Conditions: Parenteral Nutrition Associated Liver Disease PNALD Cholestasis
Keywords: PN parenteral nutrition fat emulsions omega-6 fatty acid liver disease fatty acid deficiency
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 72
Healthy volunteers: no

Brief summary:
The purpose of this study is to determine if established parenteral nutrition (PN) associated liver disease can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil as measured by normalization of serum levels of hepatic enzymes and bilirubin.

Detailed description:
The primary objective of this study is to evaluate the need for and time to small bowel transplantation or liver transplantation. This will be calculated as both the age at time of primary transplant as well as the length of time from initial evaluation to transplant.Secondary objectives are to determine if there are improvements in clinical measures associated with established parenteral nutrition- associated liver disease (PNALD). These will be determined by measurement of total and direct bilirubin levels, platelet count, serum albumin, and changes in both length and weight growth curves.

Arms:
arm 1: This study will be a prospective, non-randomized, open-label study of Omegaven™ for provision of parenteral lipid calories. The study cohort, receiving the parenteral nutrition (PN) lipid at 1g/kg/day, will be compared to historical controls at University of Nebraska Medical Center (UNMC) where parenteral lipid calories were provided exclusively through soybean-based formulations. The study is planned to enroll 100 patients. The Intestinal Rehabilitation Program at UNMC sees between 20 and 30 new pediatric patients per year, with almost all being PN-dependent and over 75% presenting with a bilirubin ≥ 2mg/dL. Based on these calculations, we estimate 4-5 years to enroll 100 patients.

Interventions: intervention 1: Omegaven™
Intervention descriptions: intervention 1: 10% Omegaven™, 50 or 100 mL bottle; 1gram/kg/day and is infused over 12-24 hours.

Protocol document:
Prot_SAP_000.pdf: