探讨激素敏感型原发性肾病综合征(PNS)患儿激素治疗巩固维持阶段不同减量方案对疾病复发率的影响。

选择2017年6月至2020年6月，在中山大学附属第一医院确诊的41例初治PNS患儿为研究对象，均对激素治疗敏感。根据激素治疗巩固维持阶段采用的不同减量方案，将其分为每日顿服组(n＝22)和隔日顿服组(n＝19)。本研究41例激素敏感型PNS患儿中，男性患儿为29例，女性为12例，男、女比例为2.4∶1，确诊时年龄为1.3~14.6岁。采取回顾性分析法对2组患儿一般临床资料、实验室检查结果进行分析。同时分析2组患儿PNS首次治疗复发时间、复发率、频复发率、加用免疫抑制剂治疗率，以及激素相关不良反应发生情况。本研究遵循的程序符合中山大学附属第一医院伦理委员会规定，通过该伦理委员会审查，并获得批准(审批文号：伦审〔2022〕334号)。

①2组患儿发病年龄、性别构成比、PNS临床分型构成比比较，差异均无统计学意义(P>0.05)。②2组患儿初治时24 h尿蛋白定量、血清白蛋白、血清胆固醇、血清肌酐、估算的肾小球过滤率(eGFR)、免疫球蛋白(Ig)G、补体C3水平，以及足量激素疗程及变态反应性疾病占比、肾功能不全占比比较，差异均无统计学意义(P>0.05)。2组患儿初治时血清IgE水平比较，差异有统计学意义(χ²＝－2.153，P＝0.031)。③本研究41例PNS患儿中，距初始治疗3、6、12个月时复发率分别为12.2%(5/41)、31.7%(13/41)、53.7%(22/41)。与隔日顿服组相比，距初始治疗时12个月内，每日顿服组患儿首次复发时间明显延长(χ²＝4.973，P＝0.026)。距初始治疗3、6个月时，每日顿服组患儿的复发率明显低于隔日顿服组，并且差异均有统计学意义(P<0.05)；距初始治疗12个月时复发率，也低于隔日顿服组[40.9%(9/22) vs 68.4%(13/19)]，但是2组比较，差异无统计学意义(P>0.05)。这41例PNS患儿中，60.7%患儿复发有明确诱因(感染或激素减量/停药)，对其采取控制感染和(或)激素加量/再次使用足量激素治疗后，尿蛋白检查结果均在4周内转阴。每日顿服组患儿激素相关性眼损害发生率高于隔日顿服组，但是差异无统计学意义(22.7% vs 5.3%，P＝0.257)。

激素敏感型PNS患儿预后良好，复发率高，巩固维持阶段采用每日顿服方案可延长复发时间，降低中期复发率。治疗期间需定期监测患儿眼压，早发现、早治疗糖皮质激素相关性眼损害。

To investigate the different dosage reduction regimens in consolidation and maintenance stage of hormone therapy for children with primary nephrotic syndrome (PNS).

A retrospective analysis was performed on 41 children who were diagnosed as PNS in the First Affiliated Hospital of Sun Yat-sen University from June 2017 to June 2020. According to the different therapeutic regimens adopted in maintenance phase, the children were divided into alternate treatment group (n=19) and daily treatment group (n=22). The clinical data, laboratory examination results, the first relapsing course, recurrence rate, glucocorticoid-induced side effects and the proportion of requirement for immunosuppressive agents as well as participants who developed frequently relapsing or steroid dependent nephrotic syndrome were recorded. Forty-one children included 29 boys and 12 girls (ratio 2.4∶1), ranging from 1.3 to 14.6 years old. The procedure followed in this study met the standards formulated by the Ethics Review Committee of First Affiliated Hospital of Sun Yat-sen University and has been approved by it (Approval No. [2022]334). Informed consent was obtained from each participant′s parents.

①There were no significant differences in the age of onset, gender ratio and clinical phenotypes between two groups (P>0.05).②Except for immunoglobulin (Ig)E (χ²=-2.153, P=0.031), there were no significant differences between two groups in terms of 24h urinary protein, serum albumin, serum cholesterol, serum creatinine, estimated glomerular filtration rate (eGFR), IgG, complement 3, remission induction therapy as well as the proportion of allergic diseases and renal insufficiency (P>0.05). ④The relapse rates at 3, 6 and 12 months from the initial treatment were 12.2% (5/41), 31.7% (13/41) and 53.7% (22/41), respectively. Compared with the alternate treatment group, the daily treatment group reduced the rate of relapse at 3 or 6 months (P<0.05) and increased the time to first relapse within a year from the initial treatment (χ²=4.973, P=0.026). Its relapse rate at 1 year from the initial treatment was still lower than that of alternate treatment group [40.9% (9/22) vs 68.4% (13/19)], without statistically significance (P>0.05). 60.7% of relapses were induced by infection, steroid reduction or withdrawal. All patients got remission within 4 weeks after infection control or the addition of steroid dosage. The incidence of eye damage caused by glucocorticoid in daily treatment group was higher than that in alternate treatment group (22.7% vs 5.3%), but the difference was not statistically significant (P=0.257).

Children with PNS generally have a good prognosis despite the high relpase rate. The daily administration of steroid regimen to maintain remission for initial episode of PNS may decrease the time to first relapse and reduce the mid-term replase rate.