达格列净治疗儿童伴蛋白尿的遗传性肾脏病的短期效果  

Short-term efficacy of dapagliflozin in children with hereditary proteinuric kidney disease

作　　者：崔静怡 刘娇娇 方晓燕 陈径 翟亦晖 徐虹 沈茜 Cui Jingyi;Liu Jiaojiao;Fang Xiaoyan;Chen Jing;Zhai Yihui;Xu Hong;Shen Qian(Department of Nephrology,Children′s Hospital of Fudan University,National Children′s Medical Center,Shanghai 201102,China)

机构地区：[1]国家儿童医学中心、复旦大学附属儿科医院肾脏科,上海201102

出　　处：《中华儿科杂志》2023年第2期164-168,共5页Chinese Journal of Pediatrics

基　　金：江苏省儿童重大疾病研究重点实验室开放项目(JKLP202101)。

摘　　要：目的探索达格列净治疗儿童伴有蛋白尿的遗传性肾脏病的短期效果和安全性。方法前瞻性队列研究。以2020年8月至2021年12月复旦大学附属儿科医院的23例遗传性肾脏病患儿为研究对象。患儿均给予达格列净治疗,体重≤30 kg患儿5 mg/d,体重>30 kg起始剂量5 mg/d,1周后增至10 mg/d。治疗期间同时服用稳定剂量的血管紧张素转换酶抑制剂。收集患儿的一般资料、原发疾病、估算肾小球滤过率(eGFR)、24 h尿蛋白及随访情况等临床指标变化。主要结局指标为治疗12(±2)周时24 h尿蛋白变化,次要结局指标包括治疗24(±2)周时24 h尿蛋白、12(±2)周和24(±2)周时eGFR变化等。使用混合线性模型比较结局指标变化。结果 23例患儿中男16例、女7例,年龄(10.8±2.9)岁。原发疾病包括Alport综合征12例、Dent病5例、蛋白尿4例、局灶节段性肾小球硬化2例。主要结局指标显示24 h尿蛋白在治疗12(±2)周时与基线相比降低[1.75(1.46,2.20)比1.84(1.14,2.54)g/m2,P<0.05]。次要结局指标显示24 h尿蛋白在24(±2)周与基线相比差异无统计学意义(P>0.05)。eGFR在治疗12(±2)周时较基线轻度降低[(107±21)比(112±28)ml/(min·1.73m2),P<0.05],24(±2)周时则差异无统计学意义(P>0.05)。血白蛋白在治疗12(±2)、24(±2)周时均较基线升高[(39±8)、(38±7)比(37±8)g/L,均P<0.05]。研究期间无低血糖事件发生。结论达格列净可短期内改善伴有蛋白尿的遗传性肾脏病患儿尿蛋白及血白蛋白水平,无低血糖等严重不良事件发生。Objective To explore the short-term efficacy and safety of dapagliflozin in children with hereditary proteinuric kidney disease.Methods This was a prospective cohort study.From August 2020 to December 2021,23 children with hereditary kidney disease from Children′s Hospital of Fudan University were enrolled.Patients received dapagliflozin 5 mg/d(weight≤30 kg)or initial dose 5 mg/d for 1 week,then 10 mg/d(weight>30 kg)and the dose of angiotensin converting enzyme inhibitors was stable during treatment.Clinical data including demographic parameters,primary diagnosis,estimated glomerular filtration rate(eGFR),24 h proteinuria and characteristics in the follow-up were collected.The primary outcome was the change in 24 h proteinuria at 12(±2)weeks,secondary outcomes included changes of 24 h proteinuria at 24(±2)weeks,eGFR at both 12(±2)and 24(±2)weeks.The data were analysed by using mixed linear model.Results Totally 23 patients were enrolled,including 16 males and 7 females.The age was(10.8±2.9)years.The primary diseases were Alport syndrome(12 cases),Dent disease(5 cases),proteinuria(4 cases),and focal segmental glomerulosclerosis(2 cases)respectively.Primary outcome showed that 24 h proteinuria decreased from baseline at 12(±2)weeks during treatment(1.75(1.46,2.20)vs.1.84(1.14,2.54)g/m2,P<0.05).Secondary outcomes showed that there was no significant difference in 24 h urine protein at 24(±2)weeks(P>0.05).eGFR decreased slightly at 12(±2)weeks((107±21)vs.(112±28)ml/(min·1.73m2),P<0.05),and there was no significant difference at 24(±2)weeks(P>0.05).Serum albumin increased at 12(±2)and 24(±2)weeks following the treatment((39±8)vs.(37±8)g/L,(38±7)vs.(37±8)g/L,both P<0.05).No hypoglycemia event was reported during the treatment.Conclusion The dapagliflozin had therapeutic effects on decreasing proteinuria and increasing serum albumin in short-term treatment in children with hereditary proteinuric kidney disease,no hypoglycemia or serious adverse events were observed.

关 键 词：肾疾病 遗传 蛋白尿 儿童 钠-葡萄糖转运体2 

分 类 号：R726.9[医药卫生—儿科]