Mercedes Martinez, MD: Advancing Pediatric Autoimmune Hepatitis

In the third and final part of the interview HCP live broadcast Mercedes Martinez, M.D., medical director of the Pediatric Abdominal Organ Transplantation and Intestinal Transplantation Program at the Center for Liver Diseases and NewYork-Presbyterian Hospital Abdominal Organ transplant center shares her wish list for progress in treating pediatric autoimmune hepatitis.

Researchers and clinicians are hampered by limited funding, awareness, and trajectory of clinical research opportunities in autoimmune hepatitis—as is often the case in the rare disease field. Martinez lamented the relative success of drug development and regulation for other chronic diseases, including psoriasis and rheumatism, both of which benefit from large and well-defined patient populations.

“There are some ideas to repurpose some drugs, such as infliximab, which is useful in IBD and we’ve been trying to use it in related IBD in some autoimmune liver diseases,” Martinez said. “It didn’t turn out that well.”

Martinez also highlighted drugs that target B cell production (associated with autoimmune hepatitis due to their antibody-boosting characteristics), including rituximab, for patients who are unresponsive to immunomodulators and steroids. Despite this, there are currently no drugs that are novel or directly target the pathology of autoimmune hepatitis in children.

In addition to drug development, there is much that needs to be developed to help hepatologists.

“First, I would like to see more specific tools to diagnose this disease,” Martinez said. “Sometimes we treat patients with autoimmune hepatitis for years and they end up developing other conditions.”

“There’s a lot of overlap with the obesity epidemic, and some obese patients may have autoimmune hepatitis… Sometimes patients are positive for autoantibodies and doctors don’t think of it,” she continued.

Martinez is also interested in developing biomarkers derived from methods that are less invasive than liver biopsies, such as blood tests. Such developments may also go some way towards a deeper understanding of autoimmune hepatitis and therefore ultimately help in the development of more targeted therapies.

“We don’t have a clear explanation,” Martinez said. “We know there is a genetic predisposition and some environmental triggers for autoimmune hepatitis, but we don’t understand the pathophysiology.”