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目的评价采用的APL2008方案治疗儿童急性早幼粒细胞白血病(APL)的治疗现状及预后。方法对2008~2014年新诊43例APL患儿临床资料进行回顾性分析,对进入APL2008方案治疗的28例患儿进行疗效统计,总结治疗方案和现状。结果本组患儿中位年龄8岁4个月,男28例,女15例,以感染、贫血、出血、发热伴肝脾淋巴结肿大为主要临床表现。标、中、高危组所占比例分别为27.9%、48.8%和23.3%。11例诊断弥散性血管内凝血(DIC)。骨髓形态学检查以早幼粒细胞(PMC)异常升高为主。37例患儿免疫分型共同特征表现为CD33、CD117、MPO高表达。43例进行PML/RARα融合基因检测,阳性率100%,同时进行细胞遗传学分析37例阳性,其中t(15;17)(q22;q11.2)经典遗传学异常28例,有9例少见核型。43例患者中,早期死亡4例,都死于颅内出血,11例早期放弃,28例可进行疗效分析APL患儿中,死亡2例,复发2例,失访1例。血液学缓解(HCR)率为96.4%,4年总生存(OS)率和无事件生存(EFS)率分别为(85.9±7.6)%和(80.4±8.8)%,去除不规范治疗患儿,2年OS和EFS率分别为(94.7±5.1)%和(88.9±7.4)%。结论 APL的临床表现以贫血、出血、发热伴浸润为主要特征。APL的PML/RARa融合基因与形态学诊断、免疫分型及染色体符合率分别为95.3%、90.2%和86.5%。APL2008方案治疗儿童APL预后较好。
Objective To evaluate the treatment status and prognosis of APL2008 in children with acute promyelocytic leukemia (APL). Methods The clinical data of 43 newly diagnosed APL children from 2008 to 2014 were analyzed retrospectively. The therapeutic effect of 28 APL patients treated with APL2008 was summarized, and the treatment plan and current status were summarized. Results The median age of this group was 8 years and 4 months. There were 28 males and 15 females. The main clinical manifestations were infection, anemia, hemorrhage and fever with hepatosplenomegaly and lymphadenopathy. The proportions of standard, middle and high-risk groups were 27.9%, 48.8% and 23.3% respectively. Eleven cases were diagnosed with disseminated intravascular coagulation (DIC). Bone marrow morphological examination with promyelocytic (PMC) abnormal increase. The common features of immunological typing in 37 children were high expression of CD33, CD117 and MPO. The positive rate of PML / RARα fusion gene was detected in 43 cases (100%), while the cytogenetic analysis was positive in 37 cases. T (15; 17) (q22; q11.2) had 28 cases of classical genetic abnormalities and 9 cases were rare Karyotype. Of the 43 patients, 4 died of early death, all died of intracranial hemorrhage, 11 were given up early, and 28 patients were evaluated for efficacy. Among the children with APL, 2 died, 2 relapsed and 1 lost. The overall rate of 4-year overall survival (OS) and event-free survival (EFS) were 96.9% for hematological response (HCR) and 85.4% for healthy children (80.4 ± 8.8% The two-year OS and EFS rates were (94.7 ± 5.1)% and (88.9 ± 7.4)%, respectively. Conclusion The clinical manifestations of APL with anemia, hemorrhage, fever with infiltration as the main feature. APL PML / RARa fusion gene and morphological diagnosis, immunophenotyping and chromosome coincidence rates were 95.3%, 90.2% and 86.5%. APL2008 program for the treatment of children with APL better prognosis.