D. Holmes Morton, MD, and Kevin A. Strauss, MD, of the Clinic for Special Children in Strasburg, Pa., collaborated with Children's physicians for months to develop the nation's first protocol to perform liver transplants as a potential cure for MSUD. Drs. Morton and Strauss have worked with many children suffering from MSUD at the clinic and brought important techniques, treatments and invaluable experience to the group.
Children's Hospital established computerized order sets -- which tell the transplant team how to proceed during every step of the transplant.
The hospital also set up a completely sterile room where raw amino acid powders can be prepared quickly in case there are complications from transplantation and the patient goes into metabolic crisis.
For each MSUD patient, Children's is prepared to make specific total parenteral nutrition (TPN) formulas based on a patient's amino acid levels. The formulas will be different for each patient and must be mixed precisely to ensure that the patient receives exactly the correct amino acids to avoid metabolic crisis. TPN is a special mixture of glucose, protein, fat, vitamins and minerals given through an intravenous tube.
"Our patients' safety and well-being is always of the utmost importance," Dr. Mazariegos said. "After one year, our results show metabolic cure."