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抗BCMA嵌合抗原受体T细胞疗法联合单倍体相合造血干细胞移植成功治疗复发/难治性髓外多发性骨髓瘤:一例报告及当代文献综述

Successful Treatment of Relapsed/Refractory Extramedullary Multiple Myeloma With Anti-BCMA CAR-T Cell Therapy Followed by Haploidentical Hematopoietic Stem Cell Transplantation: A Case Report and a Review of the Contemporary Literature.

作者信息

Qian Ying, Qian Zijun, Zhao Xiujie, Pan Wenjue, Wei Xinzheng, Meng Huimin, Yang Lin, Xiao Haowen

机构信息

Department of Hematology, Sir Run Run Shaw Hospital, Zhejiang University School of Medicine, Hangzhou, China.

Hangzhou Integrative Medicine Hospital, Hangzhou, China.

出版信息

Front Med (Lausanne). 2021 May 7;8:649824. doi: 10.3389/fmed.2021.649824. eCollection 2021.

Abstract

Extramedullary multiple myeloma (EMM) is an aggressive sub-entity of multiple myeloma (MM). Despite an excellent improvement in survival for most patients with MM over recent decades, the overall survival (OS) of patients with EMM was usually not longer than 3 years. Standard treatment for patients with EMM has not been established, and their management is particularly challenging. We presented a heavily pretreated young patient with relapsed EMM and refractoriness to a proteasome inhibitor (PI; bortezomib), a next-generation PI (ixazomib), immunomodulatory drugs (IMiDs; lenalidomide), autologous hematopoietic stem cell transplantation (ASCT), and monoclonal antibody (directed against CD38: daratumumab) and indicated that myeloablative haploidentical hematopoietic stem cell transplantation (haploidentical-HSCT) as a salvage treatment of relapse after a chimeric antigen receptor (CAR)-T cell therapy that targeted B-cell maturation antigen (BCMA) (NCT04650724) is feasible. Taken together of the contemporary literature, the promising results on the effect of anti-BCMA CAR-T cell therapy and allogeneic HSCT might present a proof-of-principle for patients with EMM, and therefore, patients with the disease need to be included in future studies.

摘要

髓外多发性骨髓瘤(EMM)是多发性骨髓瘤(MM)的一种侵袭性亚型。尽管近几十年来大多数MM患者的生存率有了显著提高,但EMM患者的总生存期(OS)通常不超过3年。EMM患者的标准治疗方案尚未确立,其治疗管理尤其具有挑战性。我们报告了一名年轻的、经过大量预处理的复发性EMM患者,该患者对蛋白酶体抑制剂(PI;硼替佐米)、新一代PI(伊沙佐米)、免疫调节药物(IMiDs;来那度胺)、自体造血干细胞移植(ASCT)以及单克隆抗体(靶向CD38:达雷妥尤单抗)均耐药,并指出清髓性单倍体相合造血干细胞移植(单倍体相合-HSCT)作为一种挽救性治疗方法,在针对B细胞成熟抗原(BCMA)的嵌合抗原受体(CAR)-T细胞治疗(NCT04650724)后复发时是可行的。综合当代文献来看,抗BCMA CAR-T细胞治疗和异基因HSCT疗效的有前景结果可能为EMM患者提供了原理验证,因此,该病患者需要纳入未来的研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/065e/8138324/00438874eee9/fmed-08-649824-g0001.jpg

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