EBMT 2022 | ARI-0001 CART19 therapy for R/R B-ALL with isolated extramedullary disease

Valentín Ortiz-Maldonado, MD, Hospital Clínic de Barcelona, Barcelona, Spain, comments on the results of a study evaluating ARI-0001, an academic second-generation chimeric antigen receptor T-cell (CAR-T) therapy in patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) with isolated extramedullary disease. Dr Ortiz-Maldonado explains that these patients are usually excluded from pivotal industry-driven CAR-T trials due to concerns about the efficacy of CAR-Ts in this population. In this study, 18 patients with extramedullary disease were treated with this CD19-directed CAR-T therapy. The study reported an overall response rate (ORR) of 94%, a complete response (CR) rate of 78%, and a two-year progression-free survival (PFS) of 49%. In addition, although half of the patients experienced cytokine release syndrome (CRS), there were no severe cases of CRS. Overall, these findings suggest that this patient population should not be excluded from studies evaluating CD19-directed commercial CAR-Ts. This interview took place at the 48th Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) 2022, which was held virtually.