Liver transplantation for pediatric metabolic disease

George Mazariegos; Benjamin Shneider; Barbara Burton; Ira J Fox; Nedim Hadzic; Priya Kishnani; D Holmes Morton; Sara McIntire; Ronald J Sokol; Marshall Summar; Desiree White; Vincent Chavanon; Jerry Vockley

doi:10.1016/j.ymgme.2014.01.006

Liver transplantation for pediatric metabolic disease

Mol Genet Metab. 2014 Apr;111(4):418-27. doi: 10.1016/j.ymgme.2014.01.006. Epub 2014 Jan 17.

Authors

Affiliations

¹ 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.
² 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.
³ 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.
⁴ 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.
⁵ King's College Hospital, Paediatric Liver Center, London, UK. Electronic address: nedim.hadzic@kcl.ac.uk.
⁶ 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.
⁷ Franklin and Marshall College, Clinic for Special Children, 535 Bunker Hill Road, Strasburg, PA 17579, USA. Electronic address: dhmorton@clinicforspecialchildren.org.
⁸ 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.
⁹ 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.
¹⁰ 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.
¹¹ Department of Psychology, Washington University, Psychology Building, Room 221, Campus Box 1125, St. Louis, MO 63130-4899, USA. Electronic address: dawhite@wustl.edu.
¹² 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.
¹³ 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.

PMID: 24495602
DOI: 10.1016/j.ymgme.2014.01.006

Abstract

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.

Keywords: Amino aciduria; Hepatocyte transplant; Inborn errors of metabolism; Liver failure; Liver transplant; Organic acidemia.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Child
Congresses as Topic
Hepatocytes / transplantation
Humans
Liver Transplantation*
Metabolic Diseases / therapy*
Treatment Outcome

Grants and funding

U01 DK 62453/DK/NIDDK NIH HHS/United States