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简化流程:FMS样酪氨酸激酶3突变型急性髓系白血病的回顾性队列研究——初治复发或难治性诊断患者的真实世界治疗模式及临床结局

Streamline: Retrospective Cohort Study of FMS-Like Tyrosine Kinase 3-Mutated Acute Myeloid Leukemia - Real-World Treatment Patterns and Clinical Outcomes of Patients in First Relapse or Refractory Diagnosis.

作者信息

Zeidan Amer M, Yu Rebekah, Wang Yuexi, Lan Ziyu, Grinblatt David L, Elsouda Dina, Spalding James, Block Alana, Touya Maelys, Walker Mark S, Pandya Bhavik J

机构信息

Department of Hematology, Yale School of Medicine and Yale Cancer Center, New Haven, Connecticut, USA.

Real World Evidence, ConcertAI, LLC, Cambridge, Massachusetts, USA.

出版信息

Acta Haematol. 2025 Apr 14:1-12. doi: 10.1159/000545384.

Abstract

INTRODUCTION

Recent advances in genomic research have expanded the treatment landscape for acute myeloid leukemia (AML). This study examined treatment patterns and clinical outcomes among relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-mutated AML patients.

METHODS

This retrospective longitudinal study included patients with confirmed AML diagnosis, FLT3 mutation, and 1st R/R event from 1/1/2015 to 1/31/2023 in the ConcertAI Oncology Dataset. Treatment patterns, FLT3 testing rates, real-world overall survival (rwOS), and real-world time to next treatment (rwTTNT) were studied.

RESULTS

Among the 336 treated patients, 50.6% received FLT3-tyrosine kinase inhibitors (FLT3-TKIs) as first treatment after R/R event, of which 51.8% received gilteritinib. High-intensity chemotherapy used as first treatment after R/R event decreased from 67.9% in 2015 to 20.0% in 2022, while FLT3-TKI utilization rose to 50% over the same period. Among the 246 patients tested for FLT3 at initial AML diagnosis, only 36% were retested at 1st R/R event. Median rwOS and rwTTNT among FLT3-TKI patients were 12.4 months and 2.9 months, respectively.

CONCLUSION

This study reveals a trend toward increasing FLT3-TKI use and highlights the need for repeated FLT3 testing among R/R AML patients. Real-world evidence is vital in understanding R/R AML patient care amidst emerging therapies.

摘要

引言

基因组研究的最新进展拓展了急性髓系白血病(AML)的治疗格局。本研究调查了复发/难治性(R/R)FMS样酪氨酸激酶3(FLT3)突变的AML患者的治疗模式和临床结局。

方法

这项回顾性纵向研究纳入了ConcertAI肿瘤学数据集中2015年1月1日至2023年1月31日确诊为AML、存在FLT3突变且发生首次R/R事件的患者。研究了治疗模式、FLT3检测率、真实世界总生存期(rwOS)和真实世界下次治疗时间(rwTTNT)。

结果

在336例接受治疗的患者中,50.6%在R/R事件后首次接受FLT3酪氨酸激酶抑制剂(FLT3-TKIs)治疗,其中51.8%接受了吉瑞替尼治疗。R/R事件后作为首次治疗的高强度化疗从2015年的67.9%降至2022年的20.0%,而同期FLT3-TKI的使用率升至50%。在246例初诊AML时检测FLT3的患者中,只有36%在首次R/R事件时进行了重新检测。FLT3-TKI治疗患者的rwOS和rwTTNT中位数分别为12.4个月和2.9个月。

结论

本研究揭示了FLT3-TKI使用增加的趋势,并强调了R/R AML患者重复进行FLT3检测的必要性。在新兴疗法中,真实世界证据对于理解R/R AML患者的治疗至关重要。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4d66/12112888/0781f21f9590/aha-2025-0000-0000-545384_F01.jpg

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