APL2008方案治疗43例儿童急性早幼粒细胞性白血病的临床分析

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A PL患儿临床资料进行回顾性分析，对进入A PL2008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A PL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A PL预后较好。

Clinical analysis of 43 childhood APL with APL2008

Objective To evaluate the treatment of current status and prognosis in childhood APL with APL2008 ,which was administrated since 2008 in our center .Methods A total of 43 children with newly diagnosed APL between 2008 to 2014 were studied retrospectively .Treatment options and current status were summarized from 28 patients who received APL2008 therapy . Results Studied 43 patients were at median age of 8 years and 4 months ,with 28 boys and 15 girls .The main clinical manifestations were infection ,anemia ,bleeding ,fever ,hepatomegaly ,splenomegaly and lymphadenopathy .The proportions of low ,intermediate and high risk groups were 27 .9% ,48 .8% and 23 .3% ,respectively .Eleven cases could be diagnosed as DIC .Bone marrow morphology showed abnormal elevation of promyelocyte .37 patients had distinctive immunophenotype such as frequent expression of CD33 , CD117 and MPO .PML/RARαfusion gene positive rate was 100% in 43 children and cytogenetic analysis were positive in 37 cases , of which specific genetic lesion in APL cells with t (15 ;17)(q22 ;q12) was found in 28 cases ,and karyotypes was found in 9 cases as infrequent chromosomal abnormalities .In 43 patients ,4 cases were early dead from intracranial hemorrhage at early stage ,and 11 cases were given up early .There were only 2 cases dead ,2 cases relapsed and 1 case lost among 28 APL children ,which enabled ef‐ficacy analysis possible .96 .4% of these 28 cases achieved HCR .The 2 year Kaplan Meier estimates of OS and EFS were 85 .9% ± 7 .6% and 80 .4% ± 8 .8% .But OS and EFS would be 94 .7% ± 5 .1% and 88 .9% ± 7 .4% if 3 patients who had non standard treat‐ment were excluded .Conclusion Childhood APL were characterized by anemia ,bleeding ,fever and infiltration .APL′s coincidence rate between PML/RARa fusion gene and morphology ,immunology and cytogenetics were 95 .3% ,90 .2% and 86 .5% ,respective‐ly .APL2008 significantly improved the prognosis of APL .