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筛查新生儿的低 T 细胞受体切除环(TRECs)无法检测免疫缺陷、着丝粒不稳定和面部异常综合征。

Screening Newborns for Low T Cell Receptor Excision Circles (TRECs) Fails to Detect Immunodeficiency, Centromeric Instability, and Facial Anomalies Syndrome.

机构信息

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany; Department of Immunology, Labor Berlin Charité-Vivantes, Berlin, Germany.

Newborn Screening Laboratory, Charité Universitätsmedizin, Berlin, Germany.

出版信息

J Allergy Clin Immunol Pract. 2023 Sep;11(9):2872-2883. doi: 10.1016/j.jaip.2023.06.006. Epub 2023 Jun 9.

Abstract

BACKGROUND

Assessment of T-cell receptor excision circles (TRECs) in dried blood spots of newborns allows the detection of severe combined immunodeficiency (SCID) (T cells <300/μL at birth) with a presumed sensitivity of 100%. TREC screening also identifies patients with selected combined immunodeficiency (CID) (T cells >300/μL, yet <1500/μL at birth). Nevertheless, relevant CIDs that would benefit from early recognition and curative treatment pass undetected.

OBJECTIVE

We hypothesized that TREC screening at birth cannot identify CIDs that develop with age.

METHODS

We analyzed the number of TRECs in dried blood spots in archived Guthrie cards of 22 children who had been born in the Berlin-Brandenburg area between January 2006 and November 2018 and who had undergone hematopoietic stem-cell transplantation (HSCT) for inborn errors of immunity.

RESULTS

All patients with SCID would have been identified by TREC screening, but only 4 of 6 with CID. One of these patients had immunodeficiency, centromeric instability, and facial anomalies syndrome type 2 (ICF2). Two of 3 patients with ICF whom we have been following up at our institution had TREC numbers above the cutoff value suggestive of SCID at birth. Yet all patients with ICF had a severe clinical course that would have justified earlier HSCT.

CONCLUSIONS

In ICF, naïve T cells may be present at birth, yet they decline with age. Therefore, TREC screening cannot identify these patients. Early recognition is nevertheless crucial, as patients with ICF benefit from HSCT early in life.

摘要

背景

通过对新生儿干血斑中的 T 细胞受体切除环(TREC)进行评估,可以检测到严重联合免疫缺陷症(SCID)(出生时 T 细胞<300/μL),其假定灵敏度为 100%。TREC 筛查还可以识别出某些联合免疫缺陷症(CID)患者(出生时 T 细胞>300/μL,但<1500/μL)。然而,一些会受益于早期发现和治疗的相关 CID 却未被检测到。

目的

我们假设出生时的 TREC 筛查无法识别随年龄发展的 CID。

方法

我们分析了 2006 年 1 月至 2018 年 11 月期间在柏林-勃兰登堡地区出生的 22 名接受造血干细胞移植(HSCT)治疗的先天性免疫缺陷症患者的 Guthrie 卡中干血斑的 TRECs 数量。

结果

所有 SCID 患者均能通过 TREC 筛查识别,但仅能识别出 6 名 CID 患者中的 4 名。其中 1 名患者患有免疫缺陷、着丝粒不稳定和面部异常综合征 2 型(ICF2)。在我们机构随访的 3 名 ICF 患者中,有 2 名患者的 TREC 数量高于出生时提示 SCID 的截断值。然而,所有患有 ICF 的患者均具有严重的临床病程,这使得早期进行 HSCT 成为必要。

结论

在 ICF 中,出生时可能存在幼稚 T 细胞,但随着年龄的增长会逐渐减少。因此,TREC 筛查无法识别这些患者。然而,早期识别仍然至关重要,因为 ICF 患者在生命早期接受 HSCT 治疗会获益。

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