title: Hepatocyte transplantation for inherited factor VII deficiency.
abstract: Hepatocyte transplantation has been investigated in patients with liver-based metabolic disorders and acute liver failure. We report the first use of hepatocyte transplantation in two brothers with severe inherited coagulation factor VII deficiency. Patient 1 received a total of 1.09x10(9) cryopreserved hepatocytes, and patient received 2.18x10(9) fresh and cryopreserved hepatocytes through a Hickman line inserted in the inferior mesenteric vein. Infusion of isolated human hepatocytes improved the coagulation defect and markedly decreased the requirement for exogenous recombinant factor VII (rFVIIa) to approximately 20% of that before cell transplantation. In both patients, episodes of line sepsis were associated with an increase in rFVIIa requirement. Six months posthepatocyte transplantation, higher rFVIIa doses were required, suggesting loss of transplanted hepatocyte function. Because of increasing problems with venous access and long-term uncertainty of the efficacy of hepatocyte transplantation, orthotopic liver transplantation was performed successfully in both cases.
authors.affiliation: Institute of Liver Studies, Guy's, King's, and St. Thomas' School of Medicine, and King's College Hospital, Denmark Hill, London, UK. anil.dhawan@kcl.ac.uk
authors.forename: Anil
authors.initials: A
authors.lastname: Dhawan
authors.forename: Ragai R
authors.initials: RR
authors.lastname: Mitry
authors.forename: Robin D
authors.initials: RD
authors.lastname: Hughes
authors.forename: Sharon
authors.initials: S
authors.lastname: Lehec
authors.forename: Claire
authors.initials: C
authors.lastname: Terry
authors.forename: Sanjay
authors.initials: S
authors.lastname: Bansal
authors.forename: Rupen
authors.initials: R
authors.lastname: Arya
authors.forename: Jim J
authors.initials: JJ
authors.lastname: Wade
authors.forename: Anita
authors.initials: A
authors.lastname: Verma
authors.forename: Nigel D
authors.initials: ND
authors.lastname: Heaton
authors.forename: Mohamed
authors.initials: M
authors.lastname: Rela
authors.forename: Giorgina
authors.initials: G
authors.lastname: Mieli-Vergani
mesh_terms: D000293:Adolescent; D000328:Adult; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D005167:Factor VII / Q000008:administration & dosage / Q000627:therapeutic use; D005168:Factor VII Deficiency / Q000235:genetics* / Q000601:surgery*; D005260:Female; D022781:Hepatocytes / Q000637:transplantation*; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D016031:Liver Transplantation; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D005167:Factor VII