pp 1-8 | Cite as
Auxiliary Partial Orthotopic Liver Transplantation for Monogenic Metabolic Liver Diseases: Single-Centre Experience
Purpose: Auxiliary partial orthotopic liver transplantation (APOLT) in metabolic liver disease (MLD) has the advantage of correcting the metabolic defect, preserving the native liver for gene therapy in the future with the possibility of withdrawal of immunosuppression.
Methods: Retrospective analysis of safety and efficacy of APOLT in correcting the underlying defect and its impact on neurological status of children with MLD.
Results: A total of 13 APOLT procedures were performed for MLD during the study period. The underlying aetiologies being propionic acidemia (PA)-5, citrullinemia type 1 (CIT1)-3 and Crigler-Najjar syndrome type 1 (CN1)-5 cases respectively. Children with PA and CIT1 had a median of 8 and 4 episodes of decompensation per year, respectively, before APOLT and had a mean social developmental quotient (DQ) of 49 (<3 standard deviations) as assessed by Vineland Social Maturity Scale prior to liver transplantation. No metabolic decompensation occurred in patients with PA and CIT1 intraoperatively or in the immediate post-transplant period on protein-unrestricted diet. Patients with CN1 were receiving an average 8–15 h of phototherapy per day before APOLT and had normal bilirubin levels without phototherapy on follow-up. We have 100% graft and patient survival at a median follow-up of 32 months. Progressive improvement in neurodevelopment was seen in children within 6 months of therapy with a median social DQ of 90.
Conclusions: APOLT is a safe procedure, which provides good metabolic control and improves the neurodevelopment in children with selected MLD.
KeywordsAuxiliary partial orthotopic liver transplantation Citrullinemia type 1 Crigler-Najjar syndrome type 1 Metabolic liver disease Propionic acidemia
- Baba C, Kasahara M, Kogure Y, Kasuya S, Ito S, Tamura T (2016) Perioperative management of living-donor liver transplantation for methylmalonic acidemia. Paediatr Anaesth 26:694–702Google Scholar
- Blankensteijn JD, Groenland TH, Baumgartner D, Vos LP, Kerkhofs LG, Terpstra OT (1990) Intraoperative hemodynamics in liver transplantation comparing orthotopic with heterotopic transplantation in the pig. Transplantation 49:665–668Google Scholar
- Fagiuoli S, Daina E, D’Antiga L, Colledan M, Remuzzi G (2013) Monogenic diseases that can be cured by liver transplantation. J Hepatol 59:595–612Google Scholar
- Govil S, Shanmugam NP, Reddy MS, Narasimhan G, Rela M (2015) A metabolic chimera: two defective genotypes make a normal phenotype. Liver Transpl 21:1453–1454Google Scholar
- Kaibori M, Egawa H, Inomata Y et al (1998) Selective portal blood flow diversion in auxiliary partial orthotopic liver transplantation to induce regeneration of the graft. Transplantation 66:935–937Google Scholar
- Kasahara M, Takada Y, Egawa H, Fujimoto Y, Ogura Y, Ogawa K (2005) Auxiliary partial orthotopic living donor liver transplantation: Kyoto University experience. Am J Transplant 5:558–565Google Scholar
- Morioka D, Kasahara M, Takada Y et al (2005a) Current role of liver transplantation for the treatment of urea cycle disorders: a review of the worldwide English literature and 13 cases at Kyoto University. Liver Transpl 11:1332–1342Google Scholar
- Morioka D, Kasahara M, Takada Y et al (2005b) Living donor liver transplantation for pediatric patients with inheritable metabolic disorders. Am J Transplant 5:2754–2763Google Scholar
- Popescu I, Dima SO (2012) Domino liver transplantation: how far can we push the paradigm? Liver Transpl 18:22–28Google Scholar
- Reddy MS, Rajalingam R, Rela M (2017) Revisiting APOLT for metabolic liver disease: a new look at an old idea. Transplantation 101:260–266Google Scholar
- Rela M, Bharathan A, Rajalingam R, Narasimhan G, Reddy MS (2013) Technique of hepatic arterial anastomosis in living donor pediatric auxiliary partial orthotopic liver transplantation. Liver Transpl 19:1046–1048Google Scholar
- Rela M, Bharathan A, Palaniappan K, Cherian PT, Reddy MS (2015) Portal flow modulation in auxiliary partial orthotopic liver transplantation. Pediatr Transplant 19:255–260Google Scholar
- Rela M, Kaliamoorthy I, Reddy MS (2016) Current status of auxiliary partial orthotopic liver transplantation for acute liver failure. Liver Transpl 22:1265–1274Google Scholar
- Shanmugam NP, Perumalla R, Gopinath R, Olithselvan A, Varghese J, Kapoor D (2011) Auxiliary liver transplantation: a form of gene therapy in selective metabolic disorders. J Clin Exp Hepatol 1:118–120Google Scholar
- Sokal EM (2006) Liver transplantation for inborn errors of liver metabolism. J Inherit Metab Dis 29:426–430Google Scholar
- Sze YK, Dhawan A, Taylor RM, Bansal S, Mieli-Vergani G, Rela M, Heaton N (2009) Paediatric liver transplantation for metabolic liver disease: experience at King’s College Hospital. Transplantation 87:87–93Google Scholar
- Trotter JF, Milliner D (2014) Auxiliary liver transplant is an ineffective treatment of primary hyperoxaluria. Am J Transplant 14:241Google Scholar