芬那酮和其他未来治疗阿尔波特综合征的选择

Helen Pearce, Holly Mabillard
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摘要

Alport综合征是一种罕见的遗传性疾病,导致基底膜IV型胶原蛋白紊乱,导致血尿、蛋白尿,并常发展为肾纤维化,导致进行性肾脏疾病。肾素-血管紧张素-醛固酮系统的治疗性阻断,可以减缓肾功能衰竭的进展,这是有强有力证据支持的。最近在慢性肾病患者中使用钠-葡萄糖共转运蛋白-2 (SGLT2)抑制剂和矿皮质激素受体拮抗剂(MRA)的临床试验已经改变了治疗前景。患有Alport综合征和进行性肾脏疾病的患者可能受益于mra治疗,因为研究表明这些药物通过多种机制具有肾保护作用,包括预防纤维化。正在进行的临床试验显示出巨大的希望,以帮助建立芬纳酮(一种MRA)的长期安全性和有效性。这篇综述讨论了MRAs作为一种潜在的治疗Alport综合征的证据,它可以减缓慢性肾脏疾病的进展并防止患者达到肾衰竭。
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Finerenone and other future therapeutic options for Alport syndrome
Abstract Alport syndrome is a rare genetic disease that results in disordered basement membrane type IV collagen resulting in haematuria, proteinuria and often development of renal fibrosis leading to progressive kidney disease. The therapeutic blockage of the renin-angiotensin-aldosterone system, which slows the progression to kidney failure, is supported by strong evidence. Recent clinical trials using sodium-glucose co-transporter-2 (SGLT2) inhibitors and mineralocorticoid receptor antagonists (MRA) in patients with chronic kidney disease have changed the therapeutic landscape. Patients with Alport syndrome and progressive kidney disease may benefit from treatment with MRAs because research has shown that these drugs are nephroprotective through a variety of mechanisms, including by preventing fibrosis. Ongoing clinical trials show great promise in order to help establish the long-term safety and efficacy of Finerenone, a MRA. This review discusses the evidence for the use of MRAs as a potential treatment in Alport syndrome that may slow the progression of chronic kidney disease and prevent patients reaching kidney failure.
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