Department of Nephrology and Rheumatology, Children's Hospital of Fudan University, Shanghai, 201102, China.
World J Pediatr. 2014 Feb;10(1):59-63. doi: 10.1007/s12519-014-0453-5. Epub 2014 Jan 25.
Idiopathic nephrotic syndrome is the most common glomerular disease in children. This study was undertaken to observe the efficacy and side-effects of rituximab (RTX) in treating children with different types of refractory primary nephrotic syndrome.
Twelve patients with steroid dependent nephrotic syndrome (SDNS), frequently relapsing nephritic syndrome (FRNS), and steroid resistant nephrotic syndrome (SRNS) were enrolled in our study. There were obvious drug side-effects, and proteinuria remained difficult to control. RTX was administered at a dose of 375 mg/m(2) body surface area, once or twice weekly.
The male to female ratio was 3:1, and the onset age was 1.6-8.9 years. There were 9 patients with steroid sensitive nephrotic syndrome (SDNS or FRNS), and 3 patients with SRNS. There were 7 patients with minimal change disease (MCD), 3 patients with focal segmental glomerular sclerosis (FSGS), 1 with focal proliferative glomerulonephritis, and 1 without renal biopsy. The total effective treatment rate of RTX was 91.67%, and for 77.78% of the patients, steroid dosage could be reduced. Six months before and after RTX infusion, the mean steroid dosage was significantly decreased (P=0.014) and the recurrence number was significantly reduced (P<0.001). The results were better in MCD patients than in FSGS patients (P=0.045). There was no significant difference between FRNS/SDNS and SRNS patients (P=0.175). During RTX administration, 3 patients developed skin rashes, 1 developed hypotension, and 1 developed a fever. One patient experienced a persistent decrease in serum immunoglobulin level but without serious infection.
RTX was effective in the treatment of refractory nephrotic syndrome, and it could significantly reduce the use of steroid and immunosuppressants.
特发性肾病综合征是儿童中最常见的肾小球疾病。本研究旨在观察利妥昔单抗（RTX）治疗不同类型难治性原发性肾病综合征的疗效和副作用。
纳入 12 例激素依赖型肾病综合征（SDNS）、频复发型肾病综合征（FRNS）和激素抵抗型肾病综合征（SRNS）患儿，均存在明显药物副作用，且蛋白尿难以控制。RTX 剂量为 375mg/m2 体表面积，每周 1 次或 2 次。
男女比为 3:1，发病年龄为 1.6-8.9 岁。9 例为激素敏感型肾病综合征（SDNS 或 FRNS），3 例为激素抵抗型肾病综合征。7 例为微小病变性肾病（MCD），3 例为局灶节段性肾小球硬化（FSGS），1 例为局灶增生性肾小球肾炎，1 例未行肾活检。RTX 总有效治疗率为 91.67%，77.78%的患者可减少激素用量。RTX 输注前、后 6 个月，平均激素用量明显减少（P=0.014），复发次数明显减少（P<0.001）。MCD 患儿的疗效优于 FSGS 患儿（P=0.045），FRNS/SDNS 与 SRNS 患儿间疗效无差异（P=0.175）。RTX 治疗期间，3 例出现皮疹，1 例出现低血压，1 例出现发热。1 例患者出现血清免疫球蛋白水平持续下降，但无严重感染。
RTX 治疗难治性肾病综合征有效，可显著减少激素和免疫抑制剂的使用。
