a Unit of Nephrology and Dialysis, Department of Internal Medicine , Messina , Italy.
b Unit of Pediatric Nephrology and Rheumatology , University of Messina , Messina , Italy.
Expert Opin Investig Drugs. 2018 Nov;27(11):839-879. doi: 10.1080/13543784.2018.1540587. Epub 2018 Oct 30.
Minimal change disease (MCD) and Focal and segmental glomerulosclerosis (FSGS) are two of the major causes of nephrotic syndrome (NS) in children and adults. According to KDIGO (Kidney Disease: Improving Global Outcomes) guidelines, the treatment of adult primary MCD and FSGS should be based on immunosuppressants and antiproteinuric drugs. Recently, Rituximab, a humanized monoclonal antibody (mAb) has emerged as a potential treatment for steroid or calcineurin inhibitor-dependent patients; it has however demonstrated lower efficacy in those with nephrotic syndrome that is resistant to the above indicated drugs.
Analysis of ongoing and already completed clinical trials, retrieved from clinicaltrials.gov, clinicaltrialsregister.eu and PubMed involving new therapies for nephrotic syndrome secondary to MCD and FSGS.
The most promising drugs under investigation for MCD and FSGS are mAbs. We are hopeful that new therapeutic options to treat multi-drug resistant MCD and FSGS will emerge from currently ongoing studies. What appears certain is the difficulty in enrolling patients affected by orphan renal diseases and the selection of valid endpoints in clinical trials, such as kidney failure.
微小病变病（MCD）和局灶节段性肾小球硬化症（FSGS）是儿童和成人肾病综合征（NS）的主要病因之一。根据 KDIGO（肾脏疾病：改善全球预后）指南，成人原发性 MCD 和 FSGS 的治疗应基于免疫抑制剂和抗蛋白尿药物。最近，利妥昔单抗（一种人源化单克隆抗体（mAb））已成为类固醇或钙调神经磷酸酶抑制剂依赖性患者的潜在治疗方法；然而，它在对上述药物耐药的肾病综合征患者中的疗效较低。
分析了从 clinicaltrials.gov、clinicaltrialsregister.eu 和 PubMed 检索到的正在进行和已完成的临床试验，这些试验涉及 MCD 和 FSGS 继发肾病综合征的新疗法。
正在研究的治疗 MCD 和 FSGS 最有前途的药物是 mAb。我们希望，目前正在进行的研究将为治疗多药耐药性 MCD 和 FSGS 带来新的治疗选择。可以肯定的是，招募患有罕见肾脏疾病的患者以及在临床试验中选择有效的终点（如肾衰竭）存在困难。
