P101 Anti-CD19 CAR-T cell therapy for refractory childhood-onset systemic lupus erythematosus

• DOI: 10.1136/lupus-2024-el.155
• Publication Date: 2024-03-15T07:35:20Z
• Authors: Emiliano Marasco; Claudia Bracaglia; Pietro Merli; Patricia Moran Al...; Rebecca Nicolai; Mattia Algeri; Maria Giuseppina ...; Marco Becilli; Fabrizio De Bened...; Franco Locatelli
• Keywords: Refractory (planetary science); Systemic therapy

<h3>Objective</h3> To report the first case of a patient with refractory childhood-onset SLE successfully treated anti-CD19 CAR-T cell therapy. <h3>Methods</h3> A single infusion fresh, autologous second-generation product (lentivirus) manufactured on Prodigy device was administered (1x10⁶ cells/kg). <h3>Results</h3> female one-year history presented at our attention age 15 years persistent clinical (hemolytic anemia, thrombocytopenia, malar rash, class II/V lupus nephritis, interstitial lung disease, pulmonary hypertension (PAH) and pericardial effusion) serological (hypocomplementemia high ANA anti-dsDNA antibodies) active disease. She received multiple treatments, including high-dose glucocorticoids (GC), hydroxychloroquine, mycophenolate (MMF), rituximab IV cyclophosphamide (CYC) without reaching remission. started sildenafil bosentan for PAH. developed osteoporosis secondary to GC therapy, 2 severe systemic infections, requiring prolonged hospitalization. Anti-CD19 therapy considered performed after lymphodepletion CYC (1500mg/m2) fludarabine (90mg/m2). Immunosuppressants (GC, MMF hydroxychloroquine) were withdrawn before After infusion, fever, rash pleural effusion as part mild cytokine release syndrome (G1) transient anemia (G2) neutropenia (G3). No infection or neurotoxicity observed. The cells expanded significantly (peak day 12, 52 cells/uL). Circulating B baseline 111 cells/uL. Complete blood B-cell depletion achieved 7. Bone marrow aspirates week 4 showed complete depletion. Urinalysis normalized 3. Complement levels 6. titers decreased (from 1:5120 1:1280 1:640 1:160 8, respectively). normal right ventricular systolic pressure NT-ProBNP found 6, allowing discontinuation. At time SLEDAI-2K score 22, which rapidly improved 6 10. Patient drug-free remission last follow-up (week 10). <h3>Conclusions</h3> In this infusion. Long-term efficacy assessment is needed.