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LEOPARD 综合征伴 PTPN11 基因突变 Y279C 及不同皮肤表现:两例病例报告及文献复习。

LEOPARD syndrome with recurrent PTPN11 mutation Y279C and different cutaneous manifestations: two case reports and a review of the literature.

机构信息

Department of Human Biology and Genetics, Institute of General and Molecular Pathology, University of Tartu, Tartu, Estonia.

出版信息

Eur J Pediatr. 2010 Apr;169(4):469-73. doi: 10.1007/s00431-009-1058-1. Epub 2009 Sep 20.

Abstract

LEOPARD syndrome (LS) is a heterogeneous disease characterised mainly by cutaneous manifestations. LEOPARD is the acronym for its major features-multiple lentigines, electrocardiographic conduction defects, ocular hypertelorism, pulmonary stenosis, abnormalities of (male) genitalia, retardation of growth and sensorineural deafness. As clinical manifestations are variable, molecular testing is supportive in the diagnosis of LS. We describe two unrelated LS cases with a common PTPN11 mutation Y279C and with completely different clinical features including distinct changes in skin pigmentation. In patient 1, the first complaint was hyperactive behaviour. First lentigines were presented at birth, but intensive growth began at the age of 2-4 years. Multiple dark lentigines were located mainly on the face and the upper part of the trunk, but the oral mucosa was spared. Patient 2 was born from induced labour due to polyhydramnion, and in the second week of life, mitral valve insufficiency and hypertrophic cardiomyopathy were diagnosed. Rapid growth of lentigines began at the age of 3 years. These are mostly located in the joint areas in the lower extremities; the face and upper trunk are spared from lentigines. In both cases, the rapid growth of lentigines made it possible to shift the diagnosis towards LS. Clinicians should give more consideration to rare genetic syndromes, especially in the case of symptoms from different clinical areas.

摘要

莱博特综合征(LS)是一种异质性疾病,主要表现为皮肤表现。LEOPARD 是其主要特征的首字母缩写-多发性痣、心电图传导缺陷、眼距过宽、肺动脉瓣狭窄、(男性)生殖器异常、生长迟缓及感觉神经性耳聋。由于临床表现多样,分子检测有助于 LS 的诊断。我们描述了两例不相关的 LS 病例,均存在共同的 PTPN11 突变 Y279C,但临床表现完全不同,包括皮肤色素沉着的明显变化。在患者 1 中,首发症状为多动行为。出生时即出现第一个痣,但 2-4 岁开始快速生长。多发性深色痣主要位于面部和上半身,但口腔黏膜未受累。患者 2 因羊水过多行引产出生,在出生后第 2 周,诊断为二尖瓣关闭不全和肥厚型心肌病。痣的快速生长始于 3 岁。这些痣大多位于下肢关节部位,面部和上半身没有痣。在这两种情况下,痣的快速生长使 LS 的诊断成为可能。临床医生应更多地考虑罕见的遗传综合征,尤其是在不同临床领域出现症状的情况下。

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