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挽救性自体干细胞移植仍是复发骨髓瘤患者可行的治疗选择吗?

Is Salvage Autologous Stem Cell Transplantation Still a Viable Treatment Option in Relapsed Myeloma Patients?

作者信息

Čemažar Luka, Škerget Matevž, Skopec Barbara

机构信息

Department of Hematology, University Medical Centre Ljubljana, 1000 Ljubljana, Slovenia.

Faculty of Medicine, University of Ljubljana, 1000 Ljubljana, Slovenia.

出版信息

Medicina (Kaunas). 2025 May 7;61(5):859. doi: 10.3390/medicina61050859.

Abstract

: The role of salvage autologous stem cell transplantation (SAT) in relapsed multiple myeloma (MM) has been increasingly questioned, particularly with the emergence of novel immunotherapies and cellular therapies. This study aimed to evaluate the efficacy of SAT in a cohort of patients with relapsed MM following their first autologous stem cell transplantation (ASCT). : A retrospective analysis was conducted on 78 patients from our institutional registry who underwent SAT for relapsed MM between April 2008 and October 2023. The primary endpoint was the progression-free survival (PFS), with secondary endpoints including the overall survival (OS) and overall response rates (ORRs) on day +100 after SAT. : The median age of the patients was 62 years (range: 48-78), and 32% were female. Most patients (81%) received reinduction therapy before SAT. The median PFS and OS from SAT were 24 months (95% CI: 20-36) and 76 months (95% CI: 48-NR), respectively. The ORR was 85%, and 65% achieved at least a very good partial response (VGPR). No significant differences in the PFS were found between subgroups based on the maintenance following SAT (hazard ratio (HR) = 0.93, 95% CI: 0.48-1.8, = 0.831), cytogenetic risk (standard vs. high-risk) (HR = 0.98, 95% CI: 0.49-1.99, = 0.969), age (<60 years vs. ≥60 years) (HR = 0.96, 95% CI: 0.5-1.85, = 0.912) or number of prior treatment lines (<3 vs. ≥3) (HR = 0.186, 95% CI: 0.95-3.61, = 0.069). The duration of remission after the first ASCT did not influence the PFS or OS following SAT (HR = 1.63, 95% CI: 0.78-3.39, = 0.193 and HR = 1.2, 95% CI: 0.46-3.09, = 0.7130). : Salvage autologous stem cell transplantation remains a viable treatment option for patients with relapsed MM, particularly in resource-limited countries or for patients who prefer short, fixed-duration therapy. However, as is known from previous studies, maintenance therapy is crucial for achieving longer PFS. In this study, no statistically significant factors were identified to predict the benefit, underscoring the need for further research to optimize patient selection.

摘要

挽救性自体干细胞移植(SAT)在复发多发性骨髓瘤(MM)中的作用越来越受到质疑,尤其是随着新型免疫疗法和细胞疗法的出现。本研究旨在评估SAT在一组首次自体干细胞移植(ASCT)后复发的MM患者中的疗效。

对2008年4月至2023年10月期间在我们机构登记处接受SAT治疗复发MM的78例患者进行了回顾性分析。主要终点是无进展生存期(PFS),次要终点包括SAT后第100天的总生存期(OS)和总缓解率(ORR)。

患者的中位年龄为62岁(范围:48 - 78岁),32%为女性。大多数患者(81%)在SAT前接受了再诱导治疗。SAT后的中位PFS和OS分别为24个月(95%CI:20 - 36)和76个月(95%CI:48 - NR)。ORR为85%,65%的患者至少达到了非常好的部分缓解(VGPR)。基于SAT后的维持治疗(风险比(HR)= 0.93,95%CI:0.48 - 1.8,P = 0.831)、细胞遗传学风险(标准风险与高风险)(HR = 0.98,95%CI:0.49 - 1.99,P = 0.969)、年龄(<60岁与≥60岁)(HR = 0.96,95%CI:0.5 - 1.85,P = 0.912)或既往治疗线数(<3与≥3)(HR = 0.186,95%CI:0.95 - 3.61,P = 0.069)的亚组之间,PFS没有显著差异。首次ASCT后的缓解持续时间对SAT后的PFS或OS没有影响(HR = 1.63,95%CI:0.78 - 3.39,P = 0.193和HR = 1.2,95%CI:0.46 - 3.09,P = 0.7130)。

挽救性自体干细胞移植仍然是复发MM患者的一种可行治疗选择,特别是在资源有限的国家或对于倾向于短疗程、固定疗程治疗的患者。然而,正如先前研究所知,维持治疗对于实现更长的PFS至关重要。在本研究中,未发现有统计学意义的因素来预测获益,这突出了进一步研究以优化患者选择的必要性。

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