Liver Transplantation As A Treatment Option For MSUD


Branched-chain amino acids are metabolized mostly in muscle, but liver transplantation has been found to provide enough enzyme activity to allow the person with MSUD to eat a normal diet and in most cases avoid a metabolic crisis when ill. Liver transplantation is not a true cure as the  gene for MSUD remains and  can still be passed to offspring. It is important to note that elevated branched chain amino acid levels have been observed in some transplanted individuals during illness. The liver from the recipient is sometimes transplanted into another person needing a liver which is called a domino transplant.

Medical therapy and transplant both carry risk. For the individual with MSUD, metabolic decompensation is always a possibility. Transplant carries risk for potential complications including rejection and the need for immunosuppressive medications.

It is important to talk to your metabolic team to determine the best option for you or your child with MSUD.



Liver Transplant for MSUD:
Outcomes, Challenges, and Long Term Perspectives

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

A Webinar Presented by the MSUD Family Support Group

Dr. Mazariegos recently addressed the MSUD community on topics related to liver transplantation in MSUD patients. To view this webinar and the accompanying slides, click the links below.

View All Presentation Slides


For additional detail regarding liver transplantation, see the scientific paper on this subject by clicking the link below:

View Scientific Paper