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评估微小残留病阴性作为多发性骨髓瘤治疗疗效替代终点:随机对照试验的荟萃分析

Evaluating Minimal Residual Disease Negativity as a Surrogate Endpoint for Treatment Efficacy in Multiple Myeloma: A Meta-Analysis of Randomized Controlled Trials.

作者信息

Ntanasis-Stathopoulos Ioannis, Filippatos Charalampos, Ntanasis-Stathopoulos Anastasios, Malandrakis Panagiotis, Kastritis Efstathios, Tsitsilonis Ourania E, Dimopoulos Meletios A, Terpos Evangelos, Gavriatopoulou Maria

机构信息

Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.

Department of Biology, School of Sciences, National and Kapodistrian University of Athens, Athens, Greece.

出版信息

Am J Hematol. 2025 Mar;100(3):427-438. doi: 10.1002/ajh.27582. Epub 2025 Jan 9.

Abstract

This meta-analysis examined the association between minimal residual disease (MRD) negativity and survival outcomes in 15 304 patients with multiple myeloma (MM) enrolled in randomized controlled trials published until June 2, 2024. Overall, there was a significant, negative and strong association between MRD negativity odds ratios and survival hazard ratios (β_PFS = -0.20, p < 0.001, β_OS = -0.12, p = 0.023). These associations remained significant for newly diagnosed patients (β_PFS = -0.35, p < 0.001), and they were consistent but not significant for relapsed/refractory patients (β_PFS = -0.06, p = 0.635). Sustained MRD negativity at 1 year was strongly correlated with prolonged PFS (β_PFS = -0.30, p < 0.001). In conclusion, this comprehensive meta-analysis supports MRD as a surrogate for survival in MM.

摘要

这项荟萃分析研究了截至2024年6月2日发表的随机对照试验中纳入的15304例多发性骨髓瘤(MM)患者的微小残留病(MRD)阴性与生存结果之间的关联。总体而言,MRD阴性比值比与生存风险比之间存在显著、负向且强烈的关联(无进展生存期β=-0.20,p<0.001,总生存期β=-0.12,p=0.023)。这些关联在新诊断患者中仍然显著(无进展生存期β=-0.35,p<0.001),而在复发/难治性患者中虽具有一致性但不显著(无进展生存期β=-0.06,p=0.635)。1年时持续的MRD阴性与延长的无进展生存期密切相关(无进展生存期β=-0.30,p<0.001)。总之,这项全面的荟萃分析支持将MRD作为MM生存的替代指标。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a60b/11803549/1dd7a7597f99/AJH-100-427-g002.jpg

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