Division of Cellular Therapy, Duke University, Durham, NC, USA.
Department of Hematology, Cambridge Institute for Medical Research, Cambridge, UK.
Bone Marrow Transplant. 2018 Jan;53(1):34-38. doi: 10.1038/bmt.2017.208. Epub 2017 Oct 30.
Single-agent high-dose melphalan (HDM, 200 mg/m) has been the most commonly used conditioning regimen prior to autologous stem cell transplant, since its introduction in 1992. We used a more aggressive alkylator-based conditioning regimen in an attempt to overcome early relapse and combat drug resistance. We present a retrospective comparison and long-term follow-up of newly diagnosed patients with multiple myeloma (MM) treated with induction followed by either high-dose carmustine (BCNU) and HDM, or HDM alone, both followed by autologous stem cell transplant (ASCT). Between 1997 and 2002, 104 patients were treated with BCNU/HDM; from 2001 to 2008, 103 patients were treated with HDM alone. Median follow-up of survivors was 78 and 68 months for the BCNU/HDM and HDM groups, respectively. The median PFS was significantly increased with the BCNU/HDM regimen (40.4 vs 20.5 months, P<0.001). Median overall survival was increased with the BCNU/HDM regimen when compared with HDM alone (88.4 vs 67.2 months, P=0.07), but the difference was not statistically significant. Transplant-related mortality was similar in both groups (2.9% with BCNU and HDM vs 3.9% with HDM alone). Our findings suggest that the BCNU/HDM preparative regimen should be investigated further and potentially compared in a prospective randomized manner with HDM alone.
单剂大剂量美法仑(HDM,200mg/m)自 1992 年引入以来,一直是自体干细胞移植前最常用的调理方案。我们使用更具侵袭性的烷化剂为基础的调理方案,试图克服早期复发和对抗药物耐药性。我们报告了一组新诊断多发性骨髓瘤(MM)患者的回顾性比较和长期随访结果,这些患者接受诱导治疗后分别接受大剂量卡莫司汀(BCNU)和 HDM 或单独 HDM 治疗,随后均进行自体干细胞移植(ASCT)。1997 年至 2002 年间,104 例患者接受 BCNU/HDM 治疗;2001 年至 2008 年间,103 例患者接受单独 HDM 治疗。BCNU/HDM 组和 HDM 组幸存者的中位随访时间分别为 78 个月和 68 个月。与 HDM 组相比,BCNU/HDM 方案的中位无进展生存期(PFS)显著延长(40.4 个月 vs 20.5 个月,P<0.001)。与单独 HDM 组相比,BCNU/HDM 方案的中位总生存期(OS)延长(88.4 个月 vs 67.2 个月,P=0.07),但差异无统计学意义。两组间移植相关死亡率相似(BCNU 和 HDM 组为 2.9%,单独 HDM 组为 3.9%)。我们的研究结果表明,BCNU/HDM 预处理方案应进一步研究,并可能与单独 HDM 进行前瞻性随机比较。