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婴儿痉挛症的新型拷贝数变异和表型。

Novel copy number variations and phenotypes of infantile epileptic spasms syndrome.

机构信息

Department of Pediatrics, Peking University First Hospital, Beijing, China.

Research and Development Center, Beijing USCI Medical Laboratory Co., Ltd, Beijing, China.

出版信息

Clin Genet. 2024 Aug;106(2):161-179. doi: 10.1111/cge.14520. Epub 2024 Mar 28.

Abstract

We summarize the copy number variations (CNVs) and phenotype spectrum of infantile epileptic spasms syndrome (IESS) in a Chinese cohort. The CNVs were identified by genomic copy number variation sequencing. The CNVs and clinical data were analyzed. 74 IESS children with CNVs were enrolled. 35 kinds of CNVs were identified. There were 11 deletions and 5 duplications not reported previously in IESS, including 2 CNVs not reported in epilepsy. 87.8% were de novo, 9.5% were inherited from mother and 2.7% from father. Mosaicism occurred in one patient with Xq21.31q25 duplication. 16.2% (12/74) were 1p36 deletion, and 20.3% (15/74) were 15q11-q13 duplication. The age of seizure onset ranged from 17 days to 24 months. Seizure types included epileptic spasms, focal seizures, tonic seizures, and myoclonic seizures. All patients displayed developmental delay. Additional features included craniofacial anomaly, microcephaly, congenital heart defects, and hemangioma. 29.7% of patients were seizure-free for more than 12 months, and 70.3% still had seizures after trying 2 or more anti-seizure medications. In conclusion, CNVs is a prominent etiology of IESS. 1p36 deletion and 15q duplication occurred most frequently. CNV detection should be performed in patients with IESS of unknown causes, especially in children with craniofacial anomalies and microcephaly.

摘要

我们总结了一个中国队列中婴儿痉挛症(IESS)的拷贝数变异(CNVs)和表型谱。通过基因组拷贝数变异测序鉴定 CNVs。分析 CNVs 和临床数据。纳入 74 例有 CNVs 的 IESS 患儿。确定了 35 种 CNVs。有 11 种缺失和 5 种以前未报道过的重复,包括 2 种未报道过的癫痫 CNVs。87.8%为新生突变,9.5%来自母亲,2.7%来自父亲。一位 Xq21.31q25 重复患者存在嵌合体。16.2%(12/74)为 1p36 缺失,20.3%(15/74)为 15q11-q13 重复。发病年龄从 17 天到 24 个月不等。发作类型包括痉挛性发作、局灶性发作、强直发作和肌阵挛发作。所有患者均有发育迟缓。其他特征包括颅面畸形、小头畸形、先天性心脏缺陷和血管瘤。29.7%的患者无癫痫发作超过 12 个月,70.3%的患者在尝试 2 种或更多抗癫痫药物后仍有癫痫发作。总之,CNVs 是 IESS 的一个突出病因。1p36 缺失和 15q 重复最常见。对于病因不明的 IESS 患者,尤其是伴有颅面畸形和小头畸形的患者,应进行 CNV 检测。

相似文献

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Infantile spasms are associated with abnormal copy number variations.婴儿痉挛症与异常拷贝数变异有关。
J Child Neurol. 2013 Oct;28(10):1191-6. doi: 10.1177/0883073812453496. Epub 2012 Aug 21.

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