Maiti Abhishek, Kantarjian Hagop M, Popat Vinita, Borthakur Gautam, Garcia-Manero Guillermo, Konopleva Marina Y, DiNardo Courtney D, Verstovsek Srdan, Andreeff Michael, Kadia Tapan M, Ajufo Helen O, Goswamy Rohit V, Blanco Carlos, Velasquez Miguel, Daver Naval G, Pemmaraju Naveen, Pierce Sherry R, Wierda William G, Kornblau Steven M, Ravandi Farhad, Cortes Jorge E
Department of Leukemia and.
Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX.
Blood Adv. 2020 Apr 28;4(8):1690-1699. doi: 10.1182/bloodadvances.2019001150.
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.
无事件生存期（EFS）作为急性髓系白血病（AML）试验终点的价值受到了质疑。我们假设，EFS的改善并非总生存期（OS）的替代指标，而是可能会减少医疗保健的使用。在这项回顾性研究中，我们确定了400例接受一线治疗试验且OS在2至36个月之间的AML患者。我们记录了从诊断到死亡或直到患者在干细胞移植（SCT）时被截尾期间的医疗保健使用情况。我们使用相关性和回归分析来确定医疗保健使用与EFS之间的关系。在新诊断的AML患者中，35%患有高危AML，48%接受了强化化疗，28%接受了去甲基化药物治疗。SCT时截尾的中位EFS为9.7个月。无论OS如何，更长的EFS都会导致医疗保健使用显著下降。所有观察指标均如此，包括总体医疗保健使用（r = -0.45）、门诊就诊、急诊就诊、住院、会诊总和（r = -0.44）、侵入性操作、实验室和影像学检查总和（r = -0.51）以及血液制品输注（r = -0.19）。对于实现完全缓解的患者，这些相关性更强，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，无论OS持续时间如何，EFS的改善都与所有医疗保健使用的减少相关。
