Huang Hsiang-Po, Chu Kai-Lin, Chien Yin-Hsiu, Wei Ming-Lee, Wu Shu-Tzu, Wang Shiao-Fang, Hwu Wuh-Liang
Departments of Pediatrics, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
J Formos Med Assoc. 2006 Nov;105(11):882-6. doi: 10.1016/S0929-6646(09)60173-X.
BACKGROUND/PURPOSE: Neonatal screening using tandem mass spectrometry (MS/MS) started in Taiwan in 2000. We evaluated the efficacy of this system by analyzing the frequency of diseases and the outcome of the patients identified.
Between August 2001 and July 2004, 199, 922 neonates were screened for 10 amino acids and acylcarnitines using MS/MS in a single center.
In total, 29 cases of inborn errors of metabolism were detected. The overall prevalence was one per 6894 births. The most common inborn error found was 3-methylcrotonyl CoA carboxylase deficiency (10 cases, 34.5%), but none of the cases needed aggressive treatment. There were two cases of type I glutaric aciduria, two cases of maple syrup urine disease, and one case of type II citrullinemia, and early therapeutic intervention was effective for all of them.
We found that MS/MS neonatal screening was valuable in the early diagnosis of severe and treatable inborn errors of metabolism such as organic acidemias and urea cycle disorders. It also detected less severe disorders that required only observation.
背景/目的:台湾于2000年开始采用串联质谱法(MS/MS)进行新生儿筛查。我们通过分析疾病发生频率及确诊患者的治疗结果来评估该系统的有效性。
2001年8月至2004年7月期间,在单一中心对199,922名新生儿采用MS/MS法筛查了10种氨基酸和酰基肉碱。
共检测出29例先天性代谢缺陷病例。总体患病率为每6894例出生中有1例。最常见的先天性代谢缺陷是3-甲基巴豆酰辅酶A羧化酶缺乏症(10例,34.5%),但无一例需要积极治疗。有2例I型戊二酸血症、2例枫糖尿症和1例II型瓜氨酸血症,早期治疗干预对所有这些病例均有效。
我们发现,MS/MS新生儿筛查对于早期诊断严重且可治疗的先天性代谢缺陷如有机酸血症和尿素循环障碍很有价值。它还检测出了仅需观察的不太严重的疾病。