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Though a regimen of steroids and immunosuppressants are well proven, alternative options may be necessary.
In the second segment of an interview with HCPLive during the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) 2023 Annual Meeting in San Diego this week, Mercedes Martinez, MD, medical director of pediatric abdominal organ transplantation as well as the Intestinal Transplant Program at the Center for Liver Disease and Abdominal Organ Transplantation at NewYork-Presbyterian, discussed first-line and second-line strategies for treating pediatric autoimmune hepatitis.
Primarily, cases will be treated first with steroids and the immunosuppressant therapy azathioprine—the latter of which decreases white blood cell counts, Martinez said. While most patients are expected to respond positively to this regimen, patients who present with low white blood cell counts often due to hyperinsulinism may not be ideal for azathioprine.
“Azathioprine is a drug that has been around for many years and I love it, because I know the side effects and I know how to look for those,” Martinez said. “Mainly, pancreatitis is a frequent side effect that has been attached to it. In patients that have a significant decrease of the white blood cell counts, then you are thinking, 'Maybe this is not the right medication for my patients.’”
Additionally, patients presenting with concurrent diabetes or obesity may not be ideal candidates for steroids.
“We need steroids, because autoimmune hepatitis is very responsive to a steroid, but also because azathioprine takes about 2-3 months to have full effects,” Martinez said. “There are other medications that can be used that work faster, like cyclosporine or tacrolimus. These are medications that are more familiar for doctors that treat patients with transplants, but actually that are very effective also for the treatment of autoimmune hepatitis.”
Second-line options may include budesonide, an alternative to prednisone that may result in lesser risk of side effects, such as steroids’ cosmetic effect on adolescent patients.
Martinez said cyclosporine or tacrolimus is associated with an approximate 30 - 50% increased response among patients who do experience response with azathioprine. However, the key second-line treatment option remains mycophenolate mofetil (MMF)—an alternative in instances when patients experience adverse events due to azathioprine.
“And you will see a good response—about 70 - 80% response,” Martinez said. “But if the indication to second-line therapy is a lack of response to azathioprine, MMF usually doesn't work, because they have the same mechanism of action.”