A Real-World Evaluation of Frontline Treatment for Acute Myeloid Leukemia With Azacitidine Plus Venetoclax

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia Pub Date : 2025-02-01 DOI:10.1016/j.clml.2025.01.024

Joseph Brandwein, David Page, Elena Liew, Mark Hnatiuk, Lauren Bolster, Marlene Hamilton, Daniel Sawler, Peng Wang

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引用次数: 0

Abstract

Background

The combination of venetoclax + azacitidine (VenAza) has become the standard frontline treatment for older unfit AML patients.

Methods

We analyzed outcomes using VenAza for previously untreated unfit AML patients at a single center between 2020-2024.

Results

The overall response rate (ORR) was 69/105 (66%), was highest for patients with NPM1 (78%) and IDH1/2 (82%) mutations and lowest with TP53 mutations (40%). The median overall survival (OS) was 9.6 months, and 16.3 months for those achieving CR/CRi. There was no significant difference in OS between those achieving CR and CRi (p = 0.077). Patients treated between 2022-24 had a lower early death rate (8% vs. 22%) and better OS (median 10.4 vs 5.8 mos, p = 0.033) than those treated between 2020-21. There was no difference in OS between by age grouping or for patients with prior hypomethylating agent exposure. Patients with FLT3-ITD/RAS or TP53 mutations had an inferior OS compared with the other patients (median OS 8.1, 1.7 and 16 months, respectively). On multivariate analysis, achievement of CR/CRi was associated with better OS (p < 0.001), and FLT3-ITS/RAS/TP53 mutations were associated with inferior OS (p = 0.003), while ELN2022 risk group was not associated with OS. The median DFS for patients achieving CR/CRi was 7.1, 4.9 and 21 mos, for those with FLT3-ITD/RAS, TP53 and others, respectively (p = 0.003).

Conclusions

This real-world analysis confirmed the prognostic importance of the mutational risk classification with VenAza treatment. OS was inferior to that reported in the VIALE A study but did improve over time.

查看原文本刊更多论文

阿扎胞苷联合Venetoclax一线治疗急性髓系白血病的现实世界评价。

背景Venetoclax+阿扎胞苷（VenAza）联合疗法已成为老年不适合AML患者的标准一线治疗方法：我们分析了一个中心在2020-2024年间使用VenAza治疗既往未经治疗的不适合AML患者的结果：总反应率（ORR）为69/105（66%），NPM1（78%）和IDH1/2（82%）突变患者的总反应率最高，TP53突变患者的总反应率最低（40%）。中位总生存期（OS）为9.6个月，达到CR/CRi的患者为16.3个月。达到CR和CRi的患者的OS无明显差异（p = 0.077）。与2020-21年间接受治疗的患者相比，2022-24年间接受治疗的患者早期死亡率较低（8% vs. 22%），OS较好（中位10.4个月 vs. 5.8个月，p = 0.033）。不同年龄组或曾接触过低甲基化药物的患者的 OS 没有差异。与其他患者相比，FLT3-ITD/RAS或TP53突变患者的OS较差（中位OS分别为8.1个月、1.7个月和16个月）。多变量分析显示，达到CR/CRi与较好的OS相关（p < 0.001），FLT3-ITS/RAS/TP53突变与较差的OS相关（p = 0.003），而ELN2022风险组与OS无关。FLT3-ITD/RAS、TP53和其他基因突变患者达到CR/CRi的中位DFS分别为7.1、4.9和21天（p = 0.003）：这项真实世界分析证实了VenAza治疗中突变风险分类对预后的重要性。OS 不如 VIALE A 研究报告的结果，但随着时间的推移确实有所改善。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Lymphoma, Myeloma & Leukemia ONCOLOGY-HEMATOLOGY

CiteScore

2.70

自引率

3.70%

发文量

1606

审稿时长

26 days

期刊介绍： Clinical Lymphoma, Myeloma & Leukemia is a peer-reviewed monthly journal that publishes original articles describing various aspects of clinical and translational research of lymphoma, myeloma and leukemia. Clinical Lymphoma, Myeloma & Leukemia is devoted to articles on detection, diagnosis, prevention, and treatment of lymphoma, myeloma, leukemia and related disorders including macroglobulinemia, amyloidosis, and plasma-cell dyscrasias. The main emphasis is on recent scientific developments in all areas related to lymphoma, myeloma and leukemia. Specific areas of interest include clinical research and mechanistic approaches; drug sensitivity and resistance; gene and antisense therapy; pathology, markers, and prognostic indicators; chemoprevention strategies; multimodality therapy; and integration of various approaches.