Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults

作者：Rebecca Gardner, Olivia Finney, Colleen Annesley, Hannah Brakke, Corinne Summers, Kasey J. Leger, Marie Bleakley, Christopher Brown, Stephanie Mgebroff, Karen S. Kelly‐Spratt, Virginia J. Hoglund, Catherine Lindgren, Assaf P. Oron, D. Li, Stanley R. Riddell, Julie R. Park, Michael C. Jensen · 发表于：Blood · 年份：2017 · DOI：10.1182/blood-2017-02-769208 · 被引用次数：1147 · 研究领域：CAR-T cell therapy research、Nanowire Synthesis and Applications

remission rate was 93% in patients who received a CAR T-cell product and 100% in the subset of patients who received fludarabine and cyclophosphamide lymphodepletion. Twenty-three percent of patients developed reversible severe cytokine release syndrome and/or reversible severe neurotoxicity. These data demonstrate that manufacturing a defined-composition CD19 CAR T cell identifies an optimal cell dose with highly potent antitumor activity and a tolerable adverse effect profile in a cohort of patients with an otherwise poor prognosis. This trial was registered at www.clinicaltrials.gov as #NCT02028455.