Chiarini Marco, Zanotti Cinzia, Serana Federico, Sottini Alessandra, Bertoli Diego, Caimi Luigi, Imberti Luisa
Laboratorio Interdipartimentale di Biologia Cellulare e Radiobiologia, Diagnostics Department, Spedali Civili of Brescia Brescia , Italy.
Clinical Biochemistry, Department of Molecular and Translational Medicine, University of Brescia , Brescia, Italy.
J Public Health Res. 2013 May 1;2(1):9-16. doi: 10.4081/jphr.2013.e3. eCollection 2013 Apr 28.
Since its introduction as a public health programme in the United States in the early 1960s, newborn blood screening (NBS) has evolved from the detection of phenylalanine levels on filter paper to the application of DNA-based technologies to identify T-cell lymphopenia in infants with severe combined immunodeficiency. This latter use of NBS has required the development of an assay for T-cell lymphopenia based on the quantification of T-cell receptor excision circles (TRECs) that could be performed on dried blood spots routinely collected from newborn infants. The TREC-based NBS was developed six years ago, and there have already been 7 successful pilot studies since then. Similarly, efforts are now being made to establish a screen for B-cell defects, in particular agammaglobulinaemia, taking advantage of the introduction of the method for the quantification of K-deleting recombination excision circles (KRECs). A further achievement of NBS could be the simultaneous recognition of T- and B-cell defects using the combined quantification of TRECs and KRECs from Guthrie card blood spots. This approach may help the early identification of infants with T- and B-cell deficiencies so that they can then be referred to specialised paediatric centres, where a precise diagnosis of severe combined immunodeficiency and agammaglobulinaemia can be performed, and where then they can immediately receive specific therapy. Simultaneous TREC and KREC quantification should also allow classification of patients into subgroups and help identify children with less serious primary immunodeficiencies. This would help avoid the opportunistic infections and frequent hospitalisations that result from a late or lack of diagnosis.
自20世纪60年代初在美国作为一项公共卫生项目推出以来,新生儿血液筛查(NBS)已从检测滤纸上的苯丙氨酸水平发展到应用基于DNA的技术来识别严重联合免疫缺陷婴儿的T细胞淋巴细胞减少症。NBS的后一种应用需要开发一种基于T细胞受体切除环(TREC)定量的T细胞淋巴细胞减少症检测方法,该方法可在从新生儿常规采集的干血斑上进行。基于TREC的NBS是六年前开发的,从那时起已经有7项成功的试点研究。同样,现在正在努力利用K缺失重组切除环(KREC)定量方法的引入,建立B细胞缺陷筛查,特别是无丙种球蛋白血症筛查。NBS的另一项成果可能是通过联合定量Guthrie卡片血斑中的TREC和KREC来同时识别T细胞和B细胞缺陷。这种方法可能有助于早期识别患有T细胞和B细胞缺陷的婴儿,以便他们随后被转诊至专门的儿科中心,在那里可以对严重联合免疫缺陷和无丙种球蛋白血症进行精确诊断,并在那里他们可以立即接受特异性治疗。同时进行TREC和KREC定量还应能将患者分类为亚组,并有助于识别原发性免疫缺陷较轻的儿童。这将有助于避免因诊断延迟或未诊断而导致的机会性感染和频繁住院。