Abstract: SA-OR088
Safety and Efficacy of Anti-CD19/B Cell Maturation Antigen (BCMA) Chimeric Antigen Receptor-Natural Killer Cell (CAR-NK) Therapy in Pediatric Patients with Refractory Systemic Lupus Erythematosus (SLE): A Preliminary Clinical Study
Session Information
- Late-Breaking Research Orals - 3
November 08, 2025 | Location: Grand Ballroom C, Convention Center
Abstract Time: 04:30 PM - 04:42 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- Meng, Hanyan, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Zheng, Chen, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Xie, Yi, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Li, Qiu-yu, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Liu, Fei, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Wang, Jingjing, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Sun, Ming, Ruitherapeutics Co., LTD, Shen Zhen, China
- Xu, Enshun, Ruitherapeutics Co., LTD, Shen Zhen, China
- Mao, Jianhua, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
Background
Pediatric SLE exhibits greater severity and organ involvement than adult-onset disease, with frequent treatment failure. While CD19 CAR-T therapy shows efficacy in refractory cases, its use is limited by toxicity risks. CAR-NK cells offer a safer alternative with reduced risks and off-the-shelf potential, improving feasibility for pediatric application.
Methods
This phase I trial utilized a dose-escalation design to evaluate the safety and efficacy of KN5601, an anti-CD19/BCMA CAR-NK cell product, in patients with active SLE (SLEDAI-2K >6) refractory to standard therapy. Following lymphodepletion with fludarabine and cyclophosphamide, patients received six infusions of KN5601 at weight-tiered doses. Primary endpoints included the incidence of DLTs and adverse events; key efficacy outcomes comprised SRI-4 response, LLDAS, and DORIS remission rates at 6 months.
Results
As of August 2025, 11 patients completed treatment and 6 reached 3-month follow-up. Three achieved SRI-4 response at 3 months; The longest follow-up was 6M; this patient attained SRI-4 at 3M, LLDAS at 4M, and DORIS remission at 6M. CRS was observed in 4 patients (3 grade I, 1 grade II), with no ICANS or rejection. B-cell reconstitution occurred by 2 months, dominated by naïve B cells.
Conclusion
In patients with refractory pediatric SLE, treatment with anti-CD19/BCMA CAR-NK cells (KN5601) appears feasible, safe, and clinically active. These preliminary findings warrant further clinical validation.
Disease characteristics and safety outcomes.
| ID | Age | Gender | Duration of Disease (Years) | Kidney biopsy | Dose | CRS | Follow up period(M) | SLEDAI- 2K Baseline | SLEDAI- 2K at present |
| 001 | 13 | F | 2 | / | 1*10^9 | / | 6 | 14 | 0 |
| 002 | 13 | F | 2 | III+V | 2*10^9 | / | 6 | 14 | 2 |
| 004 | 17 | F | 6 | III+V | 3*10^9 | / | 4 | 10 | 8 |
| 005 | 15 | M | 6 | IV+V | 3*10^9 | I | 4 | 14 | 10 |
| 006 | 17 | M | 7 | III+III-V | 4.5*10^9 | / | 3 | 14 | 8 |
| 007 | 15 | F | 5 | II | 4.5*10^9 | II | 3 | 10 | 0 |
| 008 | 14 | F | 4 | III | 3*10^9 | I | 2 | 8 | 4 |
| 009 | 15 | F | 8 | IV | 3*10^9 | I | 1 | 18 | 10 |
| 010 | 13 | M | 3 | / | 4.5*10^9 | / | 1 | 10 | 4 |
| 011 | 13 | F | 0.67 | IV+V | 3*10^9 | / | 1 | 8 | 6 |
| 012 | 12 | F | 3 | / | 4.5*10^9 | / | 1 | 10 | 6 |
Funding
- Commercial Support – Ruitherapeutics Co., Ltd.