在常规临床实践中用于CML患者的Ponatinib:PONDEROSA研究
Thomas Schenk1,2, Christian Fabisch3,4, Thomas Ernst3,4
1Abteilung Hämatologie und Internistische Onkologie, Klinik für Innere Medizin II, Universitätsklinikum Jena, Jena, Germany. thomas.schenk@med.uni-jena.de.
Annals of hematology
|January 14, 2026
概括
波纳替尼有效治疗慢性髓性白血病 (CML),包括具有T315I突变的耐药病例. 庞德罗萨研究表明其在现实世界中的有效性和安全性,强调需要个性化剂量来管理心血管风险.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 波纳替尼是慢性髓性白血病 (CML) 的铁氨酸激酶抑制剂.
- 动脉封闭事件 (AOEs) 是波纳替尼的重大安全问题.
- 在CML治疗中,T315I BCR::ABL1突变提出了挑战.
研究的目的:
- 评估ponatinib在常规临床实践中的有效性和安全性.
- 评估德国和捷克共和国用波纳提尼布治疗的CML患者的结局.
- 调查个性化剂量对疗效和安全的影响.
主要方法:
- 在99名成年CML患者的观察队列研究中,他们接受了ponatinib治疗.
- 2015年至2022年间在31个中心收集的数据.
- 随访时间中位数为22个月,分析不良事件,反应率和存活率.
主要成果:
- 64.6%的患者经历了不良事件;12.1%有严重的心血管/脑血管事件.
- 58.6%的人取得了主要分子响应 (MMR),比基线 (19.0%) 有显著改善.
- 预计2年无进展生存率为84.4%,整体生存率为85.7%.
结论:
- 波纳替尼在现实世界CML治疗中表现出临床有效性.
- 个性化剂量对于平衡ponatinib的疗效与心血管安全至关重要.
- 波纳替尼 (ponatinib) 作为一种有价值的桥梁疗法,用于等待全源干细胞移植的患者.
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