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≤40 岁初诊多发性骨髓瘤患者自体造血干细胞移植后的结局。

Outcomes of young adults (aged ≤ 40 years) with newly diagnosed multiple myeloma after up-front autologous stem cell transplant.

机构信息

Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.

Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah-Tikva, Israel.

出版信息

Br J Haematol. 2023 Aug;202(4):866-873. doi: 10.1111/bjh.18944. Epub 2023 Jun 27.

Abstract

Multiple myeloma (MM) primarily affects older patients. There are scarce data on the outcomes of young adults undergoing autologous transplantation (auto-HCT). In this single-centre analysis, we included 117 younger patients, with a median age of 37 years (range 22-40) at transplant. Seventeen (15%) patients had high-risk cytogenetics. Before transplant, 10% of patients achieved ≥CR and 44% achieved ≥VGPR. At best post-transplant response, 56% and 77% of patients achieved ≥CR and ≥VGPR respectively. With a median follow-up for survivors of 72.6 months (range 0.9-238.0), median PFS and OS were 43.1 months (95% CI 31.2-65.0) and 146.6 months (95% CI 100.0-208.1) respectively. Patients who underwent auto-HCT after 2010 had better median PFS (84.9 months vs. 28.2 months, p < 0.001) and OS (NR vs. 91.8 months, p < 0.001) compared with those transplanted earlier. In multi-variate analysis, achieving ≥CR as best post-transplant response was associated with improved PFS (HR [95% CI] 0.55 [0.32-0.95], p = 0.032), while achieving ≥VGPR was predictive of superior OS (0.32 [0.16-0.62], p < 0.001). Three patients (3%) developed a second primary malignancy. Younger MM patients had durable survival after auto-HCT, which further improved after the availability of novel anti-myeloma drugs in recent years. Depth of response following transplant remains a key predictor of survival.

摘要

多发性骨髓瘤(MM)主要影响老年患者。关于接受自体移植(auto-HCT)的年轻成年人的结局,数据稀少。在这项单中心分析中,我们纳入了 117 名中位年龄为 37 岁(范围 22-40)的年轻患者。17 名(15%)患者存在高危细胞遗传学异常。移植前,10%的患者达到了≥完全缓解(CR),44%的患者达到了≥非常好的部分缓解(VGPR)。移植后最佳缓解时,56%和 77%的患者分别达到了≥CR 和≥VGPR。对于幸存者,中位随访时间为 72.6 个月(范围 0.9-238.0),中位无进展生存期(PFS)和总生存期(OS)分别为 43.1 个月(95%CI 31.2-65.0)和 146.6 个月(95%CI 100.0-208.1)。与较早移植的患者相比,2010 年后接受 auto-HCT 的患者中位 PFS(84.9 个月 vs. 28.2 个月,p<0.001)和 OS(NR vs. 91.8 个月,p<0.001)更好。多变量分析显示,移植后达到最佳缓解的≥CR 与改善的 PFS 相关(风险比[95%CI]0.55[0.32-0.95],p=0.032),而达到≥VGPR 与更好的 OS 相关(0.32[0.16-0.62],p<0.001)。3 名患者(3%)发生了第二原发恶性肿瘤。年轻的 MM 患者在接受 auto-HCT 后有持久的生存,近年来新型抗骨髓瘤药物的应用使生存进一步改善。移植后反应的深度仍然是生存的关键预测因素。

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