通过快速基因组测序诊断新生儿肺泡毛细血管发育不良:一个新的金标准?

IF 2.6 3区 医学 Q1 PEDIATRICS Neonatology Pub Date : 2023-01-01 DOI:10.1159/000529439
Whitney S Thompson, Ellen M Bendel-Stenzel, Brendan C Lanpher, Grace M Arteaga, Raymond C Stetson, Stephanie C Mavis
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引用次数: 0

摘要

典型肺泡毛细血管发育不良伴肺静脉错位(ACDMPV)是一种罕见的先天性肺部疾病,表现为新生儿早期难治性低氧性呼吸衰竭和肺动脉高压。目前尚无有效的治疗方法。虽然明确的诊断是通过组织学,肺活检往往是具有挑战性的不稳定,危重新生儿。通过染色体微阵列和靶向基因测序实现了分子诊断;然而,每种方法都可能受到周转时间、基因组覆盖范围以及无法检测到ACDMPV所有致病变异类型的限制。我们报告了一个通过快速基因组测序诊断的ACDMPV病例,并假设快速基因组测序,包括快速外显子组和基因组测序,作为一种全面和无创的及时诊断方法,在严重新生儿呼吸衰竭中发挥着越来越大的作用。
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Neonatal Diagnosis of Alveolar Capillary Dysplasia via Rapid Genomic Sequencing: A New Gold Standard?

Classic alveolar capillary dysplasia with misalignment of pulmonary veins (ACDMPV) is a rare congenital lung disorder presenting in the early neonatal period with refractory hypoxemic respiratory failure and pulmonary hypertension. No curative treatment is currently available. Although definitive diagnosis is obtained by histology, lung biopsy is often challenging in unstable, critically ill neonates. Molecular diagnosis has been achieved with chromosomal microarray and targeted gene sequencing; however, each of these modalities can be limited by turnaround time, coverage of the genome, and inability to detect all pathogenic variant types for ACDMPV. We present a case of ACDMPV diagnosed via rapid genome sequencing and posit that rapid genomic sequencing, including both rapid exome and genome sequencing, has an expanding role in severe neonatal respiratory failure as a comprehensive and noninvasive approach to timely diagnosis.

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来源期刊
Neonatology
Neonatology 医学-小儿科
CiteScore
0.60
自引率
4.00%
发文量
91
审稿时长
6-12 weeks
期刊介绍: This highly respected and frequently cited journal is a prime source of information in the area of fetal and neonatal research. Original papers present research on all aspects of neonatology, fetal medicine and developmental biology. These papers encompass both basic science and clinical research including randomized trials, observational studies and epidemiology. Basic science research covers molecular biology, molecular genetics, physiology, biochemistry and pharmacology in fetal and neonatal life. In addition to the classic features the journal accepts papers for the sections Research Briefings and Sources of Neonatal Medicine (historical pieces). Papers reporting results of animal studies should be based upon hypotheses that relate to developmental processes or disorders in the human fetus or neonate.
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