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基于Venetoclax的治疗方案用于耐药或复发的儿科急性淋巴细胞白血病
Lin Zhang1, Zhixiao Zhang2, Aidong Lu2
1Department of Pediatrics, Peking University People's Hospital, Beijing, China, zhanglingogojiayou@163.com.
Acta haematologica
|July 8, 2025
概括
基于Venetoclax的疗法在儿科耐药性或复发性急性淋巴细胞白血病 (ALL) 中显示出高缓解率. 这种治疗是有效和安全的,特别是在早期T细胞前体ALL和KMT2A重组ALL.
科学领域:
- 儿科血液学 瘤学 儿科血液学
- 癌症治疗方法 癌症治疗方法
- 白血病研究研究 白血病研究
背景情况:
- 儿科耐药性或复发性急性淋巴细胞白血病 (R/R ALL) 存在重大治疗挑战.
- 基于Venetoclax (VEN) 的疗法正在成为难以治疗的白血病的潜在治疗选择.
研究的目的:
- 为了研究儿科R/R ALL的疗效和生存结果,用基于VEN的治疗方案治疗.
- 评估该患者群体中基于VEN的治疗的响应率和安全概况.
主要方法:
- 用基于VEN的治疗方案治疗R/R ALL的儿科患者的回顾性分析.
- 评估完全缓解 (CR),不完全恢复的CR (CRi) 和客观反应率 (ORR).
- 对生存数据和不良事件的分析.
主要成果:
- 观察到高缓解率 (77.3%),许多人获得了最小残留疾病 (MRD) 负状态.
- 对B细胞ALL (75%) 和T细胞ALL (80%) 的客观应答率均为有利.
- 基于VEN的疗法在早期T细胞前体 (ETP) ALL和KMT2A重组的ALL中表现出有效性,并具有可接受的血液学副作用.
结论:
- 基于VEN的疗法有效地实现了儿科R/R ALL的高缓解率.
- 该疗法具有良好的安全性,特别有利于特定高风险亚型,如ETP ALL和KMT2A重排 ALL.
- 基于VEN的化疗对患有R/R ALL的儿科患者来说是一个有前途的治疗选择,需要缓解诱导.
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