Hibino Satoshi, Uemura Osamu, Nagai Takuhito, Yamakawa Satoshi, Iwata Naoyuki, Ito Hidekazu, Nakano Masaru, Tanaka Kazuki
Pediatric Nephrology Aichi Children's Health and Medical Center, Aichi, Japan.
Pediatr Int. 2015;57(1):85-91. doi: 10.1111/ped.12498. Epub 2014 Nov 25.
This retrospective study was performed to assess the 3 year outcome of a unified protocol for childhood idiopathic nephrotic syndrome.
Cyclosporine A (CsA) or CsA plus mycophenolate mofetil (MMF) were used in patients without remission on high-dose steroid therapy. CsA was maintained at an area under the whole blood concentration-time curve up to 4 h after dose (AUC0-4 ) of 1500 and 2000 ng·h/mL in steroid-dependent nephrotic syndrome (SDNS) and steroid-resistant nephrotic syndrome (SRNS), respectively. Ninety-one children were enrolled in the study (SDNS, n = 64; SRNS, n = 18). Patients were divided into minimal change (MC) and focal segmental glomerulosclerosis (FSGS) groups. Three year outcome was evaluated using clinical severity defined as degree of dependence on immunosuppressive therapy for maintenance of remission.
In the SDNS group, the numbers of MC and no biopsy were 51 and 13, respectively. No patient had FSGS. Twelve SRNS patients had FSGS and six had MC. In SDNS, 15/64 patients (23%) received no medication. CsA was effective as steroid-sparing agent in 31/38 patients (82%). MMF was effective in all eight patients for whom CsA was unsuccessful. Remission rate in the SRNS group was 14/18 (78%; eight with CsA, and six with a combination of CsA + MMF). Five of the 14 SRNS remission patients received methylprednisolone pulse therapy. Four were resistant to therapy, and had impaired renal function. The clinical severity of MC and FSGS overlapped.
Treatment with CsA and combination of CsA plus MMF was useful for SDNS and for remission induction in SRNS.
本回顾性研究旨在评估儿童特发性肾病综合征统一方案的3年疗效。
对大剂量激素治疗未缓解的患者使用环孢素A（CsA）或CsA联合霉酚酸酯（MMF）。在激素依赖型肾病综合征（SDNS）和激素抵抗型肾病综合征（SRNS）中，CsA维持全血浓度-时间曲线下面积（AUC0-4）分别达到1500和2000 ng·h/mL，给药后持续4小时。91名儿童纳入研究（SDNS，n = 64；SRNS，n = 18）。患者分为微小病变（MC）组和局灶节段性肾小球硬化（FSGS）组。采用定义为维持缓解对免疫抑制治疗的依赖程度的临床严重程度评估3年疗效。
在SDNS组中，MC和未进行活检的患者数量分别为51例和13例。无患者为FSGS。12例SRNS患者为FSGS，6例为MC。在SDNS中，15/64例患者（23%）未接受药物治疗。CsA作为激素替代药物在31/38例患者（82%）中有效。MMF对CsA治疗失败的所有8例患者均有效。SRNS组的缓解率为14/18（78%；8例使用CsA，6例使用CsA + MMF联合治疗）。14例SRNS缓解患者中有5例接受了甲泼尼龙冲击治疗。4例对治疗耐药，且肾功能受损。MC和FSGS的临床严重程度重叠。
CsA及CsA联合MMF治疗对SDNS及SRNS的缓解诱导有效。
