Kristin M Zimmerman Savill, Jonathan K Kish, Choo Hyung Lee, Prathamesh Pathak, Paul D'Amico, Alexander Russell-Smith, Ajeet Gajra
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引用次数: 0
摘要
背景：Glasdegib （GLAS）和venetoclax （VEN）在美国被批准用于治疗75岁以上或伴有合并症的急性髓系白血病（AML）患者，无法进行强化诱导化疗。这些疗法的社区肿瘤学疗效有限。研究设计和方法：本回顾性图表综述了美国一线（1l） GLAS或VEN治疗AML患者的特征、治疗模式和结果。该研究没有设计或支持GLAS和VEN队列的比较。结果：在接受1 L GLAS治疗的50例患者中（82.0%采用低剂量阿糖胞苷），50.0%达到完全缓解（CR）、形态无白血病状态（MLFS）或部分缓解（PR）。中位总生存期（OS）为6.9个月（95% CI: 5.4-8.9）。试验匹配的GLAS队列占研究中所有GLAS治疗患者的80.0%。在83例接受1 L VEN治疗的患者中（94.0%使用低甲基化药物），51.8%达到CR、MLFS或PR，中位OS为8.4个月（95% CI: 5.7-16.2）， 31.3%符合关键试验资格标准。结论：这项观察性研究支持GLAS和VEN在现实世界中治疗AML患者的临床益处。
Real-world (RW) study of outcomes for acute myeloid leukemia (AML) patients treated with glasdegib or venetoclax in US community oncology practices.
Background: Glasdegib (GLAS) and venetoclax (VEN) are approved in the US for treating AML in patients aged 75+ or with comorbidities precluding intensive induction chemotherapy. Community oncology outcomes for these therapies are limited.
Research design and methods: This retrospective chart review summarized characteristics, treatment patterns, and outcomes of US patients treated with first-line (1 L) GLAS or VEN for AML using descriptive statistics. The study was not designed or powered to compare GLAS and VEN cohorts.
Results: Among 50 patients receiving 1 L GLAS (82.0% with low-dose cytarabine), 50.0% achieved complete remission (CR), morphological leukemia-free state (MLFS), or partial response (PR). Median overall survival (OS) was 6.9 months (95% CI: 5.4-8.9). A trial-matched GLAS cohort represented 80.0% of all GLAS-treated patients in the study. Among 83 patients receiving 1 L VEN (94.0% with a hypomethylating agent), 51.8% achieved CR, MLFS, or PR, median OS was 8.4 months (95% CI: 5.7-16.2), and 31.3% met pivotal trial eligibility criteria.
Conclusions: This observational study supports the clinical benefit of GLAS and VEN in treating AML patients in the real-world setting.
期刊介绍：
Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.
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