Liver transplantation for pediatric metabolic disease.
Liver transplantation (LTx) was initially developed as a therapy for liver diseases known to be associated with a high risk of near-term mortality but is based upon a different set of paradigms for inborn metabolic diseases. As overall outcomes for the procedure have improved, LTx has evolved into an attractive approach for a growing number of metabolic diseases in a variety of clinical situations. No longer simply life-saving, the procedure can lead to a better quality of life even if not all symptoms of the primary disorder are eliminated. Juggling the risk-benefit ratio thus has become more complicated as the list of potential disorders amenable to treatment with LTx has increased. This review summarizes presentations from a recent conference on metabolic liver transplantation held at the Children's Hospital of Pittsburgh of UPMC on the role of liver or hepatocyte transplantation in the treatment of metabolic liver disease.
Hillman Center for Pediatric Transplantation, Children's Hospital of Pittsburgh of UPMC, Faculty Pavilion, 4401 Penn Avenue, Pittsburgh, PA 15224, USA; University of Pittsburgh School of Medicine/UPMC Department of Surgery, Thomas E. Starzl Transplantation Institute, E1540 Biomedical Science Tower (BST), 200 Lothrop Street, Pittsburgh, PA 15261, USA. Electronic address: George.Mazariegos@chp.edu.
George
G
Mazariegos
Division of Pediatric Gastroenterology, Hepatology and Nutrition, Children's Hospital of Pittsburgh of UPMC, Rangos Research Center, 4401 Penn Avenue, 7th Floor, Pittsburgh, PA 15224, USA. Electronic address: Benjamin.Shneider@chp.edu.
Benjamin
B
Shneider
Department of Pediatrics, Northwestern University Feinberg School of Medicine/Ann & Robert H. Lurie Children's Hospital of Chicago, Box MC 59, 225 E Chicago Avenue, Chicago, IL 60611, USA. Electronic address: b-burton1@northwestern.edu.
Barbara
B
Burton
Hillman Center for Pediatric Transplantation, Children's Hospital of Pittsburgh of UPMC, Faculty Pavilion, 4401 Penn Avenue, Pittsburgh, PA 15224, USA; University of Pittsburgh School of Medicine/UPMC Department of Surgery, Thomas E. Starzl Transplantation Institute, E1540 Biomedical Science Tower (BST), 200 Lothrop Street, Pittsburgh, PA 15261, USA; McGowan Institute for Regenerative Medicine, University of Pittsburgh, Pittsburgh, PA, USA. Electronic address: Ira.Fox@chp.edu.
Ira J
IJ
Fox
King's College Hospital, Paediatric Liver Center, London, UK. Electronic address: nedim.hadzic@kcl.ac.uk.
Nedim
N
Hadzic
Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, DUMC 103856, 595 Lasalle Street, GSRB 1, 4th Floor, Room 4010, Durham, NC 27710, USA. Electronic address: priya.kishnani@duke.edu.
Priya
P
Kishnani
Franklin and Marshall College, Clinic for Special Children, 535 Bunker Hill Road, Strasburg, PA 17579, USA. Electronic address: dhmorton@clinicforspecialchildren.org.
D Holmes
DH
Morton
Department of Pediatrics, Paul C. Gaffney Diagnostic Referral Service, University of Pittsburgh School of Medicine, Children's Hospital of Pittsburgh of UPMC, 4401 Penn Avenue, Suite Floor 3, Pittsburgh, PA 15224, USA. Electronic address: mcintiresc@upmc.edu.
Sara
S
McIntire
Department of Pediatrics, University of Colorado School of Medicine and Children's Hospital Colorado, Section of Gastroenterology, Hepatology and Nutrition, 13123 E. 16th Avenue, B290, Aurora, CO 80045-7106, USA. Electronic address: ronald.sokol@childrenscolorado.org.
Ronald J
RJ
Sokol
Division of Genetics and Metabolism, George Washington University, Children's National Medical Center, Center for Genetic Medicine Research (CGMR), 111 Michigan Avenue, NW, Washington, DC 20010-2970, USA. Electronic address: MSummar@childrensnational.org.
Marshall
M
Summar
Department of Psychology, Washington University, Psychology Building, Room 221, Campus Box 1125, St. Louis, MO 63130-4899, USA. Electronic address: dawhite@wustl.edu.
Desiree
D
White
Division of Plastic and Reconstructive Surgery, Mount Sinai Hospital, 5 East 98th Street, 15th Floor, New York, NY 10029, USA. Electronic address: vincent.chavanon@mountsinai.org.
Vincent
V
Chavanon
Department of Pediatrics, University of Pittsburgh School of Medicine, 4401 Penn Avenue, Pittsburgh, PA, USA; Department of Human Genetics, University of Pittsburgh Graduate School of Public Health, Pittsburgh, PA 15261, USA; Division of Medical Genetics, Children's Hospital of Pittsburgh of UPMC, Rangos Research Center, 4401 Penn Avenue, Pittsburgh, PA 15224, USA. Electronic address: gerard.vockley@chp.edu.
Jerry
J
Vockley
D002648:Child; D003226:Congresses as Topic; D022781:Hepatocytes / Q000637:transplantation; D006801:Humans; D016031:Liver Transplantation; D008659:Metabolic Diseases / Q000628:therapy*; D016896:Treatment Outcome
Amino aciduria; Hepatocyte transplant; Inborn errors of metabolism; Liver failure; Liver transplant; Organic acidemia