to be sufficient in treating persisting diarrhea after LT [ 24 ]. Biliary drainage or rerouting after LT via SBD can amend steatosis and graft injury [ 21 , 25 , 26 , 27 ].…”

Progressive Familial Intrahepatic Cholestasis: A Descriptive Study in a Tertiary Care Center

“…Liver transplantation is a proven curative treatment for PFIC-related end-stage liver disease [ 19 , 20 , 22 , 26 ]. It is considered when patients have failed medical treatment, biliary diversion, or refractory pruritus.…”