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治疗儿童急性白血病移植后微小残留病的可行性

Feasibility of treating post-transplantation minimal residual disease in children with acute leukemia.

作者信息

Shah Nirali N, Borowitz Michael J, Robey Nancy C, Gamper Christopher J, Symons Heather J, Loeb David M, Wayne Alan S, Chen Allen R

机构信息

Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland; Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.

Department of Pathology, Johns Hopkins University, Baltimore, Maryland.

出版信息

Biol Blood Marrow Transplant. 2014 Jul;20(7):1000-7. doi: 10.1016/j.bbmt.2014.03.021. Epub 2014 Mar 27.

Abstract

Outcomes are poor for patients with hematologic malignancies who experience overt relapse after allogeneic hematopoietic stem cell transplantation (HCT). Data on outcomes of post-transplantation minimal residual disease (MRD) are limited. In this single-institution, retrospective cohort analysis of children with acute leukemia and myelodysplastic syndrome, we document the pattern of relapse with a primary focus on outcomes of post-transplantation MRD. Forty of 93 patients (43%) who underwent a first allogeneic HCT and had systematic pretransplantation and post-transplantation MRD evaluations at +30, +60, +90, +180 days and +1 and +2 years post-transplantation experienced relapse. The median time to relapse was 4.8 months post-transplantation, with a median survival of 4 months post-relapse. Despite frequent, systematic, routine post-HCT disease restaging evaluation, 31 patients (78%) presented with overt disease at the time of relapse. Seven patients with acute leukemia who had post-transplantation MRD presented at a median of 1 month post-transplantation. Owing to rapid disease progression or treatment-related mortality, there was no improvement in survival in those patients whose leukemia was detected in a state of MRD post-transplantation. Our results suggest that early intervention strategies targeting post-transplantation MRD for relapse prevention in acute leukemia may not be feasible.

摘要

对于异基因造血干细胞移植(HCT)后出现明显复发的血液系统恶性肿瘤患者,预后较差。关于移植后微小残留病(MRD)的预后数据有限。在这项针对急性白血病和骨髓增生异常综合征儿童的单机构回顾性队列分析中,我们记录了复发模式,主要关注移植后MRD的预后。93例接受首次异基因HCT并在移植后+30、+60、+90、+180天以及移植后+1年和+2年进行系统性移植前和移植后MRD评估的患者中,有40例(43%)复发。复发的中位时间为移植后4.8个月,复发后的中位生存期为4个月。尽管进行了频繁、系统的常规移植后疾病重新分期评估,但31例患者(78%)在复发时出现明显疾病。7例移植后有MRD的急性白血病患者在移植后中位1个月时出现复发。由于疾病进展迅速或与治疗相关的死亡率,那些在移植后MRD状态下被检测出白血病的患者的生存率没有改善。我们的结果表明,针对急性白血病移植后MRD进行预防复发的早期干预策略可能不可行。

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