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Specialists are hindered by limited therapies and a lack of noninvasive testing methods. Martinez shares a wishlist for her colleagues.
In the third and final 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, shared her wishlist for advancements in managing pediatric autoimmune hepatitis.
Researchers and clinicians are hindered by limited funding, awareness and trajectory of clinical investigation opportunities in autoimmune hepatitis—as is often the case with fields of rare diseases. Martinez lamented the relative success of drug development and regulation seen in other chronic diseases including psoriatic and rheumatic diseases, both of which benefit from massive and well-defined patient populations.
“There are ideas of repurposing some of the medications, like infliximab, which is useful for IBD, and we have been trying to use it for associated IBD in some of the autoimmune liver diseases,” Martinez said. “The results are not that great.”
Martinez additionally highlighted agents that target B-cell production—that which autoimmune hepatitis is associated with due to its characteristic of heightened antibodies—including rituximab, for patients unresponsive to immunomodulators and steroids. All the same, nothing novel nor directly targeting the pathology of autoimmune hepatitis for children is on the horizon.
Beyond drug development, there’s plenty more to still develop in aid of hepatologists.
“First of all, I want to see more specific tools to diagnose the disease,” Martinez said. “There are times that we treat the patient for autoimmune hepatitis for many years, and at the end, they have other conditions.”
“There is a big overlap with the epidemic of obesity, and some patients with obesity might have autoimmune hepatitis...and sometimes patient have these auto-antibodies that are positive, and the physicians don't think about,” she continued.
Martinez is also interested in the development of biomarkers derived from lesser invasive means than a liver biopsy, such as blood assays. Such a development in part could also help inform the understanding of autoimmune hepatitis—and as such, eventually contribute to the development of more targeted therapies.
“We don't have a clear explanation,” Martinez said. “We know that there is a genetic predisposition and there are some environmental triggers for autoimmune hepatitis, but we don't understand the pathophysiology.”