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泰国急性白血病工作组提供的关于伴有内部串联重复(-ITD)突变的成人急性髓系白血病的真实世界数据。

Real-world data on adult AML with -ITD mutation from the Thai acute leukemia working group.

作者信息

Rattanathammethee Thanawat, Rattarittamrong Ekarat, Wanitpongpun Chinadol, Kungwankiattichai Smith, Owattanapanich Weerapat, Chanswangphuwana Chantiya, Polprasert Chantana, Piyajaroenkij Thanakrit, Niparuck Pimjai, Saengboon Supawee, Limvorapitak Wasithep, Silpsamrit Panachai, Prayongratana Kannadit, Sriswasdi Chantrapa, Julamanee Jakrawadee, Saelue Pirun, Sasakul Aimwipa, Jit-Ueakul Dusit, Nakhakes Chajchawan, Tantiworawit Adisak

机构信息

Division of Hematology, Department of Internal Medicine, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.

Division of Hematology, Department of Internal Medicine, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.

出版信息

Front Oncol. 2025 Aug 29;15:1606943. doi: 10.3389/fonc.2025.1606943. eCollection 2025.

Abstract

BACKGROUND

-ITD mutations are among the most common genetic alterations in acute myeloid leukemia (AML) and are associated with poor clinical outcomes. However, data from low- and middle-income countries remain limited. This study aimed to investigate the prevalence, clinical characteristics, treatment patterns, and outcomes of adult AML patients with -ITD mutations in Thailand.

METHODS

We analyzed data from 360 adult patients with newly diagnosed AML, prospectively collected from 11 institutions nationwide between 2016 and 2023. -ITD mutational status, clinical features, response to therapy, and survival outcomes were compared between -ITD and -wild-type patients.

RESULTS

-ITD mutations were detected in 28.1% of patients. -ITD patients had higher white blood cell counts, bone marrow blast percentages, and NPM1 co-mutations compared to wild-type . Induction chemotherapy rates were similar, but FLT3 inhibitor use was nearly absent. Complete remission was achieved in 55.7% of -ITD patients versus 66.5% in wild-type . Median overall survival was significantly shorter in the -ITD group (8.8 vs. 13.2 months, p=0.039), while relapse-free survival was not significantly different. Multivariable analysis confirmed -ITD mutation as an independent predictor of poor overall survival.

CONCLUSIONS

In this nationwide real-world study, -ITD AML was associated with inferior outcomes despite comparable induction therapy. Limited access to FLT3-targeted treatments and stem cell transplantation may contribute to these disparities. Our findings highlight the urgent need for expanding access to molecular testing and targeted therapies in resource-limited settings.

摘要

背景

内部串联重复(ITD)突变是急性髓系白血病(AML)中最常见的基因改变之一,与不良临床预后相关。然而,低收入和中等收入国家的数据仍然有限。本研究旨在调查泰国成年AML患者中ITD突变的患病率、临床特征、治疗模式及预后。

方法

我们分析了2016年至2023年间从全国11家机构前瞻性收集的360例新诊断成年AML患者的数据。比较了ITD和野生型患者的ITD突变状态、临床特征、治疗反应及生存预后。

结果

28.1%的患者检测到ITD突变。与野生型患者相比,ITD患者白细胞计数、骨髓原始细胞百分比及NPM1共突变更高。诱导化疗率相似,但几乎未使用FLT3抑制剂。ITD患者完全缓解率为55.7%,野生型患者为66.5%。ITD组中位总生存期显著缩短(8.8个月对13.2个月,p=0.039),而无复发生存期无显著差异。多变量分析证实ITD突变是总生存期不良的独立预测因素。

结论

在这项全国性的真实世界研究中,尽管诱导治疗相当,但ITD AML的预后较差。FLT3靶向治疗和干细胞移植的可及性有限可能导致了这些差异。我们的研究结果凸显了在资源有限的环境中扩大分子检测和靶向治疗可及性的迫切需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ad7f/12425767/0a7088a94453/fonc-15-1606943-g001.jpg

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