Myeloma Unit, Division of Hematology, Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino, Torino, Italy.
Division of Hematology, Department of Cellular Biotechnologies and Hematology, Sapienza University of Roma, Roma, Italy.
Leukemia. 2017 Aug;31(8):1727-1734. doi: 10.1038/leu.2016.381. Epub 2016 Dec 23.
In newly diagnosed myeloma patients, upfront autologous transplant (ASCT) prolongs progression-free survival 1 (PFS1) compared with chemotherapy plus lenalidomide (CC+R). Salvage ASCT at first relapse may still effectively rescue patients who did not receive upfront ASCT. To evaluate the long-term benefit of upfront ASCT vs CC+R and the impact of salvage ASCT in patients who received upfront CC+R, we conducted a pooled analysis of 2 phase III trials (RV-MM-209 and EMN-441). Primary endpoints were PFS1, progression-free survival 2 (PFS2), overall survival (OS). A total of 268 patients were randomized to 2 courses of melphalan 200 mg/m and ASCT (MEL200-ASCT) and 261 to CC+R. Median follow-up was 46 months. MEL200-ASCT significantly improved PFS1 (median: 42 vs 24 months, HR 0.53; P<0.001), PFS2 (4 years: 71 vs 54%, HR 0.53, P<0.001) and OS (4 years: 84 vs 70%, HR 0.51, P<0.001) compared with CC+R. The advantage was noticed in good and bad prognosis patients. Only 53% of patients relapsing from CC+R received ASCT at first relapse. Upfront ASCT significantly reduced the risk of death (HR 0.51; P=0.007) in comparison with salvage ASCT. In conclusion, these data confirm the role of upfront ASCT as the standard approach for all young myeloma patients.
在新诊断的骨髓瘤患者中，与化疗加来那度胺（CC+R）相比， upfront autologous transplant（ASCT）可延长无进展生存期 1（PFS1）。在首次复发时进行挽救性 ASCT 仍可能有效地挽救未接受 upfront ASCT 的患者。为了评估 upfront ASCT 与 CC+R 的长期益处以及 upfront CC+R 患者接受挽救性 ASCT 的影响，我们对 2 项 3 期试验（RV-MM-209 和 EMN-441）进行了汇总分析。主要终点是无进展生存期 1（PFS1）、无进展生存期 2（PFS2）和总生存期（OS）。共有 268 名患者被随机分配至 2 个疗程的美法仑 200mg/m2 和 ASCT（MEL200-ASCT），261 名患者被随机分配至 CC+R。中位随访时间为 46 个月。与 CC+R 相比，MEL200-ASCT 显著改善了 PFS1（中位：42 与 24 个月，HR 0.53；P<0.001）、PFS2（4 年：71 与 54%，HR 0.53，P<0.001）和 OS（4 年：84 与 70%，HR 0.51，P<0.001）。该优势在预后良好和不良的患者中均可见。只有 53%的 CC+R 复发患者在首次复发时接受了 ASCT。与挽救性 ASCT 相比， upfront ASCT 显著降低了死亡风险（HR 0.51；P=0.007）。总之，这些数据证实了 upfront ASCT 作为所有年轻骨髓瘤患者标准治疗方法的作用。
