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一名患有X连锁低磷性佝偻病的韩国患者中存在致病性PHEX变异(c.1483-1G>C)。

A pathogenic PHEX variant (c.1483-1G>C) in a Korean patient with X-linked hypophosphatemic rickets.

作者信息

Jeong In Hwa, Yoo Jae-Ho, Kim Namhee

机构信息

Department of Laboratory Medicine, Dong-A University Hospital, Busan, Korea.

Department of Pediatrics, Dong-A University Hospital, Busan, Korea.

出版信息

Ann Pediatr Endocrinol Metab. 2021 Jun;26(2):130-133. doi: 10.6065/apem.2040186.093. Epub 2021 Jun 30.

DOI:10.6065/apem.2040186.093
PMID:34218635
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8255857/
Abstract

X-linked hypophosphatemic rickets is an X-linked dominantly inherited disorder characterized by defects in renal phosphate transport leading to phosphate wasting and hypophosphatemia. In this report, we describe a case of X-linked hypophosphatemic rickets in a patient with a rare pathogenic PHEX variant. The 25-year-old female patient came to our clinic for genetic counseling regarding presumed genetic disease and pregnancy. When she was 9 years old, she had been diagnosed with vitamin D-resistant rickets based on laboratory results and symptoms. She had undergone orthopedic surgery due to bowing leg deformities. Since then, she was intermittently self-prescribing oral phosphate and calcium supplements. At 25 years old, she was diagnosed with X-linked hypophosphatemic rickets with a rare pathogenic PHEX variant (c.1483-1G>C) by next-generation sequencing. This is the second report of the c.1483-1G>C variant to date, and her pathogenicity was confirmed based on the most recent guideline. Traditionally, the disease had been diagnosed mostly based on clinical findings. However, with advancements in genetic testing, genetic confirmation has become an imperative part of diagnostic workup. Herein, we report a 25-year-old female Korean patient diagnosed with X-linked hypophosphatemic rickets harboring a rare pathogenic PHEX variant.

摘要

X连锁低磷性佝偻病是一种X连锁显性遗传性疾病,其特征是肾磷酸盐转运缺陷,导致磷酸盐流失和低磷血症。在本报告中,我们描述了一例患有罕见致病性PHEX变异的X连锁低磷性佝偻病患者。这位25岁的女性患者因疑似遗传病和妊娠前来我们诊所进行遗传咨询。她9岁时,根据实验室检查结果和症状被诊断为维生素D抵抗性佝偻病。由于腿部弓形畸形,她接受了矫形手术。从那时起,她断断续续地自行服用口服磷酸盐和钙补充剂。25岁时,通过下一代测序,她被诊断为患有罕见致病性PHEX变异(c.1483-1G>C)的X连锁低磷性佝偻病。这是迄今为止关于c.1483-1G>C变异的第二篇报告,根据最新指南证实了其致病性。传统上,该疾病大多根据临床症状进行诊断。然而,随着基因检测技术的进步,基因确诊已成为诊断检查的必要组成部分。在此,我们报告一例25岁的韩国女性患者,她被诊断为患有罕见致病性PHEX变异的X连锁低磷性佝偻病。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/33ec/8255857/ae75f657b1c1/apem-2040186-093f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/33ec/8255857/ae75f657b1c1/apem-2040186-093f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/33ec/8255857/ae75f657b1c1/apem-2040186-093f1.jpg

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