Department of Medical Genetics, Bursa Yüksek İhtisas Training and Research Hospital, Bursa, Turkey.
Am J Med Genet A. 2023 Jun;191(6):1557-1564. doi: 10.1002/ajmg.a.63190. Epub 2023 Mar 25.
Clinical exome sequencing (CES) is important for the diagnosis of Mendelian diseases, which are clinically and etiologically heterogeneous. Sharing of large amounts of CES data associated with clinical findings will increase the accuracy of variant interpretation. We performed a retrospective study to state the diagnostic yield of CES in 1589 patients with a wide phenotypic spectrum. CES was performed using the Sophia Clinical Exome Sequencing Kit with 4493 genes, followed by sequencing on a NextSeq 500 system. The diagnosis rate was 36.8% when only pathogenic and likely pathogenic variants were included. Consanguineous unions and positive family history were associated with a high diagnostic yield. The neurological disease group had the highest number of patients. The groups with high diagnosis rates were ear, eye, and muscle disease groups. Seven candidate genes (EFHC2, HSPB3, FAAH2, ITGB1, GYG2, CD177, and CSTF2T) that are not yet associated with human diseases were identified. Owing to the high diagnostic yield of CES compared with that of other genetic tests, it can be used as a standard diagnostic test in patients with rare genetic disorders that require a wide differential diagnosis, especially in laboratories with limited resources.
临床外显子组测序(CES)对于孟德尔疾病的诊断很重要,这些疾病在临床和病因上具有异质性。分享与临床发现相关的大量 CES 数据将提高变异解释的准确性。我们进行了一项回顾性研究,以说明在具有广泛表型谱的 1589 名患者中 CES 的诊断率。使用包含 4493 个基因的 Sophia 临床外显子组测序试剂盒进行 CES,然后在 NextSeq 500 系统上进行测序。仅包括致病性和可能致病性变异时,诊断率为 36.8%。近亲结婚和阳性家族史与高诊断率相关。神经疾病组的患者人数最多。具有高诊断率的组是耳、眼和肌肉疾病组。确定了 7 个尚未与人类疾病相关的候选基因(EFHC2、HSPB3、FAAH2、ITGB1、GYG2、CD177 和 CSTF2T)。由于 CES 的诊断率高于其他遗传测试,因此可以将其用作需要广泛鉴别诊断的罕见遗传疾病患者的标准诊断测试,特别是在资源有限的实验室中。