title: Progressive familial intrahepatic cholestasis: a single-center experience of living-donor liver transplantation during two decades in Japan.
abstract: BACKGROUND
  Progressive familial intrahepatic cholestasis (PFIC) results in liver cirrhosis. Therefore, some PFIC patients require liver transplantation (LT). Although three types of PFIC have been identified, their etiologies include unknown mechanisms.
  
  
  METHODS
  A total of 717 recipients who underwent living-donor LT (LDLT) at <20 yr old were enrolled in this study. Among these recipients, 14 PFIC recipients comprising 11 PFIC type 1 (PFIC1) and three PFIC type 2 (PFIC2) were evaluated.
  
  
  RESULTS
  Three of 11 PFIC1 recipients died, while all three PFIC2 recipients survived. Eight of 11 PFIC1 recipients showed steatosis after LDLT. Among the eight steatosis-positive PFIC1 recipients, seven showed severe steatosis and seven were complicated with steatohepatitis. Nine of 11 PFIC1 recipients showed fibrosis after LDLT, and eight of the nine fibrosis-positive PFIC1 recipients showed severe fibrosis. In contrast to the PFIC1 recipients, the PFIC2 recipients did not show any steatosis or fibrosis after LDLT.
  
  
  CONCLUSIONS
  The clinical courses and outcomes of PFIC1 recipients after LDLT are still not sufficient owing to steatosis/fibrosis, unlike the case for PFIC2 recipients. As PFIC1 patients will require LT during the long-term progression of the disease, further strategy improvements are required for PFIC1 patients.
authors.affiliation: Division of Hepato-Biliary-Pancreatic and Transplant Surgery, Department of Surgery, Kyoto University Hospital, Sakyo-ku, Kyoto, Japan. horit@kuhp.kyoto-u.ac.jp
authors.forename: Tomohide
authors.initials: T
authors.lastname: Hori
authors.forename: Hiroto
authors.initials: H
authors.lastname: Egawa
authors.forename: Yasutsugu
authors.initials: Y
authors.lastname: Takada
authors.forename: Mikiko
authors.initials: M
authors.lastname: Ueda
authors.forename: Fumitaka
authors.initials: F
authors.lastname: Oike
authors.forename: Yasuhiro
authors.initials: Y
authors.lastname: Ogura
authors.forename: Seisuke
authors.initials: S
authors.lastname: Sakamoto
authors.forename: Mureo
authors.initials: M
authors.lastname: Kasahara
authors.forename: Kohei
authors.initials: K
authors.lastname: Ogawa
authors.forename: Aya
authors.initials: A
authors.lastname: Miyagawa-Hayashino
authors.forename: Yukihide
authors.initials: Y
authors.lastname: Yonekawa
authors.forename: Tohru
authors.initials: T
authors.lastname: Yorifuji
authors.forename: Ken-Ichiro
authors.initials: K
authors.lastname: Watanabe
authors.forename: Hiraku
authors.initials: H
authors.lastname: Doi
authors.forename: Justin H
authors.initials: JH
authors.lastname: Nguyen
authors.forename: Feng
authors.initials: F
authors.lastname: Chen
authors.forename: Ann-Marie T
authors.initials: AM
authors.lastname: Baine
authors.forename: Lindsay B
authors.initials: LB
authors.lastname: Gardner
authors.forename: Shinji
authors.initials: S
authors.lastname: Uemoto
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002780:Cholestasis, Intrahepatic / Q000150:complications / Q000401:mortality* / Q000628:therapy*; D018450:Disease Progression; D005234:Fatty Liver / Q000209:etiology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007564:Japan; D008103:Liver Cirrhosis / Q000209:etiology; D016031:Liver Transplantation; D019520:Living Donors; D008297:Male; D011379:Prognosis; D015996:Survival Rate; D013997:Time Factors