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Biology & Genetics

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

Shannon L. Maude, Theodore W. Laetsch, Jochen Buechner · ELIANA trial investigators

Published 1 February 2018 · New England Journal of Medicine · Journal article

Summary

This paper reports the pivotal global phase 2 ELIANA trial of tisagenlecleucel, an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia. It demonstrated high rates of durable remission and supported the first FDA approval of a CAR-T cell therapy. The study also characterized the safety profile, including cytokine release syndrome.

Key findings

  • Overall remission rate within 3 months was 81%, with all responders negative for minimal residual disease.
  • Event-free survival was 73% and overall survival 90% at 6 months in a heavily pretreated population.
  • Cytokine release syndrome occurred in 77% of patients and was manageable, defining the key toxicity profile of CD19 CAR-T therapy.

Subjects & keywords

Cite this paper

APA

Shannon L. Maude, Theodore W. Laetsch, & