Auxiliary liver transplantation for urea-cycle enzyme deficiencies: lessons from three cases

Kiuchi, Tetsuya; Edamoto, Yusuke; Kaibori, Masaki; Uryuhara, Kenji; Kasahara, Mureo; Üemoto, Shinji; Egawa, Hiroto; Inomata, Yukihiro; Tanaka, Kōichi

doi:10.1016/s0041-1345(98)01539-5

Cited by 13 publications

(6 citation statements)

References 4 publications

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“…For this reason, the decision to transplant an affected child who is jaundiced but otherwise healthy is often postponed, sometimes until it is too late. Auxiliary liver transplantation has been recommended to spare the native liver for possible future gene therapy or hepatocyte transplantation (24, 25). Our patient with Crigler–Najjar syndrome type I received an auxiliary left lateral segment graft at 10 months of age prior to the onset of neurologic deficiencies.…”

Section: Discussionmentioning

confidence: 99%

Long‐term survival after liver transplantation in children with metabolic disorders

Kayler

Merion

Lee

et al. 2002

Pediatric Transplantation

121

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Section: Discussionmentioning

confidence: 99%

Long‐term survival after liver transplantation in children with metabolic disorders

Kayler

Merion

Lee

et al. 2002

Pediatric Transplantation

121

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“…9 -12 There has been success with auxiliary liver transplantation in OTC deficiency, suggesting that Ͻ100% of normal liver OTC activity would be adequate to significantly improve the patient's outcome or even effect a cure. 11,13 Precisely how much activity is required is not clear; however, isolated hepatocyte transplantation may be expected to improve a patient's nitrogen tolerance. We now describe our experience with a prenatally diagnosed male infant with severe OTC deficiency, treated with hepatocyte transplantation.…”

mentioning

confidence: 99%

Isolated Hepatocyte Transplantation in an Infant With a Severe Urea Cycle Disorder

et al. 2003

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Isolated hepatocyte transplantation appeared to result in temporary relief of hyperammonemia and protein intolerance attributable to OTC deficiency. The metabolic stability achieved was lost after 11 days presumably because of rejection of the transplanted cells because of insufficient immunosuppression. Future attempts at isolated hepatocyte transplantation for inborn errors of metabolism in humans should include adequate immunosuppression and a liver biopsy as a means of proving hepatocyte engraftment and function.

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“…Graft contribution of less than 50%, according to dynamic radionucleotide studies, which have been used in differentiation between native and graft “function,” resulted in a secondary metabolic decompensation following graft insult (acute cellular rejection and hepatitis). Consequently, to provide sufficient metabolic control, including a shield during intercurrent episodes, an estimated provision of above 70% of total liver “function” was proposed . Auxiliary LT involves careful patient selection and complex technical considerations, which continue to make this a challenging decision .…”

Section: Discussionmentioning

confidence: 99%

“…The alternatives to whole liver replacement can assist in reducing pressure on the donor pool. LCT and APOLT have been shown to provide a sufficient enzyme activity and offer advantage of preserving the native liver for future gene therapies . However, the effects of LCT are often not sustained medium‐term.…”

Section: Discussionmentioning

confidence: 99%

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Liver transplantation for neonatal‐onset citrullinemia

Vara

Dhawan

Deheragoda

et al. 2018

Pediatric Transplantation

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Citrullinemia or ASS deficiency in its classical form presents in the neonatal period with poor feeding, hyperammonemia, encephalopathy, seizures, and if untreated can be fatal. Despite advances in medical therapy, neurocognitive outcomes remain suboptimal. LT has emerged as a potential management option. A retrospective single-center review identified 7 children with a median age of 1.1 years (range, 0.6-5.8) at referral. Five children presented clinically, and 2 were treated prospectively from birth due to positive family history. All patients received standard medical and dietary therapy prior to LT. The indications for LT were frequent metabolic decompensations in 4, elective in 2, and ALF in 1. The median age at LT was 2.4 years (range, 1.3-6.5). Five patients received 6 left lateral segment grafts, one a live unrelated donor left lateral segment as an APOLT graft, and one a cadaveric whole liver graft as APOLT. One child required retransplantation due to hepatic artery thrombosis. Graft and patient survival were 86% and 100%, respectively. Median follow-up is 3.1 years (range, 0.1-4.1), and the median age at follow-up is 5.5 years (range, 4.0-9.8). There have been no metabolic decompensations in 6 children, while 1 patient (with APOLT) developed asymptomatic hyperammonemia with no clinical or histological signs of liver injury, requiring additional medical therapy. Our medium-term experience following LT in citrullinemia is favorable, demonstrating a positive transformation of the clinical phenotype.

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Auxiliary liver transplantation for urea-cycle enzyme deficiencies: lessons from three cases

Cited by 13 publications

References 4 publications

Long‐term survival after liver transplantation in children with metabolic disorders

Long‐term survival after liver transplantation in children with metabolic disorders

Isolated Hepatocyte Transplantation in an Infant With a Severe Urea Cycle Disorder

Liver transplantation for neonatal‐onset citrullinemia

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