摘要
目的探讨儿童急性早幼粒细胞白血病(APL)的治疗、长期生存和预后因子。方法对该院1998年4月至2005年10月收治的APL患儿46例进行临床分析。诱导缓解采用全反式维甲酸(ATRA)+柔红霉素(DNR)或吡柔吡星(THP),巩固治疗采用大剂量阿糖胞苷与DA(柔红霉素+阿糖胞苷)、HA(高三尖杉酯碱+阿糖胞苷)、EA(足叶乙甙+阿糖胞苷)方案交替给药,维持阶段ATRA与DA,HA,EA方案交替治疗,总疗程为2.5年。结果39例患儿进行了正规治疗,36例(92.3%)获完全缓解(CR)。39例患儿1年、3年和5年总体生存率(OS)分别为(86.1±5.8)%,(76.1±7.5)%和(70.2±8.9)%,1年、3年、5年的无事件生存率(EFS)分别是(78.4±6.8)%,(63.6±8.7)%和(53.1±10.0)%。5年累积复发率(CIR)28.6%。其中WBC≤10.0×109/L者的5年OS[(81.4±10.3)%]明显高于WBC>10.0×109/L者[(51.6±14.7)%,P<0.05]。获CR的PML/RARα融合基因短型(S型)的患儿5例最后全部死亡,而长型(L型)患儿13例无死亡发生,两者差异有统计学意义(P<0.01)。结论ATRA+DNR或THP诱导缓解治疗儿童APL安全、有效,可以作为初发儿童APL的标准诱导治疗方案。联合蒽环类及阿糖胞苷等药物化疗能明显改善长期生存率。高WBC和S型PML/RARα融合基因阳性的患儿预后不佳。
Objective Acute promyelocytic leukemia （APL） is a specific type of hematopoietic malignancy, accounting for 10% of the de novo acute myeloid leukemia （AML）. The data on long-term outcome of APL in children are limited. The aim of this study was to investigate the clinical biological features, diagnosis, prognosis and long-term survival of childhood APL. Methods A total of 46 children with newly diagnosed APL from April 1998 to October 2005 were enrolled into this study. Induction treatment containing all-trans retinoic acid （ATRA） plus daunorubicin （DNR） or pirarubicin （THP） was performed on these patients, followed by 6 courses of chemotherapy consolidation： DNR, homoharringtonine or etoposide plus Ara-C. A maintenance therapy was then administered once 3-6 months. The total period of treatment was 2.5 years. Results Of the 39 patients who had completed the regular treatment, 36 （ 92.3% ） achieved a complete remission. The 5-year cumulative incidence of relapse （CIR） was 28.6%. The estimated overall survival （OS） rates at 1, 3 and 5 years were （86.1 ±5.8）% ,（76.1 ±7.5）% and （70.2 ±8.9）%respeetively, while the event free survival （EFS） rates were （ 78.4 ± 6.8 ） %, （ 63.6 ± 8.7 ） % and （ 53. 1± 10.0 ） % respectively. The 5- year OS rate of patients with WBC less than or equal to 10.0 × 10^9/L was （81.4 ± 10. 3）% , which was significantly higher than that with WBC greater than 10.0 × 10^9/L[ （51.6± 14.7） %, P 〈 0.05 ]. Five patients with RT-PCR positive for PML/RARα S （short） subtype died eventually although all of them achieved CR, but none of the 13 patients with PML/RARα L （long） subtype died. Conclusions Remission induction therapy with ATRA ＋ DNR or THP is effective and safe for newly diagnosed childhood APL. The remission induction therapy combined with chemotherapy containing high/intermediate dose Ara-C can improve the long-term survival rates of APL patients. High WBC count and S subtype of PML-RARa are two poor p
出处
《中国当代儿科杂志》
CAS
CSCD
2007年第1期28-32,共5页
Chinese Journal of Contemporary Pediatrics
关键词
白血病
早幼粒
急性
预后
生存分析
儿童
Leukemia, promyelocytic, acute
Prognosis
Survival analysis
Child
