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[一项关于自体造血干细胞移植与新药化疗治疗新诊断多发性骨髓瘤的倾向评分匹配研究]

[A Propensity Score Matching Study of Autologous Hematopoietic Stem Cell Transplantation and New Drug Chemotherapy for Newly Diagnosed Multiple Myeloma].

作者信息

Yang Fei-Yan, Wang Hua-Fang, Xia Ling-Hui, Wang Qing-Qing, Qian Chen-Jing, He Jing

机构信息

Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430022, Hubei Province, China.

Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430022, Hubei Province, China,E-mail:

出版信息

Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2022 Feb;30(1):158-165. doi: 10.19746/j.cnki.issn.1009-2137.2022.01.026.

DOI:10.19746/j.cnki.issn.1009-2137.2022.01.026
PMID:35123620
Abstract

OBJECTIVE

To compare the clinical efficacy, survival, and prognosis of autologous hematopoietic stem cell transplantation (ASCT) with new drug chemotherapy in the treatment of newly diagnosed multiple myeloma (NDMM) in the new drug era.

METHODS

The clinical data of 149 patients with NDMM treated with new drug induction regimen in Union Hospital, Tongji Medical College, Huazhong University of Science and Technology from January 2012 to December 2019 were retrospectively analyzed. Twenty-four patients who received ASCT were in ASCT group, and 125 patients who did not receive ASCT were in non-ASCT group. The median follow-up time was 43 (1-90) months. The propensity score matching (PSM) method was used to balance confounding factors, then depth of response, overall survival (OS), and progression-free survival (PFS) between the two groups were compared and subgroup analysis was performed.

RESULTS

After matching, the covariates were balanced between the two groups. Fifty-one patients (15 cases in ASCT group and 36 cases in non-ASCT group) were included. ASCT patients had a better complete response (CR) rate than non-ASCT patients receiving maintenance therapy (93.3% vs 42.3%, P=0.004), while there were no statistical differences in deep response rate and overall response rate (ORR) between the two groups (93.3% vs 65.4%, P=0.103; 93.3% vs 96.2%, P=1.000). Before matching, the 3 and 5-year PFS rate and median PFS (mPFS) in ASCT group and non-ASCT group were [89.6% vs 66.5%, P=0.024; 69.8% vs 42.7%; non-response (NR) vs 51.0 months], and the 3 and 5-year OS rate and median OS (mOS) were (100% vs 70.6%, P=0.002; 92.3% vs 49.6%; NR vs 54.0 months). After matching, the 3 and 5-year PFS rate and mPFS in ASCT group and non-ASCT group were (83.6% vs 61.7%, P=0.182; 62.7% vs 45.7%; NR vs 51.0 months), the 3 and 5-year OS rate and mOS were (100% vs 65.6%, P=0.018; 88.9% vs 46.9%; NR vs 51.0 months). Subgroup analysis showed that patients with mSMART 3.0 high risk stratification, the 3-year PFS rate and mPFS in ASCT group and non-ASCT group were (83.3% vs 41.5%, P=0.091; NR vs 34.0 months), and the 3-year OS rate and mOS were (100% vs 41.5%, P=0.034; NR vs 34.0 months). Patients with mSMART 3.0 standard risk stratification, the 3-year PFS rate and OS rate in ASCT group and non-ASCT group were (83.3% vs 76.8%, P=0.672; 100% vs 87.2%, P=0.155). The 3-year PFS and OS rate in MM patients who achieved deep response within 3 months after transplantation compared with non-ASCT patients who achieved deep response after receiving maintenance therapy were (83.1% vs 56.7%, P=0.323; 100% vs 60.5%, P=0.042), and the 3-year PFS and OS rate in patients who achieved overall response in both groups were (83.1% vs 62.5%, P=0.433; 100% vs 68.1%, P=0.082). After matching, Cox multivariate regression analysis showed that mSMART 3.0 risk stratification and ASCT were independent prognostic factors for OS.

CONCLUSION

In the new drug era, ASCT can increase CR rate and prolong OS of NDMM patients. ASCT patients who are mSMART 3.0 high risk stratification or achieved deep response within 3 months after transplantation have better OS than non-ASCT patients receiving new drug chemotherapy. ASCT and mSMART 3.0 risk stratification are independent prognostic factors for OS in NDMM patients.

摘要

目的

比较新药时代自体造血干细胞移植(ASCT)与新药化疗治疗新诊断多发性骨髓瘤(NDMM)的临床疗效、生存率及预后。

方法

回顾性分析2012年1月至2019年12月在华中科技大学同济医学院附属协和医院接受新药诱导方案治疗的149例NDMM患者的临床资料。接受ASCT的24例患者为ASCT组,未接受ASCT的125例患者为非ASCT组。中位随访时间为43(1 - 90)个月。采用倾向评分匹配(PSM)方法平衡混杂因素,然后比较两组间的缓解深度、总生存期(OS)和无进展生存期(PFS),并进行亚组分析。

结果

匹配后,两组间协变量均衡。纳入51例患者(ASCT组15例,非ASCT组36例)。接受维持治疗的ASCT患者的完全缓解(CR)率高于非ASCT患者(93.3%对42.3%,P = 0.004),而两组间的深度缓解率和总缓解率(ORR)无统计学差异(93.3%对65.4%,P = 0.103;93.3%对96.2%,P = 1.000)。匹配前,ASCT组和非ASCT组的3年和5年PFS率及中位PFS(mPFS)分别为[89.6%对66.5%,P = 0.024;69.8%对42.7%;未缓解(NR)对51.0个月],3年和5年OS率及中位OS(mOS)分别为(100%对70.6%,P = 0.002;92.3%对49.6%;NR对54.0个月)。匹配后,ASCT组和非ASCT组的3年和5年PFS率及mPFS分别为(83.6%对61.7%,P = 0.182;62.7%对45.7%;NR对51.0个月),3年和5年OS率及mOS分别为(100%对65.6%,P = 0.018;88.9%对46.9%;NR对51.0个月)。亚组分析显示,mSMART 3.0高危分层患者中,ASCT组和非ASCT组的3年PFS率及mPFS分别为(83.3%对41.5%,P = 0.091;NR对34.0个月),3年OS率及mOS分别为(100%对41.5%,P = 0.034;NR对34.0个月)。mSMART 3.0标准风险分层患者中,ASCT组和非ASCT组的3年PFS率及OS率分别为(83.3%对76.8%,P = 0.672;100%对87.2%,P = 0.155)。移植后3个月内达到深度缓解的MM患者与接受维持治疗后达到深度缓解的非ASCT患者相比,3年PFS率及OS率分别为(83.1%对56.

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