对玛利多米德-PACE作为攻击性复发性和耐火性多发性骨髓瘤的救援方案的回顾性研究
Deniz Gezer1,2, Melanie Schmitt Nogueira1,2, Martin Kirschner1,2
1Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.
European journal of haematology
|June 20, 2024
概括
波马利多米德-PACE疗法在治疗攻击性复发性耐火性多发性骨髓瘤 (RRMM) 和血细胞白血病 (PCL) 中表现有前途. 这种密集治疗在重度预治疗的患者中取得了快速反应,提供了潜在的挽救选择.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床试验 临床试验
背景情况:
- 多发性骨髓瘤 (MM) 患者对标准治疗不耐药,特别是具有侵略性或外骨髓性疾病的患者,结果不佳.
- 对于这些具有挑战性的患者群体,迫切需要新的治疗策略.
研究的目的:
- 为了评估密集组合治疗方案Pomalidomide-PACE (Pom-PACE) 的安全性和有效性,在患有复发性耐火性多发性骨髓瘤 (RRMM) 或血细胞白血病 (PCL) 的患者中.
主要方法:
- 在两个学术中心对20名连续RRMM或PCL患者进行了追溯分析,这些患者接受了Pom-PACE治疗.
- 患者确诊复发,有足够的器官功能,并且进行了大量的预治疗,通常是多种先前疗法和多种药物类别的折射性.
- 关于疗效 (反应率,存活率) 和安全性 (毒性) 的数据从医疗图表中收集.
主要成果:
- 研究群体 (75%的外骨髓性疾病患者) 接受过大量预治疗,平均接受过四次前期治疗,并且患有双重 (80%) 或三重类 (40%) 折射性高.
- 整体响应率 (ORR) 为68%,其中31%的患者获得了非常好的部分响应,64%的患者仅在一个周期后就得到了响应.
- 中位数无进展生存时间为8.9个月,中位数整体生存时间为11.8个月. 所有患者都经历了4级血液毒性,但没有发生与治疗相关的死亡.
结论:
- 波马利多米德-PACE在重度预治疗,激进的RRMM和PCL患者中显示出诱导快速反应的能力.
- 该疗法提供了可管理的毒性特征,作为一种有效的救援疗法,并成为其他先进治疗方法 (如CAR T细胞疗法) 的潜在桥梁.
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