A Multi-Center Group to Study Acute Liver Failure in Children

University of Pittsburgh

Status: Completed

Conditions: Hepatic Encephalopathy; Acute Liver Failure

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT00986648

U01DK072146 (U.S. NIH Grant/Contract)

2U01DK072146-06 (U.S. NIH Grant/Contract)

1U01DK072146-01

Details and patient eligibility

About

The PALF study group began with 20 sites and now continues with 12 sites (11 in the United States and 1 in Canada) in the new funding period. The primary objective of the Pediatric Acute Liver Failure (PALF) study is to collect, maintain, analyze, and report clinical, epidemiological, and outcome data in children with ALF, including information derived from biospecimens.

Full description

The PALF study group will collect clinical, epidemiological and outcome data on children with ALF. This information will be used to develop methods to predict whether a child will recover from the illness without the need for a liver transplant or other life-saving procedure. We believe the methods to predict survival will vary with different patient age groups, but that diagnosis, multi-system organ failure, degree of encephalopathy and level of coagulopathy will be important regardless of patient age. Biological samples, such as blood and liver tissue, will provide opportunities to identify subgroups of patients who have unique treatment requirements and outcomes. In addition, we hope to identify unrecognized mechanisms of liver injury resulting in ALF in children. Eligible study participants will be invited to participate in neurocognitive testing. Since patients that develop acute liver failure experience varying levels of hepatic encephalopathy and cerebral edema, we suspect that there may be residual sub-clinical neurological injury that compromises long-term neurocognitive function. Detailed neurocognitive testing has never been performed in a cohort of children that survive acute liver failure and this study seeks to close that information gap by defining the spectrum of neurocognitive outcomes in this population.

Enrollment: 158 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Written informed consent/assent

• Birth through 17 years of age

• Biochemical evidence of acute liver injury

• Coagulopathy not corrected by vitamin K (or other intervention intended to correct coagulopathy)

  • The presence of encephalopathy (ENC) is required if the INR is at least 1.5 and less than 2.0
  • If INR is at least 2.0, the presence of ENC is not required

Exclusion criteria

• Known chronic underlying liver disease
• Multi-organ system failure following heart surgery or ECMO
• Solid organ or bone marrow transplantation
• Acute trauma
• Previously enrolled in the PALF Cohort Study
• Other severe illness, condition, or other reason in the opinion of the investigator that would make the patient unsuitable for the study