Hospital de Niños Ricardo Gutiérrez, Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina.
Nefrologia. 2012 May 14;32(3):321-8. doi: 10.3265/Nefrologia.pre2012.Jan.11093. Epub 2012 Apr 17.
Persistent nephrotic syndrome that does not respond to treatment may cause progression to kidney failure. We designed a therapeutic protocol with sirolimus for this group of patients. We conducted a prospective, interventional, time series, cohort study lasting 20 months. Thirteen patients were enrolled, with a mean age of 10 years (range: 8-18 years old) with steroid-resistant primary nephrotic syndrome and a histological diagnosis of focal and segmental glomerulosclerosis. We administered sirolimus 3.6mg/m2/day. The duration of this regimen was 12 months in responsive patients. The protocol's efficacy was assessed according to reduction of proteinuria (3 response levels: total, partial, or no response). Severity of histological renal damage and mean time from clinical diagnosis to protocol initiation were also assessed. Nine of 13 patients responded to the treatment with sirolimus, and mean progression time and the severity of histological renal damage influenced response to therapy. We believe that sirolimus is a valid treatment option in patients with steroid-resistant nephrotic syndrome, even though this regimen probably requires an earlier treatment.
持续的肾病综合征对治疗没有反应可能会导致肾功能衰竭。我们为这组患者设计了一种使用西罗莫司的治疗方案。我们进行了一项为期 20 个月的前瞻性、干预性、时间序列、队列研究。共纳入 13 名患者，平均年龄为 10 岁（范围：8-18 岁），患有激素抵抗性原发性肾病综合征和局灶节段性肾小球硬化的组织学诊断。我们给予西罗莫司 3.6mg/m2/天。对有反应的患者，该方案的疗程为 12 个月。根据蛋白尿减少（3 个反应水平：完全、部分或无反应）评估方案的疗效。还评估了组织学肾损伤的严重程度和从临床诊断到方案开始的平均时间。13 名患者中有 9 名对西罗莫司治疗有反应，治疗反应受进展时间和组织学肾损伤严重程度的影响。我们认为，即使这种方案可能需要更早的治疗，西罗莫司也是激素抵抗性肾病综合征患者的有效治疗选择。
