Department of Pediatric Endocrinology and Genetics, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Institute for Pediatric Research, Shanghai 200092, China.
Department of Pediatric Endocrinology and Genetics, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Institute for Pediatric Research, Shanghai 200092, China.
Clin Biochem. 2021 Aug;94:27-34. doi: 10.1016/j.clinbiochem.2021.04.012. Epub 2021 Apr 18.
Glucose-6-phosphate dehydrogenase (G6PD) deficiency is the most common inherited disease. Current neonatal screening methods for G6PD deficiency primarily rely on the use of biochemical tests. However, only 15%-20% of female carriers were estimated to have been detected using these tests. As a better alternative, DNA-based tests could be used for G6PD deficiency screening. We aimed to develop a matrix-assisted laser desorption/ionization-time of flight mass spectrometry (MALDI-TOF MS) assay for G6PD variant detection.
A MALDI-TOF MS assay with multiprimer extension (multi-PEX) was developed to rapidly and accurately detect the 29 common G6PD variants in the Chinese population using a dried blood spot as a template. A parallel study screening 571 unrelated neonatal samples using the MALDI-TOF MS and fluorescence quantitative enzymatic assays was performed. All results were confirmed by Sanger sequencing in a blind study.
In 571 unrelated neonatal samples, 34 positive samples, including 26 samples from hemizygous males and eight samples from heterozygous females, were correctly identified, yielding a clinical sensitivity of 100%. The results were validated using Sanger sequencing with 100% concordance. In contrast, the fluorescence quantitative enzymatic assay had a 75% false negative and 88.8% false positive rate for the detection of heterozygous G6PD deficient females.
We established a reliable MALDI-TOF MS assay for G6PD deficiency screening in the Chinese population maximizing the chance of detection of heterozygous G6PD deficient females and reducing the false negative and false positive rates associated with routinely used newborn screening procedures.
葡萄糖-6-磷酸脱氢酶(G6PD)缺乏症是最常见的遗传性疾病。目前,G6PD 缺乏症的新生儿筛查方法主要依赖生化检测。然而,据估计,这些检测方法仅能检测出 15%-20%的女性携带者。作为更好的替代方法,可以使用基于 DNA 的检测方法进行 G6PD 缺乏症筛查。我们旨在开发基质辅助激光解吸/电离飞行时间质谱(MALDI-TOF MS)检测方法用于 G6PD 变异检测。
开发了一种多重引物延伸(multi-PEX)MALDI-TOF MS 检测方法,用于使用干血斑作为模板快速准确地检测中国人群中 29 种常见的 G6PD 变异体。对 571 例无关新生儿样本进行了 MALDI-TOF MS 和荧光定量酶联免疫吸附试验平行筛查。所有结果均在盲法研究中通过 Sanger 测序进行确认。
在 571 例无关新生儿样本中,34 例阳性样本,包括 26 例半合子男性样本和 8 例杂合子女性样本,被正确识别,临床灵敏度为 100%。使用 Sanger 测序进行验证,结果完全一致。相比之下,荧光定量酶联免疫吸附试验检测杂合子 G6PD 缺乏女性的假阴性率为 75%,假阳性率为 88.8%。
我们建立了一种可靠的 MALDI-TOF MS 检测方法,用于中国人群的 G6PD 缺乏症筛查,最大限度地提高了检测杂合子 G6PD 缺乏女性的机会,并降低了与常规新生儿筛查程序相关的假阴性和假阳性率。