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诱导方案对急性髓系白血病患者生存结局的有效性:来自2001年至2015年的真实世界数据。

Effectiveness of induction regimens on survival outcome in acute myeloid leukemia patients: a real-world data from 2001 to 2015.

作者信息

Hou Hsin-An, Tzeng Huey-En, Liu Hung-Yi, Chou Wen-Chien, Tien Hwei-Fang, Chien Li-Nien

机构信息

Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.

Division of Hematology and Oncology, Department of Internal Medicine, Taipei Medical University Hospital, Taipei, Taiwan.

出版信息

Ann Hematol. 2022 Jan;101(1):109-118. doi: 10.1007/s00277-021-04670-1. Epub 2021 Oct 13.

Abstract

Since patients with acute myeloid leukemia (AML) in the real world have a much different clinical picture than patients recruited in the clinical trials, obtaining real-world evidence of medication adoption is important for therapeutic efficiency and safety. This study used three population-based data in Taiwan, the National Health Insurance Research Database, Taiwan Cancer Registry, and National Death Registry, between 2001 and 2015, to investigate the effect of conventional chemotherapy (CCT) versus non-conventional chemotherapy (NCCT) on the overall survival (OS) of patients with AML (n = 7,763). Cox proportional hazard regression was used to estimate the hazard ratios (HR) of different treatments on the risk of mortality. To reduce the potential selection bias, we used the inverse probability of treatment weighting based on the propensity score to balance the baseline characteristics between patients receiving CCT and NCCT. The median survival time for CCT and NCCT arms was 10.2 months (95% confidence interval (95% CI): 9.7-10.9) and 4.1 months (95% CI: 3.8-4.5), respectively. Compared to the patients received NCCT, those receiving CCT had a lower risk of mortality (HR 0.63 (95% CI: 0.59-0.67, P < 0.001). Subgroup analysis showed that CCT did benefit patients in different gender, age, comorbidity, and socioeconomic status (SES) groups. In conclusion, the real-world population-based data exhibited CCT were more likely to be prescribed for patients with AML of younger age, fewer comorbidities, diagnosed recently (2011-2015), and higher SES. In fact, CCT had better treatment outcomes than NCCT in terms of OS for adult patients diagnosed with AML.

摘要

由于现实世界中急性髓系白血病(AML)患者的临床情况与临床试验中招募的患者有很大不同,获取药物使用的真实世界证据对于治疗效果和安全性很重要。本研究使用了2001年至2015年间台湾的三个基于人群的数据,即国民健康保险研究数据库、台湾癌症登记处和国民死亡登记处,来调查传统化疗(CCT)与非传统化疗(NCCT)对AML患者(n = 7763)总生存期(OS)的影响。采用Cox比例风险回归来估计不同治疗对死亡风险的风险比(HR)。为了减少潜在的选择偏倚,我们使用基于倾向评分的治疗权重逆概率来平衡接受CCT和NCCT患者之间的基线特征。CCT组和NCCT组的中位生存时间分别为10.2个月(95%置信区间(95%CI):9.7 - 10.9)和4.1个月(95%CI:3.8 - 4.5)。与接受NCCT的患者相比,接受CCT的患者死亡风险更低(HR 0.63(95%CI:0.59 - 0.67,P < 0.001)。亚组分析表明,CCT确实使不同性别、年龄、合并症和社会经济地位(SES)组的患者受益。总之,基于真实世界人群的数据显示,CCT更有可能被开给年龄较小、合并症较少、近期(2011 - 2015年)诊断且SES较高的AML患者。事实上,就成年AML患者的OS而言,CCT的治疗效果优于NCCT。

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