如今的Alagille综合征:“一、无一、十万”

M. Arrabito, S. D'Amico, C. Gulizia, L. Marino, G. Parisi, M. Papale, S. Manti, S. Leonardi
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引用次数: 0

摘要

Alagille综合征是一种常染色体显性遗传的复杂多系统疾病,包括广泛的临床方面,最常见于婴儿期或儿童早期。它主要是由参与Notch信号通路的基因突变引起的。临床主要表现在肝脏,但其他器官,如心脏、眼睛和骨骼也可能受损。在此,我们报道了一个家族的故事,其中四名成员被诊断患有ALGS,导致相同的基因突变。我们病例的特点在于,他们在一生中的不同时间表现出疾病,并有许多不同的症状,这突出了ALGS令人印象深刻的临床变异性,以及在肝损伤疾病的鉴别诊断中考虑它的重要性。
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Alagille Syndrome Nowadays: “One, no-One and One Hundred Thousand”
Alagille syndrome [ALGS] is an autosomal dominant, complex multisystem disorder that includes a wide range of clinical aspects, most commonly manifest in infancy or early childhood. It is mostly caused by mutations of genes involved in the Notch signaling pathway. The major of clinic manifestations occur in liver, but also other organs, like heart, eye and skeleton can be impaired. Herein we report the story of a family in which four members were diagnosed with ALGS, leading the same gene mutation. The peculiarity of our case lies in the fact that they manifested the disease in different time of their life and with many different symptoms, highlighting the impressive clinical variability of the ALGS and the importance of considering it in the differential diagnosis of liver impairment disease.
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