Stefanski H, Eaton A, Baggott C, et al. Higher Doses of Tisagenlecleucel Associated with Improved Outcomes: A Report from the Pediatric Real-World CAR Consortium. Blood Advances. 2022; (doi: 10.1182/bloodadvances.2022007246).
Patients with refractory/relapsed B-cell acute lymphoblastic leukemia (B-ALL) who received higher doses of tisagenlecleucel had increased overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS), according to a study from the Pediatric Real World CAR Consortium. The chimeric antigen receptor T cell (CAR-T) therapy targeting CD19 is approved by the U.S. Food and Drug Administration for patients up to age 26 years with refractory/relapsed B-ALL. Patients receive one dose of tisagenlecleucel, with a broad dose range of 0.2- 5.0 x 106 CAR T cells/kg for patients ≤ 50 kg, or 0.1- 2.5 x 108 CAR T cells for patients > 50 kg. Researchers sought to determine the effect of CAR-T cell dose on OS, EFS, and RFS. Retrospective data for 185 patients infused with commercial tisagenlecleucel shows that the median dose of viable transduced CAR T cells was 1.7 x106 CAR T cells/kg. Responders were divided into four dose groups: 0.134-1.300 x 106 (n=48), 1.301-1.700 x 106 (n=46), 1.701-2.400 x 106 (n=43), and 2.401-5.100 x 106 (n=43). Improved OS, EFS, and RFS was observed in patients who received higher doses of tisagenlecleucel without increase in toxicity. Results of this study suggest that targeting higher doses of tisagenlecleucel be utilized to optimize outcomes for patients