Despite a lack of medical interventions to treat children with non-alcoholic fatty liver disease (NAFLD), physicians at the Cincinnati Children’s Steatohepatitis Center are optimistic about what the future holds for these patients. Last year, to enhance patient care, the multidisciplinary center added faculty; introduced new, non-invasive diagnostic technologies; and broadened its research efforts.

NAFLD is a growing problem in the United States, where almost one in 10 children has the disease. Among obese children, the number is one in three. About one-quarter to one-third of patients with NAFLD can develop non-alcoholic steatohepatitis (NASH), which can progress to cirrhosis and liver failure. NASH has become the second-most common indication for liver transplantation in adults.

“These diseases have their roots in childhood, and tackling them early, well before end-stage liver disease occurs, is critical,” says Stavra Xanthakos, MD, a pediatric gastroenterologist and director of the Steatohepatitis Center. “Weight loss is currently the only treatment for NAFLD, but it can be very difficult to achieve due to individual, societal and biological factors. However, promising new medications are being developed and tested in adults and children. Pharmaceutical companies are finally directing their attention to this problem, and we expect that approved medications will be available in the next few years.”

Since 2008, more than 850 patients have been seen in the Steatohepatitis Center at Cincinnati Children’s (more than 300 in FY17 alone), where they have received care from a multidisciplinary team of physicians, nurses, dietitians, psychologists and research coordinators. Two new faculty members – Marialena Mouzaki, MD, and Ana Catalina Arce-Clachar, MD – were recruited in 2017 to join Xanthakos and Kristin Bramlage, MD, on the steatohepatitis team. Mouzaki has been a pioneer in investigating the role of the intestinal microbiome in modulating the development and progression of NASH. Arce-Clachar will be expanding and improving services to Hispanic patients, who are disproportionately affected by an increasing burden of NAFLD.

The bariatric surgery option 
Recognizing that weight loss offers significant benefits for children with NAFLD, the steatohepatitis team works closely with colleagues in the Cincinnati Children’s Surgical Weight Loss Program for Teens to identify patients who may benefit from bariatric surgery. The surgical weight loss program has seen significant growth in recent years, with more than 300 bariatric surgeries performed in total since 2001. “Research indicates that only 2 percent of severely obese teenagers are able to lose weight and keep it off without surgery,” says Xanthakos, who serves as medical director of the Surgical Weight Loss Program for Teens. “Bariatric surgery is shown to benefit pediatric patients with NAFLD, and it is likely that patients with NASH would experience similar health benefits. We are currently leading a National Institutes of Health-funded study to establish to what degree bariatric surgery can reverse the effects of NASH and identify predictors of improvement.”

The center offers access to several other NIH-funded clinical studies designed to determine outcomes and identify better treatments for NAFLD and NASH, including one observational cohort study to track the natural progression of NAFLD. Cincinnati Children’s is one of only 11 pediatric sites nationwide in the NASH Clinical Research Network, which is funded by the National Institute of Diabetes and Digestive and Kidney Diseases.

Novel imaging technologies
In 2017, Cincinnati Children’s became one of the first pediatric centers in the country to offer both magnetic resonance elastography and advanced ultrasound technology, such as acoustic radiation force impulse elastography, to clinically assess NAFLD. These technologies can provide non-invasive monitoring of liver disease severity and progression. Xanthakos and her team work closely with pediatric radiologists at Cincinnati Children’s to optimize the use of these technologies in order to minimize the need for liver biopsies, and accelerate testing and identification of better therapies for children with NAFLD.