Department of Pediatric Nephrology, University Hospital of Leuven, Herestraat 49, 3000, Leuven, Belgium,
Pediatr Nephrol. 2013 Nov;28(11):2089-96. doi: 10.1007/s00467-012-2384-5. Epub 2013 Jan 24.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of kidney failure. It is a systemic disorder, not only affecting the kidneys, but also associated with cyst formation in other organs such as the liver, spleen, pancreas, and seminal vesicles. Other extra-renal symptoms may consist of intracranial arterial aneurysms, cardiac valvular defects, abdominal and inguinal hernias and colonic diverticulosis. Very little is known regarding bone involvement in ADPKD; however, recent evidence has revealed the potential role of fibroblast growth factor 23 (FGF23). FGF23 is an endocrine fibroblast growth factor acting in the kidney as a phosphaturic hormone and a suppressor of active vitamin D with key effects on the bone/kidney/parathyroid axis, and has been shown to increase in patients with ADPKD, even with normal renal function. The aim of this review is to provide an overview of bone and mineral abnormalities found in experimental models and in patients with ADPKD, and to discuss the possible role of FGF23 in this disease.
常染色体显性多囊肾病(ADPKD)是最常见的肾脏衰竭的单基因病因。它是一种全身性疾病,不仅影响肾脏,还与肝脏、脾脏、胰腺和精囊等其他器官的囊肿形成有关。其他肾脏外症状可能包括颅内动脉动脉瘤、心脏瓣膜缺陷、腹部和腹股沟疝以及结肠憩室病。关于 ADPKD 的骨骼受累知之甚少;然而,最近的证据揭示了成纤维细胞生长因子 23(FGF23)的潜在作用。FGF23 是一种内分泌成纤维细胞生长因子,在肾脏中作为一种排磷激素和活性维生素 D 的抑制剂发挥作用,对骨骼/肾脏/甲状旁腺轴有重要影响,并已在 ADPKD 患者中显示增加,即使肾功能正常。本综述的目的是概述在实验模型和 ADPKD 患者中发现的骨骼和矿物质异常,并讨论 FGF23 在这种疾病中的可能作用。