遗传性佝偻病:儿科医生快速指南》。

IF 1.3 Q3 PEDIATRICS
Abdulmajeed AlSubaihin, Jennifer Harrington
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引用次数: 0

摘要

随着越来越多与维生素 D 代谢以及钙磷平衡调节有关的基因被发现,越来越多的遗传性佝偻病现已被确认。这些遗传性佝偻病可分为钙生成性佝偻病和磷原性佝偻病。钙生成型遗传性佝偻病是由于基因突变改变了维生素 D 激活途径中的酶活性或损害了维生素 D 受体的作用而引起的。另一方面,遗传性磷酸化性佝偻病是由于基因突变导致 FGF23 激素表达增加或损害了近端肾小管对磷酸盐的吸收能力。由于获得性佝偻病和遗传性佝偻病的临床表现存在重叠,因此识别遗传性佝偻病患儿具有挑战性。清楚地了解遗传性佝偻病的分子基础及其相关的生化模式,可以让医疗服务提供者做出正确的诊断,避免无效的干预,并缩短这些儿童的诊断时间。在这篇微型综述中,将讨论在线人类孟德尔遗传数据库(Online Mendelian Inheritance in Man)中列出的已知遗传性佝偻病形式。此外,还提出了识别和诊断遗传性佝偻病儿童的临床方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Hereditary Rickets: A Quick Guide for the Pediatrician.

With the increased discovery of genes implicated in vitamin D metabolism and the regulation of calcium and phosphate homeostasis, a growing number of genetic forms of rickets are now recognized. These are categorized into calciopenic and phosphopenic rickets. Calciopenic forms of hereditary rickets are caused by genetic mutations that alter the enzymatic activity in the vitamin D activation pathway or impair the vitamin D receptor action. Hereditary forms of phosphopenic rickets, on the other hand, are caused by genetic mutations that lead to increased expression of FGF23 hormone or that impair the absorptive capacity of phosphate at the proximal renal tubule. Due to the clinical overlap between acquired and genetic forms of rickets, identifying children with hereditary rickets can be challenging. A clear understanding of the molecular basis of hereditary forms of rickets and their associated biochemical patterns allow the health care provider to assign the correct diagnosis, avoid non-effective interventions and shorten the duration of the diagnostic journey in these children. In this mini-review, known forms of hereditary rickets listed on the Online Mendelian Inheritance in Man database are discussed. Further, a clinical approach to identify and diagnose children with hereditary forms of rickets is suggested.

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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
66
期刊介绍: Current Pediatric Reviews publishes frontier reviews on all the latest advances in pediatric medicine. The journal’s aim is to publish the highest quality review articles dedicated to clinical research in the field. The journal is essential reading for all researchers and clinicians in pediatric medicine.
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