By Wendy S. Meyer
Non-alcoholic fatty liver disease (NAFLD) is the most common chronic liver disease in children, largely due to the rising obesity epidemic. It is the second leading indication of liver transplantation in adults, and by 2030, experts project it will be the leading indication. Several drugs are in development but none are approved by the FDA. The only effective treatment is weight loss and exercise, which is very difficult for most patients to achieve or maintain. Unless researchers can develop an effective way to treat NAFLD, we will be facing a public health crisis in the near future.
However, pediatric gastroenterologist Shikha Sundaram, MD is about to publish a study on a novel therapy to help treat NAFLD in children.
“As I have been studying this disease, one of the things I have noticed is that some children, in addition to having fatty liver disease, also have sleep apnea,” says Sundaram. “Kids who have hypoxia [a result of sleep apnea] at night have more severe fatty liver disease that is also more progressive.”
Sundaram, with the help of the CCTSI’s pediatric Clinical and Translational Research Center at Children's Hospital Colorado, has been leading a clinical trial where children with NAFLD and sleep apnea use a CPAP machine at night. (CPAP stands for Continuous Positive Airway Pressure, and it is considered the standard of care for treating obstructive sleep apnea.)
“We have been following this cohort of 12 children since 2009. As we studied them, we found that in kids with sleep apnea who used CPAP to improve oxygenation, their liver disease got better. Their biomarkers also got better; their liver enzymes got better; inflammatory markers got better; their markers of oxidative injury got better,” Sundaram says.
Sundaram and her collaborators Ann Halbower, MD, and Jelena Klawitter, PhD, will soon publish the results of their study in The Journal of Pediatrics.
She calls her work a pilot study because the group of participants is so small. Sundaram is now in the midst of putting together an R01 grant proposal to the NIH to study a larger population of patients.
The dramatic results from the CPAP therapy in obese children with NAFLD use may or may not have an indication for adult patients. She cites mixed results of adults with NAFLD who use CPAP machines. “Kids’ physiology may be different and their ability to remodel their livers may be different,” she says. “Even if something didn’t work in adults, it might be worth trying in pediatrics.”
CCTSI Director Ron Sokol, MD, a co-author on this study, commented that although only 12 research participants were reported in this study, the results are so consistent that a major pediatric research journal accepted the paper for publication.
“Because the work is very novel, we dug in pretty deep, that makes it interesting,” Sundaram says. “I want people to know that there is a real possibility that this may be a treatment strategy in the future for a difficult- to almost-impossible-to-treat disease.”