predicting transplant outcomes

Humans were once helpless to the unknown progressions of disease. But we have evolved in how we react to unpredictable events; we now have hope for survival.

We didn't know which children would suddenly develop Pediatric Acute Liver Failure (PALF)*, which can progress quickly and lead to devastating outcomes. Forty-five percent of children who develop PALF will either need a liver transplant or will not survive.

Before, when a child developed PALF, it was difficult to determine the most effective treatment. Because liver transplant can be a daunting, complicated and expensive process for all those involved, caregivers hesitated to choose this treatment if they weren't sure it was absolutely necessary. There was no simple way to predict which children would survive and which would need a liver transplant.

But now there is a way. Ronald Sokol, M.D.,** a Children's Colorado physician, and his colleagues at Children's Colorado developed a blood-test based mathematical formula (called the LIU score) 14 years ago to predict clinical outcomes for PALF.

Working with the Pediatric Acute Liver Failure Study Group, Dr. Sokol and his colleagues tested the formula on 400 pediatric patients throughout the country. In December 2012, Dr. Sokol and his colleagues published the results in The Journal of Pediatrics, which, according to Dr. Sokol, shows that the score "is very promising in terms of predicting who needs a liver transplant and who can survive without transplant."

The application of this formula has the potential to relieve hesitation for treatment and fast-track patients to the best treatment.

"It's a big advance now that we've validated the formula in the largest group of children with ALF ever studied," Dr. Sokol said. "Hopefully this will be adopted by others."

*A predictive test for ALF has been used in adults, but it does not work well in children.

**Ronald Sokol, M.D., Arnold Silverman, M.D., Endowed Chair in Digestive Health; Professor and Vice Chair of Pediatrics; Director, Colorado Clinical and Translational Sciences Institute; Chief, Section of Pediatric Gastroenterology, Hepatology and Nutrition and the Digestive Health Institute

Results, from The Journal of Pediatrics: "In this study, using a large multicenter multinational cohort of children with PALF, the LIU score was shown to be strongly predictive of transplant-free survival (c-index 0.81)."

"The LIU scoring system adds a quantitative predictive model based on these tests that may contribute objective criteria useful for the decision-making process regarding listing and timing for liver transplant."

Who needs a transplant?