Ann Surg 1999 Apr;229(4):565-9

Auxiliary partial orthotopic liver transplantation for Crigler-Najjar syndrome type I

Rela M, Muiesan P, Vilca-Melendez H, Dhawan A, Baker A, Mieli-Vergani G, Heaton
ND
Department of Child Health, King's College Hospital, London, United Kingdom.

Abstract
OBJECTIVE: To determine if auxiliary partial orthotopic liver transplantation
(APOLT) has the long-term potential to correct the underlying abnormality in
Crigler-Najjar syndrome type 1 (CNS1) without the need for total liver
replacement. BACKGROUND: Orthotopic liver transplantation has been used
successfully to replace the defective enzyme in CNS1. Experimental studies have
shown that only 1% to 2% of the normal hepatocyte mass is needed for bilirubin
conjugation. If APOLT corrects the underlying metabolic abnormality, it has the
advantage of preserving the native liver, which would serve as a "safety net"
should the graft fail, and there is the potential for gene therapy in the future
with possible withdrawal of immunosuppression. METHODS: Seven APOLT procedures
were performed in six recipients with CNS1. Median age at transplantation was
10.5 years. Six transplants were performed as a left auxiliary liver transplant,
and one was performed as a right auxiliary liver transplant. Median serum
bilirubin level at transplantation was 320 micromol/L. All patients required 12
to 16 hours of phototherapy daily before the transplant to maintain serum
bilirubin levels between 250 and 350 micromol/L. RESULTS: Median serum bilirubin
level was 50 micromol/L at day 5 after the transplant and 23 micromol/L at a
median follow-up of 32 months. In four children, early severe acute rejection
developed, requiring conversion to tacrolimus; one underwent a second transplant
for chronic rejection and graft atrophy but died from lymphoproliferative
disease 6 months after the second transplant. CONCLUSIONS: This report shows
that APOLT is technically feasible and provides adequate hepatocyte mass to
correct the underlying metabolic abnormality in CNS1