Clinical StudiesBCMA CAR-T Therapy Is Safe and Effective for Refractory/Relapsed Multiple Myeloma With Central Nervous System InvolvementWang, Yiyun*,†,‡,§; Zu, Cheng*,†,‡,§; Teng, Xinyi*,†,‡,§; Yang, Li*,†,‡,§; Zhang, Mingming*,†,‡,§; Hong, Ruimin*,†,‡,§; Zhao, Houli*,†,‡,§; Cui, Jiazhen*,†,‡,§; Xu, Huijun*,†,‡,§; Hongsheng, Alex Chang*,†,‡,§; Hu, Yongxian*,†,‡,§; Huang, He*,†,‡,§ Author Information *Bone Marrow Transplantation Center, The First Affiliated Hospital, Zhejiang University School of Medicine †Institute of Hematology, Zhejiang University ‡Zhejiang Province Engineering Laboratory for Stem Cell and Immunity Therapy §Zhejiang Laboratory for Systems & Precision Medicine, Zhejiang University Medical Center, Hangzhou, Zhejiang Province, China Y.W., C.Z., and X.T. contributed equally. Reprints: He Huang, Bone Marrow Transplantation Center, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou 311121, Zhejiang Province, China (e-mail: [email protected]). Journal of Immunotherapy: January 2022 - Volume 45 - Issue 1 - p 25-34 doi: 10.1097/CJI.0000000000000391 Buy Metrics Abstract Central nervous system (CNS) involvement is a rare complication of multiple myeloma (MM) that portends an extremely poor prognosis. Although chimeric antigen receptor (CAR)-T cell therapy is considered a promising strategy for patients with MM, the role of CAR-T cell therapy in MM involving the CNS has not been fully elucidated. In this study, we retrospectively analyzed 4 cases of B-cell maturation antigen CAR-T cell therapy for patients with relapsed/refractory MM involving the CNS. Patients received a range of 2–7 lines of prior therapy, including 1 autologous hematopoietic stem cell transplant. The most common adverse event was cytokine release syndrome, which was observed in all 4 patients, including 2 with grade 1 and 2 with grade 2. No patient was complicated with immune effector cell–associated neurotoxicity syndrome. Within the follow-up (median: 257 d, range: 116–392 d), 3 of 4 patients reached complete remission (CR), and 1 patient reached partial response. At the data cutoff, 1 patient continued to remain in CR at day 220, and the patient with partial response died at day 116. The other 2 patients relapsed at 317 and 111 days with CR durations of 287 and 81 days, respectively. Our results show promising effectiveness and acceptable safety of CAR-T cell therapy for heavily pretreated patients with CNS MM. Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved.