Pieragostino Damiana, Cicalini Ilaria, Di Michele Silvia, Fusilli Paola, Cotugno Giovanna, Ferrante Rossella, Bucci Ines, Dionisi-Vici Carlo, Stuppia Liborio, De Laurenzi Vincenzo, Rossi Claudia
Center for Advanced Studies and Technology (CAST), University "G. d'Annunzio" of Chieti-Pescara, 66100 Chieti, Italy.
Department of Medical, Oral and Biotechnological Sciences, University ''G. d'Annunzio'' of Chieti-Pescara, 66100 Chieti, Italy.
Metabolites. 2020 Jan 24;10(2):44. doi: 10.3390/metabo10020044.
Phenylketonuria (PKU) is a rare autosomal recessive condition affecting about 1 in 10,000 people in the Europe, with a higher rate in some countries, like Ireland and Italy. In Italy, newborn screening (NBS) by MS/MS allows the diagnostic suspicion of PKU and its variants (Hyperphenylalaninemia (HPA), Tetrahydrobiopterin (BH4) synthesis deficiency, and Tetrahydrobiopterin (BH4) recycling deficiency) through the quantification of Phenylalanine (Phe) and the Phenylalanine/Tyrosine (Phe/Tyr) ratio in dried blood Spot (DBS) samples. Here, we report a case of an HPA whose suspicion was possible with expanded NBS, even if the normal-weight newborn was in total parenteral nutrition (TPN). It is known that TPN may present metabolic alterations, mainly for amino acids at NBS in MS/MS, frequently causing false positives. Actually, TPN is considered a special protocol in NBS, requiring several sample collections. In particular, a DBS sample is required before TPN, at basal time point (48 h after birth) and 72 h after the end of the procedure. In the case we report, even if the first DBS sample (before TPN) resulted negative, the repeated NBS tests revealed increased levels of Phe and dramatically high Phe/Tyr ratio. Thus, the newborn was recalled, and the NBS test was repeated several times before that HPA suspicion was confirmed by other specific biochemical tests. This case highlights the importance of Phe/Tyr ratio, only detectable by MS/MS analysis, in supporting the diagnostic suspicion during amino acids administration in the neonatal period.
苯丙酮尿症(PKU)是一种罕见的常染色体隐性疾病,在欧洲约每10000人中就有1人受其影响,在爱尔兰和意大利等一些国家发病率更高。在意大利,通过串联质谱法进行的新生儿筛查(NBS)能够通过对干血斑(DBS)样本中苯丙氨酸(Phe)的定量以及苯丙氨酸/酪氨酸(Phe/Tyr)比值,对PKU及其变异型(高苯丙氨酸血症(HPA)、四氢生物蝶呤(BH4)合成缺陷和四氢生物蝶呤(BH4)再循环缺陷)进行诊断性怀疑。在此,我们报告一例HPA病例,即使该体重正常的新生儿处于全胃肠外营养(TPN)状态,通过扩展的NBS仍有可能怀疑此病。众所周知,TPN可能会出现代谢改变,主要是在串联质谱法进行NBS检测时氨基酸方面的改变,经常导致假阳性结果。实际上,TPN在NBS中被视为一种特殊方案,需要多次采集样本。具体而言,在TPN之前、基础时间点(出生后48小时)以及该操作结束后72小时都需要采集DBS样本。在我们报告的这个病例中,即使第一个DBS样本(TPN之前)结果为阴性,但重复进行的NBS检测显示Phe水平升高且Phe/Tyr比值极高。因此,该新生儿被召回,在通过其他特定生化检测确认HPA怀疑之前,多次重复进行NBS检测。这个病例突出了只有通过串联质谱分析才能检测到的Phe/Tyr比值在新生儿期氨基酸给药期间支持诊断性怀疑方面的重要性。