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单倍体相合造血干细胞移植治疗复发/难治性髓外多发性骨髓瘤:一种塞利尼索与螺旋断层放疗联合预处理方案——病例报告及文献综述

Haploidentical Hematopoietic Stem Cell Transplantation for Relapsed/Refractory Extramedullary Multiple Myeloma: A Conditioning Regimen Combining Selinexor and Helical Tomotherapy-A Case Report and Literature Review.

作者信息

Chen Bin, Guo Rongrong, Niu Yanyan, Guo Wei, Wang Tao

机构信息

Department of Hematology, Third Hospital of Shanxi Medical University, Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Tongji Shanxi Hospital, Taiyuan, 030032, People's Republic of China.

出版信息

J Blood Med. 2025 Apr 2;16:177-185. doi: 10.2147/JBM.S495256. eCollection 2025.

DOI:10.2147/JBM.S495256
PMID:40196041
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11972968/
Abstract

Relapsed/refractory multiple myeloma (RRMM) and extramedullary multiple myeloma (EMM) present significant challenges for patients with multiple myeloma (MM) after their disease progresses.Despite notable advancements in treatments like autologous hematopoietic stem cell transplantation (ASCT) and chimeric antigen receptor (CAR)-T-cell therapy, most patients with RRMM and EMM face a short survival period. Currently, there are no effective treatments available. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the treatment options for MM. Reduced-intensity conditioning (RIC) regimens have largely replaced myeloablative conditioning (MAC) regimens. RIC is now preferred because it significantly lowers transplant-related mortality, which has dropped to 10-20%. However, RIC regimens are linked to higher relapse rates compared to MAC. To enhance the efficacy of allo-HSCT, it is essential to identify a safer and more effective conditioning regimen. We report a case of EMM involving the breast, supraclavicular region, mediastinum, and pleural effusion, among other sites. The patient did not respond to several treatments, including a proteasome inhibitor (PI) like bortezomib, immunomodulatory drugs (IMiDs) such as lenalidomide, and a monoclonal antibody targeting CD38, like daratumumab. Consequently, we recommended haploidentical hematopoietic stem cell transplantation as a salvage treatment. After undergoing allo-HSCT with a conditioning regimen that mainly included selinexor and helical tomotherapy, the patient achieved a complete remission(CR) and enjoyed long-term disease-free survival for 11 months. Along with existing literature, this case provides encouraging insights for future research on RRMM and EMM, and we anticipate more reports on allo-HSCT cases in the future.

摘要

复发/难治性多发性骨髓瘤(RRMM)和髓外多发性骨髓瘤(EMM)在疾病进展后给多发性骨髓瘤(MM)患者带来了重大挑战。尽管自体造血干细胞移植(ASCT)和嵌合抗原受体(CAR)-T细胞疗法等治疗方法取得了显著进展,但大多数RRMM和EMM患者的生存期较短。目前,尚无有效的治疗方法。异基因造血干细胞移植(allo-HSCT)是MM的治疗选择之一。减低强度预处理(RIC)方案已在很大程度上取代了清髓性预处理(MAC)方案。目前更倾向于使用RIC,因为它显著降低了移植相关死亡率,该死亡率已降至10%-20%。然而,与MAC相比,RIC方案的复发率更高。为了提高allo-HSCT的疗效,确定一种更安全、更有效的预处理方案至关重要。我们报告了一例EMM病例,病变累及乳房、锁骨上区域、纵隔和胸腔积液等部位。该患者对多种治疗均无反应,包括蛋白酶体抑制剂(PI)如硼替佐米、免疫调节剂(IMiDs)如来那度胺以及靶向CD38的单克隆抗体如达雷妥尤单抗。因此,我们建议进行单倍体相合造血干细胞移植作为挽救治疗。在接受了主要包括塞利尼索和螺旋断层放疗的预处理方案的allo-HSCT后,患者实现了完全缓解(CR),并享受了11个月的长期无病生存。结合现有文献,该病例为RRMM和EMM的未来研究提供了令人鼓舞的见解,我们预计未来会有更多关于allo-HSCT病例的报告。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/2ae2d64d4f51/JBM-16-177-g0004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/8d0abd91ee76/JBM-16-177-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/8e075e1cd1d0/JBM-16-177-g0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/258fc3d07ce8/JBM-16-177-g0003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/2ae2d64d4f51/JBM-16-177-g0004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/8d0abd91ee76/JBM-16-177-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/8e075e1cd1d0/JBM-16-177-g0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/258fc3d07ce8/JBM-16-177-g0003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fe32/11972968/2ae2d64d4f51/JBM-16-177-g0004.jpg

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本文引用的文献

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