title: Wilson's disease with severe hepatic insufficiency: beneficial effects of early administration of D-penicillamine.
abstract: BACKGROUND
  Wilson's disease, heralded by severe hepatic insufficiency, is a rare disorder for which emergency liver transplantation is considered to be the only effective therapy.
  
  
  OBJECTIVE
  To report the features of Wilson's disease with severe hepatic insufficiency in a series of 17 patients and, during the second period of the study, to assess the efficacy of a policy consisting of early administration of D-penicillamine.
  
  
  METHODS
  Seventeen consecutive patients with Wilson's disease were studied. During the first period of the study (up to 1979), none of the patients received D-penicillamine. During the second period (after 1979), all patients without encephalopathy at admission received D-penicillamine.
  
  
  RESULTS
  The four patients observed during the first period who did not have encephalopathy at admission and did not receive D-penicillamine progressed to encephalopathy and died. Among the 13 consecutive patients observed during the second period, two patients with encephalopathy at admission did not receive D-penicillamine and were transplanted. The 11 remaining patients all received D-penicillamine. Ten of these patients survived without the need for transplantation and returned to compensated liver disease without liver insufficiency. In one patient, liver insufficiency progressed and transplantation had to be performed.
  
  
  CONCLUSIONS
  In most patients with Wilson's disease heralded by severe hepatic insufficiency and without encephalopathy at admission, early administration of D-penicillamine was associated with survival without transplantation. These results suggest the importance of early diagnosis of this form of Wilson's disease before the onset of encephalopathy, and favour early administration of D-penicillamine which could avoid the need for transplantation in most cases.
authors.affiliation: Service d'Hépatologie, Hôpital Beaujon, Clichy, France. francois.durand@bjn.ap-hop-paris.fr
authors.forename: F
authors.initials: F
authors.lastname: Durand
authors.forename: J
authors.initials: J
authors.lastname: Bernuau
authors.forename: E
authors.initials: E
authors.lastname: Giostra
authors.forename: G
authors.initials: G
authors.lastname: Mentha
authors.forename: D
authors.initials: D
authors.lastname: Shouval
authors.forename: C
authors.initials: C
authors.lastname: Degott
authors.forename: J P
authors.initials: JP
authors.lastname: Benhamou
authors.forename: D
authors.initials: D
authors.lastname: Valla
mesh_terms: D058186:Acute Kidney Injury / Q000209:etiology / Q000628:therapy; D000293:Adolescent; D000328:Adult; D002614:Chelating Agents / Q000627:therapeutic use*; D002648:Child; D005260:Female; D006501:Hepatic Encephalopathy / Q000209:etiology; D006527:Hepatolenticular Degeneration / Q000150:complications / Q000175:diagnosis / Q000628:therapy*; D006801:Humans; D016031:Liver Transplantation / Q000379:methods; D008297:Male; D018579:Patient Selection; D010396:Penicillamine / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D002614:Chelating Agents; D010396:Penicillamine