儿童急性髓系白血病伴骨髓增生异常相关改变临床特征及预后分析

Clinical features and prognosis of childhood acute myeloid leukemia with myelodysplasia-related changes

目的探讨儿童急性髓系白血病伴骨髓增生异常相关改变(AML-MRC)的临床特征及其预后。方法收集2014年6月至2020年3月确诊14例儿童AML-MRC的临床资料,对其临床特征、实验室检查、预后结果进行回顾性分析。结果14例AML-MRC患儿中位发病年龄11岁(范围1~17岁),男9例,女5例,初诊中位白细胞计数8.3×10^(9)/L(范围2.0×10^(9)/L~191.0×10^(9)/L),中位血红蛋白73 g/L(范围44~86 g/L),中位血小板计数75×10^(9)/L(4×10^(9)/L~231×10^(9)/L)。按照FAB分型,AML-M5占71%(10/14)。14例患儿中,多系发育异常4例,具有骨髓增生异常综合征(MDS)病史2例,携带MDS细胞遗传学改变5例,多系发育异常伴MDS细胞遗传学改变2例,MDS病史伴多系发育异常1例。14例患儿中位随访期10.6个月(范围0.4~54.4个月),2例患儿诊断后即放弃治疗,12例患儿可评估疗效。2年总体生存(OS)率为50%±15%;2年无病生存(DFS)率为33%±13%。7例患儿接受造血干细胞移植(HSCT),且均为单倍体造血干细胞移植,5例无病存活,2例死亡,2年OS率为71%±17%,2年DFS率为43%±19%;5例单纯化疗患儿,1例无病存活,3例死亡,1例失访,2年OS率为40%±30%,2年DFS率为30%±24%。移植组及化疗组生存分析结果差异无统计学意义(P>0.05)。结论儿童AML-MRC男性患儿多发,FAB分型以AML-M5多见,预后不良。HSCT可有望改善儿童AML-MRC不良预后,但是由于病例数较少,还需增加病例数进一步观察。

Objective To study the clinical features and prognosis of childhood acute myeloid leukemia with myelodysplasia-related changes(AML-MRC).Methods A retrospective analysis was performed on the medical data of 14 children who were diagnosed with AML-MRC from June 2014 to March 2020,including clinical features,laboratory examination results,and prognosis.Results Among the 14 children with AML-MRC,there were 9 boys and 5 girls,with a median age of 11 years(range:1-17 years),a median leukocyte count of 8.3×10^(9)/L[range:(2.0-191.0)×10^(9)/L],a median hemoglobin level of 73 g/L(range:44-86 g/L),and a median platelet count of 75×10^(9)/L[range:(4-213)×10^(9)/L]at diagnosis.According to the FAB classification,the children with AML-M5 accounted for 71%(10/14).Among the 14 children,4 had multi-lineage dysplasia(MLD),2 had a history of myelodysplastic syndrome(MDS),5 had MDS-related cytogenetic changes,2 had MLD with MDS-related cytogenetic changes,and 1 had a history of MDS with MLD.The median follow-up time was 10.6 months(range:0.4-54.4 months)for 14 children,among whom 2 gave up treatment immediately after diagnosis and 12 had an evaluable treatment outcome.The 2-year overall survival(OS)rate was 50%±15%,and the 2-year disease-free survival(DFS)rate was 33%±13%.Of the 12 children,7 underwent haploidentical hematopoietic stem cell transplantation(HSCT),among whom 5 achieved DFS and 2 died,with a 2-year OS rate of 71%±17%and a 2-year DFS rate of 43%±19%;5 children underwent chemotherapy alone,among whom 1 achieved DFS,3 died,and 1 was lost to follow-up,with a 2-year OS rate of 40%±30%and a 2-year DFS rate of 30%±24%.There was no significant difference in the survival condition between the transplantation and chemotherapy groups(P>0.05).Conclusions Childhood AML-MRC is often observed in boys,and AML-M5 is the most common type based on FAB classification.Such children tend to have a poor prognosis.HSCT is expected to improve the poor prognosis of children with AML-MRC.However due to the small number of cases,it is necessary to increase the number of cases for further observation.