甲基泼尼松龙冲击治疗小儿激素敏感肾病综合征的临床随机对照研究

A prospective clinical randomized controlled trial on pulse methylprednisolone therapy in children with steroid-sensitive nephrotic syndrome

目的客观评价甲基泼尼松龙(甲泼尼龙)冲击与传统的口服足量泼尼松对小儿肾病综合征的疗效及不良反应,为临床应用提供可参考的证据。方法以原发性肾病综合征、激素敏感复发或初发初治的病例为研究对象,采用分层区组随机有效对照,开放试验,以甲泼尼龙冲击为治疗组,口服泼尼松治疗为对照组,比较两者的疗效及不良反应。结果甲泼尼龙组13例患儿中2例冲击治疗期间因感染停止治疗,11例完成治疗并缓解,尿蛋白转阴时间为3～7d,中位时间5d;泼尼松组11例患儿均完成治疗并缓解,尿蛋白转阴时间为5～28d,中位时间8d(P=0.0008);甲泼尼龙冲击治疗组患儿2周内的不良反应有感染、情绪改变、消化道症状和浮肿加重,不良反应的发生率较泼尼松组高;缓解后3个月内的再次复发两组无差别;治疗后3个月的体质量和皮下脂肪厚度,泼尼松组较治疗前增加,身高和骨密度两组治疗前后均无差别;全部患儿的肾上腺皮质功能治疗后2周均受到抑制,至治疗后3个月绝大多数仍未恢复。结论与口服足量泼尼松治疗相比,甲泼尼龙冲击治疗显示了可使激素敏感的原发性肾病综合征患儿尿蛋白更快转阴的趋势,但治疗期间可能出现感染等不良反应;甲泼尼龙冲击治疗不能减少缓解后3个月内的再次复发,对患儿体质量和皮下脂肪分布的影响可能更小。

Objective A prospective study was designed to further evaluate the efficacy and safety of pulse methylprednisolone therapy （PMT） and to compare its effects with oral prednisone in children with steroid-sensitive nephrotic syndrome.Methods A prospective clinical randomized controlled trial was conducted.Stratified randomization,standard treatment control and open trial design was performed.Children with steroid-sensitive nephrotic syndrome were treated by PMT （PMT group） or oral prednisone （control group）.Intention-to-treat analysis was employed to deal the data,while Kaplan-Meier survival analysis,Log Rank test and t-test were used for statistical analysis.Results Thirtyone children were enrolled with only 24 cases suitable for evaluation,including 13 cases in the PMT group and 11 cases in control group.The median remission times were 5 days in PMT group and 8 days in control group,respectively.PMT induced a more rapid remission than oral prednisone （P=0.000 8）.More adverse effects were found in PMT group during the first two weeks of treatment,with slower increase of weight and subcutaneous fat thickness over the subsequent 3 months.There was no significant difference in relapse rates between the two groups over the subsequent 3 months.Conclusions Compared with oral prednisone,PMT could induce complete remission more rapidly in children with steroid-sensitive nephrotic syndrome,but some adverse effects might appear during the treatment,with slower increase of weight and subcutaneous fat thickness over the subsequent 3 months.PMT could not prevent relapsing again in the subsequent 3 months.