George Mazariegos, MD

George Mazariegos, MD, Center for Rare Disease Therapy
George V. Mazariegos, MD, FACS
Chief, Pediatric Transplantation
Professor of Surgery and Critical Care, University of Pittsburgh School of Medicine

When transplant surgeon Dr. George Mazariegos began working with children, it didn’t take long for him to recognize that he had found his calling.

“I realized that we could make an impact for a lifetime,” he recalls. “We could have a part in restoring these children to the lives they were intended to live.”

As director of pediatric transplantation at UPMC Children's Hospital of Pittsburgh, Dr. Mazariegos specializes in liver and intestinal transplantation. He also cares for children who develop acute liver failure and may require specialized support therapies until they recover or until donor organs are available.

Dr. Mazariegos graduated from Northwestern University Medical School in 1986. After completing his residency training at Michigan State University in 1991, he came to the University of Pittsburgh, where he completed a fellowship in critical care medicine in 1992 and a fellowship in liver and multi-organ transplantation in 1993. He joined the Thomas E. Starzl Transplantation Institute in 1994 and came to Children's in 1997 as co-director of pediatric liver and intestine transplantation.

At Children’s Dr. Mazariegos led a team of doctors that developed a novel therapy, approved in 2001, that eliminates the routine use of steroids following a transplant to prevent rejection of the transplanted organ. “Steroids are potentially toxic and have many side effects,” he says. “With our novel approach, we have been able to maximize the benefits that children get from transplantation while minimizing the procedure’s risks and complications.”

This new approach made transplantation an option for children in whom the risks had previously been deemed too great. In 2004, under Dr. Mazariegos’s leadership and in collaboration with doctors at the Clinic for Special Children in Strasburg, PA, Children’s became the first hospital in the world to establish a liver transplantation protocol for patients with maple syrup urine disease (MSUD).

This rare metabolic condition gets its name from the characteristic burnt-sugar smell of patients’ urine. MSUD (also known as branched-chain ketoaciduria) is caused by a genetic error that prevents the body from breaking down certain amino acids, the building blocks of protein. The build-up of these amino acids in the body causes symptoms that may include seizures, brain swelling, and brain damage. The standard treatment for MSUD is a strict protein- and dairy-free diet. It’s a challenge for patients and families stick to the diet and, even when they succeed in doing so, it may not be enough to prevent the development of MSUD complications.

A liver transplant can cure MSUD, says Dr. Mazariegos, but prior to 2004 the procedure was considered too risky to be justifiable in patients who were not at imminent risk of death from liver failure. “By focusing intently on minimizing post-transplant complications, we have now successfully performed more than 50 liver transplants in patients with MSUD–more than any other center in the world,” he says.

Dr. Mazariegos directs a research program focused on further refining treatments to minimize the risks of post-transplant complications and on understanding what characteristics enable patients who have received a transplant to be successfully withdrawn from medications that suppress the immune system. He is an author or co-author of 10 book chapters and more than 70 articles in medical journals. His work has earned him memberships in numerous professional and scientific societies, including the Society of University Surgeons and the International Pediatric Transplant Association. He also serves on the Scientific Advisory Board of Studies in Pediatric Liver Transplantation.

The Center for Rare Disease Therapy offers “a special environment,” says Dr. Mazariegos. “The level of multidisciplinary collaboration is amazing. Everyone is focused on caring for the patient and the family, educating them about all the potential treatment options, making a shared decision about the option that’s best for that child, and following up to assess how effective the treatment has been and what to do next. Everything we do is focused on helping children live as normal a life as possible.”