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病因不明的慢性肾脏病 5 期年轻患者的遗传学研究。GENSEN 研究设计。

Genetic study in young patients with chronic kidney disease stage G5 from unknown etiology. The GENSEN study design.

机构信息

Servicio de Nefrología y Trasplante Renal, Centro de Referencia en Enfermedad Glomerular Compleja del Sistema Nacional de Salud (CSUR), IDIBAPS, Hospital Clínic, Universitat de Barcelona, Barcelona, Spain; RICORS2040, Madrid, Spain.

RICORS2040, Madrid, Spain; IIS-La Princesa, Servicio de Nefrología, Hospital Universitario de la Princesa, Madrid, Spain.

出版信息

Nefrologia (Engl Ed). 2024 Jul-Aug;44(4):568-575. doi: 10.1016/j.nefroe.2023.09.010. Epub 2024 Jul 25.

Abstract

INTRODUCTION

Chronic kidney disease (CKD) of non-inherited etiology is one of the main causes of renal replacement therapy in our setting. Previous studies in other territories suggest that hereditary diseases could be one of the potential causes of this pathology, especially in younger patients. The GENSEN study will evaluate the presence of pathogenic genetic variants in subjects who have developed CKD category G5 before the age of 46 years, of non-inherited etiology.

METHODS

Observational, prospective, multicenter study, which evaluates the diagnostic utility of massive high-throughput sequencing (HTS) directed to a set of genes, in the identification of the cause of CKD. Patients from all over Spain will be included, from whom a blood or saliva sample will be taken and a panel of 529 genes associated with hereditary kidney disease will be analyzed. This publication communicates the study protocol.

CONCLUSION

The GENSEN study will make it possible to evaluate the diagnostic performance of the gene panel study in young subjects in our setting with the development of CKD category G5 without a clear cause. An etiological diagnosis would offer potential benefits for patients and relatives (targeted therapies, clinical trials, detection of extrarenal manifestations, evaluation of relatives for live donation, estimation of the risk of recurrence in the renal graft, genetic counseling, among others) and would allow to apply this genetic study to the nephrology of our country.

摘要

简介

非遗传性病因导致的慢性肾脏病(CKD)是我们治疗环境中肾脏替代治疗的主要原因之一。其他地区的先前研究表明,遗传性疾病可能是导致这种病理的潜在原因之一,尤其是在年轻患者中。GENSEN 研究将评估在 46 岁之前患有非遗传性病因导致的 CKD G5 类别的患者中,是否存在致病性遗传变异。

方法

这是一项观察性、前瞻性、多中心研究,评估了针对一组基因的大规模高通量测序(HTS)在确定 CKD 病因方面的诊断效用。该研究将纳入来自西班牙各地的患者,采集其血液或唾液样本,并分析与遗传性肾脏疾病相关的 529 个基因。本出版物介绍了该研究方案。

结论

GENSEN 研究将评估在我们的治疗环境中,对病因不明的发展为 CKD G5 类别的年轻患者进行基因panel 研究的诊断性能。病因诊断将为患者和亲属带来潜在的益处(靶向治疗、临床试验、发现肾外表现、评估活体供者、估计肾移植复发风险、遗传咨询等),并使该基因研究可应用于我国的肾病学。

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