无明显家族史的常染色体显性多囊肾病:单中心经验。

IF 1.1 4区 医学 Q3 UROLOGY & NEPHROLOGY
Marrissa Samuels, Omar Maarouf, Wing Zeng, Mehrdad Hamrahian, Neera K Dahl, Jingjing Zhang
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引用次数: 0

摘要

常染色体显性多囊肾病(ADPKD)是最常见的遗传性肾病,占肾衰竭患者的近5%。由于新生突变或其他原因,近25%临床诊断为PKD的患者没有明显的家族史。这对肾病医师做出准确诊断提出了挑战。我们评估了有和没有家族病史的ADPKD患者,并比较了基因检测、肾脏成像和肾功能结果。我们回顾了2020年至2024年间托马斯杰斐逊大学医院54名PKD患者的基因检测结果。共有38例(70%)患者有ADPKD家族史。在这些患者中,92%有PKD1或PKD2致病变异。16例(30%)无家族史。在这些患者中,56%有PKD1或PKD2致病变异,19%有与ADPKD相关的次要基因变异。5例患者(9%)没有遗传诊断。我们提出了一种算法,以帮助分类患者与ADPKD表型但没有家族史。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Autosomal dominant polycystic kidney disease without apparent family history: A single-center experience.

Autosomal dominant polycystic kidney disease (ADPKD) - the most common hereditary kidney disease - accounts for nearly 5% of patients with kidney failure. Due to de novo mutations or other causes, nearly 25% of patients with a clinical diagnosis of PKD have no apparent family history. This poses a challenge for practicing nephrologists in making an accurate diagnosis. We evaluated ADPKD patients with and without a family history and compared genetic testing, kidney imaging, and kidney function results. We reviewed the genetic testing results of 54 PKD patients at Thomas Jefferson University Hospital between 2020 and 2024. A total of 38 patients (70%) had a family history of ADPKD. Of these patients, 92% had either a PKD1 or PKD2 disease-causing variant. 16 patients (30%) had no family history. Of these patients, 56% had either a PKD1 or PKD2 disease-causing variant and 19% had variants in minor genes associated with ADPKD. Five patients (9%) had no genetic diagnosis. We propose an algorithm to help classify patients with an ADPKD phenotype but without family history.

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来源期刊
Clinical nephrology
Clinical nephrology 医学-泌尿学与肾脏学
CiteScore
2.10
自引率
9.10%
发文量
138
审稿时长
4-8 weeks
期刊介绍: Clinical Nephrology appears monthly and publishes manuscripts containing original material with emphasis on the following topics: prophylaxis, pathophysiology, immunology, diagnosis, therapy, experimental approaches and dialysis and transplantation.
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