Evaluation of Omegaven™ Parenteral Nutrition in Patients With Total Parenteral Nutrition (TPN)-Induced Cholestasis

Phase 2

Completed

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.

Recruitment & Eligibility

Inclusion Criteria

Patients must be enrolled in the Intestinal Rehabilitation Program at the University of Nebraska Medical Center, AND:
Be unable to meet nutritional needs solely by enteral nutrition and be expected to require PN for at least another 30 days
Have clinical evidence of parenteral nutrition associated liver disease (PNALD) as defined as a direct bilirubin of 2 mg/dl or more. A liver biopsy is desirable but not necessary for treatment
Signed patient informed consent

Exclusion Criteria

Parent or guardian or child unwilling to provide consent or assent
Inability or unwillingness on the part of parent/guardian or child to follow clinical recommendations of the Intestinal Rehabilitation Program
Allergies or clinical conditions precluding safe use of Omegaven™

Arm && Interventions

Group	Intervention	Description
Omegaven™	Omegaven™	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.

Primary Outcome Measures

Name	Time	Method
Number of Patients With Progression to Small Bowel Transplantation.	Bi-weekly x4, then monthly until the completion of the study, for an overall average of 5.5 years follow-up for the cohort	Whether or not the subject had an intestine-containing transplant or not

Secondary Outcome Measures

Name	Time	Method
Number of Subjects With Reversal of Biochemical Cholestasis	weekly x 4, then bi-weekly x4, then monthly until the completion of the study, with an average follow-up of 5.5 years for the study cohort	Of patients starting with elevated total serum bilirubin, the number that progress to normalization of serum total bilirubin in routine bloodwork