Franklin Andrew D, Chaudhari Bimal P, Koboldt Daniel C, Machut Kerri Z
Division of Neonatology, NorthShore University HealthSystem, Evanston, IL, United States.
Division of Genetic and Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, United States.
Front Genet. 2021 Jun 14;12:664278. doi: 10.3389/fgene.2021.664278. eCollection 2021.
A 32-week premature infant presented with respiratory failure, later progressing to pulmonary hypertension (PH), liver failure, lactic acidosis, and encephalopathy. Using exome sequencing, this patient was diagnosed with a rare Polymerase Gamma (POLG)-related mitochondrial DNA (mtDNA) depletion syndrome. This case demonstrates that expanding the differential to uncommon diagnoses is important for complex infants, even in premature neonates whose condition may be explained partially by their gestational age (GA). It also shows that patients with complex neonatal diseases with significant family history may benefit from exome sequencing for diagnosis.
一名32周早产婴儿出现呼吸衰竭,随后发展为肺动脉高压(PH)、肝功能衰竭、乳酸性酸中毒和脑病。通过外显子组测序,该患者被诊断为一种罕见的与聚合酶γ(POLG)相关的线粒体DNA(mtDNA)耗竭综合征。该病例表明,对于病情复杂的婴儿,即使是那些病情可能部分由其孕周(GA)解释的早产儿,扩大鉴别诊断范围以考虑罕见诊断也很重要。它还表明,有明显家族病史的复杂新生儿疾病患者可能受益于外显子组测序以进行诊断。