TBM预处理方案自体造血干细胞移植治疗高危神经母细胞瘤患儿疗效分析  

Clinical efficacy of autologous hematopoietic stem cell transplantation based on TBM preparative regimen in the treatment of children with high-risk neuroblastoma

作　　者：李雪国 姚彤 陈芬 徐艳芳 王如民 罗新辉 郎涛[1] LI Xueguo;YAO Tong;CHEN Fen(Department of Hematology,People’s Hospital of Xinjiang Uygur Autonomous Region,Xinjiang,Urumqi 830001,China;不详)

机构地区：[1]新疆维吾尔自治区人民医院血液病科,乌鲁木齐市830001 [2]新疆儿童医院血液肿瘤科

出　　处：《河北医药》2024年第18期2835-2838,共4页Hebei Medical Journal

基　　金：新疆维吾尔自治区人民医院医疗新技术项目(编号:XJS2018080701)。

摘　　要：目的研究TBM[即塞替派(thiotepa)+白舒菲(busulfan)+美法伦(melphalan)]预处理方案自体造血干细胞移植治疗高危神经母细胞瘤患儿的临床疗效。方法选取2021年11月5日至2022年9月8日于新疆维吾尔自治区人民医院血液病科移植中心接受TBM预处理自体造血干细胞移植患儿7例为研究对象,评估造血重建情况及移植相关不良反应。分析7例高危神经母细胞瘤患儿临床疗效和疗效相关指标,以及随访结果和治疗前后的营养指标。结果患儿治疗后及末次随访不同时期的临床疗效比较,差异无统计学意义(P>0.05)。7例高危神经母细胞瘤患儿中位年龄6.2岁(2~13岁),所有患儿均移植回输前期采集冻存自体干细胞,中位计数单个核细胞(MNC)为12.17×10^(8)/kg[(8.65~28.81)×10^(8)/kg],CD34^(+)为4.15×10^(6)/kg[(2.11~13.12)×10^(6)/kg]。所有患儿均获得造血重建,中性粒细胞中位植入时间为11 d(9~18 d),血小板植入时间为16 d(13~31)d。随访期间所有患儿均存活,其中发生Ⅲ级以上黏膜炎4例,发生率为51.7%;发生Ⅲ~Ⅳ级呕吐腹泻等胃肠道不良反应5例,发生率为71.4%;发生药物性肝损伤2例,发生率为28.5%;未见神经系统、肾脏以及肝窦隙阻塞综合征等预处理相关毒性反应。患儿治疗前后的前白蛋白(PAB)、清蛋白(ALB)及血红蛋白(Hb)等营养指标比较,差异无统计学意义(P>0.05)。结论TBM方案用于高危神经母细胞瘤患儿自体造血干细胞移植预处理中的疗效较佳,且不良反应常见可控,然而仍需进一步扩大病例数临床验证。Objective To analyze the clinical efficacy of autologous hematopoietic stem cell transplantation(ASCT)based on TBM(thiotepa+busulfan+melphalan)preparative regimen in the treatment of children with high-risk neuroblastoma.Methods Seven children with high-risk neuroblastoma who received ASCT based on TBM preparative regimen at the Transplantation Center of the Department of Hematology,People’s Hospital of Xinjiang Uygur Autonomous Region from November 5,2021 to September 8,2022 were enrolled.The hematopoietic reconstruction and transplantation-related adverse events were evaluated.The clinical efficacy,efficacy related indexes,follow-up results,nutritional indicators in children were analyzed.Results The 7 high-risk neuroblastoma children had a median age of 6.2(2-13)years.Autologous stem cells were collected from all children and frozen for transplantation.The median mononuclear cell count(MNC)and CD34^(+)cells were 12.17(8.65-28.81)×10^(8)/kg and 4.15(2.11-13.12)×10^(6)/kg respectively.Hematopoietic reconstruction was achieved in all patients,with a median implantation time of neutrophils and platelets of 11(9-18)days,and 16(13-31)days,respectively.All children survived during the follow-up period,with 4(51.7%)developed gradeⅢmucositis or above.Five(71.4%)cases had gradeⅢ-Ⅳvomiting diarrhea and other gastrointestinal adverse reactions.There were 2(28.5%)cases of drug-induced liver injury.No preconditioning-related toxic reactions in the nervous system and kidney,and hepatic sinusoidal obstruction syndrome were observed.Nutritional indicators,such as prealbumin(PAB),albumin(ALB)and hemoglobin(HB)before and after treatment were comparable without significant differences(P>0.05).Conclusion TBM protocol is effective in the preconditioning of autologous hematopoietic stem cell transplantation in children with high-risk neuroblastoma,and the adverse reactions are common and controllable.

关 键 词：塞替派 白舒菲 美法伦 自体造血干细胞移植 高危神经母细胞瘤 

分 类 号：R730.264.4[医药卫生—肿瘤]