Sato Mai, Ito Shuichi, Ogura Masao, Kamei Koichi
Division of Nephrology and Rheumatology, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo, 157-8535, Japan.
Pediatr Nephrol. 2014 Aug;29(8):1373-9. doi: 10.1007/s00467-014-2792-9. Epub 2014 Mar 7.
Children with steroid-dependent nephrotic syndrome (SDNS) often suffer from serious adverse events, including growth retardation and obesity. Rituximab (RTX) is a promising therapeutic option to overcome steroid dependency. We have examined the impact of RTX on growth and obesity in children with SDNS.
Thirteen pediatric patients with SDNS who were refractory despite treatment with multiple immunosuppressive agents received RTX infusions. Mean follow-up was 2.3 years from the first administration of RTX. Improvement in the height and obesity indexes from prior to the initial RTX infusion through to the last visit was assessed.
After RTX, the number of relapses [2.8 (before RTX) vs. 0.8/year (after RTX); p = 0.0008] and the prednisolone dose (287.9 vs. 70.7 mg/kg/year, respectively; p = 0.0002) were significantly decreased. Marked improvement in the height standard deviation score (SDS) was achieved by ten of the 13 patients (77 %) [n = 13; -1.6 (before RTX) vs. -0.8 SDS (after RTX); p = 0.03]. Notably, the height SDS of seven of the eight patients whose height was less than average at the first RTX improved from -2.6 to -1.4 SDS with RTX therapy. At the same time, the obesity index of 12 of the 13 patients (92 %) significantly improved (n = 13; 16.9 vs. 3.1 %; p = 0.004).
Therapy with RTX may contribute to an improvement in the growth and obesity indexes in some patients suffering from severe side effects of steroids.
患有激素依赖型肾病综合征（SDNS）的儿童常遭受严重不良事件，包括生长发育迟缓及肥胖。利妥昔单抗（RTX）是克服激素依赖的一种有前景的治疗选择。我们研究了RTX对SDNS儿童生长及肥胖的影响。
13例尽管接受多种免疫抑制剂治疗仍难治的儿科SDNS患者接受了RTX输注。自首次给予RTX起平均随访2.3年。评估从初始RTX输注前至最后一次就诊时身高及肥胖指数的改善情况。
RTX治疗后，复发次数[2.8次（RTX治疗前）对0.8次/年（RTX治疗后）；p = 0.0008]及泼尼松龙剂量（分别为287.9 vs. 70.7 mg/kg/年；p = 0.0002）显著降低。13例患者中有10例（77%）身高标准差评分（SDS）显著改善[n = 13；-1.6（RTX治疗前）对-0.8 SDS（RTX治疗后）；p = 0.03]。值得注意的是，8例首次RTX治疗时身高低于平均水平的患者中有7例身高SDS从-2.6改善至-1.4 SDS。同时，13例患者中有12例（92%）肥胖指数显著改善（n = 13；16.9%对3.1%；p = 0.004）。
RTX治疗可能有助于改善一些因类固醇严重副作用而患病的患者的生长及肥胖指数。
