Objective: To investigate the efficacy and safety of adrenocorticotropic hormone (ACTH) in children with frequently relapsing or steroid-dependent nephrotic syndrome. Methods: The clinical data of 38 children with frequently relapsing or steroid-dependent nephrotic syndrome who were admitted to the Department of Nephrology, the Children Hospital, Zhejiang University School of Medicine from January 2015 to December 2020 were retrospectively analyzed. The general information, clinical manifestations, laboratory data of the children and follow-up (till 12 months after treatment) were collected. The patients were divided into ACTH group and Glucocorticoid (GC) group according to treatment plan. Cumulative remission, average recurrence rate, GC dosage, height and weight change and peripheral blood CD19+B lymphocyte count were compared between the two groups to evaluate the efficacy and adverse reactions of ACTH. Fisher's exact test, t test or rank sum test was used for comparison between groups. Results: Among the 38 patients, 28 were male and 10 were female, aged 84 (24, 180) months; 19 were in ACTH group and 19 were in GC group. The cumulative remission rate of 12 months in ACTH group was higher than that in GC group (9/19 vs. 2/19,χ²=6.81,P=0.009), the average recurrence rate was lower than that in GC group ((0.7±0.8) vs. (1.7±1.1) times, t=-3.27, P=0.011), and the average dosage of GC was lower than that in GC group ((0.27±0.16) vs. (0.51±0.27) mg/(kg·d), t=-3.21, P=0.014). The increase in height was higher than that in the GC group (4 (3,5) vs. 3 (2, 3) cm/year, Z=2.58, P=0.010), and the peripheral blood CD19+B lymphocyte count was lower than that in the GC group ((223±149)×106 vs. (410±213)×106/L,t=-3.35, P=0.009). In safety, 19 cases had transient decreased urine volume, 7 cases had hyperglycemia, and 3 cases had hypertension during the infusion of ACTH, which could be relieved after drug withdrawal. Conclusion: ACTH has a better effect on children with frequently relapsing or steroid-dependent nephrotic syndrome, which can improve cumulative sustained remission rate, lower relapses rate and decrease the dosage of GC, with good safety.
目的： 探讨促肾上腺皮质激素（ACTH）对儿童频复发或激素依赖型肾病综合征的疗效及安全性。 方法： 回顾性分析2015年1月至2020年12月就诊于浙江大学医学院附属儿童医院肾内科的38例频复发或激素依赖型肾病综合征患儿的临床资料。收集患儿的一般情况、临床表现、实验室检查、治疗、随访（至治疗后12个月）等资料。根据治疗方案分为ACTH组和糖皮质激素（GC）组，比较2组病例累积缓解情况、平均复发率、GC用量、身高体重变化及外周血CD19+B淋巴细胞计数等，评价ACTH的疗效和不良反应。组间比较采用Fisher确切概率法、t检验或秩和检验。 结果： 38例患儿中男28例、女10例，年龄84（24，180）月龄。ACTH组19例，GC组19例，ACTH组治疗12个月的累积持续缓解率高于GC组（9/19比2/19，χ²=6.81，P=0.009），复发次数低于GC组［（0.7±0.8）比（1.7±1.1）次，t=-3.27，P=0.011］，GC用量低于GC组［（0.27±0.16）比（0.51±0.27）mg/（kg·d），t=-3.21，P=0.014］，身高增长高于GC组［4（3，5）比3（2，3）cm/年，Z=2.58，P=0.010］，外周血CD19+B淋巴细胞计数低于GC组［（223±149）×106比（410±213）×106/L，t=-3.35，P=0.009］。安全性方面，19例患儿在输注ACTH过程中出现一过性尿量减少，7例血糖升高，3例血压升高，停药后可缓解。 结论： ACTH对频复发或激素依赖型肾病综合征患儿有较好的疗效，可提高临床缓解率，减少复发率，减少糖皮质激素的用量，安全性好。.