TLS/ERG阳性儿童急性髓系白血病临床特点及预后

Clinical features and prognosis of acute myeloid leukemia with TLS/ERG positive in children

目的探讨TLS/ERG基因阳性儿童急性髓系白血病(AML)的临床特征及预后。方法回顾分析2008年6月至2018年12月收治的6例TLS/ERG基因阳性AML患儿的临床资料,并汇总分析国内外文献报道的62例类似患儿的预后资料。结果6例TLS/ERG阳性AML患儿占同期收治的0~18岁AML患儿的1.3%。男4例、女2例,中位年龄9.1岁(4.0~14.0岁)。1例患儿起病时合并中枢神经系统白血病。免疫表型除表达髓系标记CD117、CD13、CD33及CD34外,4例(66.7%)患儿表达CD56。3例患儿诱导化疗后未缓解。5例接受异基因造血干细胞移植患儿中3例出现骨髓复发,中位复发时间为移植后11.6月(3.0~22.0月);1例移植后出现中枢神经系统白血病复发同时骨髓持续缓解;1例骨髓持续缓解。汇总文献既往报道的TLS/ERG阳性AML患儿预后资料,化疗组全部复发,移植组复发率为69.2%,其中1例于复发后行二次移植后持续缓解长期存活。结论TLS/ERG阳性AML发病率极低,其免疫表型具有容易合并CD56表达特点,总体预后差,造血干细胞移植可改善预后,仍作为首次缓解期的推荐治疗,但移植后复发率仍高,亟需改善治疗方法,寻找新的治疗方案。

Objective To explore the clinical features and prognosis of acute myeloid leukemia(AML)with positive TLS/ERG gene in children.Methods The clinical data of TLS/ERG gene positive AML in 6 children admitted from June 2008 to December 2018 were retrospectively analyzed,and the prognosis data of 62 similar cases of children reported in domestic and foreign literature were summarized and analyzed.Results Six AML children with positive TLS/ERG gene was 1.3%of the AML children from 0 to 18 years admitted in the same period.They were 4 boys and 2 girls with a median age of 9.1 years(4.0-14.0 years).One case was complicated with central nervous system(CNS)leukemia at the onset.In addition to expressed myeloid markers CD117,CD 13,CD 33,and CD 34,immunophenotypic expression of CD 56 was found in 4(66.7%)children.There was no remission after induction chemotherapy in 3 children.Bone marrow recurrence occurred in 3 of 5 children receiving allogeneic hematopoietic stem cell transplantation,and the median recurrence time was 11.6 months(3.0-22.0 months)after transplantation.After transplantation,one patient relapsed in the form of CNS leukemia,but the bone marrow was in sustained remission.Bone marrow had the sustained remission in 1 case.By summarizing the prognostic data of children with TLS/ERG positive AML reported in the literature,it was found that all patients in the chemotherapy group experienced recurrence,and the recurrence rate in the transplantation group was 69.2%.One of the patients had sustained remission and long-term survival after a second transplant following recurrence.Conclusions The incidence of TLS/ERG positive AML is extremely low,most of its immunophenotypes are associated with CD56 expression,and the overall prognosis is poor.Hematopoietic stem cell transplantation can improve the prognosis,which is still the recommended treatment for the first remission period,but the recurrence rate after transplantation is still high.It is needed to improve treatment methods and find new treatment options.