2018
Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia
Abstract: In this global study of CAR T-cell therapy, a single infusion of tisagenlecleucel provided durable remission with long-term persistence in pediatric and young adult patients with relapsed or refractory B-cell ALL, with transient high-grade toxic effects. (Funded by Novartis Pharmaceuticals; ClinicalTrials.gov number, NCT02435849 .).
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Cited by 5,290 publications
(4,844 citation statements)
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“…23 Novel treatment options for B-cell ALL, including monoclonal antibodies and CAR T cells, may also have therapeutic potential in these patients. 24,25 However, our case does not confirm these observations. Considering patient´s refraction to given treatment, the chance for finding new possible drug sensitivities could lay in performing drug response profiling studies.…”
Section: Discussioncontrasting
confidence: 99%
“…23 Novel treatment options for B-cell ALL, including monoclonal antibodies and CAR T cells, may also have therapeutic potential in these patients. 24,25 However, our case does not confirm these observations. Considering patient´s refraction to given treatment, the chance for finding new possible drug sensitivities could lay in performing drug response profiling studies.…”
Section: Discussioncontrasting
confidence: 99%
“…The safety profile of tisagenlecleucel observed in this long-term follow-up analysis was consistent with published reports, 4,5 and no new AEs or treatment-related mortality were observed (Data Supplement). The proportion of patients with grade 3/4 AEs declined over time (83.5% of patients < 8 weeks after infusion, 49% from 8 weeks to 1 year, and 29% > 1 year).…”
Section: Resultssupporting
confidence: 91%
“…Consistent with this, we demonstrate that primary NHL samples display a remarkable amount of heterogeneity in CD19 expression, which could account for the lower reported response rates in this disease compared with B-ALL (1,5,7,9,10,60). Using the well-described NALM6 preclinical model, we demonstrate the limited activity of CD19 CARs against low-antigen-density tumor cells, in line with previously reported data for various CARs (16)(17)(18)(19)(20)56).…”
Section: The Cd28 H/t Domain Results In Faster Tumor-cell Killing Andsupporting
confidence: 89%
