Department of Hematology, Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, 1095 Jie-Fang Avenue, Wuhan, 430030, Hubei, People's Republic of China.
Immunotherapy Research Center for Hematologic Diseases of Hubei Province, 1095 Jie-Fang Avenue, Wuhan, 430030, Hubei, People's Republic of China.
J Hematol Oncol. 2018 Oct 22;11(1):128. doi: 10.1186/s13045-018-0672-7.
POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome still has no standard treatment. On the basis that both POEMS syndrome and myeloma have an underlying plasma cell dyscrasia, anti-myeloma therapy can be expected to be useful for POEMS syndrome. Chimeric antigen receptor T (CAR-T) cells targeting B cell maturation antigen (BCMA) has been used in the treatment of relapsed and refractory multiple myeloma (RRMM). No POEMS syndrome cases treated with anti-BCMA CAR-T cells have been reported.
Here, we, for the first time, report a POEMS syndrome case treated with anti-BCMA CAR-T cells. A 49-year-old female with incapacitating POEMS syndrome that progressed on lenalidomide treatment was enrolled in a phase I study involving anti-BCMA CAR-T cells (ChiCTR-OPC-16009113). Another patient with RRMM who had undergone six prior lines treatments was also enrolled in the study. They received infusions of anti-BCMA CAR-T cells. Both patients achieved a stringent complete response. Complete remission persisted in the patient with POEMS syndrome and lasted for 7.6 months before a relapse in RRMM patient. Both patients had toxicity consistent with the grade 1 cytokine release syndrome.
This is the first report of treatment by anti-BCMA CAR-T cells in POEMS syndrome. Our findings demonstrate the anti-BCMA CAR-T cell treatment may be a feasible therapeutic option for patients with POEMS syndrome and RRMM who do not respond well to traditional therapies.
ChiCTR-OPC, ChiCTR-OPC-16009113 . Registered 29 August 2016.
POEMS（多发性神经病、器官肿大、内分泌病、单克隆丙种球蛋白病和皮肤改变）综合征仍然没有标准的治疗方法。鉴于 POEMS 综合征和骨髓瘤都存在浆细胞异常增生，抗骨髓瘤治疗有望对 POEMS 综合征有效。靶向 B 细胞成熟抗原（BCMA）的嵌合抗原受体 T（CAR-T）细胞已用于治疗复发性和难治性多发性骨髓瘤（RRMM）。尚未有报道使用抗-BCMA CAR-T 细胞治疗 POEMS 综合征的病例。
在此，我们首次报告了一例使用抗-BCMA CAR-T 细胞治疗的 POEMS 综合征病例。一名 49 岁女性患有进行性 POEMS 综合征，在接受来那度胺治疗后进展，入组了一项涉及抗-BCMA CAR-T 细胞的 I 期研究（ChiCTR-OPC-16009113）。另一名患有 RRMM 的患者，已接受过六线治疗，也入组了该研究。他们接受了抗-BCMA CAR-T 细胞输注。两名患者均达到严格的完全缓解。POEMS 综合征患者的完全缓解持续了 7.6 个月，随后 RRMM 患者复发。两名患者的毒性与 1 级细胞因子释放综合征一致。
这是首例使用抗-BCMA CAR-T 细胞治疗 POEMS 综合征的报告。我们的研究结果表明，抗-BCMA CAR-T 细胞治疗可能是对传统治疗反应不佳的 POEMS 综合征和 RRMM 患者的一种可行的治疗选择。
ChiCTR-OPC，ChiCTR-OPC-16009113。注册于 2016 年 8 月 29 日。
