Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common genetic diseases implicated in the development of end stage renal disease (ESRD). Although FDA has recently approved a drug against ADPKD, there is still a great need for development of alternative management strategies for ADPKD. Understanding the different mechanisms that lead to cystogenesis and cyst expansion in ADPKD is imperative to develop new therapies against ADPKD. Recently, we demonstrated that caloric restriction can prevent the development of cystic disease in animal models of ADPKD and through these studies identified a new role for pregnancy associated plasma protein-A (PAPP-A), a component of the insulin-like growth factors (IGF) pathway, in the pathogenesis of this disease. The PAPP-A-IGF pathway plays an important role in regulation of cell growth, differentiation, and transformation and dysregulation of this pathway has been implicated in many diseases. Several indirect studies support the involvement of IGF-1 in the pathogenesis of ADPKD. However, it was only recently that we described a direct role for a component of this pathway in pathogenesis of ADPKD, opening a new avenue for the therapeutic approaches for this cystic disease. The present literature review will critically discuss the evidence that supports the role of components of IGF pathway in the pathogenesis of ADPKD and discuss the pharmacological implications of PAPP-A-IGF axis in this disease.

常染色体显性多囊肾病（ADPKD）是最常见的遗传性疾病之一，与终末期肾病（ESRD）的发展有关。尽管 FDA 最近批准了一种针对 ADPKD 的药物，但仍然非常需要开发针对 ADPKD 的替代管理策略。了解导致 ADPKD 囊肿发生和囊肿扩张的不同机制对于开发针对 ADPKD 的新疗法至关重要。最近，我们证明热量限制可以预防 ADPKD 动物模型中囊性疾病的发展，并通过这些研究确定了妊娠相关血浆蛋白 A (PAPP-A) 的新作用，PAPP-A 是胰岛素样生长因子的一个组成部分。 IGF）途径，在这种疾病的发病机制中。PAPP-A-IGF 通路在细胞生长调节中发挥重要作用，该途径的分化、转化和失调与许多疾病有关。一些间接研究支持 IGF-1 参与 ADPKD 的发病机制。然而，直到最近，我们才描述了该通路的一个组成部分在 ADPKD 发病机制中的直接作用，为这种囊性疾病的治疗方法开辟了新途径。本文献综述将批判性地讨论支持 IGF 通路成分在 ADPKD 发病机制中的作用的证据，并讨论 PAPP-A-IGF 轴在该疾病中的药理学意义。直到最近，我们才描述了该通路的一个组成部分在 ADPKD 发病机制中的直接作用，为这种囊性疾病的治疗方法开辟了新途径。本文献综述将批判性地讨论支持 IGF 通路成分在 ADPKD 发病机制中的作用的证据，并讨论 PAPP-A-IGF 轴在该疾病中的药理学意义。直到最近，我们才描述了该通路的一个组成部分在 ADPKD 发病机制中的直接作用，为这种囊性疾病的治疗方法开辟了新途径。本文献综述将批判性地讨论支持 IGF 通路成分在 ADPKD 发病机制中的作用的证据，并讨论 PAPP-A-IGF 轴在该疾病中的药理学意义。