双重高剂量化疗和自身干细胞移植用于骨肉瘤与初始肺转移的肺癌
Hyun Jin Park1,2, Kyung Taek Hong1,2, Jung Yoon Choi1,2
1Department of Pediatrics, Seoul National University College of Medicine, Seoul, Republic of Korea.
Pediatric blood & cancer
|July 19, 2025
概括
双重高剂量化疗与自身干细胞移植 (HDC/ASCT) 提供了转移性骨髓瘤的潜在治疗方法. 然而,需要修改治疗方案以减轻毒性,特别是严重的静脉闭塞性疾病.
科学领域:
- 儿科瘤学 儿科瘤学
- 血液学 血液学 血液学
- 干细胞移植 干细胞移植
背景情况:
- 转移性骨肉瘤的预后不佳,尽管局部疾病治疗的进展.
- 在高风险骨髓瘤中,高剂量化疗与自身干细胞移植 (HDC/ASCT) 的疗效和最佳调节方案仍在研究中.
- 这项研究评估了新诊断的患有肺转移的骨髓瘤患者的双重HDC/ASCT.
研究的目的:
- 评估高剂量化疗与自身干细胞移植 (HDC/ASCT) 在患有肺转移的儿科骨髓瘤患者的双重高剂量化疗结果.
- 在这个患者队列中评估特定调节疗法的安全性和有效性 (梅尔法兰,埃托波西德,卡博普拉丁,其次是布苏尔法兰,梅尔法兰).
主要方法:
- 在诊断时,对11名骨髓瘤患者进行了肺转移的双重HDC/ASCT治疗的回顾性分析.
- 在第一个HDC中使用了梅尔法兰,埃托波西德和碳金,随后在第二个HDC中使用了布苏尔法兰和梅尔法兰.
- 随访时间的中位数为57.4个月.
主要成果:
- 5年无事件生存率为41.6%,整体生存率为68.2%.
- 在所有患者中都实现了移植;然而,36.4%的患者在第二次HDC/ASCT期间经历了严重的静脉封闭性疾病 (VOD).
- 与治疗相关的死亡发生在两名患者身上 (VOD和骨髓质疏松综合征),两人死于骨髓瘤进展.
结论:
- 双重HDC/ASCT可能是肺转移骨肉瘤患者的可行治疗选择,与历史数据相比,显示出有希望的生存率.
- 显著的毒性,特别是在第二次移植期间严重的VOD,需要修改调节方案.
- 需要进一步的研究来优化HDC/ASCT协议,以提高转移性骨髓瘤的安全性和疗效.
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