12例利妥昔单抗治疗儿童激素耐药型原发性局灶节段性肾小球硬化症的疗效及安全性分析

Efficacy and safety of rituximab dosing for steroid-resistant idiopathic focal segmental glomerulosclerosis:a report of 12 cases

目的分析利妥昔单抗(rituximab,RTX)治疗儿童激素耐药型原发性局灶节段性肾小球硬化症(focal segmental glomerulosclerosis,FSGS)的疗效和安全性。方法回顾性分析2014年1月至2020年12月于南京大学医学院附属金陵医院(东部战区总医院)儿科接受RTX治疗的激素耐药型原发性FSGS患儿的临床资料。结果共纳12例患儿,初始激素耐药10例,迟发激素耐药2例,首次接受RTX治疗的年龄为8.00(5.15,15.25)岁。随访第六个月,3例完全缓解,1例部分缓解。末次随访时,1例完全缓解,2例部分缓解。接受治疗次数≥3次的患儿的缓解率高于<3次的患儿(随访第六个月,66.7%比25.0%;末次随访,66.7%比12.5%)。迟发激素耐药患儿的缓解率高于初始激素耐药型患儿(随访第六个月,100%比20.0%;末次随访,100%比10.0%)。伴有非肾病范围尿蛋白的患儿的缓解率高于伴有肾病范围尿蛋白的患儿(随访第六个月,50.0%比16.7%;末次随访,33.3%比16.7%)。治疗随访过程中1例患儿出现输液反应,3例出现重症肺炎。结论RTX对部分儿童激素耐药型原发性FSGS有效,但对初始激素耐药型或伴有肾病范围尿蛋白的患儿疗效不佳。治疗次数的增加可能有利于稳定临床缓解率。RTX治疗后需密切随访,预防感染。

Objective To evaluate the efficacy and safety of rituximab(RTX)for steroid-resistant idiopathic focal segmental glomerulosclerosis(FSGS)in children.Methods From 2014 to 2020,the relevant clinical data were retrospectively reviewed for 12 children of steroid-resistant idiopathic FSGS.Results Steroid resistance was initial(n=10)and delayed(n=2).The age of initial RTX dosing was 8.00(5.15,15.25)years.During a follow-up period of 6 months,complete remission(CR,n=3,)and partial remission(PR,n=1)were obtained.At the last follow-up,there were CR(n=1)and PR(n=2).Remission rate was higher in children on≥3 doses than in those on<3 doses(6-month follow-ups:66.7%vs 25.0%;last follow-up:66.7%vs 12.5%).Remission rate was higher in children with delayed steroid resistance than in those with initial steroid resistance(6-month follow-ups:100.0%vs 20.0%;last follow-up:100.0%vs 10.0%).Remission rate was higher in children with a non-nephrotic range of urinary protein than in those with a nephrotic range of urinary protein(6-month follow-ups:50.0%vs 16.7%;last follow-up:33.3%vs 16.7%).During treatment and follow-ups,there were infusion reaction(n=1)and severe pneumonia(n=3).Conclusion RTX is efficacious for primary steroid-resistant FSGS in select children.However,it responds poorly to primary steroid-resistant FSGS or a nephrotic range of urinary protein.A greater number of doses may stabilize clinical response rates.Close follow-ups are required after RTX dosing for preventing infection.