研究概要
RIC 目前更受青睐,因为它显著降低了移植相关死亡率,该死亡率已降至 10-20%。
中文摘要
复发/难治性多发性骨髓瘤(RRMM)和髓外多发性骨髓瘤(EMM)在疾病进展后仍给患者带来重大挑战。尽管自体造血干细胞移植(ASCT)和CAR-T 细胞疗法等治疗已有显著进展,多数RRMM和EMM患者生存期仍较短,目前有效治疗选择有限。异基因造血干细胞移植(allo-HSCT)是多发性骨髓瘤的治疗选择之一;减低强度预处理(RIC)因将移植相关死亡率降至10%–20%而逐渐取代清髓性预处理(MAC),但其复发率高于MAC。为提高allo-HSCT疗效,仍需寻找更安全有效的预处理方案。本文报告一例EMM,累及乳房、锁骨上区、纵隔并伴胸腔积液等部位。患者对多种治疗均无应答,包括蛋白酶体抑制剂硼替佐米、免疫调节药物来那度胺及抗CD38单克隆抗体达雷妥尤单抗,因此接受单倍体相合造血干细胞移植作为挽救治疗。患者接受主要包括塞利尼索和螺旋断层放疗的预处理方案后行allo-HSCT,达到完全缓解(CR),并保持无病生存11个月。结合既往文献,该病例为RRMM和EMM的后续研究提供了有希望的线索。
展开英文摘要原文
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.
论文信息
- 作者
- Chen B、Guo R、Niu Y、Guo W、Wang T
- 单位
- 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.China
- 文献类型
- 病例报告
- 期刊
- Journal of blood medicine2025