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儿科急性髓系白血病的改善预后:造血干细胞移植的进展。

Improved Outcome in Pediatric Acute Myeloid Leukemia: Progress With Hematopoietic Cell Transplantation.

机构信息

Department of Pediatric Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland

Department of Pediatric Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland.

出版信息

Anticancer Res. 2022 Sep;42(9):4499-4504. doi: 10.21873/anticanres.15951.

DOI:10.21873/anticanres.15951
PMID:36039434
Abstract

BACKGROUND/AIM: Pediatric acute myeloid leukemia (AML) is a heterogenous disease; however, development of diagnostic methods has led to a better understanding of its nature and improvement in therapy outcomes. In this study, we evaluated treatment results in children with AML treated in a single oncology center in comparison with international results.

PATIENTS AND METHODS

Data from 77 children with AML treated in a single oncology center between 1994 and 2020 were analyzed. Patients had been treated according to five consecutive therapy protocols: AML NOPHO 88; ANLL 98; BFM AML 2004; BFM AML 2012, and BFM AML 2019. Five-year overall survival (OS), 5-year event-free survival (EFS) and 5-year relapse-free survival (RFS) were evaluated for each therapy protocol to determine prognostic factors and clarify differences between international and individual center results.

RESULTS

During the observational period, 5-year OS increased from 55.6% to 85.7%, 5-year EFS from 45.7% to 87.5% and 5-year RFS from 51.4% to 85.7%. Hematopoietic cells transplantation (HCT) introduction in early 2000' improved treatment outcomes in the high-risk group, what has been mostly seen in the results of 5-year EFS. Treatment-related mortality was the most frequent cause of death in the analyzed group.

CONCLUSION

Despite the significant improvement in therapy of pediatric AML, treatment outcomes remain unsatisfying. Introduction of HCT relevantly improved therapy results, especially in the high-risk group. International cooperation is crucial because of the small patient numbers in individual oncology centers.

摘要

背景/目的:小儿急性髓细胞白血病(AML)是一种异质性疾病;然而,诊断方法的发展使其本质得到更好的理解,并改善了治疗效果。在本研究中,我们评估了在单一肿瘤中心治疗的 AML 患儿的治疗结果,并与国际结果进行比较。

患者和方法

分析了 1994 年至 2020 年在单一肿瘤中心治疗的 77 例 AML 患儿的数据。根据五个连续的治疗方案对患者进行治疗:AML NOPHO 88;ANLL 98;BFM AML 2004;BFM AML 2012 和 BFM AML 2019。评估每个治疗方案的 5 年总生存率(OS)、5 年无事件生存率(EFS)和 5 年无复发生存率(RFS),以确定预后因素,并阐明国际与个别中心结果之间的差异。

结果

在观察期间,5 年 OS 从 55.6%增加到 85.7%,5 年 EFS 从 45.7%增加到 87.5%,5 年 RFS 从 51.4%增加到 85.7%。2000 年代初引入造血细胞移植(HCT)改善了高危组的治疗结果,这在 5 年 EFS 的结果中最为明显。治疗相关死亡率是分析组中死亡的最常见原因。

结论

尽管小儿 AML 的治疗有了显著改善,但治疗结果仍不尽如人意。HCT 的引入显著改善了治疗结果,特别是在高危组。由于个别肿瘤中心的患者数量较少,国际合作至关重要。

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