利妥昔单抗治疗儿童激素耐药型肾病综合征的临床疗效和安全性  

Efficacy and safety of rituximab in the treatment of steroid-resistant nephrotic syndrome in children

作　　者：万灵 陈朝英[1] 涂娟[1] 李华荣[1] 孙金山[1] 夏华 Wan Ling;Chen Chaoying;Tu Juan;Li Huarong;Sun Jinshan;Xia Hua(Department of Nephrology,Children's Hospital Affiliated to Capital Institute of Pediatrics,Bejing 100020,China)

机构地区：[1]首都儿科研究所附属儿童医院肾脏内科,北京100020

出　　处：《中华肾脏病杂志》2025年第2期107-113,共7页Chinese Journal of Nephrology

基　　金：首都儿科研究所附属儿童医院临床培育专项基金(LCYJ-2023-24)。

摘　　要：目的:评价利妥昔单抗(rituximab,RTX)治疗激素耐药型肾病综合征(steroid-resistant nephrotic syndrome,SRNS)患儿的临床疗效及安全性。方法:该研究为回顾性观察性研究,回顾性分析2021年7月至2023年8月首都儿科研究所附属儿童医院收治并接受过RTX治疗的SRNS患儿的临床资料,比较患儿RTX用药前后尿蛋白量、肾功能、血清白蛋白、免疫球蛋白等指标的变化,评估RTX治疗SRNS的临床疗效及安全性。结果:共14例SRNS患儿入选该研究,男女比例为6∶8,肾病发病年龄(4.36±3.12)岁,有8例接受过肾活检,其中6例肾脏病理表现为局灶节段性肾小球硬化,2例为微小病变型肾病。14例患儿首次使用RTX年龄为(8.45±3.98)岁,剂量375 mg/m 2,最大剂量500 mg,使用2剂、3剂、4剂、5剂RTX的患儿例数分别为6例、6例、1例及1例。RTX给药的同时予复方磺胺甲噁唑口服预防感染,首剂RTX后4.0(2.5,6.5)个月后停用糖皮质激素(激素)及免疫抑制剂。随访时间10.5(6.0,18.3)个月,至随访3个月、6个月及随访终点,患儿的肾病完全缓解率分别为100.0%(14/14)、85.7%(12/14)和64.3%(9/14),有5例患儿出现肾病复发。与首剂RTX治疗前比较,随访终点时患儿的血清白蛋白和身高显著增加,体重指数显著下降,差异有统计学意义(均P<0.05),而尿蛋白量、肾功能、IgG水平的差异无统计学意义。14例患儿在RTX治疗过程中均未出现输液反应,随访过程中无严重感染,1例确诊低丙种球蛋白血症。结论:RTX可提高SRNS的缓解率、降低复发率,减少激素和免疫抑制剂用量及药物相关不良反应,改善患儿生长发育情况,不良反应较少。对于激素联合免疫抑制剂治疗肾病不缓解的SRNS患者可考虑应用RTX。Objective:To evaluate the efficacy and safety of rituximab(RTX)in children with steroid resistant nephrotic syndrome(SRNS).Methods:The was a retrospective observational study.A retrospective analysis was conducted on the clinical data of 14 children with SRNS who received RTX treatment in the Department of Nephrology,Children's Hospital Affiliated to Capital Institute of Pediatrics from July 2021 to August 2023.The changes in urinary protein content,renal function,serum albumin,immunoglobulin and other indicators before and after RTX medication were compared to evaluate the clinical efficacy and safety of RTX treatment for SRNS.Results:A total of 14 children with SRNS were selected for this study,with a male to female ratio of 6:8.The age of onset of kidney disease was(4.36±3.12)years,and 8 of them underwent kidney biopsy.Among them,6 cases showed focal segmental glomerulosclerosis in renal pathology,and 2 cases showed minimal change disease.The age of 14 children who first used RTX was(8.45±3.98)years old,with a dose of 375 mg/m 2 and a maximum dose of 500 mg.The number of children who had used 2,3,4,and 5 doses of RTX was 6,6,1,and 1,respectively.RTX was administered orally with compound sulfamethoxazole to prevent infection.Glucocorticoids and immunosuppressants were discontinued 4.0(2.5,6.5)months after the first dose of RTX.The median follow-up time was 10.5(6.0,18.3)months.By the end of the 3-month,6-month,and follow-up endpoints,the complete remission rates of kidney disease in the children were 100.0%(14/14),85.7%(12/14),and 64.3%(9/14),respectively.Five children experienced kidney disease recurrence.Compared with before the first dose of RTX treatment,the serum albumin and height significantly increased,while body mass index significantly decreased at the end of follow-up(all P<0.05).There was no statistically significant difference in urinary protein content,renal function,and IgG(all P>0.05).During the RTX treatment,all 14 children did not experience any infusion reaction,and there were no serious in

关 键 词：利妥昔单抗 肾病综合征 肾病 脂性 肾小球硬化症 局灶节段性 儿童 糖皮质激素耐药 

分 类 号：R726.9[医药卫生—儿科]