A simple and cheap regimen of Vitamin E is an effective method to treat a severe form of fatty liver disease in children, according to a recent clinical trial led by Joel Lavine, MD, PhD, director of the Division of Gastroenterology, Hepatology and Nutrition in Pediatrics. The results of the clinical trial were published in the April 27 issue of JAMA.
About 10 percent of children have some form of fatty liver disease, a condition that stems from excess fat stored in the liver. In its mildest form, the liver appears to tolerate the extra fat, but in its most severe form – called nonalcoholic steatohepatitis, or NASH – the excess fat causes inflammation, scarring and liver damage. People with NASH are more likely to develop liver cirrhosis, liver failure or liver cancer in the future.
NASH is most often caused by obesity, but aside from losing weight, there are no other treatments.
When Lavine first started investigating NASH 17 years ago, he hypothesized that anti-oxidants might help heal the liver because fat appears to be a substrate for oxidants.
In the 1990s, Lavine conducted the first clinical trial of Vitamin E and NASH – a small pilot study with 11 patients – and found that Vitamin E reduced the levels of a serum enzyme, called ALT, that is typically elevated in children with NASH.
The JAMA study put Vitamin E to a stronger test. In a placebo-controlled, multi-center trial, 173 children with fatty liver disease were randomized to receive Vitamin E, metformin, or a placebo. Almost all the children were obese and 121 had NASH, which must be diagnosed by liver biopsy.
Lavine and his colleagues found that 96 weeks of daily Vitamin E reduced the level of ALT, but ALT levels also declined in the placebo group. The difference between the two groups was not statistically significant.
But more important, says Lavine, were the changes seen in the livers of children with NASH. In these children, Vitamin E reduced the signs of injury in the liver, particularly the massive swelling of the liver’s hepatocytes that is the major feature of NASH.
Among children diagnosed with NASH at the beginning of the study, liver biopsies showed that 58 percent no longer had the disease by the end of treatment, compared to 28% improvement in the placebo group. This was statistically significant.
Metformin, a drug used to treat type 2 diabetes in children, did not reduce ALT levels or eliminate NASH compared to placebo.
“The take home message is that doctors should try Vitamin E if their patients with NASH do not succeed with exercise and diet,” Lavine says. “If it were my child, I’d certainly prescribe Vitamin E for NASH.”
Diagnosing NASH raises risk-benefit issues, however. The risks of performing a liver biopsy – mainly internal bleeding – may outweigh the benefits of therapy.
Lavine is now looking for non-invasive markers in blood that could diagnose NASH or predict who will be responsive to therapy.
Finally, Lavine cautions that parents should not give Vitamin E to their children unless under doctor’s orders. Only some formulations of Vitamin E are effective, and though Vitamin E is relatively safe, there are risks with any treatment.
The trial was conducted by the NASH Clinical Research Network, a group of academic researchers supported by the National Institutes of Health.
To make an appointment, or to learn more about studies enrolling patients with fatty liver disease, email firstname.lastname@example.org, or call 212 305-NASH.