Med (New York, N.Y.) · 2026
Anti-CD19 CAR T cell therapy for refractory SLE-ITP.
Clinical StudyСтатьяНазвание и аннотация: Перевод в очереди
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Li Mengtao, Zhao Jiuliang, Lu Zhi John, Ning Chun, Jiang Nan, Zhang Yan, Li Yu, Feng Yi, Wang Qian, Xu Dong, Leng Xiaomei, Li Jing, Zhang Shangzhu, Wang Ziqian, Peng Liying, Tian Xinping, Lv Lulu, Zhou Daobin, Zeng Xiaofeng
Аннотация
BACKGROUND: Patients with systemic lupus erythematosus (SLE) complicated by immune thrombocytopenia (SLE-ITP) exhibit an increased incidence of bleeding events and significantly reduced long-term survival, with poor responses to conventional therapies as well as biologic agents.
METHODS: In an investigator-initiated, single-arm, dose-escalation trial, six patients with refractory SLE-ITP received anti-CD19 chimeric antigen receptor (CAR) T cells after lymphodepleting chemotherapy. The primary outcome was safety. Secondary outcomes included overall response rate at time points (complete remission [CR]: platelet count ≥ 100 × 109/L; partial remission [PR]: ≥30 × 109/L with 2-fold baseline increase).
FINDINGS: All patients achieved clinical response (CR or PR)-with three attaining CR-and discontinued immunosuppressants, with a median follow-up of 12 months. Treatment-related adverse events were limited to grade 1 cytokine release syndrome in two patients, and no immune effector cell-associated neurotoxicity syndrome (ICANS) occurred. Continuously accumulating bone marrow plasma cells, overactivated bone marrow B cell signaling pathways, and weakened activity of the megakaryocyte maturation pathway, along with dysregulated transcription factors, were observed in three PR patients by exploratory single-cell multi-omics.
CONCLUSION: These results preliminarily support CD19 CAR T cell therapy as a promising and safe treatment for refractory SLE-ITP. Large-scale trials are needed to verify these conclusions (ClinicalTrials.gov: NCT05930314).
FUNDING: This study was supported by the Chinese National Key Technology R&D Program, Ministry of Science and Technology (2021YFC2501300); the CAMS Innovation Fund for Medical Sciences (CIFMS) (2021-I2M-1-005); and National High Level Hospital Clinical Research Funding (2025-PUMCH-D-001, 2022-PUMCH-B-013, D-009). This study was also funded and supported by Juventas Cell Therapy Ltd.
METHODS: In an investigator-initiated, single-arm, dose-escalation trial, six patients with refractory SLE-ITP received anti-CD19 chimeric antigen receptor (CAR) T cells after lymphodepleting chemotherapy. The primary outcome was safety. Secondary outcomes included overall response rate at time points (complete remission [CR]: platelet count ≥ 100 × 109/L; partial remission [PR]: ≥30 × 109/L with 2-fold baseline increase).
FINDINGS: All patients achieved clinical response (CR or PR)-with three attaining CR-and discontinued immunosuppressants, with a median follow-up of 12 months. Treatment-related adverse events were limited to grade 1 cytokine release syndrome in two patients, and no immune effector cell-associated neurotoxicity syndrome (ICANS) occurred. Continuously accumulating bone marrow plasma cells, overactivated bone marrow B cell signaling pathways, and weakened activity of the megakaryocyte maturation pathway, along with dysregulated transcription factors, were observed in three PR patients by exploratory single-cell multi-omics.
CONCLUSION: These results preliminarily support CD19 CAR T cell therapy as a promising and safe treatment for refractory SLE-ITP. Large-scale trials are needed to verify these conclusions (ClinicalTrials.gov: NCT05930314).
FUNDING: This study was supported by the Chinese National Key Technology R&D Program, Ministry of Science and Technology (2021YFC2501300); the CAMS Innovation Fund for Medical Sciences (CIFMS) (2021-I2M-1-005); and National High Level Hospital Clinical Research Funding (2025-PUMCH-D-001, 2022-PUMCH-B-013, D-009). This study was also funded and supported by Juventas Cell Therapy Ltd.