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[二级检测纳入加泰罗尼亚新生儿筛查项目及其他国际项目的影响。]

[Impact of the inclusion of second-tier tests in the newborn screening program of Catalonia and in other international programs.].

作者信息

Pajares García Sonia, López Galera Rosa Mª, Marín Soria Jose Luis, Argudo Ramírez Ana, González de Aledo-Castillo Jose Manuel, Ribes Rubió Antonia, Prats Viedma Blanca, Asso Ministral Laia, García-Villoria Judit

机构信息

Sección Errores Congénitos del Metabolismo-IBC. Servicio de Bioquímica y Genética Molecular. Hospital Clínic de Barcelona. Barcelona. España.

Ciber de Enfermedades Raras (CIBERER). España.

出版信息

Rev Esp Salud Publica. 2020 Dec 16;94:e202012158.

Abstract

OBJECTIVE

Newborn screening programmes (NBSP) have experienced a qualitative breakthrough due to the implementation of tandem mass spectrometry. However, the tests used give rise to false positives (FP) generating an excessive request for second samples with the consequent anxiety of the families. In order to avoid this problem several programmes have developed second-tier tests (2TT).

METHODS

This article presents our experience in the implementation of 2TT in the NBSP of Catalonia, as well as in other international programmes.

RESULTS

From 2004 to the present, 2TT tests have been developed for more than 30 diseases. The use of 2TT helps to decrease the FP rate and increase the positive predictive value (PPV). In the NBSP of Catalonia, the implementation of 2TT for the detection of methylmalonic and propionic acidemias, homocystinurias, maple syrup disease and citrulinaemia, has managed to increase the PPV to 95% and decrease the PF rate to less than 0.01%. In cystic fibrosis, the application of 2TT slightly increases PPV but with a significant decrease in the request for second samples and in the number of cases referred to clinical units.

CONCLUSIONS

The introduction of 2TT in the NBSP allows to reduce considerably the FP, decreases the number of requested samples, as well as both anxiety and stress of the families, at the same time that the hospital costs are reduced and the PPV is increased, improving notably the efficiency of the NBSP.

摘要

目的

由于串联质谱技术的应用,新生儿筛查项目(NBSP)实现了质的突破。然而,所使用的检测方法会产生假阳性(FP),导致对二次样本的过度需求,进而使家庭产生焦虑。为避免这一问题,多个项目已开发出二级检测(2TT)。

方法

本文介绍了我们在加泰罗尼亚新生儿筛查项目以及其他国际项目中实施二级检测的经验。

结果

从2004年至今,已针对30多种疾病开发了二级检测。二级检测的使用有助于降低假阳性率并提高阳性预测值(PPV)。在加泰罗尼亚的新生儿筛查项目中,实施用于检测甲基丙二酸血症和丙酸血症、高胱氨酸尿症、枫糖浆病和瓜氨酸血症的二级检测,已成功将阳性预测值提高到95%,并将假阳性率降低至低于0.01%。在囊性纤维化方面,二级检测的应用使阳性预测值略有提高,但二次样本的需求以及转诊至临床科室的病例数量显著减少。

结论

在新生儿筛查项目中引入二级检测可大幅减少假阳性,减少所需样本数量,减轻家庭的焦虑和压力,同时降低医院成本并提高阳性预测值,显著提高新生儿筛查项目的效率。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/53ae/11583059/296fa63e6661/1135-5727-resp-94-e202012158-g001.jpg

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