Department of Physical Medicine and Rehabilitation, Incheon St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.
Medical Genetics and Rare Disease Center, Incheon St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.
Sci Rep. 2023 Dec 20;13(1):22807. doi: 10.1038/s41598-023-50285-z.
Despite the current widespread use of chromosomal microarray analysis (CMA) and exome/genome sequencing for the genetic diagnosis of unexplained intellectual disability (ID) in children, gaining improved diagnostic yields and defined guidelines remains a significant challenge. This is a cohort study of children with unexplained ID. We analyzed the diagnostic yield and its correlation to clinical phenotypes in children with ID who underwent concurrent CMA and clinical exome sequencing (CES). A total of 154 children were included (110 [71.4%] male; mean [SD] age, 51.9 [23.1] months). The overall diagnosis yield was 26.0-33.8%, with CMA contributing 12.3-14.3% and CES contributing 13.6-19.4%, showing no significant difference. The diagnostic rate was significantly higher when gross motor delay (odds ratio, 6.69; 95% CI, 3.20-14.00; P < 0.001), facial dysmorphism (odds ratio, 9.34; 95% CI 4.29-20.30; P < 0.001), congenital structural anomaly (odds ratio 3.62; 95% CI 1.63-8.04; P = 0.001), and microcephaly or macrocephaly (odds ratio 4.87; 95% CI 2.05-11.60; P < 0.001) were presented. Patients with only ID without any other concomitant phenotype (63/154, 40.9%) exhibited a 6.3-11.1% diagnostic rate.
尽管目前广泛应用染色体微阵列分析(CMA)和外显子/基因组测序来对儿童不明原因智力障碍(ID)进行遗传诊断,但提高诊断率和制定明确的指南仍然是一个重大挑战。这是一项对不明原因 ID 儿童进行的队列研究。我们分析了同时进行 CMA 和临床外显子组测序(CES)的 ID 儿童的诊断率及其与临床表型的相关性。共纳入 154 名儿童(110 名[71.4%]男性;平均[标准差]年龄为 51.9[23.1]个月)。整体诊断率为 26.0-33.8%,CMA 贡献 12.3-14.3%,CES 贡献 13.6-19.4%,无显著差异。当存在粗大运动发育迟缓(比值比,6.69;95%置信区间,3.20-14.00;P<0.001)、面部畸形(比值比,9.34;95%置信区间,4.29-20.30;P<0.001)、先天性结构异常(比值比,3.62;95%置信区间,1.63-8.04;P=0.001)和小头畸形或大头畸形(比值比,4.87;95%置信区间,2.05-11.60;P<0.001)时,诊断率显著升高。仅 ID 而无其他任何伴随表型的患者(63/154,40.9%)的诊断率为 6.3-11.1%。