爆发期骨髓扩散性瘤:当代审查和2024年治疗算法
Ayalew Tefferi1, Hassan Alkhateeb2, Naseema Gangat2
1Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, Minnesota, USA. tefferi.ayalew@mayo.edu.
Blood cancer journal
|July 17, 2023
概括
在骨髓增殖性新生瘤 (MPN) 中的白血病转变的预后不好. 同源干细胞移植可以改善存活率,针对性疗法如venetoclax和低甲基化剂对符合条件的患者显示有前途.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 干细胞移植 干细胞移植
背景情况:
- 白血病转变或爆发期 (MPN-BP) 是髓增殖性新生体 (MPN) 的严重并发症.
- MPN-BP与预后不佳有关,在1年后的生存率低于20%,在3年后的生存率低于5%.
- 目前的化疗方案为MPN-BP提供了有限的长期生存益处.
研究的目的:
- 审查当前管理多余爆发 (MPN-BP和MPN-AP) 的骨髓增殖性瘤的情况.
- 讨论全源造血干细胞移植 (AHSCT) 在改善结果中的作用.
- 评估新型治疗策略的有效性,包括桥梁化疗和向药物.
主要方法:
- 对MPN-BP和MPN-AP管理的回顾性数据和临床指南的审查.
- 分析与不同治疗方式相关的生存结果.
- 评估新兴疗法,如venetoclax (Ven),低甲基化剂 (HMA) 和IDH抑制剂.
主要成果:
- 全基性造血干细胞移植 (AHSCT) 提供了长期存活的唯一潜力 (>30%在3年).
- 用Ven-HMA进行过桥化疗有利于MPN-BP,特别是在没有复杂心类型和TET2突变的患者中.
- 对于IDH突变的患者,可以考虑使用IDH抑制剂,单独或与Ven-HMA一起使用.
结论:
- AHSCT是MPN-BP的首选治疗方法,最好在慢性阶段进行.
- 移植前的化疗选择应该是个性化的,平衡潜在的益处与危害移植资格的风险.
- 进一步的受控研究对于优化移植前和移植后的管理以及减少发病率和复发至关重要.
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