Metabolic Control and “Ideal” Outcomes in Liver Transplantation for Maple Syrup Urine Disease - 22/09/21
Abstract |
Objectives |
To assess outcomes following liver transplantation for maple syrup urine disease by determining attainment and sustainability of metabolic control and apply an “ideal” outcome composite in long-term survivors.
Study design |
A single center, retrospective review collected clinical data including branched-chain amino acid (leucine, isoleucine, and valine) levels following liver transplant and determined achievement of an ideal long-term outcome profile of a first allograft stable on immunosuppression monotherapy, normal growth, and absence of common transplant-related sequelae.
Results |
Of 77 patients meeting inclusion criteria identified, 23 were long-term (≥10-year) survivors and were additionally assessed for ideal outcome attainment. Patient and graft survival were 100% and 99%, respectively, and all patients were on an unrestricted protein intake diet. Although significant variation was noted in mean isoleucine (P < .01) and leucine (P < .05) levels postliver transplantation, no difference was seen in valine (P = .29) and overall clinical impact was likely negligible as metabolic stability was achieved and sustained beyond 3 years postliver transplantation and no metabolic crises were identified. Of 23 long-term survivors with available data, 9 (39%) achieved all composite metrics determined to define “ideal” outcomes in pediatric postliver transplantation populations.
Conclusions |
Liver transplant enables long-term metabolic stability for patients with maple syrup urine disease. A combination of experience and improvement in both pre- and postliver transplantation care has enabled excellent survival and minimal comorbidities following transplant.
Le texte complet de cet article est disponible en PDF.Abbreviations : ADHD, BCAA, BCKDH, BUN, GGT, ISO, LEU, MSUD, SPLIT, VAL
Plan
The authors declare no conflicts of interest. |
Vol 237
P. 59 - octobre 2021 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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