Auxiliary Partial Orthotopic Liver Transplantation for Monogenic Metabolic Liver Diseases: Single-Centre Experience

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.

Appendices

Synopsis

Auxiliary partial orthotopic liver transplantation provides good metabolic control and improves the neurodevelopment in children with selected metabolic liver diseases while retaining a part of the native liver for future gene therapy.

Compliance with Ethics Guidelines

Conflict of Interest

Naresh P. Shanmugam, Joseph J. Valamparampil, Khoula Julenda Al Said, Khalid Al-Thihli, Nadia Al-Hashmi, Emtithal Al-Jishi, Hasan Mohamed Ali Isa, Anil B. Jalan and Mohammed Rela declare that they have no conflict of interest.

Informed Consent

All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000 (5). Informed consent was obtained from all patients for being included in the study.

This article does not contain any studies with animal subjects performed by the any of the authors.

Contributions of Individual Authors

Naresh P. Shanmugam and Joseph J. Valamparampil – collection of clinical information, literature review and manuscript writing. Khoula Julenda Al Said, Khalid Al-Thihli, Nadia Al-Hashmi, Emtithal Al-Jishi, Hasan Mohamed Ali Isa and Anil B. Jalan – literature review and review of manuscript. Prof. Mohammed Rela oversaw all aspects of the manuscript preparation and edited the manuscript. Prof. Mohamed Rela will be the guarantor for the article.