Precision Medicine, Nemours Children's Health, Jacksonville, Florida, USA.
Department of Pharmacotherapy and Translational Research, The University of Florida College of Pharmacy, Jacksonville, Florida, USA.
Clin Transl Sci. 2023 Aug;16(8):1352-1358. doi: 10.1111/cts.13539. Epub 2023 Jul 6.
The risk of severe adverse events related to thiopurine therapy can be reduced by personalizing dosing based on TPMT and NUDT15 genetic polymorphisms. However, the optimal genetic testing platform has not yet been established. In this study, we report on the TPMT and NUDT15 genotypes and phenotypes generated from 320 patients from a multicenter pediatric healthcare system using both Sanger sequencing and polymerase chain reaction genotyping (hereafter: genotyping) methods to determine the appropriateness of genotyping in our patient population. Sanger sequencing identified variant TPMT alleles including *3A (8, 3.2% of alleles), *3C (4, 1.6%), and *2 (1, 0.4%), and NUDT15 alleles including *2 (5, 3.6%) and *3 (1, 0.7%). For genotyped patients, variants identified in TPMT included *3A (12, 3.1%), *3C (4, 1%), *2 (2, 0.5%), and *8 (1, 0.25%), whereas NUDT15 included *4 (2, 1.9%) and *2 or *3 (1, 1%). Between Sanger sequencing and genotyping, no significant difference in allele, genotype, or phenotype frequency was identified for either TPMT or NUDT15. All patients who were tested using Sanger sequencing would have been accurately phenotyped for either TPMT (124/124), NUDT15 (69/69), or both genes (68/68) if they were assayed using the genotyping method. Considering 193 total TPMT and NUDT15 Sanger Sequencing tests reviewed, all tests would have resulted in an appropriate clinical recommendation if the test had instead been conducted using the comparison genotyping platforms. These results suggest that, in this study population, genotyping would be sufficient to provide accurate phenotype calls and clinical recommendations.
基于 TPMT 和 NUDT15 基因多态性的个体剂量调整可以降低与硫嘌呤治疗相关的严重不良事件风险。然而,尚未建立最佳的基因检测平台。在这项研究中,我们报告了来自多中心儿科医疗保健系统的 320 名患者的 TPMT 和 NUDT15 基因型和表型,使用 Sanger 测序和聚合酶链反应基因分型(以下简称基因分型)方法生成,以确定基因分型在我们患者群体中的适用性。Sanger 测序鉴定了变异 TPMT 等位基因,包括3A(8,3.2%的等位基因)、3C(4,1.6%)和2(1,0.4%),以及 NUDT15 等位基因,包括2(5,3.6%)和3(1,0.7%)。对于基因分型患者,TPMT 中鉴定的变异包括3A(12,3.1%)、3C(4,1%)、2(2,0.5%)和8(1,0.25%),而 NUDT15 包括4(2,1.9%)和2 或3(1,1%)。在 Sanger 测序和基因分型之间,TPMT 或 NUDT15 的等位基因、基因型或表型频率均无显著差异。如果使用基因分型方法检测,所有使用 Sanger 测序检测的患者都将能够准确地对 TPMT(124/124)、NUDT15(69/69)或两个基因(68/68)进行表型分析。考虑到 193 次总 TPMT 和 NUDT15 Sanger 测序测试,所有测试如果使用比较基因分型平台进行,都会得出适当的临床建议。这些结果表明,在本研究人群中,基因分型足以提供准确的表型分析和临床建议。