Department of Pediatrics, Division of Nephrology, Duke University Medical Center, Durham, North Carolina, USA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA.
Department of Pediatrics, Division of Nephrology, Duke University Medical Center, Durham, North Carolina, USA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA.
Kidney Int. 2023 May;103(5):839-841. doi: 10.1016/j.kint.2023.02.017.
Currently, no evidence-based guidelines exist for treatment of children with monogenic steroid-resistant nephrotic syndrome. A retrospective study on 141 patients from Malakasioti et al. revealed that 27.6% responded to calcineurin inhibitor (CNI) treatment, and 75% of responders maintained stable kidney function. Virtually all CNI nonresponders developed progressive loss of kidney function. This study emphasized roles for CNIs in patients with monogenic steroid-resistant nephrotic syndrome, and the need for future studies to identify CNI response biomarkers.
目前，尚无针对单基因激素耐药性肾病综合征患儿的循证治疗指南。Malakasioti 等人的一项回顾性研究显示，141 例患者中 27.6%对钙调磷酸酶抑制剂（CNI）治疗有反应，75%的反应者保持稳定的肾功能。几乎所有 CNI 无反应者的肾功能均逐渐丧失。这项研究强调了 CNI 在单基因激素耐药性肾病综合征患者中的作用，以及未来研究确定 CNI 反应生物标志物的必要性。
