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小眼畸形综合征9:一例患有肺发育不全、膈膨出、小眼畸形、心脏缺陷和重度原发性肺动脉高压的新生儿病例报告

Microphthalmia Syndrome 9: Case Report of a Newborn Baby with Pulmonary Hypoplasia, Diaphragmatic Eventration, Microphthalmia, Cardiac Defect and Severe Primary Pulmonary Hypertension.

作者信息

Andijani Abdurahman A, Shajira Eman S, Abushaheen Amani, Al-Matary Abdulrahman

机构信息

Neonatal Intensive Care Unit, King Fahad Medical City, Children Specialized Hospital, Riyadh, Saudi Arabia.

Department of Pediatrics, Bahrain Defense Force Hospital, Riffa, Bahrain.

出版信息

Am J Case Rep. 2019 Mar 18;20:354-360. doi: 10.12659/AJCR.912873.

Abstract

BACKGROUND The pulmonary hypoplasia/agenesis, diaphragmatic hernia/eventration, anophthalmia/microphthalmia, and cardiac defect (PDAC) syndrome is a rare medical condition presumably of autosomal recessive way of inheritance with only a few reported cases. Recessive mutations in the STRA6 and both recessive and dominant mutations in RARB gene have been identified as the cause of anophthalmia/microphthalmia and other abnormalities included in the PDAC spectrum. However, those mutations have not been found in all PDAC syndrome cases reviewed from the literature. CASE REPORT We report a case of a full-term living male infant with pulmonary hypoplasia, left diaphragmatic eventration, bilateral microphthalmia, congenital cardiac defects, and severe pulmonary hypertension. CONCLUSIONS The main feature in the reported cases was anophthalmia/microphthalmia. Therefore, screening for the other associated congenital anomalies is highly suggested. Mutations in STRA6 and RARB genes are commonly encountered in this spectrum. However, whole exome sequencing of suspected cases and their parents is recommended to detect possible de novo mutations. Further reports are needed to identify risk factors and prognosis of this rare syndrome.

摘要

背景

肺发育不全/肺不发育、膈疝/膈膨出、无眼/小眼、心脏缺陷(PDAC)综合征是一种罕见的医学病症,可能以常染色体隐性方式遗传,仅有少数病例报道。STRA6基因的隐性突变以及RARB基因的隐性和显性突变已被确定为无眼/小眼及PDAC谱系中其他异常的病因。然而,在从文献中回顾的所有PDAC综合征病例中并未发现这些突变。病例报告:我们报告一例足月存活男婴,患有肺发育不全、左侧膈膨出、双侧小眼、先天性心脏缺陷和严重肺动脉高压。结论:所报告病例的主要特征是无眼/小眼。因此,强烈建议筛查其他相关先天性异常。在该谱系中常见STRA6和RARB基因的突变。然而,建议对疑似病例及其父母进行全外显子测序以检测可能的新发突变。需要更多报告来确定这种罕见综合征的危险因素和预后。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1862/6434610/27a3135d7722/amjcaserep-20-354-g001.jpg

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