What is PALF?
Sudden onset in a previously healthy child
Yellowing of skin and eyes and loss of liver function
Change in behavior and level of consciousness
One third of children with PALF require liver transplantation
Pediatric Acute Liver Failure is a disorder of childhood that occurs suddenly in previously healthy children. Affected children develop loss of liver function over a period of days to weeks. The liver provides many vital functions for the body and loss of liver function is a life-threatening condition. There are several well-established causes of PALF which include viral liver infections, drug related liver injury, inherited defects in metabolism and autoimmune inflammatory conditions. However, in more than half the children that develop PALF a cause is never determined. Approximately one third of children affected with PALF require a liver transplantation and one in five children with PALF die.
How is PALF treated?
All patients with PALF require intensive support, typically in an intensive care unit designed for children. As the liver fails it can affect other organs such as the lungs, heart, brain and kidneys. If other organs are affected, children may require supportive care measures such as a breathing machine, dialysis or intravenous medications to improve their blood pressure. These supportive measures help protect the other organs as the liver tries to heal. In liver injury that is due to a treatable condition, such as autoimmune liver injury or some drug injuries, there may be specific medical treatments that will speed recovery of liver function. Frequently a liver biopsy must be performed to determine these diagnoses. In children who do not have an identifiable cause for their liver failure, all we can offer is supportive care.
There is no specific treatment for most cases
Supportive care in an intensive care unit is frequently required
Injury to other organs is common
What is TRIUMPH?
TRIUMPH is a clinical trial funded by the National Institutes of Health for children with acute liver failure
The trial tests two different therapies compared to supportive care alone
Many pediatric hospitals across the country are participating in the trial
TRIUMPH is a NIH sponsored clinical trial (clinicaltrials.gov) testing two different types of therapies as compared to supportive care alone for children who do not have a treatable cause for their liver failure. The trial is testing whether medications that dampen the body’s immune response to sudden, severe liver injury will allow the liver to recover more quickly. We believe that in some patients with PALF the body mounts an inflammatory response to the liver injury that is overactive and that the resulting inflammation may be a barrier to effective healing of the liver cells. This trial is offered at 20 large pediatric hospitals across the country and will be enrolling patients between 2022 and 2025.
My child has PALF,
what can I do?
The first step is to listen to your doctors and ask the right questions to get information that you need. You want to be sure your child will get the care they need as soon as they need it.
Does the hospital caring for you have a dedicated pediatric intensive care unit?
Do they have a pediatric liver transplant program? If not, could they facilitate a transfer to a center that can provide that care if your child needs it?
Do the doctors know the cause of the liver injury? What causes are they testing for?
Are they planning any specific therapies?
Children with acute liver failure can change rapidly, either for the better or for the worse. Be sure that you stay in frequent communication with the doctors who are making the treatment decisions so that you can have the most up to date information.
What should I do if I want my child to be considered for the TRIUMPH trial?
Link to the 20 medical centers listed on the Trial Sites page and ask your doctors if it would be possible for your child to be transferred to one of those centers to be evaluated as a candidate for the trial. Once you connect with a TRIUMPH trial site, the TRIUMPH team will guide you through the rest.
Ann & Robert H. Lurie Children’s Hospital of Chicago
Estella M. Alonso, MD, FAASLD