Nishiyama Risa, Kagoo Toshiya, Ueno Hironori, Yokoyama Akihiro
Division of Hematology, Department of Internal Medicine, National Hospital Organization Tokyo Medical Center, Tokyo, Japan
Division of Hematology, Department of Internal Medicine, National Hospital Organization Tokyo Medical Center, Tokyo, Japan.
In Vivo. 2025 Jan-Feb;39(1):340-345. doi: 10.21873/invivo.13833.
BACKGROUND/AIM: Autologous stem cell transplantation (ASCT) is the standard strategy after induction therapy for newly diagnosed transplant-eligible multiple myeloma. High-dose melphalan (HDM) conditioning has been the recommended treatment regimen for a long time. No other conditioning regimen has been proven safer and more effective. Because bortezomib has a synergistic effect with melphalan, bortezomib with HDM (Bor-HDM) as a conditioning regimen has shown favorable outcomes, improved complete response rates after ASCT, and no prolonged hematological toxicities. However, few studies have reported long-term follow-up data. This study aimed to evaluate the long-term progression-free survival (PFS) and overall survival (OS) of patients receiving Bor-HDM conditioning, compared to those treated with HDM alone.
This single-center retrospective study included 36 patients newly diagnosed with transplant-eligible myeloma from 2008 to 2020. In total, 15 patients received a Bor-HDM regimen, while 21 patients received HDM as a conditioning regimen. The probabilities of PFS and OS were plotted using the Kaplan-Meier method. All statistical analyses were performed using EZR software.
After a median follow up of 77 months, no severe hematological toxicities were observed. The PFS and OS rates in the Bor-HDM group as compared with the HDM group were 0.762 vs. 0.60 (p=0.409) and 0.80 vs. 0.904 (p=0.476) respectively. No significant differences were observed between the two groups.
These long-term results show that Bor-HDM is a safe and effective option for ASCT conditioning regimens.
背景/目的：自体干细胞移植（ASCT）是新诊断的适合移植的多发性骨髓瘤诱导治疗后的标准策略。大剂量美法仑（HDM）预处理长期以来一直是推荐的治疗方案。尚无其他预处理方案被证明更安全、更有效。由于硼替佐米与美法仑具有协同作用，硼替佐米联合HDM（Bor-HDM）作为预处理方案已显示出良好的效果，提高了ASCT后的完全缓解率，且无延长的血液学毒性。然而，很少有研究报告长期随访数据。本研究旨在评估接受Bor-HDM预处理的患者与仅接受HDM治疗的患者相比的长期无进展生存期（PFS）和总生存期（OS）。
本单中心回顾性研究纳入了2008年至2020年新诊断的适合移植的骨髓瘤患者36例。共有15例患者接受Bor-HDM方案，21例患者接受HDM作为预处理方案。采用Kaplan-Meier法绘制PFS和OS概率图。所有统计分析均使用EZR软件进行。
中位随访77个月后，未观察到严重血液学毒性。Bor-HDM组与HDM组的PFS率分别为0.762和0.60（p=0.409），OS率分别为0.80和0.904（p=0.476）。两组之间未观察到显著差异。
这些长期结果表明，Bor-HDM是ASCT预处理方案的一种安全有效的选择。
