UCSF Researchers Shed Light on Silent Menace of Pediatric Obesity in Nationwide Study

Philip Rosenthal counseling one of his patients.
Philip Rosenthal counseling one of his patients.

By Kate Rauch

In efforts to address childhood obesity, much of the focus is on well-known risk factors such as diabetes, high blood pressure and heart disease. UCSF researchers though, as part of a major multi-site effort, are investigating a lesser understood but equally worrisome obesity-related illness: nonalcoholic steatohepatitis (NASH).

NASH is a chronic, degenerative liver disease marked by small deposits of fat in the liver tissue. A silent menace, because it can be symptomless until it is severe, leading to liver failure.


“Some patients, including children and adolescents, have required a liver transplant for this condition, so  it is serious and it is definitely increasing,”said Philip Rosenthal, MD, the Pediatric Principal Investigator of UCSF’s clinical site of the NASH Collaborative Research Network (CRN).

But there is no standard treatment. A recent study, published in the Journal of the American Medical Association (JAMA), focused on children and found that two fairly common treatments for the condition, vitamin E and metformin, showed no better outcome than placebo.

“While vitamin E and metformin have been used by physicians for treating NASH, neither drug clearly demonstrated a significant benefit over two years of therapy,” said  Rosenthal. "Without a standard treatment for NASH and with little research on the condition in children, pediatricians can't offer their patients clear choices. More research is needed, but this is a significant step in caring for patients with this condition.

The double-blind study followed 173 children at ten university medical centers for two years, randomly assigning them vitamin E, metformin or placebo. Neither the patients nor their doctors knew which they were receiving. Liver biopsies, the only way to clearly diagnose NASH, were compared from the beginning and end of the study period.

About 20 children were enrolled in the study through UCSF’s Clinical and Translational Science Institute (CTSI) with care provided by the Clinical Research Services, including diagnostic tests, specimen processing, and biopsy hospital stays. “This service made our work so much easier,” Rosenthal said.

The results are not likely to halt pediatricians from using vitamin E or metformin, since they have so few tools for treating NASH, Rosenthal said. But the large, well-executed project sets a precedent for further investigation. “The study was historic.”

It also reinforces a proven nugget of medical advice for NASH: “Diet and exercise remain currently the best therapy for this disorder in children.” 

CTSI at UCSF is a member of the national, NIH-funded CTSA network focusing on accelerating research to improve health. The CTSI Clinical Research Services are among a wide range of CTSI resources and services that support research at every stage.