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新型药物时代高危多发性骨髓瘤的串联自体干细胞移植与单次自体干细胞移植:一项中国的真实世界研究

Tandem Versus Single Autologous Stem Cell Transplantation for High-Risk Multiple Myeloma in the Era of Novel Agents: A Real-World Study of China.

作者信息

Dou Xuelin, Ren Juan, Li Jiangtao, Liu Xiaodan, Bao Li, Chen Yuan, Zhao Peng, Zhong Yuping, Peng Nan, Wen Lei, Cao Leqing, Liu Yang, Deng Daoxing, Wang Fengrong, Wang Liru, Liu Hui, Huang Xiaojun, Mo Xiaodong, Lu Jin

机构信息

Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University, Beijing, China.

Department of Hematology, The First Affiliated Hospital of Xi'an Jiao Tong University, Shanxi, China.

出版信息

Cancer Med. 2025 Jan;14(1):e70573. doi: 10.1002/cam4.70573.

DOI:10.1002/cam4.70573
PMID:39744915
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11694139/
Abstract

BACKGROUND

This study compares the efficacy and safety of single autologous stem cell transplantation (ASCT) versus tandem ASCT for multiple myeloma (MM) patients in the era of novel agents.

METHODS

A total of 112 high-risk MM patients were included (single ASCT, (n = 57) or tandem ASCT(n = 55) in this retrospective multicenter study. Responses and outcomes were evaluated.

RESULTS

At 100 days after ASCT1 and ASCT2, 36 (63.2%) versus 45 (81.8%) patients achieved sCR/CR, 16 (28.1%) versus 7 (12.7%) patients achieved VGPR, and 5 (8.8%) versus 1 (1.8%) patient achieved PR, respectively, in the single and tandem ASCT cohorts. The 3-year cumulative incidence of non-relapse mortality and disease progression was 0% versus 7.3% (p = 0.083), and 45.8% versus 25.8% (p = 0.039), respectively, for the single and tandem ASCT cohort. The tandem ASCT cohort showed a trend of better 3-year probability of PFS (58.1% vs. 64.7%, p = 0.064) compared with the single ASCT cohort. In multivariate analysis, ultra high-risk and achieving<VGPR response after ASCT1 were associated with an inferior PFS. Ultra high-risk was also associated with an inferior OS.

CONCLUSIONS

Tandem ASCT demonstrated improved outcomes compared to single ASCT in high-risk MM patients receiving triplet or quadruplet induction and maintenance therapy. However, patients with ultra high-risk cytogenetics may require innovative therapeutic approaches, as tendem ASCT does not overcome their adverse prognosis.

摘要

背景

本研究比较了在新型药物时代,单倍自体干细胞移植(ASCT)与串联ASCT治疗多发性骨髓瘤(MM)患者的疗效和安全性。

方法

在这项回顾性多中心研究中,共纳入112例高危MM患者(单倍ASCT,n = 57;或串联ASCT,n = 55)。评估反应和结果。

结果

在ASCT1和ASCT2后100天,单倍和串联ASCT队列中分别有36例(63.2%)和45例(81.8%)患者达到严格完全缓解/完全缓解,16例(28.1%)和7例(12.7%)患者达到非常好的部分缓解,5例(8.8%)和1例(1.8%)患者达到部分缓解。单倍和串联ASCT队列的3年非复发死亡率和疾病进展累积发生率分别为0%和7.3%(p = 0.083),以及45.8%和25.8%(p = 0.039)。与单倍ASCT队列相比,串联ASCT队列显示出3年无进展生存期概率更高的趋势(58.1%对64.7%,p = 0.064)。在多变量分析中,超高危以及在ASCT1后达到<非常好的部分缓解与较差的无进展生存期相关。超高危也与较差的总生存期相关。

结论

在接受三联或四联诱导和维持治疗的高危MM患者中,串联ASCT与单倍ASCT相比显示出更好的结果。然而,具有超高危细胞遗传学特征的患者可能需要创新的治疗方法,因为串联ASCT并不能克服其不良预后。

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Circulating plasma cells as a predictive biomarker in Multiple myeloma: an updated systematic review and meta-analysis.循环血浆细胞作为多发性骨髓瘤的预测性生物标志物:一项更新的系统评价和荟萃分析。
Ann Med. 2024 Dec;56(1):2338604. doi: 10.1080/07853890.2024.2338604. Epub 2024 Apr 10.
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自体造血干细胞移植治疗缓解不佳的骨髓瘤患者的疗效:一项多中心回顾性分析。
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Single versus tandem autologous stem-cell transplantation in patients with newly diagnosed multiple myeloma and high-risk cytogenetics. A retrospective, open-label study of the PETHEMA/Spanish Myeloma Group (GEM).新诊断的伴有细胞遗传学高危因素的多发性骨髓瘤患者中单倍体与双次自体造血干细胞移植的比较。PETHEMA/Spanish Myeloma Group(GEM)的一项回顾性、开放性研究。
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The epidemiological landscape of multiple myeloma: a global cancer registry estimate of disease burden, risk factors, and temporal trends.多发性骨髓瘤的流行病学概况:全球癌症登记处对疾病负担、风险因素和时间趋势的估计。
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The Importance of FISH Signal Cut-off Value and Copy Number Variation for 1q21 in Newly Diagnosed Multiple Myeloma: Is it Underestimated?新发多发性骨髓瘤中 FISH 信号截断值和 1q21 拷贝数变异的重要性:是否被低估了?
Clin Lymphoma Myeloma Leuk. 2022 Jul;22(7):535-544. doi: 10.1016/j.clml.2022.01.013. Epub 2022 Jan 31.
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Carfilzomib with cyclophosphamide and dexamethasone or lenalidomide and dexamethasone plus autologous transplantation or carfilzomib plus lenalidomide and dexamethasone, followed by maintenance with carfilzomib plus lenalidomide or lenalidomide alone for patients with newly diagnosed multiple myeloma (FORTE): a randomised, open-label, phase 2 trial.卡非佐米联合环磷酰胺和地塞米松或来那度胺和地塞米松联合自体移植,或卡非佐米联合来那度胺和地塞米松,随后用卡非佐米联合来那度胺或来那度胺维持治疗新诊断的多发性骨髓瘤患者(FORTE):一项随机、开放标签、2 期试验。
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