Muronova Ludmila, Soucek Ondrej, Zihala David, Sevcikova Tereza, Popkova Tereza, Plonkova Hana, Venglar Ondrej, Pour Ludek, Stork Martin, Rihova Lucie, Bezdekova Renata, Minarik Jiri, Látal Vojtech, Novak Martin, Jungova Alexandra, Dekojova Tereza, Straub Jan, Spacek Martin, Rezacova Vladimira, Maisnar Vladimir, Radocha Jakub, Hajek Roman, Jelinek Tomas
Department of Hematooncology, University Hospital Ostrava, Ostrava, Czech Republic.
Department of Hematooncology, Faculty of Medicine, University of Ostrava, Ostrava, Czech Republic.
Eur J Haematol. 2025 Jan;114(1):155-163. doi: 10.1111/ejh.14316. Epub 2024 Oct 10.
Minimal residual disease (MRD) is one of the most important prognostic factors in multiple myeloma (MM) and a valid surrogate for progression-free survival (PFS) and overall survival (OS). Recently, MRD negativity was approved as an early clinical endpoint for accelerated drug approval in MM. Nevertheless, there is limited evidence of MRD utility in real-world setting. In this retrospective multicenter study, we report outcomes of 331 newly diagnosed MM patients with MRD evaluation at Day+100 after autologous stem cell transplantation using flow cytometry with a median limit of detection of 0.001%. MRD negativity was reached in 47% of patients and was associated with significantly prolonged median PFS (49.2 months vs. 18.4 months; hazard ratios (HR) = 0.37; p < 0.001) and OS (not reached vs. 74.9 months; HR = 0.50; p = 0.007). Achieving MRD negativity was associated with PFS improvements regardless of age, International Staging System (ISS) stage, lactate dedydrogenase (LDH) level, or cytogenetic risk. Importantly, MRD positive patients benefited from lenalidomide maintenance versus no maintenance (18-months PFS: 81% vs. 46%; HR = 0.24; p = 0.002) while in MRD negative patients such benefit was not observed (p = 0.747). The outcomes of our real-world study recapitulate results from clinical trials including meta-analyses and support the idea that MRD positive patients profit more from lenalidomide maintenance than MRD negative ones.
微小残留病(MRD)是多发性骨髓瘤(MM)最重要的预后因素之一,也是无进展生存期(PFS)和总生存期(OS)的有效替代指标。最近,MRD阴性被批准作为MM加速药物批准的早期临床终点。然而,在现实世界中,关于MRD效用的证据有限。在这项回顾性多中心研究中,我们报告了331例新诊断的MM患者在自体干细胞移植后第100天使用流式细胞术进行MRD评估的结果,检测限中位数为0.001%。47%的患者达到MRD阴性,这与显著延长的中位PFS(49.2个月对18.4个月;风险比(HR)=0.37;p<0.001)和OS(未达到对74.9个月;HR=0.50;p=0.007)相关。无论年龄、国际分期系统(ISS)分期、乳酸脱氢酶(LDH)水平或细胞遗传学风险如何,实现MRD阴性均与PFS改善相关。重要的是,MRD阳性患者从来那度胺维持治疗中获益,而未接受维持治疗的患者则不然(18个月PFS:81%对46%;HR=0.24;p=0.002),而在MRD阴性患者中未观察到这种获益(p=0.747)。我们的现实世界研究结果重现了包括荟萃分析在内的临床试验结果,并支持MRD阳性患者比MRD阴性患者从来那度胺维持治疗中获益更多的观点。