文章：

来那度胺时代后多药联合方案是否改善复发难治性多发性骨髓瘤的预后？——来自多发性骨髓瘤真实世界创新研究（REAL IMM）的发现

Does the Simultaneous Introduction of Several Pharmaceuticals in the Post-Lenalidomide Era Translate to Better Outcomes in Relapse Refractory Multiple Myeloma? Findings from the Real-World Innovation in Multiple Myeloma (REAL IMM) Study

原文发布日期：14 December 2023

DOI: 10.3390/cancers15245846

类型: Article

开放获取: 是

英文摘要：

Newer methodologies are needed to assess the real-world comparative effectiveness of a “generation” of pharmaceutical innovation versus the prior standard of care. This chart review study aimed to first evaluate the cumulative clinical benefits of pharmaceutical innovation in everyday relapse/refractory multiple myeloma before analyzing findings in the context of respective real-world outcomes from the bortezomib/lenalidomide era. Study endpoints included the 52-week PFS rate in second and third line of therapy (LOT), mPFS-2 across the first and second LOT, the ORR, reasons for discontinuation, and the treatment duration per therapeutic algorithm. Data from 107 patients were collected. The median follow-up was 2.0 years. Of the subjects who met the selection criteria for the second LOT, 72.2% maintained the PFS at 52 weeks. In the third-line setting, the PFS rate at 52 weeks was 63.5%. The mPFS across the first and second, the second, and the third LOTs were 26, 17, and 15 months, respectively. The ORR was 76.1% in the second and 69.7% in the third LOT. After non-response or progression, the main reason for drug discontinuation was treatment intolerability. The second-line ORR and the 52-week PFS rate were similar to previous real-world findings from the bortezomib/lenalidomide era. The cumulative mPFS across the second and third LOTs was higher than the respective mPFS across the first and second LOTs. Despite its limitations, the methodology and findings from this study may be used in future clinical and economic evaluations across all hematological malignancies.

摘要翻译： 

需要新的方法来评估新一代药物创新相较于既往标准治疗在真实世界中的相对有效性。本研究通过病历回顾，旨在分析硼替佐米/来那度胺时代相应真实世界结局之前，首先评估日常复发/难治性多发性骨髓瘤治疗中药物创新的累积临床获益。研究终点包括二线及三线治疗52周无进展生存率、前两线治疗中位无进展生存期、总缓解率、停药原因及各治疗方案持续时间。共收集107例患者数据，中位随访时间2.0年。符合二线治疗筛选标准的患者中，72.2%在52周时保持无进展生存状态；三线治疗中52周无进展生存率为63.5%。一线至二线、二线及三线治疗的中位无进展生存期分别为26、17和15个月。二线治疗总缓解率为76.1%，三线治疗为69.7%。在无应答或疾病进展后，停药的主要原因为治疗不耐受。二线治疗总缓解率和52周无进展生存率与硼替佐米/来那度胺时代的既往真实世界研究结果相似。二线至三线治疗的累积中位无进展生存期高于一线至二线治疗的相应数值。尽管存在局限性，本研究的方法和结果可为未来所有血液系统恶性肿瘤的临床经济学评估提供参考。

原文链接：

Does the Simultaneous Introduction of Several Pharmaceuticals in the Post-Lenalidomide Era Translate to Better Outcomes in Relapse Refractory Multiple Myeloma? Findings from the Real-World Innovation in Multiple Myeloma (REAL IMM) Study