ArticleMolecular genetics and metabolism2010
Early orthotopic liver transplantation in urea cycle defects: follow up of a developmental outcome study.
Article in Molecular genetics and metabolism, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
19 citing papers in PubMed.
- Transplantation as disease modifying therapy in the era of gene therapy medicinal products - health policy considerations.Orphanet journal of rare diseases · 2026Review
- Early continuous blood purification and timely liver transplantation in a neonatal-onset ornithine transcarbamylase deficiency: a case report.Translational pediatrics · 2025Article
- Long-term neurodevelopmental outcomes following liver transplantation for metabolic disease-a single centre experience.Journal of inherited metabolic disease · 2025Article
- Liver transplantation in ornithine transcarbamylase deficiency: A retrospective multicentre cohort study.Molecular genetics and metabolism reports · 2023Article
- Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency.Biomedicines · 2023Review
- Urea cycle disorders and indications for liver transplantation.Frontiers in pediatrics · 2023Article
- Liver transplantation in rare late-onset ornithine transcarbamylase deficiency with central nervous system injury: A case report and review of the literature.Brain and behavior · 2022Review
- Identification of rare variants causing urea cycle disorders: A clinical, genetic, and biophysical study.Journal of cellular and molecular medicine · 2021Article
- Clinical and molecular characteristics of 69 Chinese patients with ornithine transcarbamylase deficiency.Orphanet journal of rare diseases · 2020Article
- Article
- Citrullinemia Type 1: Behavioral Improvement with Late Liver Transplantation.Indian journal of pediatrics · 2019Article
- Polish Experience with Liver Transplantation and Post-Transplant Outcomes in Children with Urea Cycle Disorders.Annals of transplantation · 2017Article
- Improving long term outcomes in urea cycle disorders-report from the Urea Cycle Disorders Consortium.Journal of inherited metabolic disease · 2016Article
- Inherited metabolic disorders presenting as acute liver failure in newborns and young children: King's College Hospital experience.European journal of pediatrics · 2015Article
- Neurologic outcome of urea cycle disorder liver transplant recipients may be predicted by pretransplant neurological imaging.Pediatric transplantation · 2015Article
- Postanalytical tools improve performance of newborn screening by tandem mass spectrometry.Genetics in medicine : official journal of the American College of Medical Genetics · 2014Article
- Therapeutic hepatocyte transplant for inherited metabolic disorders: functional considerations, recent outcomes and future prospects.Journal of inherited metabolic disease · 2014Review
- Pediatric liver transplantation for urea cycle disorders and organic acidemias: United Network for Organ Sharing data for 2002-2012.Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society · 2014Article
- Suggested guidelines for the diagnosis and management of urea cycle disorders.Orphanet journal of rare diseases · 2012Review
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
Patients with neonatal urea cycle defects (UCDs) typically have high mortality and poor neurological outcome unless they receive liver transplantation. Neurologic outcome may be better with liver transplantation before age one year. We report on a follow up on an initial prospective study performed to assess developmental outcome after early liver transplant using the Griffiths Scales. Developmental testing up to 7years after transplantation showed average developmental quotients (DQs) of 69 for four children who underwent transplantation before one year of age (latest DQs were 47, 63, 95 and 96), and 80 for a patient who underwent transplantation at 3years of age (latest DQ was 88). We conclude that a combination of early liver transplantation, aggressive metabolic management and early childhood intervention improve the neurologic outcome of children with UCDs.
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