儿童非高危急性早幼粒细胞白血病诱导期加一剂蒽环类治疗的疗效和安全性SCCLG/SCCCG-APL协作组  

Efficacy and safety of adding a dose of anthracyclines for induction treatment in pediatric non high-risk acute promyelocytic leukemia

作　　者：樊重 杨良春[2] 陈以乔[3] 万伍卿[4] 麦惠容[5] 杨丽华[6] 兰和魁[6] 周敦华[7] 李婉丽[8] 郭碧赟[9] 何飞[10] 廖柳华[11] 陈惠芹[12] 甄子俊[13] 贺湘玲[14] 冯晓勤[15] 陈国华[16] 吉训琦[17] 陈沫[18] 黎巧茹 龙兴江[20] 田川 符莹 罗杰思 黄丹萍 张晓莉[1] 唐燕来 王丽娜[1] 李毓[1] 李彬[23] 黄礼彬[1] 罗学群[1] FAN Zhong;YANG Liangchun;CHEN Yiqiao;WAN Wuqing;MAI Huirong;YANG Lihua;LAN Hekui;ZHOU Dunhua;LI Wanli;GUO Biyun;HE Fei;LIAO Liuhua;CHEN Huiqin;ZHEN Zijun;HE Xiangling;FENG Xiaoqin;CHEN Guohua;JI Xunqi;CHEN Mo;LI Qiaoru;LONG Xingjiang;TIAN Chuan;FU Ying;LUO Jiesi;HUANG Danping;ZHANG Xiaoli;TANG Yanlai;WANG Lina;LI Yu;LI Bin;HUANG Libin;LUO Xuequn(The First Affiliated Hospital,Sun Yat-sen University,Guangzhou;Xiangya Hospital,Central South University,Changsha;Fujian Medical University Union Hospital,Fuzhou;Second Xiangya Hospital,Central South University,Changsha;Shenzhen Children′s Hospital,Shenzhen;Zhujiang Hospital,Southern Medical University,Guangzhou;Sun Yat-sen Memorial Hospital,Sun Yat-sen University,Guangzhou;Hunan Children′s Hospital,Changsha;First Affiliated Hospital of Xiamen University,Xiamen;Jiangxi Children′s Hospital,Nanchang;Huizhou Central People′s Hospital,Huizhou;Third Affiliated Hospital,Sun Yat-sen University,Guangzhou;Sun Yat-sen University Cancer Center,Guangzhou;Hunan Provincial People′s Hospital,Changsha;Nanfang Hospital,Southern Medical University,Guangzhou;First People′s Hospital of Huizhou;Hainan Provincial People′s Hospital,Haikou;Fifth Affiliated Hospital,Sun Yat-sen University,Zhuhai;Zhongshan People′s Hospital,Zhongshan;Liuzhou People′s Hospital,Liuzhou;First Affiliated Hospital,Guangdong Medical University,Zhanjiang;Shunde Women and Children′s Hospital,Guangdong Medical University,Shunde;Biostatistics Team,Clinical Trials Unit,The First Affiliated Hospital,Sun Yat-sen University,Guangzhou,China)

机构地区：[1]中山大学附属第一医院,广州510080 [2]中南大学湘雅医院,长沙410119 [3]福建医科大学附属协和医院,福州350001 [4]中南大学湘雅二医院,长沙410011 [5]深圳市儿童医院,深圳518038 [6]南方医科大学珠江医院,广州510145 [7]中山大学孙逸仙纪念医院,广州510120 [8]湖南省儿童医院,长沙410007 [9]厦门大学附属第一医院,厦门361003 [10]江西省儿童医院,南昌330006 [11]惠州市中心人民医院,惠州516001 [12]中山大学附属第三医院,广州510630 [13]中山大学附属肿瘤医院,广州510060 [14]湖南省人民医院,长沙410005 [15]南方医科大学南方医院,广州510510 [16]惠州市第一人民医院,惠州516003 [17]海南省人民医院,海口570311 [18]中山大学附属第五医院,珠海519000 [19]中山市人民医院,中山528400 [20]柳州市人民医院,柳州545006 [21]广东医科大学附属医院,湛江524001 [22]广东医科大学附属顺德妇女儿童医院,顺德528300 [23]中山大学附属第一医院临床研究中心,广州510080

