title: Plasma exchange and chelator therapy rescues acute liver failure in Wilson disease without liver transplantation.
abstract: OBJECTIVE
  Wilson disease (WD) in patients with a New Wilson Index (NWI) score ≥ 11 is fatal, and these patients are good candidates for liver transplantation (LT). However, plasma exchange and chelator therapy are indispensable and effective even for WD with a score ≥ 11. Moreover, continuous hemodiafiltration (CHDF) with these treatments is essential for acute liver failure (ALF) in WD with hepatic encephalopathy because CHDF can exclude toxic metabolites that may cause damage to the brain. Here, we describe four rescued patients presenting with ALF in WD and discuss the available treatment options.
  
  
  METHODS
  We have experienced 11 male and 8 female patients presenting with WD at the Department of Pediatrics, Kumamoto University Hospital between 1999 and 2014. A male and 4 female patients were diagnosed as WD with ALF using a combination of clinical findings and biochemical tests.
  
  
  RESULTS
  The NWI score was ≥ 11 in cases 1 to 3. Cases 1 and 2 with hepatic encephalopathy received plasma exchange, CHDF, coagulation factor replacement treatment (CFRT) and LT. Cases 3 and 4 without encephalopathy obtained stable status without LT by plasma exchange, blood infusion, and CFRT.
  
  
  CONCLUSIONS
  It is better to undergo LT for WD patients with a NWI score ≥ 11, however, there is a possibility of remission by plasma exchange and medical therapy even without LT. WD patients with a NWI score ≥ 11can be rescued by conservative therapy when the ALF of WD does not present with ALF and hepatic encephalopathy. Therefore, ALF with hepatic encephalopathy itself is an indication for LT in WD.
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Jun
authors.initials: J
authors.lastname: Kido
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Shirou
authors.initials: S
authors.lastname: Matsumoto
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Ken
authors.initials: K
authors.lastname: Momosaki
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.|Department of Transplantation and Pediatric Surgery, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Rieko
authors.initials: R
authors.lastname: Sakamoto
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Mitsubuchi
authors.affiliation: Department of Transplantation and Pediatric Surgery, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Yukihiro
authors.initials: Y
authors.lastname: Inomata
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Fumio
authors.initials: F
authors.lastname: Endo
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto City, Japan.
authors.forename: Kimitoshi
authors.initials: K
authors.lastname: Nakamura
keywords: Continuous hemodiafiltration; New Wilson Index; prothrombin time