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Pediatric Liver Transplantation for Metabolic Diseases: Analysis of Children's Hospital of Pittsburgh (CHP) and United Network for Organ Sharing (UNOS) Databases

A. Ganoza, B. Shneider, R. Sindhi, K. Soltys, G. Bond, G. Vockley, Q. Sun, G. Mazariegos.

Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA.

Meeting: 2015 American Transplant Congress

Abstract number: 49

Keywords: Liver transplantation, Metabolic disease, Pediatric

Session Information

Date: Sunday, May 3, 2015

Session Name: Concurrent Session: Liver: Pediatrics

Session Time: 2:15pm-3:45pm

 Presentation Time: 2:27pm-2:39pm

Location: Room 115-C

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Purpose: To analyze long term patient survival outcomes after primary LTx for metabolic disease in children <21 years at CHP (1981-2014) and the multi-center UNOS database (1988-2013) based on functional subcategories of inborn error of metabolism (IEM) and genetic metabolic disease (GMD).

Methods: Functional subcategories are:

IEM: 1) Liver disease curable by LTx, e.g. Crigler Najjar, 2) Systemic disease curable by LTx, without acute crisis, e.g. Tyrosinemia, 3) Systemic disease curable by LTx, with acute crisis e.g. MSUD, Urea cycle disorders, 4) Systemic potentially progressive disease, e.g. GSD, Methyl/Malonic Acidemia.

GMD: 1) Liver disease curable by LTx, e.g. familial cholestasis, 2) Systemic disease curable by LTx, e.g. alpha-1-anti-trypsin deficiency, 3) Systemic potentially progressive disease, e.g. cystic fibrosis.

Other variables analyzed were transplant era (era 1= pre-1997, era 2=1998-2005, and era 3= 2006-2013) recipient age and gender, and donor type-living vs. cadaveric.

Results: The distribution of IEM and GMD was 126:161 in CHP and 617:836 in the UNOS database. At CHP, 10 year patient survival for IEM disease categories 1 through 4 was 99.9%, 69.2%, 92.6% and 58.3% respectively, HR=1.67, p=0.003 and for GMD disease categories 1 through 3 was 73.9%, 76.7% and 65.8%, respectively, HR=1.13, p=0.072. Five-year patient survival after LTx for IEM at CHP was significantly better in the most recent era compared with the first era (97 vs 59%, p=0.001) and GMD (93.8 vs 72.6%, p=0.024). In backward stepwise logistic regression analysis of the CHP dataset, Transplant era emerged as the only significant predictor of patient survival outcomes after LTx for IEM (p=0.001) but not for GMD (p=0.085, NS). In the larger UNOS dataset, significant predictors of patient survival after LTx for IEM were Tx era (p=0.004,) disease category (p=0.004), age <1 year (p=0.005) and male gender (p=0.02), and for GMD were disease category (p=0.001) and age <1 year (p=0.001).

Conclusions: Progressive functional disease subcategories of IEM and GMD showed inferior patient survival in both datasets, which was significant in the larger UNOS dataset. In multivariate analyses, LTx in the most recent era remains the most significant determinant of improved patient survival in children with IEM but not GMD in single and multi-center datasets.

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To cite this abstract in AMA style:

Ganoza A, Shneider B, Sindhi R, Soltys K, Bond G, Vockley G, Sun Q, Mazariegos G. Pediatric Liver Transplantation for Metabolic Diseases: Analysis of Children's Hospital of Pittsburgh (CHP) and United Network for Organ Sharing (UNOS) Databases [abstract]. Am J Transplant. 2015; 15 (suppl 3). https://atcmeetingabstracts.com/abstract/pediatric-liver-transplantation-for-metabolic-diseases-analysis-of-childrens-hospital-of-pittsburgh-chp-and-united-network-for-organ-sharing-unos-databases/. Accessed January 19, 2021.

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