GD2抗体达妥昔单抗β治疗儿童神经母细胞瘤临床分析  

Clinical analysis of the GD2 antibody Dinutuximab beta in pediatric neuroblastoma treatment

作　　者：杨帆[1] 唐汝泽 赵东亮 吴琳琳 黄顺根[1] 卢俊[2] YANG Fan;TANG Ruze;ZHAO Dongliang;WU Linlin;HUANG Shungen;LU Jun(Department of General Surgery,2Department of Hematology and Oncology,Children's Hospital of Soochow University,Jiangsu 215000)

机构地区：[1]苏州大学附属儿童医院普外科,江苏215000 [2]苏州大学附属儿童医院血液肿瘤科,江苏215000

出　　处：《南通大学学报(医学版)》2024年第6期556-560,共5页Journal of Nantong University(Medical sciences)

基　　金：江苏省卫生健康委员会重点项目(ZD2021006);苏州市“科教兴卫”青年科技项目(KJXW2021026)。

摘　　要：目的:分析达妥昔单抗β治疗儿童高危及复发/难治性神经母细胞瘤(neuroblastoma,NB)的效果及不良反应,为NB的免疫治疗提供参考。方法:对苏州大学附属儿童医院2022年6月—2024年6月应用达妥昔单抗β治疗的6例NB患者的病例资料进行回顾性分析。结果:应用达妥昔单抗β治疗6例患者,其中男2例,女4例,中位年龄为5.54岁[四分位数(interquartile range,IQR),3.62,8.33岁]。高危NB 4例,复发/难治性NB 2例,在接受达妥昔单抗β治疗前,2例达到完全缓解(complete response,CR),4例达到非常好的部分缓解(very good partial response,VGPR)。6例患者治疗27个周期,随访时间185.5 d(152.25,403.75 d),5例持续缓解,1例因治疗2个周期后疾病进展停止治疗。27个周期治疗中,不良反应最早出现于治疗后5 h左右,最常见的不良反应依次为发热14例次(51.9%)、低蛋白血症13例次(48.1%)、肝功能异常12例次(44.4%)、胃肠道反应11例次(40.7%),过敏反应9例次(33.3%),疼痛6例次(22.2%),最常见的3级不良反应为发热、胃肠道反应、电解质紊乱,均经对症支持治疗后好转,无患者因不良反应终止治疗,未发生治疗相关死亡事件。结论:达妥昔单抗β可改善高危及复发/难治性NB患儿预后,但部分患儿预后不佳,仍需进一步临床研究。治疗相关不良反应总体可控,最早出现于用药后5 h左右,需做好严密监测。Objective:To analyze the clinical characteristics of the efficacy and adverse reactions of Dinutuximab beta,and provide valuable insights for immunotherapy strategies targeting high-risk relapsed and refractory neuroblastoma(NB).Methods:The clinical data of patients who received treatment with Dinutuximab beta at Children's Hospital of Soochow University were retrospectively analyzed from June 2022 to June 2024.Results:A total of 6 patients were treated with Dinutuximab beta,among whom 2 cases were male and 4 cases were female.The median age was 5.54 years[interquartile range(IQR),3.62,8.33 years].4 cases were high-risk NB,and 2 cases were relapsed/refractory NB.Before receiving Dinutuximab beta,2 cases achieved complete response(CR),and 4 cases achieved very good partial response(VGPR).Six patients completed a total of 27 treatment cycles,with a median follow-up duration of 185.5 days(152.25,403.75 days).Five patients achieved sustained remission,while one patient discontinued treatment due to disease progression after two cycles.The earliest adverse events were observed approximately 5 hours after the initiation of treatment during a total of 27 treatment cycles.The most frequently adverse reactions included fever(14 cases,51.9%),hypoalbuminemia(13 cases,48.1%),liver function abnormalities(12 cases,44.4%),gastrointestinal reactions(11 cases,40.7%),allergic reactions(9 cases,33.3%),and pain(6 cases,22.2%).The most frequently observed grade 3 adverse reactions included fever,gastrointestinal reactions,and electrolyte disturbances,all of which demonstrated improvement with symptomatic supportive treatment.No patients discontinued treatment due to adverse reactions,nor treatment-related deaths occurred.Conclusions:The administration of Dinutuximab beta has demonstrated potential in enhancing the prognosis of pediatric patients with relapsed/refractory NB;however,a subset of children still exhibits unfavorable outcomes,necessitating further investigation through clinical studies.The management of treatment-related a

关 键 词：神经母细胞瘤 达妥昔单抗β 双唾液酸神经节苷脂 有效性 安全性 儿童 

分 类 号：R739.4[医药卫生—肿瘤]