CAR T-Cell Therapy in Acute Lymphoblastic Leukemia—Is Limited Persistence Enough?
Mené sur 261 enfants atteints d'une leucémie lymphoblastique aiguë à cellules B (âge médian : 8,2 ans ; durée médiane de suivi : 35,7 mois), cet essai de phase II détermine la dose maximale tolérée d'une immunothérapie bicistronique à base de lymphocytes CAR-T anti-CD19/CD22 puis évalue son efficacité du point de vue de la survie sans événement
In this issue of JAMA Oncology, Wan et al report a cohort of 261 pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) treated with bicistronic anti-CD19/CD22 chimeric antigen receptor (CAR) T-cell therapy. This nonrandomized clinical trial achieved remarkable remission rates of 99.2% (95% CI, 97.3%-99.8%) and 2-year event-free survival (EFS) of 57.9% (95% CI, 51.6%-65.0%) among patients who did not undergo consolidative hematopoietic stem cell transplant (HSCT). Intriguingly, these outcomes were achieved despite a median CAR T-cell persistence of only approximately 2 months (median [IQR] time to B-cell recovery [≥1% normal B cells in peripheral blood and/or bone marrow], 62 [52-92] days), challenging the prevailing paradigm that prolonged CAR T-cell–mediated immune surveillance is required for durable remission in ALL. Furthermore, Wan et al provide important insights into a particularly underrepresented population: patients with isolated extramedullary disease, 60% of whom remained disease-free at 2 years after CAR T-cell therapy without additional consolidation or local irradiation.
JAMA Oncology , éditorial, 2026