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

Gardner, Rebecca; Finney, Olivia; Annesley, Colleen; Brakke, Hannah; Summers, Corinne; Leger, Kasey J.; Bleakley, Marie; Brown, Christopher; Mgebroff, Stephanie; Kelly‐Spratt, Karen S.; Hoglund, Virginia J.; Lindgren, Catherine; Oron, Assaf P.; Li, D.; Riddell, Stanley R.; Park, Julie R.; Jensen, Michael C.

doi:10.1182/blood-2017-02-769208

articleBloodApr 13, 2017BRONZE OA

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

RGRebecca Gardner OFOlivia Finney CAColleen Annesley HBHannah Brakke CSCorinne Summers

Seattle Children's Hospital · University of Washington · +1 more institution

PubMed

Indexed incrossrefpubmed

Abstract

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.

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Topics

Keywords

Fludarabine
Medicine
Cytokine release syndrome
Cyclophosphamide
Internal medicine
Adverse effect
Minimal residual disease
CD8

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