Clinical value of event-free survival in acute myeloid leukemia
Clinical value of event-free survival in acute myeloid leukemia
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DOI：
10.1182/bloodadvances.2019001150
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发表时间：
2020-04-28
期刊：
影响因子：
7.5
通讯作者：
Cortes, Jorge E.
中科院分区：
文献类型：
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作者：
Maiti, Abhishek;Kantarjian, Hagop M.;Cortes, Jorge E.
无事件生存期（EFS）作为急性髓性白血病（AML）试验终点的价值受到质疑。我们假设EFS的改善可能会减少医疗保健的使用，而不是总生存期（OS）的替代品。在这项回顾性研究中，我们确定了400例接受一线治疗试验的AML患者，OS在2至36个月之间。我们记录了从诊断到死亡或直到患者在干细胞移植（SCT）时被删失的医疗保健使用情况。我们使用相关和回归分析来确定医疗保健使用和EFS之间的关系。在新诊断的AML患者中，35%有不良风险的AML，48%接受了强化化疗，28%接受了低甲基化药物。SCT时删失的中位EFS为9.7个月。无论OS如何，较长的EFS导致医疗保健使用显著下降。这适用于所有观察结果，包括整体医疗保健使用（r = -0.45），门诊、急诊、住院、咨询的总和（r = -0.44）、侵入性操作、实验室和影像学检查的总和（r = -0.51）和血液制品输注（r = -0.19）。这些相关性在达到完全缓解的患者中更强，并且在年龄，治疗和疾病风险亚组中均成立。在新诊断的AML患者中，EFS的改善与所有医疗保健使用的减少相关，无论OS持续时间如何。
The value of event-free survival (EFS) as an end point in acute myeloid leukemia (AML) trials has been questioned. We hypothesized that rather than a surrogate for overall survival (OS), improvement in EFS may decrease the use of health care. In this retrospective study, we identified 400 patients with AML who were treated on first-line therapy trials and had OS between 2 and 36 months. We captured health care use from diagnosis until death or until the patient was censored at stem cell transplantation (SCT). We used correlation and regression analysis to determine the relation between health care use and EFS. Among patients with newly diagnosed AML, 35% had adverse-risk AML, 48% received intensive chemotherapy, and 28% received hypomethylating agents. The median EFS censored at SCT was 9.7 months. Longer EFS led to a significant decline in health care use regardless of OS. This held true for all observations, including overall health care use (r = -0.45), sum of clinic visits, emergency room visits, hospitalizations, consultations (r = -0.44), sum of invasive procedures, laboratory and imaging studies (r = -0.51), and blood product transfusions (r = -0.19). These correlations were stronger for patients who achieved a complete remission and held true across age, treatment, and disease risk subgroups. In patients with newly diagnosed AML, improvement in EFS correlates with a decrease in all health care use irrespective of OS duration.