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面对缩短诊断探索之旅的挑战:哥伦比亚疑似罕见病队列的首次全基因组测序经验。

Facing the challenges to shorten the diagnostic odyssey: first Whole Genome Sequencing experience of a Colombian cohort with suspected rare diseases.

机构信息

Personalized Medicine Group, Unidad de Bioentendimiento, Bioscience Center, Ayudas Diagnósticas SURA, Medellín, Colombia.

CENTOGENE GmbH, Rostock, Germany.

出版信息

Eur J Hum Genet. 2024 Oct;32(10):1327-1337. doi: 10.1038/s41431-024-01609-8. Epub 2024 Jun 22.

DOI:10.1038/s41431-024-01609-8
PMID:38909121
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11499989/
Abstract

Exome and genome sequencing (ES/GS) are routinely used for the diagnosis of genetic diseases in developed countries. However, their implementation is limited in countries from Latin America. We aimed to describe the results of GS in patients with suspected rare genetic diseases in Colombia. We studied 501 patients from 22 healthcare sites from January to December 2022. GS was performed in the index cases using dried blood spots on filtercards. Ancestry analysis was performed under iAdmix. Multiomic testing was performed when needed (biomarker, enzymatic activity, RNA-seq). All tests were performed at an accredited genetic laboratory. Ethnicity prediction data confirmed that 401 patients (80%) were mainly of Amerindian origin. A genetic diagnosis was established for 142 patients with a 28.3% diagnostic yield. The highest diagnostic yield was achieved for pathologies with a metabolic component and syndromic disorders (p < 0.001). Young children had a median of 1 year of diagnostic odyssey, while the median time for adults was significantly longer (15 years). Patients with genetic syndromes have spent more than 75% of their life without a diagnosis, while for patients with neurologic and neuromuscular diseases, the time of the diagnostic odyssey tended to decrease with age. Previous testing, specifically karyotyping or chromosomal microarray were significantly associated with a longer time to reach a definitive diagnosis (p < 0.01). Furthermore, one out of five patients that had an ES before could be diagnosed by GS. The Colombian genome project is the first Latin American study reporting the experience of systematic use of diagnostic GS in rare diseases.

摘要

外显子组和基因组测序 (ES/GS) 已被常规用于发达国家遗传疾病的诊断。然而,在拉丁美洲国家,其应用受到限制。本研究旨在描述哥伦比亚疑似罕见遗传疾病患者进行 GS 的结果。我们对 2022 年 1 月至 12 月期间来自 22 个医疗保健地点的 501 名患者进行了研究。使用滤纸片上的干血斑对索引病例进行 GS 检测。在 iAdmix 下进行祖先分析。在需要时进行多组学检测(生物标志物、酶活性、RNA-seq)。所有检测均在经过认证的遗传实验室进行。种族预测数据证实,401 名患者(80%)主要来自美洲印第安人。142 名患者确定了遗传诊断,诊断率为 28.3%。具有代谢成分和综合征的病理诊断率最高(p < 0.001)。幼儿的中位诊断探索时间为 1 年,而成年人的中位时间明显更长(15 年)。有遗传综合征的患者一生中超过 75%的时间没有得到诊断,而对于神经和神经肌肉疾病的患者,诊断探索时间随着年龄的增长而趋于减少。之前的检测,特别是核型分析或染色体微阵列分析,与达到明确诊断的时间较长显著相关(p < 0.01)。此外,进行 ES 检测的患者中有五分之一可以通过 GS 进行诊断。哥伦比亚基因组计划是首个报告在罕见疾病中系统使用诊断性 GS 的拉丁美洲研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/336410dc4c10/41431_2024_1609_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/7dcb1aa8df50/41431_2024_1609_Fig1_HTML.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/336410dc4c10/41431_2024_1609_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/7dcb1aa8df50/41431_2024_1609_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/44fcf5263e9e/41431_2024_1609_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/4f6d3d9f7591/41431_2024_1609_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7235/11499989/948db4c7e769/41431_2024_1609_Fig4_HTML.jpg
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