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患者存在股骨发育不全、精神运动发育迟缓、心脏缺损和面部畸形,存在纯从头 16p13.3 重复和扩增——病例报告及部分 16p13.3 三体综合征文献复习。

A pure de novo 16p13.3 duplication and amplification in a patient with femoral hypoplasia, psychomotor retardation, heart defect, and facial dysmorphism-a case report and literature review of the partial 16p13.3 trisomy syndrome.

机构信息

Department of Medical Genetics, Poznan University of Medical Sciences, Rokietnicka 8, 60-806, Poznań, Poland.

Centers for Medical Genetics GENESIS, Dąbrowskiego 77A, 60-529, Poznań, Poland.

出版信息

J Appl Genet. 2023 Feb;64(1):125-134. doi: 10.1007/s13353-022-00743-7. Epub 2022 Dec 31.

Abstract

Partial 16p trisomy syndrome is a rare disorder typically characterized by psychomotor retardation, prenatal and postnatal growth deficiency, cleft palate, and facial dysmorphism, with some patients also presenting with heart defects and urogenital anomalies. Pure 16p13.3 duplications usually occur de novo, while those duplications that associate with partial monosomy result rather from parental chromosomal translocations. Due to the large size of the aberrations, the majority of patients are identified by standard chromosome analysis. In all published cases, the minimal-causative duplicated region encompasses the CREBBP gene. Here, we report on the patient presenting with psychomotor retardation, femoral hypoplasia, and some features of the partial 16p trisomy syndrome, who carries a complex de novo terminal 16p13.3 microduplication with an overlapping region of amplification without translocation or associated monosomy. In contrast to the previously reported cases, the duplicated region of the patient does not involve CREBBP and other neighboring genes; still, the observed pattern of dysmorphic features of the index is characteristic of the described syndrome. Based on the animal studies and other published cases, we discuss the possible role of the PDK1 and IGFALS genes in the development of limb anomalies, while IFT140 could contribute both to the observed femoral phenotype and heart abnormalities in the patient. To the best of our knowledge, we present a proband harboring the smallest terminal 16p13.3 duplication of the size below 3 Mb. Therefore, our proband with her detailed phenotypic description may be helpful for clinicians who consult patients with this syndrome.

摘要

部分 16p 三体综合征是一种罕见的疾病,其典型特征为精神运动发育迟缓、产前和产后生长发育不良、腭裂以及面部畸形,部分患者还存在心脏缺陷和泌尿生殖系统异常。纯 16p13.3 重复通常为新发突变,而那些与部分单体性相关的重复则源于父母染色体易位。由于这些畸变较大,大多数患者可通过标准染色体分析得到识别。在所有已发表的病例中,最小致病重复区域均包含 CREBBP 基因。在此,我们报告了一位表现为精神运动发育迟缓、股骨发育不全以及部分 16p 三体综合征特征的患者,该患者携带一个复杂的、新发的、末端 16p13.3 微重复,该重复与未发生易位或相关单体性的扩增区域存在重叠。与之前报道的病例不同,患者的重复区域不包含 CREBBP 和其他相邻基因;尽管如此,该患者的畸形特征表现与所描述的综合征一致。基于动物研究和其他已发表的病例,我们讨论了 PDK1 和 IGFALS 基因在肢体异常发育中的可能作用,而 IFT140 可能导致患者出现所观察到的股骨表型和心脏异常。据我们所知,我们提出了一个携带小于 3Mb 的最小末端 16p13.3 重复的先证者。因此,我们的先证者及其详细的表型描述可能有助于咨询该综合征患者的临床医生。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0fe8/9837002/c68062e2903e/13353_2022_743_Fig1_HTML.jpg

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