利妥昔单抗治疗儿童原发性局灶节段性肾小球硬化症疗效分析

Analysis of efficacy of rituximab in treating primary focal segmental glomerulosclerosis in children

目的利妥昔单抗(RTX)治疗儿童原发性局灶节段性肾小球硬化症(FSGS)的研究报道罕见。文中旨在分析10例接受单剂量RTX治疗的原发性FSGS患儿的不良作用及安全性,为RTX治疗FSGS提供参考。方法回顾性分析2014年4月至2017年8月南京军区南京总医院儿科住院的10例FSGS患儿临床资料。统计分析患儿给予RTX治疗后的24 h尿蛋白、血清清蛋白、外周血CD20+B细胞水平、RTX不良反应等指标。结果 10例患儿中激素耐药型肾病综合征(SRNS)6例,激素依赖/频复发肾病综合征(FRNS/SDNS)4例。经RTX治疗后10例患儿中5例完全缓解、2例部分缓解、3例未缓解。其中6例SRNS患儿治疗后2例完全缓解、1例部分缓解、3例未缓解;4例FRNS/SDNS患儿治疗后3例完全缓解、1例部分缓解。经RTX治疗后第3个月,10例患儿尿蛋白从治疗前[2.41(0.89~6.82)g/24 h]降低至[0.43(0.05~1.1)g/24 h],血清清蛋白从治疗前[31.60(13.00~38.22)g/L]升高至[38.30(27.18~53.20)g/L]。1例患者出现不良反应,表现为发热、寒战、胸闷,输液速度降低后缓解。随访期间1例在RTX治疗1个月后发生严重肺部感染及蛋白尿,未见其他患儿感染次数增加,无呼吸道、消化道及神经系统的异常表现。结论 RTX治疗原发性FSGS安全性较高,无严重不良反应,是FSGS患儿的有效治疗手段之一。

Objective There are few studies on children with primary focal segmental glomerulosclerosis（ FSGS） treated with rituximab（ RTX）. This study aimed to analyze the side effects and safety of the single dose of RTX in 10 children with primary FSGS,and further provide reference for the RTX treatment of FSGS. Methods We retrospectively analysed the clinical data of 10 FSGS children who hospitalized in the department of pediatrics in Nanjing General Hospital of Nanjing Military Area Command from April 2014 to August 2017. 24 hours urinary protein,serum albumin,the count of circulating CD20＋B cells and the RTX adverse reactions were analyzed after the treatment of RTX. Results From the 10 cases,6 cases were steroid-resistant nephrotic syndrome（ SRNS）,and 4 cases were frequently relapsing/steroid dependent nephrotic syndrome（ FRNS/SDNS）. After single dose of RTX treatment,5 cases a-chieved complete remission（ CR）,2 partial remission（ PR）,and 3 non-remission（ NR）. Among the 6 cases of SRNS,2,1,3 cases achieved CR,PR,NR respectively; Among the 4 cases of FRNS/SDNS,CR was achieved in 3 patients,PR was achieved in 1 patient.3 months after RTX treatment,urinary protein decreased from [2.41（ 0.89-6.82） g/24 h] to [0.43（ 0.05-1.1） g/24 h],serum albumin increased from [31.60（ 13.00-38.22） g/L] to [38.30（ 27.18-53.20） g/L]. Adverse reactions occurred in 1 case including fever,chills,and chest tightness. These adverse reactions relieved after the decreased of RTX infusion speed. One developed severe pneumonia and proteinuria one month after RTX treatment. There was no increase in the number of infections in other children and no abnormalities in the respiratory tract,digestive tract,and nervous system during follow-up. Conclusion RTX treatment of primary FSGS has high security and has no serious adverse reactions. It is one of the effective treatments for children with FSGS.