Nephrology Unit, Great Ormond Street Hospital for Children, Great Ormond Street, London, UK.
Nephrology Unit, Great Ormond Street Hospital for Children, Great Ormond Street, London, UK.
Lancet Child Adolesc Health. 2018 Dec;2(12):880-890. doi: 10.1016/S2352-4642(18)30283-9. Epub 2018 Oct 18.
More than 85% of children and adolescents (majority between 1-12 years old) with idiopathic nephrotic syndrome show complete remission of proteinuria following daily treatment with corticosteroids. Patients who do not show remission after 4 weeks' treatment with daily prednisolone are considered to have steroid-resistant nephrotic syndrome (SRNS). Renal histology in most patients shows presence of focal segmental glomerulosclerosis, minimal change disease, and (rarely) mesangioproliferative glomerulonephritis. A third of patients with SRNS show mutations in one of the key podocyte genes. The remaining cases of SRNS are probably caused by an undefined circulating factor. Treatment with calcineurin inhibitors (ciclosporin and tacrolimus) is the standard of care for patients with non-genetic SRNS, and approximately 70% of patients achieve a complete or partial remission and show satisfactory long-term outcome. Additional treatment with drugs that inhibit the renin-angiotensin axis is recommended for hypertension and for reducing remaining proteinuria. Patients with SRNS who do not respond to treatment with calcineurin inhibitors or other immunosuppressive drugs can show declining kidney function and are at risk for end-stage renal failure. Approximately a third of those who undergo renal transplantation show recurrent focal segmental glomerulosclerosis in the allograft and often respond to combined treatment with plasma exchange, rituximab, and intensified immunosuppression.
超过 85%的儿童和青少年（多数为 1-12 岁）患有特发性肾病综合征，经皮质类固醇每日治疗后蛋白尿完全缓解。经泼尼松龙每日治疗 4 周后未缓解的患者被认为患有激素抵抗性肾病综合征（SRNS）。大多数患者的肾脏组织学显示局灶节段性肾小球硬化、微小病变病和（罕见）系膜增生性肾小球肾炎。三分之一的 SRNS 患者显示一种关键足细胞基因的突变。其余的 SRNS 病例可能是由未定义的循环因子引起的。对于非遗传性 SRNS 患者，使用钙调神经磷酸酶抑制剂（环孢素和他克莫司）治疗是标准治疗，约 70%的患者达到完全或部分缓解，并显示出满意的长期结果。对于高血压和减少剩余蛋白尿，建议加用抑制肾素-血管紧张素轴的药物治疗。对于未对钙调神经磷酸酶抑制剂或其他免疫抑制剂治疗有反应的 SRNS 患者，肾功能可能会下降，并存在终末期肾衰竭的风险。大约三分之一接受肾移植的患者在同种异体移植物中出现复发性局灶节段性肾小球硬化，通常对血浆置换、利妥昔单抗和强化免疫抑制联合治疗有反应。
