不适合强化治疗的急性髓系白血病患者一线阿扎胞苷或地西他滨联合或不联合维奈托克的真实世界疗效。

Real-world effectiveness of first-line azacitidine or decitabine with or without venetoclax in acute myeloid leukemia patients unfit for intensive therapy.

机构信息

Department of Medicine II, Hematology and Oncology, Goethe University Frankfurt, University Hospital, Frankfurt, Germany.

Carreras Leukemia Center, Hematology, Oncology, Immunology, Philipps University Marburg, and University Hospital Giessen and Marburg, Marburg, Germany.

出版信息

Eur J Haematol. 2024 Nov;113(5):623-630. doi: 10.1111/ejh.14278. Epub 2024 Jul 18.

DOI:10.1111/ejh.14278

PMID:39023132

Abstract

BACKGROUND

First-line treatment in patients with acute myeloid leukemia (AML) unfit for intensive therapy is the combination of a hypomethylating agent (HMA) with venetoclax (VEN). However, retrospective data confirming the benefits of this regimen outside of clinical trials have shown conflicting results.

METHODS

We performed a multicenter retrospective analysis of outcomes with first-line HMA-VEN versus HMA in AML patients unfit for intensive chemotherapy.

RESULTS

A total of 213 patients were included from three German hospitals (125 HMA-VEN, 88 HMA). Median overall survival in the HMA-VEN cohort was 7.9 months (95% confidence interval [CI], 5.1-14.7) versus 4.9 months (3.1-7.1) with HMA. After 1 year, 42% (95% CI, 33-54) and 19% (12-30) of patients were alive, respectively (hazard ratio [HR] for death, 0.64; 95% CI, 0.46-0.88). After adjusting for clinical and molecular baseline characteristics, treatment with HMA-VEN remained significantly associated with both prolonged survival (HR, 0.48; 95% CI, 0.29-0.77) and time to next treatment (HR, 0.63; 95% CI, 0.47-0.85). Patients who achieved recovery of peripheral blood counts had a favorable prognosis (HR for death, 0.52; 95% CI, 0.33-0.84).

DISCUSSION

These data align with findings from the pivotal VIALE-A trial and support the use of HMA-VEN in patients unfit for intensive therapy.

摘要

背景

不适合强化治疗的急性髓系白血病（AML）患者的一线治疗是低甲基化药物（HMA）联合 venetoclax（VEN）。然而，临床试验之外的回顾性数据证实了该方案的益处，但结果相互矛盾。

方法

我们对不适合强化化疗的 AML 患者进行了一线 HMA-VEN 与 HMA 的多中心回顾性分析。

结果

共纳入来自德国三家医院的 213 例患者（HMA-VEN 组 125 例，HMA 组 88 例）。HMA-VEN 组的中位总生存期为 7.9 个月（95%置信区间[CI]，5.1-14.7），HMA 组为 4.9 个月（3.1-7.1）。1 年后，42%（95%CI，33-54）和 19%（12-30）的患者存活，死亡风险比（HR）分别为 0.64（95%CI，0.46-0.88）。在调整了临床和分子基线特征后，HMA-VEN 治疗与生存时间延长（HR，0.48；95%CI，0.29-0.77）和下一次治疗时间（HR，0.63；95%CI，0.47-0.85）均显著相关。外周血细胞计数恢复的患者预后良好（HR 为 0.52；95%CI，0.33-0.84）。