Department of Nephrology, Tokyo Metropolitan Children's Medical Center, Fuchu, Japan.
Pediatr Nephrol. 2013 May;28(5):765-71. doi: 10.1007/s00467-012-2393-4. Epub 2013 Jan 13.
Cyclosporine has improved remission rates in children with steroid-resistant nephrotic syndrome (SRNS). However, little prospective long-term follow-up data is available.
We prospectively followed and analyzed 5-year outcomes of all 35 patients enrolled in our previous prospective multicenter trial with cyclosporine and steroids in children with SRNS. At enrollment, 23 cases were classified as minimal change (MC), five as diffuse mesangial proliferation (DMP), and seven as focal segmental glomerulosclerosis (FSGS).
Renal survival at 5 years (median 7.7 years) was 94.3 %. Patient status was complete remission (CR) in 31 (88.6 %) (MC/DMP, 25; FSGS, 6); partial remission in one (FSGS); and non-remission in three (MC/DMP), including chronic kidney disease and end-stage kidney disease in one each. Among 31 patients with CR, 22 (71.0 %) were receiving treatment with immunosuppressants at 5 years, including cyclosporine in 19, and seven of these 22 continued to show frequent relapse. Response to cyclosporine at 4 months predicted 5-year outcome in 31 of 35 patients.
Although SRNS treatment with cyclosporine provides high renal survival and remission rates, many children require ongoing immunosuppression. Management has advanced from the prevention of end-stage kidney disease to the long-term maintenance of remission and management of relapse after induction therapy.
环孢素提高了激素耐药性肾病综合征（SRNS）患儿的缓解率。但是，很少有前瞻性的长期随访数据。
我们前瞻性地随访并分析了既往前瞻性多中心试验中纳入的 35 例接受环孢素和激素治疗的 SRNS 患儿的 5 年结局。入组时，23 例患者被分类为微小病变（MC），5 例为弥漫性系膜增生（DMP），7 例为局灶节段性肾小球硬化（FSGS）。
5 年肾脏存活率（中位数 7.7 年）为 94.3%。31 例（88.6%）患者达到完全缓解（CR）（MC/DMP，25 例；FSGS，6 例）；1 例部分缓解（FSGS）；3 例未缓解（MC/DMP），包括慢性肾脏病和终末期肾病各 1 例。在 31 例 CR 患者中，22 例（71.0%）在 5 年后继续接受免疫抑制剂治疗，其中 19 例接受环孢素治疗，这 22 例中有 7 例仍频繁复发。4 个月时对环孢素的反应预测了 35 例患者中的 5 年结局。
尽管环孢素治疗 SRNS 可提供较高的肾脏存活率和缓解率，但许多患儿仍需要持续的免疫抑制治疗。管理策略已从预防终末期肾病进展为长期维持缓解和管理诱导治疗后的复发。
