Department of Urology, UCSF, San Francisco, CA, USA.
Nat Rev Urol. 2022 Mar;19(3):137-146. doi: 10.1038/s41585-021-00543-4. Epub 2021 Dec 8.
Primary hyperoxalurias are a devastating family of diseases leading to multisystem oxalate deposition, nephrolithiasis, nephrocalcinosis and end-stage renal disease. Traditional treatment paradigms are limited to conservative management, dialysis and combined transplantation of the kidney and liver, of which the liver is the primary source of oxalate production. However, transplantation is associated with many potential complications, including operative risks, graft rejection, post-transplant organ failure, as well as lifelong immunosuppressive medications and their adverse effects. New therapeutics being developed for primary hyperoxalurias take advantage of biochemical knowledge about oxalate synthesis and metabolism, and seek to specifically target these pathways with the goal of decreasing the accumulation and deposition of oxalate in the body.
原发性高草酸尿症是一组严重的疾病,会导致多系统草酸沉积、肾结石、肾钙质沉着症和终末期肾病。传统的治疗模式仅限于保守治疗、透析和肾脏与肝脏联合移植,而肝脏是草酸生成的主要来源。然而,移植存在许多潜在并发症,包括手术风险、移植物排斥、移植后器官衰竭,以及终身免疫抑制药物及其不良反应。正在开发用于原发性高草酸尿症的新疗法利用了关于草酸合成和代谢的生化知识,并寻求通过专门针对这些途径来减少体内草酸的积累和沉积。