Session Time: 3:15pm-4:45pm
Presentation Time: 4:15pm-4:27pm
*Purpose: Determine outcomes and branch-chain amino acid (BCAA – leucine [LEU], isoleucine [ISO], and valine [VAL]) levels at 1-3, 4-7, and 8-10 years for patient’s status-post liver transplant (LT) for MSUD.
*Methods: Single center, retrospective chart review of patients with MSUD s/p LTx at the Children’s Hospital of Pittsburgh. BCAA levels at 1-3, 4-7, and 8-10 years post-liver transplantation were obtained with data related to clinical outcomes. Normal reference ranges (umol/L) for ISO, LEU, and VAL were 30 – 100, 30 – 200, and 70 – 350, respectively. Student t-test and single factor ANOVA analysis were performed.
*Results: 88 LT were performed for MSUD from 1/1/2004 – 6/30/2019. 10 patients were excluded from the analysis due to inadequate records for review (n=6) or did not meet minimum follow-up period (1 yr) from LT for inclusion (n=4) thus 78 patients (male/female 40/38) were included in the analysis. Mean age at LT was 11.7 yrs. Following LT, all patients were managed with an unrestricted protein diet. Patient and graft survival were 100% and 99% respectively. One patient underwent re-transplant for hepatic artery thrombosis and subsequent biliary fibrosis. Mean ISO levels (umol/L) and standard deviation at 1-3, 4-7, and 8-10 yrs post-LT were 71.2 (SD 84.1), 101.9 (SD 81.5), and 95.7 (SD 87.3) (p=0.12). Mean LEU levels (umol/L) and standard deviation at 1-3, 4-7, and 8-10 yrs post-LT were 85.1 (SD 99.4), 136.7 (SD 110.0), and 131.1 (SD 117.3) (p=0.02). Mean VAL levels (umol/L) at 1-3, 4-7, and 8-10 yrs post-LT were 150.6 (SD 185.8), 226.2 (SD 183.3), and 207.8 (SD 181.8) (p=0.08). (Figure 1)
*Conclusions: LT for MSUD enables excellent long-term survival and metabolic control on an unrestricted protein diet. While significant increases were noted in mean LEU levels (p=0.02) and trends were seen in both ISO (p=0.12) and VAL (p=0.08), overall mean values were near or below normal lab values. Variations in post-LT BCAA levels were not associated with any metabolic crises.
To cite this abstract in AMA style:Ewing C, Soltys K, Strauss K, Mazariegos G, Sindhi R, Vockley J, McKiernan P, Squires R, Bond G, Khanna A, Squires J. Long-Term Metabolic Control and Health Outcomes in Maple Syrup Urine Disease Following Liver Transplantation in Children and Young Adults [abstract]. Am J Transplant. 2020; 20 (suppl 3). https://atcmeetingabstracts.com/abstract/long-term-metabolic-control-and-health-outcomes-in-maple-syrup-urine-disease-following-liver-transplantation-in-children-and-young-adults/. Accessed October 26, 2020.
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