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T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial.

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      Abstract

      Chimeric antigen receptor (CAR) modified T cells targeting CD19 have shown activity in case series of patients with acute and chronic lymphocytic leukaemia and B-cell lymphomas, but feasibility, toxicity, and response rates of consecutively enrolled patients treated with a consistent regimen and assessed on an intention-to-treat basis have not been reported. We aimed to define feasibility, toxicity, maximum tolerated dose, response rate, and biological correlates of response in children and young adults with refractory B-cell malignancies treated with CD19-CAR T cells.

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      Affiliations
      [1 ] Pediatric Oncology Branch, Bethesda, MD, USA.
      [2 ] Experimental Transplantation and Immunology Branch, Bethesda, MD, USA.
      [3 ] Laboratory of Pathology, Bethesda, MD, USA.
      [4 ] Surgery Branch, Bethesda, MD, USA.
      [5 ] Cell Processing Section, Department of Transfusion Medicine, Clinical Center, National Institutes of Health, Bethesda, MD, USA.
      [6 ] Biostatistics and Data Management Section, Office of the Clinical Director, National Cancer Institute, Bethesda, MD, USA.
      [7 ] National Institutes of Health Medical Student Training Program, The University of Nevada School of Medicine, Reno, NV, USA.
      [8 ] Children's Center for Cancer and Blood Diseases, Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
      [9 ] Pediatric Oncology Branch, Bethesda, MD, USA. Electronic address: mackallc@mail.nih.gov.
      Journal
      Lancet
      Lancet (London, England)
      1474-547X
      0140-6736
      Feb 7 2015
      : 385
      : 9967
      S0140-6736(14)61403-3
      10.1016/S0140-6736(14)61403-3
      25319501
      Copyright © 2015 Elsevier Ltd. All rights reserved.

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