摘要
目的 研究异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)治疗伴混合谱系白血病-部分串联重复(mixed lineage leukemia-partial tandem duplications,MLL-PTD)重排的急性髓系白血病(acute myeloid leukemia,AML)患者的临床特征及预后.方法 回顾性分析2020年04月—2023年12月在苏州大学附属第一医院行allo-HSCT治疗的14 例MLL-PTD重排AML患者的临床资料.结果 14 例MLL-PTD重排AML患者中,男性4 例,女性10 例,中位年龄44.5(17～65)岁.14 例患者中,染色体正常核型为11 例,异常核型为3 例.采用标准IA方案诱导治疗8 例,1 疗程诱导治疗获完全缓解(complete remission,CR)1 例,获部分缓解(partial remission,PR)3 例,1 疗程CR+PR共占50.0%(4/8);采用VA方案诱导治疗6 例,1 疗程诱导治疗获CR 5 例,获PR 0 例,1 疗程CR+PR共占83.3%(5/6).巩固治疗后,1 疗程诱导治疗获CR/PR的9 例患者,7 例患者MLL-PTD定量转阴,2 例患者MLL-PTD持续阳性,后续均序贯allo-HSCT.5 例诱导治疗未缓解(no remission,NR)的患者,经IA或VA再诱导1～2 疗程均获CR,其中仅1 例患者MLL-PTD定量转阴,4 例患者MLL-PTD定量持续阳性,后续均序贯allo-HSCT.移植后3 例患者血液学复发,其中2 例患者移植前MLL-PTD定量阳性,1 例患者MLL-PTD定量阴性,复发中位时间333(151～511)d.移植前MLL-PTD阴性组、阳性组患者1 年总生存(OS)率分别为(51.4±20.4)%、(21.4±17.8)%,但差异无统计学意义(P=0.236).结论 MLL-PTD重排AML患者采用VA方案诱导化疗缓解率高,移植前获得MLL-PTD定量阴性有望减少移植后复发,改善患者预后.
Abstract
Objective To study the clinical characteristics and prognosis of patients with allogeneic hematopoietic stem cell transplantation(allo-HSCT)for acute myeloid leukemia(AML)with mixed lineage leukemia-partial tandem duplications(MLL-PTD).Methods The clinical data of 14 patients with MLL-PTD rearrangement AML who underwent allo-HSCT from April 2020 to December 2023 in First Affiliated Hospital of Soochow University were retrospectively analyzed.Results Of the 14 patients with MLL-PTD rearrangement AML,4 were male and 10 were female,with a median age of 44.5(17-65)years.Of the 14 patients,11 had normal karyotypes of chromosomes and 3 had abnormal karyotypes.There were 8 cases of induction therapy with IA regimen,1 case of complete remission(CR)and 3 cases of partial remission(PR)with 1 course of induction therapy,accounting for 50.0%(4/8)of CR+PR with 1 course of therapy.There were 6 cases of induction therapy with VA regimen,5 cases of CR and 0 cases of PR with 1 course of induction therapy,accounting for 83.3%(5/6)of CR+PR with 1 course of therapy.After consolidation therapy,9 patients who obtained CR/PR with 1 course of induction therapy,7 patients with quantitatively negative MLL-PTD,and 2 patients with persistently positive MLL-PTD were followed by sequential allo-HSCT.In 5 patients with induction therapy for no remission(NR),CR was obtained after 1-2 courses of reinduction by IA or VA,of which only 1 patient had a quantitative negative MLL-PTD,and 4 patients had a positive MLL-PTD quantification,and all of them followed with allo-HSCT.3 patients had hematologic relapses after transplantation,2 patients with positive pre-transplant MLL-PTD quantification and 1 patient with negative MLL-PTD quantification,with a median time to relapse of 333(151-511)days.The 1-year overall survival(OS)rates of patients in the pre-transplantation MLL-PTD-negative and positive groups were(51.4±20.4)%and(21.4±17.8)%,respectively,but the difference was not statistically significant(P=0.236).Conclusion Patients with MLL-PTD rearrangement AML have a high remission rate with induction chemotherapy using the VA regimen,and obtaining negative MLL-PTD quantification prior to transplantation is expected to reduce post-transplantation relapse and improve patient prognosis.
维普
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