Autologous CD19 CAR T-cell therapy for pediatric and adult systemic lupus erythematosus: a phase 1/2 trial - PubMed
3 hours ago
- #CAR T-cell therapy
- #Clinical trial
- #Systemic lupus erythematosus
- The trial evaluates autologous CD19 CAR T-cell therapy for systemic lupus erythematosus in a phase 1/2 design.
- Primary endpoints focus on dose-limiting toxicity and adverse events in phase 1, and response rates at 3 and 6 months in phase 2.
- Eighteen patients were enrolled, mostly pediatric, with 15 having a SLEDAI-2K score ≥4.
- No dose-limiting toxicities occurred, and the recommended dose for phase 2 was 1×10^6 CAR T cells/kg.
- Adverse events included grade 1 cytokine release syndrome in 72%, neurotoxicity in 17%, and infections in 17%.
- Response rates at month 3 and 6 for patients with SLEDAI-2K ≥4 were 9 out of 15 and 12 out of 15 achieving SRI-S response, with some achieving LLDAS or DORIS remission.
- Median follow-up was 10.2 months, with most responders maintaining response without immunosuppressants, and CAR T cells persisted for a median of 56 days.