In the second segment of the interview, HCP Live At the North American Society of Pediatric Gastroenterology, Liver Diseases, and Nutrition (NASPGHAN) 2023 Annual Meeting in San Diego this week, Mercedes Martinez, MD, medical director of the Center for Liver Diseases’ Pediatric Abdominal Organ Transplant and Intestinal Transplant Program, said: , NewYork-Presbyterian Abdominal Organ Transplant, discussed first- and second-line strategies for treating pediatric autoimmune hepatitis.
Primarily, cases are first treated with steroids and the immunosuppressive therapy azathioprine, the latter of which reduces white blood cell counts, Martinez said. Although most patients are expected to respond positively to this regimen, azathioprine may not be ideal for patients who often have low white blood cell counts due to hyperinsulinemia.
“Azathioprine is a drug that’s been around for years, and I love it because I know the side effects and I know how to test for them,” Martinez said. “Primarily, there are often side effects associated with pancreatitis. If you have a patient who has a significantly reduced white blood cell count, you think, ‘Maybe this isn’t the right drug for my patient.'”
Additionally, patients with concomitant diabetes or obesity may not be ideal candidates for steroids.
“Steroids are necessary because autoimmune hepatitis is very responsive to steroids, and it takes about two to three months for azathioprine to take full effect,” Martinez said. “There are other drugs that work faster, like cyclosporine and tacrolimus. These are familiar drugs to doctors who treat transplant patients, but they’re also very effective in treating autoimmune hepatitis.”
Second-line options may include budesonide, an alternative to prednisone that can reduce the risk of side effects such as the cosmetic effects of steroids in adolescent patients.
Martinez said cyclosporine or tacrolimus is associated with an increased response in about 30 to 50 percent of patients who experienced a reaction with azathioprine. However, an important second-line treatment option remains mycophenolate mofetil (MMF), which provides an alternative if a patient experiences adverse events from azathioprine.
“And we’re going to see about a 70 to 80 percent good response,” Martinez said. “However, if the indication for second-line therapy is lack of response to azathioprine, MMF is usually ineffective, as MMF has the same mechanism of action.”
