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中东和北非地区儿童常染色体隐性多囊肾病的流行病学和结局。

Epidemiology and outcomes of pediatric autosomal recessive polycystic kidney disease in the Middle East and North Africa.

机构信息

CTI Clinical Trial and Consulting Services, Cairo, Egypt.

Smart Health Unit, University of East London, London, E16 2, UK.

出版信息

Pediatr Nephrol. 2024 Sep;39(9):2569-2578. doi: 10.1007/s00467-024-06281-0. Epub 2024 Jan 23.

DOI:10.1007/s00467-024-06281-0
PMID:38261064
Abstract

The incidence of rare diseases is expected to be comparatively higher in the Middle East and North Africa (MENA) region than in other parts of the world, attributed to the high prevalence of consanguinity. Most MENA countries share social and economic statuses, cultural relativism, religious beliefs, and healthcare policies. Polycystic kidney diseases (PKDs) are the most common genetic causes of kidney failure, accounting for nearly 8.0% of dialysis cases. The development of PKDs is linked to variants in several genes, including PKD1, PKD2, PKHD1, DZIP1L, and CYS1. Autosomal recessive PKD (ARPKD) is the less common yet aggressive form of PKD. ARPKD has an estimated incidence between 1:10,000 and 1:40,000. Most patients with ARPKD require kidney replacement therapy earlier than patients with autosomal dominant polycystic kidney disease (ADPKD), often in their early years of life. This review gathered data from published research studies and reviews of ARPKD, highlighting the epidemiology, phenotypic presentation, investigations, genetic analysis, outcomes, and management. Although limited data are available, the published literature suggests that the incidence of ARPKD may be higher in the MENA region due to consanguineous marriages. Patients with ARPKD from the MENA region usually present at a later disease stage and have a relatively short time to progress to kidney failure. Limited data are available regarding the management practice in the region, which warrants further investigations.

摘要

中东和北非(MENA)地区的罕见病发病率预计比世界其他地区高,这归因于近亲结婚的高发率。大多数 MENA 国家具有相似的社会和经济地位、文化相对论、宗教信仰和医疗保健政策。多囊肾病(PKD)是导致肾衰竭的最常见遗传原因,占透析病例的近 8.0%。PKD 的发展与几个基因的变异有关,包括 PKD1、PKD2、PKHD1、DZIP1L 和 CYS1。常染色体隐性多囊肾病(ARPKD)是 PKD 中较为罕见但侵袭性较强的形式。ARPKD 的发病率估计在 1:10000 至 1:40000 之间。大多数 ARPKD 患者需要比常染色体显性多囊肾病(ADPKD)患者更早进行肾脏替代治疗,通常在生命的早期。本综述从已发表的 ARPKD 研究和综述中收集数据,重点介绍了 ARPKD 的流行病学、表型表现、检查、基因分析、结果和管理。尽管可用数据有限,但已发表的文献表明,由于近亲结婚,ARPKD 的发病率在 MENA 地区可能更高。来自 MENA 地区的 ARPKD 患者通常在疾病后期出现,并在较短时间内进展为肾衰竭。关于该地区管理实践的可用数据有限,需要进一步调查。

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本文引用的文献

1
Phenotypic Variability in Siblings With Autosomal Recessive Polycystic Kidney Disease.常染色体隐性多囊肾病患者兄弟姐妹的表型变异性
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PRETERM DETECTION OF CONGENITAL ANOMALIES BY ULTRASOUND AND CORRELATION WITH POSSIBLE ASSOCIATED RISK FACTORS.超声对先天性畸形的产前检测及其与可能相关的危险因素的相关性。
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Clinical and genetic characteristics of autosomal recessive polycystic kidney disease in Oman.阿曼常染色体隐性多囊肾病的临床和遗传特征。
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Polycystic kidney disease.多囊肾病。
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Diabetes Mellitus After Pediatric Kidney Transplant.小儿肾移植后的糖尿病
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Young-Adult Polycystic Kidney Disease is Associated with Major Cardiovascular Complications.青年型多囊肾病与主要心血管并发症相关。
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Genetics of Autosomal Recessive Polycystic Kidney Disease and Its Differential Diagnoses.常染色体隐性多囊肾病的遗传学及其鉴别诊断。
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