出　　处：《中国小儿血液与肿瘤杂志》2024年第2期86-90,96,共6页Journal of China Pediatric Blood and Cancer

基　　金：广东省科技计划项目(2014A020221008)。

摘　　要：目的成人非高危急性早幼粒细胞白血病(NHR-APL)的去化疗诱导治疗,其疗效和安全性在儿童患者中尚未得到验证,需开展儿童的研究。方法数据来自华南儿童APL协作组的前瞻性研究资料,≤16岁、完成诱导治疗的NHR-APL患儿纳入分析。在砷剂+全反式维甲酸诱导治疗的基础上,比较加或不加1剂量去甲氧柔红霉素/米托蒽醌10mg/m^(2)(化疗或无化疗组)的缓解情况和不良事件率。结果化疗组139例,无化疗组27例,两组的诱导治疗结果比较:达血液学完全缓解的中位天数分别为26.0(22.0,31.5)d和31.0(27.5,35.0)d(P=0.002);分子学完全缓解分别有29.5%和3.7%(P=0.005);外周血白细胞计数>10×10^(9)/L分别有43.2%和74.1%(P=0.003);最高白细胞中位数分别为8.6(IQR 4.9,18.4)×10^(9)/L和22.7(IQR 16.0,41.7)×10^(9)/L(P<0.001);确诊分化综合征分别有5.8%和33.3%(P<0.001);出凝血事件发生率分别为14.4%和51.9%(P<0.001)。感染、颅内高压、心和肝功能异常等不良事件的发生率两组相似(均P>0.1)。结论儿童NHR-APL可能有别于成人患者,与无化疗比较,诱导治疗加1剂量蒽环类疗效更好,并发症的风险更低。Objective The efficacy and safety of chemotherapy-free therapy for adult non high-risk acute promyelocytic leukemia(NHR-APL)have not been validated in children,and pediatric research is needed.Methods The data came from the prospective study of the South China Children′s Leukemia/Cancer Group,and NHR-APL patients who completed induction therapy were included in the analysis.The remission and adverse event rates with or without 1 dose of idarubicin/mitoxantrone 10mg/m^(2)(chemotherapy or non-chemotherapy group)were compared,on the basis of induction therapy with arsenic and all trans retinoic acid.Results There were 139 patients in the chemotherapy group and 27 patients in the non chemotherapy group.Comparison of induction results between chemotherapy and non chemotherapy groups:the median time of hematological complete remission were 26.0(22.0,31.5)days and 31.0(27.5,35.0)days respectively(P=0.002),and there were 29.5%and 3.7%of cases achieved molecular complete remission(P=0.005).The incidence of white blood cells(WBC)>10×10^(9)/L during induction was 43.2%and 74.1%(P=0.003).The median highest WBC was 8.6(4.9,18.4)×10^(9)/L,and 22.7(16.0,41.7)×10^(9)/L(P<0.001).In two groups,5.8%and 33.3%of cases were diagnosed with differentiation syndrome,respectively(P<0.001).The incidence of coagulation events was 14.4%and 51.9%(P<0.001).In addition,the incidence of adverse events such as infection,intracranial hypertension,heart and liver disfunction,and gastrointestinal symptoms was similar in the two groups(All P>0.1).Conclusions Pediatric APL may be different from adult patients.Compared to non chemotherapy regimen,NHR APL patients only received one dose of anthracycline during induction have better induction response and lower complications.

关 键 词：急性早幼粒细胞白血病 儿童 化疗 

分 类 号：R733.71[医药卫生—肿瘤]