Whitney S Thompson, Ellen M Bendel-Stenzel, Brendan C Lanpher, Grace M Arteaga, Raymond C Stetson, Stephanie C Mavis
{"title":"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?","authors":"Whitney S Thompson, Ellen M Bendel-Stenzel, Brendan C Lanpher, Grace M Arteaga, Raymond C Stetson, Stephanie C Mavis","doi":"10.1159/000529439","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":18924,"journal":{"name":"Neonatology","volume":"120 3","pages":"395-399"},"PeriodicalIF":2.6000,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Neonatal Diagnosis of Alveolar Capillary Dysplasia via Rapid Genomic Sequencing: A New Gold Standard?\",\"authors\":\"Whitney S Thompson, Ellen M Bendel-Stenzel, Brendan C Lanpher, Grace M Arteaga, Raymond C Stetson, Stephanie C Mavis\",\"doi\":\"10.1159/000529439\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":18924,\"journal\":{\"name\":\"Neonatology\",\"volume\":\"120 3\",\"pages\":\"395-399\"},\"PeriodicalIF\":2.6000,\"publicationDate\":\"2023-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Neonatology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1159/000529439\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neonatology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1159/000529439","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
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.
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.