Luo Tie-Mei, Yu Jie, An Xi-Zhou
Department of Hematology and Oncology, Children's Hospital of Chongqing Medical University/National Clinical Research Center for Child Health and Disorders/Ministry of Education Key Laboratory of Child Development and Disorders/Chongqing Key Laboratory of Pediatrics, Chongqing 401122, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2021 Jun;23(6):613-620. doi: 10.7499/j.issn.1008-8830.2101008.
To study the clinical features and prognosis of children with acute megakaryocytic leukemia (AMKL) and the clinical effect of acute myeloid leukemia 03 (AML03) regimen for the treatment of pediatric AMKL.
The clinical data were collected from 47 children with AMKL who were diagnosed from May 2011 to December 2019. The treatment outcomes and prognostic factors were analyzed. The Kaplan-Meier method and the log-rank test were used for survival analysis.
Among the 47 children with AMKL, 22 with non-Down syndrome-AMKL were treated by the AML03 regimen, with a median follow-up time of 11.4 months. For the 22 non-Down syndrome-AMKL patients, the remission rate of bone marrow cytology was 85% and the negative rate of minimal residual disease (MRD) was 79% after induction Ⅱ, with a 2-year overall survival (OS) rate of (50±13)% and a 2-year event-free survival (EFS) rate of (40±12)%. The group with positive immunophenotypic marker CD56 had significantly lower 2-year EFS and OS rates than the group with negative CD56 ( < 0.05). The group without remission of bone marrow cytology after induction Ⅱ had significantly lower 2-year EFS and OS rates than the group with remission ( < 0.05). The group with positive MRD after induction Ⅱ had a significantly lower 2-year EFS rate than the group with negative MRD ( < 0.05). There was no significant difference in 2-year OS and EFS rates between the patients with transplantation and those without transplantation ( > 0.05).
Children with AMKL tend to have a low remission rate and a poor prognosis. Positive immunophenotypic marker CD56, bone marrow cytology during early treatment response, and MRD results are important factors influencing the prognosis. Allogeneic hematopoietic stem cell transplantation has no significant effect on the prognosis of AMKL.
研究儿童急性巨核细胞白血病（AMKL）的临床特征及预后，以及急性髓系白血病03（AML03）方案治疗儿童AMKL的临床疗效。
收集2011年5月至2019年12月确诊的47例AMKL患儿的临床资料，分析治疗效果及预后因素，采用Kaplan-Meier法和log-rank检验进行生存分析。
47例AMKL患儿中，22例非唐氏综合征AMKL患儿采用AML03方案治疗，中位随访时间11.4个月。22例非唐氏综合征AMKL患儿诱导Ⅱ期后骨髓细胞学缓解率为85%，微小残留病（MRD）阴性率为79%，2年总生存（OS）率为（50±13）%，2年无事件生存（EFS）率为（40±12）%。免疫表型标志物CD56阳性组的2年EFS率和OS率显著低于CD56阴性组（<0.05）。诱导Ⅱ期后骨髓细胞学未缓解组的2年EFS率和OS率显著低于缓解组（<0.05）。诱导Ⅱ期后MRD阳性组的2年EFS率显著低于MRD阴性组（<0.05）。移植患者与未移植患者的2年OS率和EFS率差异无统计学意义（>0.05）。
AMKL患儿缓解率低，预后差。免疫表型标志物CD56阳性、早期治疗反应时的骨髓细胞学及MRD结果是影响预后的重要因素。异基因造血干细胞移植对AMKL的预后无显著影响。
