Naresh P Shanmugam1,2, Joseph J Valamparampil1,2, Mettu Srinivas Reddy1,2, Khoula Julenda Al Said3, Khalid Al-Thihli4, Nadia Al-Hashmi3, Emtithal Al-Jishi5, Hasan Mohamed Ali Isa6,7, Anil B Jalan8, Mohamed Rela9,10,11. 1. Institute of Liver Disease and Transplantation, Gleneagles Global Health City, Chennai, India. 2. Institute of Liver Disease and Transplantation, Dr. Rela Institute & Medical Centre, Bharat Institute of Higher Education & Research, Chennai, India. 3. Royal Hospital, Muscat, Oman. 4. Genetic and Developmental Medicine Clinic, Sultan Qaboos University, Muscat, Oman. 5. Paediatric and Inborn Error of Metabolism, Salmaniya Medical Complex, Manama, Bahrain. 6. Salmaniya Medical Complex, Manama, Bahrain. 7. Arabian Gulf University, Manama, Bahrain. 8. Paediatric and Inborn Error of Metabolism, Navi Mumbai Institute of Research in Mental and Neurological Handicap, Navi Mumbai, India. 9. Institute of Liver Disease and Transplantation, Gleneagles Global Health City, Chennai, India. mohamed.rela@gmail.com. 10. Institute of Liver Disease and Transplantation, Dr. Rela Institute & Medical Centre, Bharat Institute of Higher Education & Research, Chennai, India. mohamed.rela@gmail.com. 11. Institute of Liver Studies, King's College Hospital, London, UK. mohamed.rela@gmail.com.
Abstract
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.
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.
Entities:
Keywords:
Auxiliary partial orthotopic liver transplantation; Citrullinemia type 1; Crigler-Najjar syndrome type 1; Metabolic liver disease; Propionic acidemia