Abstract: SA-PO0659
Reduced Intensity Lymphodepletion and Dose Optimization of Autologous CD19 Chimeric Antigen Receptor T Cell (CAR-T) Therapy in Pediatric Refractory Systemic Lupus Erythematosus
Session Information
- Pediatric Nephrology: Transplantation, Hypertension, AKI, Genetics, and Developmental Diseases
November 08, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- He, Xue, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Liu, Fei, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Zhang, Yingzi, Chongqing Precision Biotechnology Co., LTD, Chongqing, China
- Zheng, Chen, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Li, Qiu-yu, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Wang, Jingjing, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Feng, Chunyue, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Fu, Haidong, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Zhang, Juan, Chongqing Precision Biotechnology Co., LTD, Chongqing, China
- Shen, Junjie, Chongqing Precision Biotechnology Co., LTD, Chongqing, China
- Xiao, Huijie, Peking University First Hospital, Beijing, China
- Lu, Meiping, Department of Allergy Immunology and Rheumatology, Children’s Hospital of Zhejiang University School of Medicine, Hangzhou, China
- Qian, Peng Qian, Xuzhou Children’s Hospital, Xuzhou Medical University, Jiangsu, China
- Wang, Yulong, Department of Pediatrics, The Second Hospital of Shandong University, Shandong, China
- Xiao, Jing, Department of Pediatric, Affiliated Hospital of Guangdong Medical University, Guangdong, China
- Yu, Shengyou, Department of Pediatrics, Guangzhou First People’s Hospital, South China University of Technology, Guangzhou, China
- Jiang, Xiaoyun, The First Affiliated Hospital of Sun Yat-sen University, Guangzhou, Guangdong, China
- Qian, Cheng, Chongqing Precision Biotechnology Co., LTD, Chongqing, China
- Mao, Jianhua, Nephrology Department, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
- Ye, Qing, Department of Laboratory Medicine, Children’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
Background
Autologous CD19 CAR-T cell therapy demonstrates efficacy in adults with refractory SLE, yet pediatric data remain limited. Thus, optimal lymphodepletion regimens and cell doses for children demand exploration.
Methods
Severe pediatric SLE patients with inadequate response to ≥4 prior therapies received autologous CD19 CAR-T cells at doses of 0.3×105/kg, 1×105/kg, or 3×105/kg. All patients underwent lymphodepleting chemotherapy with fludarabine and cyclophosphamide, with reduced-intensity lymphodepletion allowed. Immunosuppressants stopped 1 week pre-infusion. Efficacy measured by SRI-4, LLDAS, and DORIS. Safety monitored for CRS and ICANS.
Results
Among the 22 patients evaluated, 8 patients received full-dose lymphodepletion, while 14 received reduced-intensity lymphodepletion (2 with a 2/3 dose, 2 with a 1/2 dose and 10 with a 1/3 dose). Furthermore, 6 patients received 0.3×105/kg cell doses, 12 received 1×105/kg cell doses, and 4 received 3×105/kg cell doses. CAR-T expansion and efficacy were independent of dose or conditioning. At a median 6.4-month follow-up (3.1–13.1) 86.4 % achieved SRI-4, 45.5 % reached LLDAS and 31.8 % attained DORIS remission. CRS occurred in 20/22 (grade 1 = 19, grade 3 = 1) and ICANS in 4/22 (grade 1 = 3, grade 4 = 1). Three patients (13.6 %) relapsed 5–9 months post-infusion.
Conclusion
This study revealed that autologous CD19 CAR-T cell therapy with reduced lymphodepletion and lower cell doses is effective and safe for pediatric patients with refractory SLE.
Funding
- Other NIH Support