Evolution of tisagenlecleucel use for the treatment of pediatric and young adult relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL): Center for International Blood & Marrow Transplant Research (CIBMTR) registry results.

• DOI: 10.1200/jco.2024.42.16_suppl.10016
• Publication Date: 2024-06-03T19:38:10Z
• Authors: Rayne H. Rouce; Susanne H.C. Baum...; Kevin J. Curran; Vanessa A. Fabrizio; Erin M. Hall; Emily M. Hsieh; Nicole A. Karras; Amy K. Keating; Amy Moskop; Marcelo C. Pasquini; Christine L. Phil...; Michael A. Pulsipher; Marja Nuortti; Jennifer Willert; Roberto Ramos; Samuel John; Stephan A. Grupp
• Keywords: Refractory (planetary science); Blinatumomab; Bone marrow transplant; Acute lymphocytic leukemia; Bone transplantation

10016 Background: Tisagenlecleucel is an autologous CD19-directed chimeric antigen receptor (CAR) T-cell immunotherapy indicated for patients (pts) up to 25 y of age with B-ALL that refractory or in second later relapse. Since the pivotal ELIANA trial, pt characteristics now include pts < 3 y, isolated central nervous system relapse, and leukemia burden 5%. Here we examine impact tisagenlecleucel on treatment journey since FDA approval 2017. Methods: Data were collected as a part noninterventional, prospective, longitudinal study using CIBMTR registry. Pts treated United States, Canada, Korea, Taiwan. Results: As May 4, 2023, 974 received tisagenlecleucel. Primary disease history has evolved Notably, prior infusion decreased (≥50% blasts: 18% 2018, 4% 2022) higher proportion while morphological complete remission (34% 51% 2022). Between 2018 2022, who third greater relapse (14% vs 2%, respectively). ≥18 had more exposure blinatumomab inotuzumab compared 18 y: 27% 16% 17% 7%, respectively. The undergoing ≥1 hematopoietic stem cell transplantation (HSCT) before (37% 15% 2022), coinciding use earlier lines therapy. Reporting B-cell recovery was suboptimal. In total, 34.5% (314/911) postinfusion HSCT (reasons not captured most pts); 8.5% (77/911) treat persistent/progressive disease, positive minimal residual disease. Although overall rate did change, high-risk cytogenetics showed decrease frequency. Previously, KMT2A rearrangement HSCT. 2017, only (12/75) despite 72% (54/75) having rearrangement. Furthermore, rearrangement, 43% (23/53) postinfusion. With censoring HSCT, median RFS improved: mo 27 2020, estimable 2021. OS substantially affected by censoring; 36-mo probabilities (95% CI) without 66 (61-71) 62 (57-66), Conclusions: Pediatric young adult r/r are receiving course their treatment, reducing prolonging RFS. both real-world clinical trial data supporting curative potential grow, after CAR-T should be carefully evaluated.