Division of Neuropediatrics and Inherited Metabolic Disorders, Department of Pediatrics, Innsbruck Medical University, Anichstrasse 35, 6020, Innsbruck, Austria.
Amino Acids. 2010 May;38(5):1473-81. doi: 10.1007/s00726-009-0356-2. Epub 2009 Oct 1.
The objective of the study is to analyze plasma amino acid concentrations in propionic acidemia (PA) for the purpose of elucidating possible correlations between propionyl-CoA carboxylase deficiency and distinct amino acid behavior. Plasma concentrations of 19 amino acids were measured in 240 random samples from 11 patients (6 families) with enzymatically and/or genetically proven propionic acidemia (sampling period, January 2001-December 2007). They were compared with reference values from the literature and correlated with age using the Pearson correlation coefficient test. Decreased plasma concentrations were observed for glutamine, histidine, threonine, valine, isoleucine, leucine, phenylalanine and arginine. Levels of glycine, alanine and aspartate were elevated, while values of serine, asparagine, ornithine and glutamate were normal. For lysine, proline and methionine a clear association was not possible. Significant correlations with age were observed for 13 amino acids (positive correlation: asparagine, glutamine, proline, alanine, histidine, threonine, methionine, arginine; negative correlation: leucine, phenylalanine, ornithine, glutamate and aspartate). This study gives new insight over long-term changes in plasma amino acid concentrations and may provide options for future therapies (e.g., substitution of anaplerotic substances) in PA patients.
本研究的目的是分析丙酸血症(PA)患者的血浆氨基酸浓度,以阐明丙酰辅酶 A 羧化酶缺乏与不同氨基酸行为之间的可能相关性。在 2001 年 1 月至 2007 年 12 月期间,对 11 例(6 个家系)经酶学和/或遗传学证实的丙酸血症患者的 240 个随机样本进行了 19 种氨基酸的血浆浓度检测。将这些值与文献中的参考值进行比较,并使用 Pearson 相关系数检验与年龄进行相关分析。结果显示,患者的血浆谷氨酸、组氨酸、苏氨酸、缬氨酸、异亮氨酸、亮氨酸、苯丙氨酸和精氨酸浓度降低。甘氨酸、丙氨酸和天冬氨酸水平升高,而丝氨酸、天冬酰胺、鸟氨酸和谷氨酸水平正常。赖氨酸、脯氨酸和蛋氨酸的情况则不太明确。13 种氨基酸与年龄呈显著相关(正相关:天冬酰胺、谷氨酸、脯氨酸、丙氨酸、组氨酸、苏氨酸、蛋氨酸、精氨酸;负相关:亮氨酸、苯丙氨酸、鸟氨酸、谷氨酸和天冬氨酸)。本研究提供了关于丙酸血症患者血浆氨基酸浓度长期变化的新见解,并可能为未来的治疗方案(例如,补充生酮物质)提供依据。