1. Results of ARI-0001 CART19 cell therapy in patients with relapsed/refractory CD19-positive acute lymphoblastic leukemia with isolated extramedullary disease.
- Author
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Ortiz-Maldonado V, Alonso-Saladrigues A, Español-Rego M, Martínez-Cibrián N, Faura A, Magnano L, Català A, Benítez-Ribas D, Giné E, Díaz-Beyá M, Correa JG, Rovira M, Montoro-Lorite M, Martínez-Roca A, Rodríguez-Lobato LG, Cabezón R, Cid J, Lozano M, Garcia-Rey E, Conde N, Pedrals G, Rozman M, Torrebadell M, Setoain X, Rodríguez S, Esteve J, Pascal M, Urbano-Ispizua Á, Juan M, Delgado J, and Rives S
- Subjects
- Adult, Antigens, CD19 therapeutic use, Child, Clinical Trials as Topic, Cytokine Release Syndrome epidemiology, Humans, Multicenter Studies as Topic, Positron Emission Tomography Computed Tomography, Immunotherapy, Adoptive adverse effects, Immunotherapy, Adoptive methods, Precursor Cell Lymphoblastic Leukemia-Lymphoma diagnostic imaging, Precursor Cell Lymphoblastic Leukemia-Lymphoma therapy
- Abstract
We evaluated outcomes of 18 patients with isolated extramedullary disease (iEMD) relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) treated with the CD19-directed CAR T cells ARI-0001 in two centers (adult and pediatric), including patients treated in the CART19-BE-01 trial and the consecutive compassionate use program. iEMD was detected by PET-CT in 78% (14/18), and/or by cerebrospinal fluid analysis in 28% (5/18). Patients received cyclophosphamide and fludarabine followed by 1 × 10
6 ARI-0001 cells/kg, initially as a single dose (first patient) and later split into three fractions (10%, 30%, and 60%). Cytokine release syndrome (CRS) occurred in 50% (9/18) of patients, with no cases of grade ≥3 CRS, and 1 case (6%) of grade 1 neurotoxicity. Tocilizumab was used in 6% of patients (1/18). Procedure-related mortality was 0% at 2 years. Objective responses were seen in 94% (95% confidence interval [CI]: 73%-99%) of patients, with complete responses (CR) seen in 78% (95% CI: 52%-94%) of them. Progression-free and overall survival were 49% (95% CI: 30%-79%) and 61% (95% CI: 40%-92%) at 2 years. In conclusion, the use of ARI-0001 cells in patients with R/R ALL and iEMD was associated with a safety and efficacy profile that is comparable with what is observed in patients with marrow involvement and in line with other CART19 products., (© 2022 Wiley Periodicals LLC.)- Published
- 2022
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