高三尖杉酯碱和维甲酸联合As_2O_3治疗初发儿童急性早幼粒细胞白血病的临床研究  

Homoharringtonine with retinoic acid and arsenic trioxide for newly diagnosed acute promyelocytic leukemia in children

作　　者：裴仁治[1] 李双月[1] 陆滢[1] 张丕胜[1] 刘旭辉[1] 杜小红[1] 马俊霞[1] 陈冬[1] 沙科娅[1] 陈列光[1] 

机构地区：[1]浙江省宁波大学医学院附属鄞州医院血液科,315040

出　　处：《中华全科医学》2014年第12期1970-1972,共3页Chinese Journal of General Practice

基　　金：浙江省宁波市农业与社会发展科技计划项目(2008-2-22)

摘　　要：目的观察高三尖杉酯碱(HHT)联合全反式维甲酸(ATRA)和三氧化二砷(As2O3)治疗初发儿童急性早幼粒细胞白血病(APL)的临床疗效以及诱导治疗期毒副反应。方法回顾性分析宁波大学医学院附属鄞州医院血液科2006年1月—2013年6月期间HHT联合ATRA和As2O3方案治疗18例初治儿童APL的临床疗效、诱导治疗期毒副反应。结果 1所有患儿PML/RARa融合基因均阳性,其中L型11例(61.1%),S型7例(38.9%);参照Sanz危险度分层,18例患儿低危、中危和高危分别有6例(33.3%)、7例(38.9%)和5例(27.8%)。218例初发APL儿童CR率为100%(18/18),无一例患儿因出血、感染等原因导致早期死亡;获得CR的时间为(29.7±4.9)d;诱导缓解后,PML/RARa融合基因的阴性率为77.8%(14/18);缓解后序贯实施第一周期治疗结束后PML/RARa融合基因的阴性率为94.4%(17/18),截止随访日期,18例患儿PML/RARa融合基因全部阴性。318例初发APL儿童5年OS为100.0%、5年DFS为(90.9±8.7)%。4诱导治疗期间,毒副反应给予对症支持治疗后均控制并恢复正常。结论 HHT联合ATRA和As2O3治疗初发儿童APL疗效好,长期生存率高,且价格经济,是APL治疗的合理选择之一。Objective To study the clinical efficacy and side effects of homoharringtonine（HHT） combined with all-trans retinoie acid（ATRA） and arsenic trioxide （As203 ） in the treatment of newly diagnosed acute promyelocytic leukemia （APL） in children. Methods From January 2006 to June 2013,18 newly diagnosed children with APL without prior ex- posure to any anti-leukemic therapy were recruited ;the therapeutic effects and side effects during induction therapy were analyzed. Results （1）The fusion gene PML/RARa was detected in all patients, in which 11 cases were with L isoform （61.1%） ,7 cases with S isoform（38.9% ）. According to the Sanz score, the patients were separated into three groups： low risk group （33.3%） ,intermediate risk group（38.9% ） and high risk group（27.8% ）. （2）All 18 patients achieved complete remission（ 100% ）. The time of achieved CR was （29.7 ± 4.9） days. 77.8% （14/18） was negative for PML- RARer fusion gene at the time of CR,94.4% （17/18） was negative at the end of the first cycle treatment. （3）The 5-years overall survival（OS） and disease free survival（DFS） were 100.0% and（90.9 ± 8.7 ）%, respectively. （4）During induction therapy, the incidence of side effects included dryness of the mouth 66.7% （ 12/18 ）, headache 27.8% （ 5/18 ）, nausea and vomiting 38.9% （7/18） ,hepatic toxicity 33.3% （6/18） ,facial edema 27.8% （5/18） ,infection 11.1 （2/18 and differentiation syndrome 5.6% （ 1/18 ）, all were cured by supportive treatments. Conclusion HHT with ATRA and As2 03 regimen is effective, economic and safe in the treatment of children with newly diagnosed APL, with a high long term survival rate.

关 键 词：儿童 白血病 早幼粒细胞 急性 高三尖杉酯碱 维甲酸 三氧化二砷 

分 类 号：R733.71[医药卫生—肿瘤] R730.53[医药卫生—临床医学]