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.
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.
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采用抗 BCMA CAR-T 细胞疗法及单倍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：病例报告及当代文献综述
DOI：
10.3389/fmed.2021.649824
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发表时间：
2021
影响因子：
3.9
通讯作者：
Xiao H
中科院分区：
文献类型：
--
作者：
Qian Y;Qian Z;Zhao X;Pan W;Wei X;Meng H;Yang L;Xiao H
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患者的生存率有了很好的改善，但EMM患者的总生存期（OS）通常不超过3年。EMM患者的标准治疗尚未建立，其管理特别具有挑战性。我们介绍了一个经过大量预治疗的年轻患者，复发性EMM和对蛋白酶体抑制剂无效（PI;硼替佐米），下一代PI（ixazomib），免疫调节药物（IMiDs;来那度胺）、自体造血干细胞移植（ASCT）和单克隆抗体（针对CD 38：daratumumab），并表明清髓性单倍体相合造血干细胞移植因此，在靶向B细胞成熟抗原（BCMA）的嵌合抗原受体（CAR）-T细胞疗法（NCT 04650724）之后，将HSCT（单倍体同一性-HSCT）作为复发的挽救治疗是可行的。综合当代文献，抗BCMA CAR-T细胞治疗和同种异体HSCT效果的有希望的结果可能为EMM患者提供了原理证明，因此，需要将该疾病患者纳入未来的研究中。
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.
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DOI：
10.1038/nrclinonc.2017.148
发表时间：
2018-01
期刊：
Nature reviews. Clinical oncology
影响因子：
--
作者：
Neelapu SS;Tummala S;Kebriaei P;Wierda W;Gutierrez C;Locke FL;Komanduri KV;Lin Y;Jain N;Daver N;Westin J;Gulbis AM;Loghin ME;de Groot JF;Adkins S;Davis SE;Rezvani K;Hwu P;Shpall EJ
通讯作者：
Shpall EJ
影响因子：
4.3
作者：
Gagelmann, Nico;Eikema, Diderik-Jan;Kroeger, Nicolaus
通讯作者：
Kroeger, Nicolaus
影响因子：
20.3
作者：
Touzeau, Cyrille;Moreau, Philippe
通讯作者：
Moreau, Philippe
影响因子：
11.4
作者：
Gandhi, Ujjawal H.;Cornell, Robert F.;Costa, Luciano J.
通讯作者：
Costa, Luciano J.
影响因子：
3.1
作者：
Pick, Marjorie;Vainstein, Vladimir;Gatt, Moshe E.
通讯作者：
Gatt, Moshe E.