HNF1B 缺陷的全貌:一个尚未充分探索的综合征。
The Landscape of HNF1B Deficiency: A Syndrome Not Yet Fully Explored.
机构信息
Department of Medical Sciences, University of Turin, 10126 Turin, Italy.
Division of Liver and Transplant Pathology, University of Pittsburgh, Pittsburgh, PA 15232, USA.
出版信息
Cells. 2023 Jan 13;12(2):307. doi: 10.3390/cells12020307.
The hepatocyte nuclear factor 1β (HNF1B) gene is involved in the development of specialized epithelia of several organs during the early and late phases of embryogenesis, performing its function mainly by regulating the cell cycle and apoptosis pathways. The first pathogenic variant of HNF1B (namely, R177X) was reported in 1997 and is associated with the maturity-onset diabetes of the young. Since then, more than 230 different HNF1B variants have been reported, revealing a multifaceted syndrome with complex and heterogenous genetic, pathologic, and clinical profiles, mainly affecting the pediatric population. The pancreas and kidneys are the most frequently affected organs, resulting in diabetes, renal cysts, and a decrease in renal function, leading, in 2001, to the definition of HNF1B deficiency syndrome, including renal cysts and diabetes. However, several other organs and systems have since emerged as being affected by HNF1B defect, while diabetes and renal cysts are not always present. Especially, liver involvement has generally been overlooked but recently emerged as particularly relevant (mostly showing chronically elevated liver enzymes) and with a putative relation with tumor development, thus requiring a more granular analysis. Nowadays, HNF1B-associated disease has been recognized as a clinical entity with a broader and more variable multisystem phenotype, but the reasons for the phenotypic heterogeneity are still poorly understood. In this review, we aimed to describe the multifaceted nature of HNF1B deficiency in the pediatric and adult populations: we analyzed the genetic, phenotypic, and clinical features of this complex and misdiagnosed syndrome, covering the most frequent, unusual, and recently identified traits.
肝细胞核因子 1β(HNF1B)基因参与胚胎发生的早期和晚期阶段几个器官的特化上皮的发育,主要通过调节细胞周期和细胞凋亡途径发挥其功能。HNF1B 的第一个致病变异体(即 R177X)于 1997 年报道,与青年发病的成年型糖尿病有关。此后,已经报道了超过 230 种不同的 HNF1B 变异体,揭示了一种具有复杂和异质性遗传、病理和临床特征的多面综合征,主要影响儿科人群。胰腺和肾脏是最常受影响的器官,导致糖尿病、肾囊肿和肾功能下降,这导致 2001 年 HNF1B 缺乏综合征的定义,包括肾囊肿和糖尿病。然而,此后已经出现了几个其他器官和系统受到 HNF1B 缺陷的影响,而糖尿病和肾囊肿并不总是存在。特别是,肝脏受累通常被忽视,但最近被认为特别相关(主要表现为慢性升高的肝酶),并与肿瘤发展有关,因此需要更细致的分析。如今,HNF1B 相关疾病已被认为是一种具有更广泛和更多变的多系统表型的临床实体,但表型异质性的原因仍知之甚少。在这篇综述中,我们旨在描述 HNF1B 缺乏在儿科和成人中的多面性质:我们分析了这种复杂和误诊综合征的遗传、表型和临床特征,涵盖了最常见、不常见和最近发现的特征。