儿童4期神经母细胞瘤联合治疗的临床疗效观察  

Clinical efficacy of combined therapy in children with stage 4 neuroblastoma

作　　者：梁伟玲 叶小帆 钟共 陈建军 戴康临 卓家良[4,5] 莫姝 王博深 李春雨 蒋轩竹 徐志渊 周黎[1] 陈秀慧 陈健良 朱知梅 李珮华 陈志峰 LIANG Wei-Ling;YE Xiao-Fan;ZHONG Gong;CHEN Jian-Jun;DAI Kang-Lin;CHEUK Ka Leung Daniel;MO Shu;WANG Bo-Shen;LI Chun-Yu;JIANG Xuan-Zhu;XU Zhi-Yuan;ZHOU Li;CHAN Irene;CHEN Jian-Liang;CHU Patrick;LEE Pui Wah Pamela;CHAN Chi Fung Godfrey(Department of Pediatrics,Hong Kong University-Shenzhen Hospital,Shenzhen,Guangdong 518000,China)

机构地区：[1]香港大学深圳医院儿科,广东深圳518000 [2]香港大学深圳医院神经外科,广东深圳518000 [3]香港大学深圳医院小儿外科,广东深圳518000 [4]香港大学玛丽医院儿童及青少年科学系,中国香港 [5]香港儿童医院,中国香港 [6]香港大学大学深圳医院放疗科,广东深圳518000 [7]香港大学深圳医院移植科,广东深圳518000

出　　处：《中国当代儿科杂志》2022年第7期759-764,共6页Chinese Journal of Contemporary Pediatrics

摘　　要：目的观察4期神经母细胞瘤联合治疗的早期疗效,探讨提高生存率及改善生活质量的治疗方案。方法对2016年1月至2021年6月在香港大学深圳医院确诊的14例4期神经母细胞瘤患儿的临床资料、治疗及随访情况进行回顾性分析。结果14例患儿中位发病年龄为3岁7.5个月。骨髓检查阳性9例,N-Myc基因扩增4例,神经元特异性烯醇化酶增高13例,尿香草扁桃酸增高7例。病理结果:分化型6例,未分化型1例,混合型1例,分化差型6例。N7方案化疗10例(含2例N7方案+三氧化二砷化疗患儿),Rapid COJEC方案化疗4例。手术13例;自体造血干细胞移植14例;放疗10例。Ch14.18/CHO免疫治疗8例,其中1例因在治疗过程中出现过敏性休克而停止免疫治疗;其余7例完成治疗,期间未观察到严重不良反应。完成免疫治疗患儿中1例复发后进行3次Lu177 Dotatate治疗,目前仍在化疗中。14例患儿中位随访时间为45个月。2年内复发4例,2年内总生存率100%;3年内复发4例,3年内无病存活7例。结论儿童4期神经母细胞瘤建议选择多学科联合方案治疗,使4期神经母细胞瘤患儿获得更好的生存及预后。Objective To study the early clinical efficacy of combined therapy of stage 4 neuroblastoma.Methods A retrospective analysis was performed on the medical data and follow-up data of 14 children with stage 4 neuroblastoma who were diagnosed in Hong Kong University-Shenzhen Hospital from January 2016 to June 2021.Results The median age of onset was 3 years and 7.5 months in these 14 children.Among these children,9 had positive results of bone marrow biopsy,4 had N-Myc gene amplification,13 had an increase in neuron-specific enolase,and 7 had an increase in vanilmandelic acid in urine.Based on the results of pathological examination,differentiated type was observed in 6 children,undifferentiated type in one child,mixed type,in one child and poorly differentiated type in 6 children.Of all the children,10 received chemotherapy with the N7 regimen(including 2 children receiving arsenic trioxide in addition)and 4 received chemotherapy with the Rapid COJEC regimen.Thirteen children underwent surgery,14 received hematopoietic stem cell transplantation,and 10 received radiotherapy.A total of 8 children received Ch14.18/CHO immunotherapy,among whom 1 child discontinued due to anaphylactic shock during immunotherapy,and the other 7 children completed Ch14.18/CHO treatment without serious adverse events,among whom 1 child was treated with Lu177 Dotatate 3 times after recurrence and is still undergoing chemotherapy at present.The median follow-up time was 45 months for all the 14 children.Four children experienced recurrence within 2 years,and the 2-year overall survival rate was 100%;4 children experienced recurrence within 3 years,and 7 achieved disease-free survival within 3 years.Conclusions Multidisciplinary combined therapy is recommended for children with stage 4 neuroblastoma and can help them achieve better survival and prognosis.

关 键 词：神经母细胞瘤 自体造血干细胞移植 Ch14.18/CHO 儿童 

分 类 号：R739.4[医药卫生—肿瘤]