自体造血干细胞移植治疗高危神经母细胞瘤临床分析  

Clinical analysis of high risk neuroblastoma treated by autologous hematopoietic stem cell transplantation

作　　者：张朝霞[1] 钟笛箫[1] 李君惠[1] 胡涛[1] 李娟娟[1] 宋泽亮[1] 师晓东[1] 刘嵘[1] Zhang Zhaoxia;Zhong Dixiao;Li Junhui;Hu Tao;Li Juanjuan;Song Zeliang;Shi Xiaodong;Liu Rong(Department of Hematology/Oncology,Children's Hospital Affiliated to Capital Institute of Pediatrics,Beijing 100020,China)

机构地区：[1]首都儿科研究所附属儿童医院血液肿瘤科,北京100020

出　　处：《北京医学》2020年第11期1075-1079,共5页Beijing Medical Journal

摘　　要：目的总结应用大剂量化疗联合自体造血干细胞移植(hematopoietic stem cell transplantation,HSCT)的高危神经母细胞瘤患儿的临床特点及预后。方法选取2012年9月至2020年5月首都儿科研究所附属儿童医院血液肿瘤科收治的经病理诊断为神经母细胞瘤、临床危险度为高危且进行了大剂量化疗联合自体HSCT的13例患儿,总结分析其临床特征、移植过程及随访结果。结果13例患儿中卡铂/依托泊苷/马法兰(carboplatin/etoposide/melphalan,CEM)方案预处理8例,白消安/马法兰(busulfan/melphalan,Bu/Mel)方案5例。13例患儿造血功能均获得满意重建,所有患儿预处理期间均未发生Ⅲ级以上器官毒性,发生Ⅰ~Ⅱ级黏膜毒性4例(30.8%),I级消化道毒性3例(23.1%),移植期间肠道感染1例(7.7%),巨细胞病毒(cytomegalovirus,CMV)血症2例(15.4%),念珠菌血症1例(7.7%)。移植后持续完全缓解8例,疾病稳定带瘤生存2例,体格及智力发育同正常同龄儿,心肝肾肺功能均正常;复发3例,均为移植前部分缓解患儿,分别于移植后半年、1年及2年复发,复发后均死于疾病进展。结论高危神经母细胞瘤患儿以CEM或Bu/Mel为预处理方案进行自体HSCT安全有效,未见远期并发症发生,Bu/Mel方案较CEM方案的不良反应发生率低。移植前缓解状态影响预后。Objective To summarize the clinical features and long term follow-up results of high-risk neuroblastoma(NB)treated by autologous hematopoietic stem cell transplantation(HSCT).Methods A retrospective analysis was conducted in 13 children admitted to Children’s Hospital Affiliated to Capital Institute of Pediatrics,from September 2012 to May 2020.The patients were diagnosed with NB by pathology,had a high clinical risk and received high-dose chemotherapy combined with autologous HSCT.The clinical features,transplantation process and follow-up results were studied.Results Among the 13 children,carboplatin/etoposide/melphalan(CEM)regimen was carried in eight cases,busulfan/melphalan(Bu/Mel)regimen was carried in five.All the children had satisfactory hematopoietic recovery.No gradeⅢor higher organ toxicity occurred during pretreatment.GradeⅠ-Ⅱmucosal toxicity occurred in four cases(30.8%),grade I gastrointestinal toxicity in three cases(23.1%),intestinal infection in one case(7.7%),cytomegaloviremia in two cases(15.4%)and candidiasis in one case(7.7%).Eight cases got continuous complete response(CR)after transplantation,two cases were in stable and three cases relapsed.The physical and mental development of the CR children were the same as normal age,and the functions of heart,liver,kidney and lung were normal.Three patients with partial response before transplantation relapsed at six months,one year and two years after transplantation,and all of them died of disease progression.Conclusions It is safe and effective for high-risk NB children treated with CEM or Bu/Mel as pretreatment regimen followed by autologous HSCT.No long-term complications are observed.Bu/Mel regimen has less side effect than CEM regimen.Disease status before transplantation affects prognosis.

关 键 词：神经母细胞瘤 自体造血干细胞移植 高危 

分 类 号：R739.4[医药卫生—肿瘤]