Fecarotta S, Parenti G, Vajro P, Zuppaldi A, Della Casa R, Carbone M T, Correra A, Torre G, Riva S, Dionisi-Vici C, Santorelli F M, Andria G
Department of Pediatrics, Federico II University, Via S Pansini 5, 80131 Naples, Italy.
J Inherit Metab Dis. 2006 Feb;29(1):186-9. doi: 10.1007/s10545-006-0120-7.
We report a 3-year-old Italian patient with the hyperornithinaemia, hyperammonaemia, homocitrullinuria (HHH) syndrome who presented with neurological deterioration after an intercurrent infection. Hyperammonaemia, coagulopathy and moderate hypertransaminasaemia were detected on hospital admission. Severe hepatocellular necrosis with hypertransaminasaemia (aspartate aminotransferase 20,000 UI/L, alanine aminotransferase 18,400 UI/L) and coagulopathy (PT < 5%) rapidly developed within few days, prompting evaluation for liver transplantation. A protein-restricted diet and arginine supplementation were immediately started, with a rapid improvement of the patient's neurological conditions and normalization of liver function tests and blood ammonia. The diagnosis of HHH syndrome was based on the presence of the typical metabolic abnormalities. Molecular analysis of the SLC25A15 gene showed that the patient was heterozygous for two novel mutations (G113C and M273K). The diagnosis of HHH syndrome should be considered in patients with fulminant hepatitis-like presentations. Early identification and treatment of these patients can be life-saving and can avoid liver transplantation.
我们报告了一名3岁的意大利患者,患有高鸟氨酸血症、高氨血症、同型瓜氨酸尿症(HHH)综合征,在一次并发感染后出现神经功能恶化。入院时检测到高氨血症、凝血功能障碍和中度转氨酶升高。在几天内迅速出现严重的肝细胞坏死伴转氨酶显著升高(天冬氨酸转氨酶20,000 UI/L,丙氨酸转氨酶18,400 UI/L)和凝血功能障碍(凝血酶原时间<5%),促使对患者进行肝移植评估。立即开始限制蛋白质饮食并补充精氨酸,患者的神经状况迅速改善,肝功能检查和血氨恢复正常。HHH综合征的诊断基于典型代谢异常的存在。对SLC25A15基因的分子分析表明,该患者存在两个新的杂合突变(G113C和M273K)。对于暴发性肝炎样表现的患者应考虑HHH综合征的诊断。对这些患者的早期识别和治疗可挽救生命并避免肝移植。