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三联疗法、移植和维持治疗多发性骨髓瘤直至疾病进展可显著延长生存期
作者:小柯机器人 发布时间:2022/6/7 12:44:04

美国丹娜-法伯癌症研究所Paul G. Richardson团队研究了三联疗法、移植和维持治疗多发性骨髓瘤直至疾病进展的效果。相关论文于2022年6月5日发表在《新英格兰医学杂志》上。

对于新诊断的多发性骨髓瘤患者,在三联疗法(来那度胺、硼替佐米和地塞米松[RVD])中加入自体干细胞移植(ASCT),然后在疾病进展前进行来那度胺维持治疗的效果尚不清楚。

在这项3期临床试验中,患有症状性骨髓瘤的成年人(18-65岁)接受了一个周期的RVD。研究组以1:1的比例随机分配这些患者,分别接受两个额外的RVD周期加上干细胞动员,然后再接受五个额外的RVD周期(仅RVD组)或高剂量美法仑加ASCT,然后再接受两个额外的RVD周期(移植组)。两组均接受来那度胺治疗,直到疾病进展或出现不可接受的副作用,或两者兼而有之。主要终点是无进展生存率。

仅RVD组357名患者和移植组365名患者接受了平均76.0个月的随访,共发生328起疾病进展或死亡事件;仅RVD组的风险比移植组高53%,危险比为1.53,差异显著;中位无进展生存期分别为46.2个月和67.5个月。仅RVD组部分缓解或更好的患者占比为95.0%,移植组为97.5%;两组分别有42.0%和46.8%的患者完全缓解或更好。仅RVD组和移植组3级及以上治疗相关不良事件发生率分别为78.2%和94.2%;5年生存率分别为79.2%和80.7%,死亡危险比为1.10。

研究结果表明,在成人多发性骨髓瘤患者中,与单纯RVD相比,RVD加ASCT与更长的无进展生存期相关。但未观察到总体生存益处。

附:英文原文

Title: Triplet Therapy, Transplantation, and Maintenance until Progression in Myeloma | NEJM

Author: Paul G. Richardson, M.D.,, Susanna J. Jacobus, M.Sc., M.B.A.,, Edie A. Weller, Ph.D.,, Hani Hassoun, M.D.,, Sagar Lonial, M.D.,, Noopur S. Raje, M.D.,, Eva Medvedova, M.D.,, Philip L. McCarthy, M.D.,, Edward N. Libby, M.D.,, Peter M. Voorhees, M.D.,, Robert Z. Orlowski, M.D., Ph.D.,, Larry D. Anderson, Jr., M.D., Ph.D.,, Jeffrey A. Zonder, M.D.,, Carter P. Milner, M.D.,, Cristina Gasparetto, M.D.,, Mounzer E. Agha, M.D.,, Abdullah M. Khan, M.B., B.S.,, David D. Hurd, M.D.,, Krisstina Gowin, D.O.,, Rammurti T. Kamble, M.D.,, Sundar Jagannath, M.D.,, Nitya Nathwani, M.D.,, Melissa Alsina, M.D.,, R. Frank Cornell, M.D.,, Hamza Hashmi, M.D.,, Erica L. Campagnaro, M.D.,, Astrid C. Andreescu, M.D.,, Teresa Gentile, M.D., Ph.D.,, Michaela Liedtke, M.D.,, Kelly N. Godby, M.D.,, Adam D. Cohen, M.D.,, Thomas H. Openshaw, M.D.,, Marcelo C. Pasquini, M.D.,, Sergio A. Giralt, M.D.,, Jonathan L. Kaufman, M.D.,, Andrew J. Yee, M.D.,, Emma Scott, M.D.,, Pallawi Torka, M.D.,, Amy Foley, M.A.,, Mariateresa Fulciniti, Ph.D.,, Kyle Hebert, M.S.,, Mehmet K. Samur, Ph.D.,, Kelly Masone, B.A.,, Michelle E. Maglio, M.B.A.,, Andrea A. Zeytoonjian, M.B.A.,, Omar Nadeem, M.D.,, Robert L. Schlossman, M.D.,, Jacob P. Laubach, M.D., M.P.P.,, Claudia Paba-Prada, M.D.,, Irene M. Ghobrial, M.D.,, Aurore Perrot, M.D., Ph.D.,, Philippe Moreau, M.D.,, Hervé Avet-Loiseau, M.D., Ph.D.,, Michel Attal, M.D., Ph.D.,, Kenneth C. Anderson, M.D.,, and Nikhil C. Munshi, M.D.

Issue&Volume: 2022-06-05

Abstract:

Background

In patients with newly diagnosed multiple myeloma, the effect of adding autologous stem-cell transplantation (ASCT) to triplet therapy (lenalidomide, bortezomib, and dexamethasone [RVD]), followed by lenalidomide maintenance therapy until disease progression, is unknown.

Methods

In this phase 3 trial, adults (18 to 65 years of age) with symptomatic myeloma received one cycle of RVD. We randomly assigned these patients, in a 1:1 ratio, to receive two additional RVD cycles plus stem-cell mobilization, followed by either five additional RVD cycles (the RVD-alone group) or high-dose melphalan plus ASCT followed by two additional RVD cycles (the transplantation group). Both groups received lenalidomide until disease progression, unacceptable side effects, or both. The primary end point was progression-free survival.

Results

Among 357 patients in the RVD-alone group and 365 in the transplantation group, at a median follow-up of 76.0 months, 328 events of disease progression or death occurred; the risk was 53% higher in the RVD-alone group than in the transplantation group (hazard ratio, 1.53; 95% confidence interval [CI], 1.23 to 1.91; P<0.001); median progression-free survival was 46.2 months and 67.5 months. The percentage of patients with a partial response or better was 95.0% in the RVD-alone group and 97.5% in the transplantation group (P=0.55); 42.0% and 46.8%, respectively, had a complete response or better (P=0.99). Treatment-related adverse events of grade 3 or higher occurred in 78.2% and 94.2%, respectively; 5-year survival was 79.2% and 80.7% (hazard ratio for death, 1.10; 95% CI, 0.73 to 1.65).

Conclusions

Among adults with multiple myeloma, RVD plus ASCT was associated with longer progression-free survival than RVD alone. No overall survival benefit was observed.

DOI: 10.1056/NEJMoa2204925

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2204925

 

期刊信息

The New England Journal of Medicine:《新英格兰医学杂志》,创刊于1812年。隶属于美国麻省医学协会,最新IF:70.67
官方网址:http://www.nejm.org/
投稿链接:http://www.nejm.org/page/author-center/home