三氧化二砷治疗急性早幼粒细胞性白血病7年随访

Clinical Observation and Follow up of Acute Promyelocytic Leukemia in Children Treated with Arsenic Trioxide

目的研究治疗剂量三氧化二砷（As2O3）对小儿急性早幼粒细胞性白血病（APL）的疗效和特点。方法对21例初治APL患儿静脉滴注As2O3进行诱导缓解,诱导剂量0.15-0.33 mg/（kg.d）,诱导总量45-720 mg。诱导治疗时间为30-106 d。巩固治疗主要应用化疗,主要以As2O3进行维持治疗,总疗程2-3 a。系统观察其不良反应。随访持续时间5-88个月。结果21例初治APL患儿达完全缓解（CR）13例（61.9%）;部分缓解（PR）7例（33.3%）,经化疗最后均达CR;不缓解（NR）1例。21例治疗期间无死亡,复发率9.5%,无瘤生存率（DFS）为85.7%。18例APL均处于CR中,随访持续时间5-88（37.3±23.6）个月,其中12例已停止用药,停药时间1-51个月,8例停药者进行了PML/RARα融合基因检测,均为阴性。未见髓外白血病发生。主要不良反应包括胃肠道反应、肝功能损害、高白细胞综合征等。治疗相关不良反应轻,多能耐受。结论As2O3治疗小儿APL的CR率和长期生存率高,复发率低,不良反应轻,与全反式维A酸和其他化疗药物无交叉耐药,并可单独用于巩固和维持治疗,是目前治疗小儿APL较理想的药物。

Objective To study the clinical efficacy of acute promyelocytic leukemia（APL）in children with arsenic trioxide（As2O3） of therapeutic dose.Methods Twenty-one children with APL were treated in 7 years with As2O3 to induce,consolidate and maintain.The induce dose was 0.15-0.33 mg/（kg·d）,total induce dose was 45-720 mg.The time of induction was 30-106 days.Course of treatment was 2 to 3 years.Adverse effects were observed.Duration of follow up was 5-88 months.Results The complete remission（CR） were 13 cases（61.9%）,partial remission（PR） were 7 cases（33.3%） and non remission（NR） were 1 case for the previously untreated group.During the treatment none of the 21children was dead,relapse rate was 9.5% and disease free survival was 85.7%.Eighteen cases were in complete remission,12 of them had stopped taking drugs for 1 to 51 months.Eight cases had the fusion gene detection,all of them were negative.The main adverse effect included gastric-intestinal reaction,hepatic demage,high white blood cell syndrome and so on.There were mild side effects,which were tolerant.Extramedullary leukemia was not occurred.Conclusions The CR rate and long-term survival are high and the relapse rate is low in APL children treated with As2O3.It can also be used separately in maintenance treatment.As2O3 is a more ideally agent treating APL in children at present.