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科斯特洛综合征中的呼吸系统受累情况。

Respiratory system involvement in Costello syndrome.

作者信息

Gomez-Ospina Natalia, Kuo Christin, Ananth Amitha Lakshmi, Myers Angela, Brennan Marie-Luise, Stevenson David A, Bernstein Jonathan A, Hudgins Louanne

机构信息

Division of Medical Genetics, Stanford University School of Medicine, Stanford, California.

Division of Pulmonary Medicine, Stanford University School of Medicine, Stanford, California.

出版信息

Am J Med Genet A. 2016 Jul;170(7):1849-57. doi: 10.1002/ajmg.a.37655. Epub 2016 Apr 22.

Abstract

Costello syndrome (CS) is a multisystem disorder caused by heterozygous germline mutations in the HRAS proto-oncogene. Respiratory system complications have been reported in individuals with CS, but a comprehensive description of the full spectrum and incidence of respiratory symptoms in these patients is not available. Here, we report the clinical course of four CS patients with respiratory complications as a major cause of morbidity. Review of the literature identified 56 CS patients with descriptions of their neonatal course and 17 patients in childhood/adulthood. We found that in the neonatal period, respiratory complications are seen in approximately 78% of patients with transient respiratory distress reported in 45% of neonates. Other more specific respiratory diagnoses were reported in 62% of patients, the majority of which comprised disorders of the upper and lower respiratory tract. Symptoms of upper airway obstruction were reported in CS neonates but were more commonly diagnosed in childhood/adulthood (71%). Analysis of HRAS mutations and their respiratory phenotype revealed that the common p.Gly12Ser mutation is more often associated with transient respiratory distress and other respiratory diagnoses. Respiratory failure and dependence on mechanical ventilation occurs almost exclusively with rare mutations. In cases of prenatally diagnosed CS, the high incidence of respiratory complications in the neonatal period should prompt anticipatory guidance and development of a postnatal management plan. This may be important in cases involving rarer mutations. Furthermore, the high frequency of airway obstruction in CS patients suggests that otorhinolaryngological evaluation and sleep studies should be considered. © 2016 Wiley Periodicals, Inc.

摘要

科斯特洛综合征(CS)是一种由HRAS原癌基因杂合种系突变引起的多系统疾病。已有报道称CS患者会出现呼吸系统并发症,但目前尚无对这些患者呼吸系统症状的全貌和发生率的全面描述。在此,我们报告了4例以呼吸系统并发症为主要发病原因的CS患者的临床病程。文献回顾确定了56例有新生儿期病程描述的CS患者以及17例儿童期/成人期患者。我们发现,在新生儿期,约78%的患者出现呼吸系统并发症,45%的新生儿有短暂性呼吸窘迫的报告。62%的患者有其他更具体的呼吸系统诊断,其中大多数包括上、下呼吸道疾病。CS新生儿有上气道梗阻症状的报告,但在儿童期/成人期更常被诊断(71%)。对HRAS突变及其呼吸表型的分析表明,常见的p.Gly12Ser突变更常与短暂性呼吸窘迫和其他呼吸系统诊断相关。呼吸衰竭和对机械通气的依赖几乎仅发生于罕见突变的情况。在产前诊断为CS的病例中,新生儿期呼吸系统并发症的高发生率应促使进行预期指导并制定产后管理计划。这在涉及罕见突变的病例中可能很重要。此外,CS患者气道梗阻的高频率表明应考虑进行耳鼻喉科评估和睡眠研究。© 2016威利期刊公司

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