威尼斯人
低甲基化剂
髓系白血病
医学
耐受性
肿瘤科
人口
内科学
白血病
不利影响
慢性淋巴细胞白血病
基因
DNA甲基化
生物化学
基因表达
化学
环境卫生
作者
Yasmin Abaza,Eric S. Winer,Guru Subramanian Guru Murthy,Rory M. Shallis,Andrew Matthews,Talha Badar,Emily Geramita,Vamsi Kota,Alok Swaroop,Peter H. Doukas,Danielle Bradshaw,Irene Helenowski,Yingzhe Liu,Hui Zhang,Annie Im,Mark R. Litzow,Alexander E. Perl,Ehab Atallah,Jessica K. Altman
摘要
Abstract Venetoclax (VEN) combined with hypomethylating agents (HMAs) is the standard of care for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) unfit for intensive chemotherapy. To date, real‐world data published on HMAs plus VEN have been either single‐center studies or using community‐based electronic databases with limited details on mutational landscape, tolerability, and treatment patterns in elderly patients. Therefore, we conducted a multicenter retrospective study to assess the real‐world experience of 204 elderly patients (≥75 years) with newly diagnosed AML treated with HMAs plus VEN from eight academic centers in the United States. Overall, 64 patients achieved complete remission (CR; 38%) and 43 CR with incomplete count recovery (CRi; 26%) for a CR/CRi rate of 64%, with a median duration of response of 14.2 months (95% CI: 9.43, 22.1). Among responders, 63 patients relapsed (59%) with median overall survival (OS) after relapse of 3.4 months (95% CI, 2.4, 6.7). Median OS for the entire population was 9.5 months (95% CI, 7.85–13.5), with OS significantly worse among patients with TP53 ‐mutated AML (2.5 months) and improved in patients harboring NPM1 , IDH1 , and IDH2 mutations (13.5, 18.3, and 21.1 months, respectively). The 30‐day and 60‐day mortality rates were 9% and 19%, respectively. In conclusion, HMAs plus VEN yielded high response rates in elderly patients with newly diagnosed AML. The median OS was inferior to that reported in the VIALE‐A trial. Outcomes are dismal after failure of HMAs plus VEN, representing an area of urgent unmet clinical need.
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