Call to action: Kids need fatty liver research, diagnostics, treatment

WASHINGTON — With up to 10% of children worldwide diagnosed with nonalcoholic fatty liver disease and even nonalcoholic steatohepatitis, pediatric hepatologists face an unmet need for medical treatment and non-invasive diagnostics, according to a presenter at The Liver Meeting 2017.

“There’s more data coming out about transplantation for NASH in young adults and that is what keeps me up at night and has me worried about these kids,” Miriam Vos, MD, assistant professor of pediatrics at Emory University School of Medicine, and physician on staff at Children’s Healthcare of Atlanta, said during the Global Forum on NAFLD. “NASH is the most rapidly increasing indication for LT in young adults in the U.S.”

Vos showed that a recent study looked at 330 young adults transplanted for NASH and found that 30% (n = 100) of those transplanted were dead within 4 years. Of those who lived, 11% underwent a second transplant.

“Unfortunately, pediatric NAFLD is common across the world ... The prevalence is increasing. From the late 80s to now, it’s more than doubled” in the U.S., she said.

The progression of metabolic disease seems to be accelerated, Vos added. She explained while fatty liver can be inherited from a mother with gestational diabetes, the progression of a person from that to type 2 diabetes and further to cirrhosis has changed.

“What is worrisome is that now what we’re seeing is the type 2 diabetes is moving earlier. And the cirrhosis is moving earlier,” Vos said. “Genetics don’t explain the rapid acceleration of this phenotype in children.”

She told the story of a child in early adolescence who plays soccer yet has NASH with stage 2 fibrosis.

“I don’t know what else you do with a patient who already plays soccer and doesn’t drink sugary drinks. We definitely need medications,” Vos said.

Currently, it’s just “lifestyle, lifestyle, lifestyle,” she said, but that even focuses more on prevention and inhibiting progression to type 2 diabetes. Vos pointed to the gut-liver axis and the microbiome as a possible target for ongoing studies.

The microbiome “is an important area to keep studying in pediatric NAFLD,” she said and she reminded the audience that “the microbiome might differ greatly from region to region and country to country.”

Vos added that while imaging studies are progressing, pediatric patients do not respond the same way to noninvasive fibrosis scoring systems.

“The existing scores don’t work for children,” she said.

So more noninvasive measurements are needed and require validation, Vos said, along with more natural history studies on a global scale.

“We need to collaborate globally on these natural history studies. ... So we know what we’re talking about,” Vos said. “We need to understand differences by region. ... We need to validate all of these things in children.” – by Katrina Altersitz

 

Reference:

Vos M. Global Forum on NAFLD. Presented at: The Liver Meeting; Oct. 20-24, 2017; Washington, D.C.

 

Disclosure: Vos reports participating in advisory committees or review panels for Aegerion, Immuron and Target Pharmasolutions; consulting for Boehringer Ingelheim, Bristol Myers Squibb, Intercept and Shire; and receiving grant or research support from Resonance Health.

Editor's note: This item has been revised based on input from the presenter.