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.

IF 2.1 Q3 HEMATOLOGY
Journal of Blood Medicine Pub Date : 2025-04-02 eCollection Date: 2025-01-01 DOI:10.2147/JBM.S495256
Bin Chen, Rongrong Guo, Yanyan Niu, Wei Guo, Tao Wang
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引用次数: 0

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.

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,但大多数复发性/难治性多发性骨髓瘤(RRMM)和髓外多发性骨髓瘤(EMM)患者的生存期仍然很短。目前,还没有有效的治疗方法。异基因造血干细胞移植(allo-HSCT)是治疗MM的方法之一。降低强度调理(RIC)方案已在很大程度上取代了骨髓溶解调理(MAC)方案。RIC是目前的首选方案,因为它能显著降低移植相关死亡率,目前已降至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,必须找到一种更安全、更有效的调理方案。我们报告了一例累及乳房、锁骨上区、纵隔和胸腔积液等部位的EMM病例。患者对多种治疗方法均无反应,包括硼替佐米等蛋白酶体抑制剂(PI)、来那度胺等免疫调节药物(IMiD)以及达拉单抗等靶向CD38的单克隆抗体。因此,我们建议采用单倍体造血干细胞移植作为挽救治疗。患者接受了异基因造血干细胞移植(allo-HSCT),并接受了主要包括西利奈索和螺旋断层治疗在内的调理方案,最终获得了完全缓解(CR),并长期无病生存了11个月。结合现有文献,本病例为今后研究RRMM和EMM提供了令人鼓舞的启示,我们期待今后有更多关于allo-HSCT病例的报道。
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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
94
审稿时长
16 weeks
期刊介绍: The Journal of Blood Medicine is an international, peer-reviewed, open access, online journal publishing laboratory, experimental and clinical aspects of all topics pertaining to blood based medicine including but not limited to: Transfusion Medicine (blood components, stem cell transplantation, apheresis, gene based therapeutics), Blood collection, Donor issues, Transmittable diseases, and Blood banking logistics, Immunohematology, Artificial and alternative blood based therapeutics, Hematology including disorders/pathology related to leukocytes/immunology, red cells, platelets and hemostasis, Biotechnology/nanotechnology of blood related medicine, Legal aspects of blood medicine, Historical perspectives. Original research, short reports, reviews, case reports and commentaries are invited.
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