Journal
PEDIATRICS INTERNATIONAL
Volume 59, Issue 4, Pages 422-426Publisher
WILEY
DOI: 10.1111/ped.13163
Keywords
ammonia; carbamoyl phosphate synthetase I deficiency; citrulline; ornithine transcarbamylase deficiency; urea cycle disorder
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Funding
- Ministry of Education, Culture, Sports, Science and Technology
- Ministry of Health, Labor and Welfare
- Grants-in-Aid for Scientific Research [17K10055] Funding Source: KAKEN
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BackgroundThe amino acid l-citrulline is used as a therapeutic agent for urea cycle disorders (UCD) including ornithine transcarbamylase deficiency (OTCD), carbamoyl phosphate synthetase I deficiency (CPSD), and N-acetylglutamate synthase deficiency. There are few reports, however, on the use of l-citrulline in Japan and little consensus regarding the effects of l-citrulline. MethodsWe conducted a questionnaire survey of patients undergoing l-citrulline treatment for a UCD to evaluate the current status of this therapy. The survey included patient background, details of l-citrulline treatment, clinical examination data, treatment, frequency of vomiting, and liver transplantation. ResultsWe retrospectively investigated 43 questionnaire respondents (OTCD, n = 33; CPSD, n = 10). The weight of male OTCD patients improved by +0.79 SD, and the ammonia level decreased by a mean of 44.3 mol/L in all patients. The protein intake of all patients and of male OTCD patients increased by 0.14 g/kg/day and 0.17 g/kg/day, respectively. Conclusionsl-Citrulline effectively reduced ammonia level, increased protein intake, and improved weight gain in UCD patients. l-Citrulline should be considered a standard therapy in OTCD and CPSD patients.
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