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台湾地区 1996 年至 2019 年儿童急性髓系白血病的治疗结果:一项回顾性分析。

Treatment outcomes of pediatric acute myeloid leukemia: a retrospective analysis from 1996 to 2019 in Taiwan.

机构信息

Department of Laboratory Medicine, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, Taiwan.

Department of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, Taiwan.

出版信息

Sci Rep. 2021 Mar 15;11(1):5893. doi: 10.1038/s41598-021-85321-3.

DOI:10.1038/s41598-021-85321-3
PMID:33723338
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7960737/
Abstract

Improvement in outcomes of children with acute myeloid leukemia (AML) is attributed to several refinements in clinical management. We evaluated treatment outcomes of Taiwanese pediatric AML patients in the past 20 years. Overall, 860 de novo AML patients aged 0-18 years and registered in the Childhood Cancer Foundation of R.O.C during January 1996-December 2019 were included. Survival analysis was performed to identify factors that improved treatment outcomes. Regardless of treatment modalities used, patients during 2008-2019 had better 5-year event-free survival (EFS) and overall survival (OS) rates than patients during 1996-2007. For patients received the TPOG-AML-97A treatment, only 5-year OS rates were significantly different between patients diagnosed before and after 2008. Patients with RUNX1-RUNX1T1 had similar relapse-free survival rates, but 5-year OS rates were better during 2008-2019. However, the survival of patients who received hematopoietic stem-cell transplantations (HSCT) did not differ significantly before and after 2008. For patients without relapse, the 5-year OS improved during 2008-2019. Non-relapse mortality decreased annually, and cumulative relapse rates were similar. In conclusion, 5-year EFS and OS rates improved during 2008-2019, though intensities of chemotherapy treatments were similar before and after 2008. Non-relapse mortality decreased gradually. Further treatment strategies including more intensive chemotherapy, novel agents' use, identification of high-risk patients using genotyping and minimal residual disease, early intervention of HSCT, and antibiotic prophylaxis can be considered for future clinical protocol designs in Taiwan.

摘要

儿童急性髓细胞白血病(AML)治疗结果的改善归因于临床管理的多项改进。我们评估了过去 20 年来台湾儿科 AML 患者的治疗结果。共有 860 名年龄在 0-18 岁之间、1996 年 1 月至 2019 年 12 月期间在中华民国儿童癌症基金会注册的初诊 AML 患者被纳入本研究。采用生存分析来确定改善治疗结果的因素。无论使用何种治疗方式,2008-2019 年期间的患者 5 年无事件生存率(EFS)和总生存率(OS)均优于 1996-2007 年期间的患者。对于接受 TPOG-AML-97A 治疗的患者,仅在 2008 年前后诊断的患者之间 5 年 OS 率存在显著差异。RUNX1-RUNX1T1 患者的无复发生存率相似,但 2008-2019 年期间的 5 年 OS 率更好。然而,接受造血干细胞移植(HSCT)的患者在 2008 年前后的生存情况没有显著差异。对于无复发的患者,2008-2019 年期间 5 年 OS 有所提高。非复发死亡率逐年下降,累积复发率相似。总之,尽管 2008 年前后化疗治疗强度相似,但 2008-2019 年期间 5 年 EFS 和 OS 率有所提高。非复发死亡率逐渐下降。未来可以考虑在台湾的临床方案设计中采用包括更强化疗、新型药物的使用、采用基因分型和微小残留病识别高危患者、HSCT 的早期干预和抗生素预防等在内的进一步治疗策略。

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