在既往未经治疗的成年急性髓系白血病患者中，使用低甲基化剂联合维奈克拉治疗的最佳反应结果。

Outcomes by best response with hypomethylating agent plus venetoclax in adults with previously untreated acute myeloid leukemia.

作者信息

Jain Akriti G, Volpe Virginia O, Wang Chen, Ball Somedeb, Tobon Katherine, Chan Onyee, Padron Eric, Kuykendall Andrew, Lancet Jeffrey E, Komrokji Rami, Sallman David A, Sweet Kendra L

机构信息

Leukemia and Myeloid Disorders , Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA.

Department of Malignant Hematology, Dana Farber Cancer Institute, Boston, MA, USA.

出版信息

Ann Hematol. 2025 Jan;104(1):307-315. doi: 10.1007/s00277-024-05976-6. Epub 2024 Sep 7.

DOI:10.1007/s00277-024-05976-6

PMID:39243312

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11868202/

Abstract

INTRODUCTION

We aimed to compare outcomes of patients with AML treated with frontline hypomethylating agent and venetoclax (HMA + Ven) who achieved complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), or morphologic leukemia-free state (MLFS) as defined by ELN 2022.

METHODS

Patients with AML seen at Moffitt Cancer Center between 2018 and 2022 and treated with HMA + Ven were retrospectively evaluated.

RESULTS

About 120 patients achieved blast clearance with best response of CR in 52 (43.3%), CRh in 22 (18.3%), CRi in 31 (25.8%) and MLFS in 15 (12.5%) patients. Greater proportion of patients with MLFS had a prior myeloid malignancy (p = 0.003) and were treated with prior HMA (p < 0.001). Patients that achieved MLFS as their best response had inferior OS compared to the CR/CRh/CRi cohort (8 months vs. 27 months; p < 0.001). RFS was also worse for the MLFS cohort.

CONCLUSION

To the best of our knowledge, this is the largest study analyzing differences in outcomes of AML patients treated with HMA + Ven based on best response. We noted that prior myeloid malignancy and use of HMA led to more MLFS as best response compared to CR/CRi. The OS and RFS were inferior for MLFS cohort.

摘要

引言

我们旨在比较接受一线低甲基化药物和维奈克拉（HMA+Ven）治疗且达到完全缓解（CR）、部分血液学恢复的完全缓解（CRh）、血液学恢复不完全的完全缓解（CRi）或根据2022年欧洲白血病网络（ELN）定义的形态学无白血病状态（MLFS）的急性髓系白血病（AML）患者的预后。

方法

对2018年至2022年在莫菲特癌症中心就诊并接受HMA+Ven治疗的AML患者进行回顾性评估。

结果

约120例患者达到原始细胞清除，最佳反应为CR的有52例（43.3%），CRh的有22例（18.3%），CRi的有31例（25.8%），MLFS的有15例（12.5%）。MLFS患者中既往有髓系恶性肿瘤的比例更高（p=0.003），且曾接受过HMA治疗（p<0.001）。与CR/CRh/CRi队列相比，以MLFS作为最佳反应的患者总生存期较差（8个月对27个月；p<0.001）。MLFS队列的无复发生存期也更差。