학술논문
CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+Relapsed/Refractory Malignancies
Document Type
Article
Author
Ortíz-Maldonado, Valentín; Rives, Susana; Castellà, Maria; Alonso-Saladrigues, Anna; Benítez-Ribas, Daniel; Caballero-Baños, Miguel; Baumann, Tycho; Cid, Joan; Garcia-Rey, Enric; Llanos, Cristina; Torrebadell, Montserrat; Villamor, Neus; Giné, Eva; Díaz-Beyá, Marina; Guardia, Laia; Montoro, Mercedes; Català, Albert; Faura, Anna; González, E. Azucena; Español-Rego, Marta; Klein-González, Nela; Alsina, Laia; Castro, Pedro; Jordan, Iolanda; Fernández, Sara; Ramos, Federico; Suñé, Guillermo; Perpiñá, Unai; Canals, Josep M.; Lozano, Miquel; Trias, Esteve; Scalise, Andrea; Varea, Sara; Sáez-Peñataro, Joaquín; Torres, Ferran; Calvo, Gonzalo; Esteve, Jordi; Urbano-Ispizua, Álvaro; Juan, Manel; Delgado, Julio
Source
Molecular Therapy; February 2021, Vol. 29 Issue: 2 p636-644, 9p
Subject
Language
ISSN
15250016; 15250024
Abstract
We evaluated the administration of ARI-0001 cells (chimeric antigen receptor T cells targeting CD19) in adult and pediatric patients with relapsed/refractory CD19+malignancies. Patients received cyclophosphamide and fludarabine followed by ARI-0001 cells at a dose of 0.4–5 × 106ARI-0001 cells/kg, initially as a single dose and later split into 3 fractions (10%, 30%, and 60%) with full administration depending on the absence of cytokine release syndrome (CRS). 58 patients were included, of which 47 received therapy: 38 with acute lymphoblastic leukemia (ALL), 8 with non-Hodgkin’s lymphoma, and 1 with chronic lymphocytic leukemia. In patients with ALL, grade ≥3 CRS was observed in 13.2% (26.7% before versus 4.3% after the amendment), grade ≥3 neurotoxicity was observed in 2.6%, and the procedure-related mortality was 7.9% at day +100, with no procedure-related deaths after the amendment. The measurable residual disease-negative complete response rate was 71.1% at day +100. Progression-free survival was 47% (95% IC 27%–67%) at 1 year: 51.3% before versus 39.5% after the amendment. Overall survival was 68.6% (95% IC 49.2%–88%) at 1 year. In conclusion, the administration of ARI-0001 cells provided safety and efficacy results that are comparable with other academic or commercially available products. This trial was registered as ClinicalTrials.gov: NCT03144583.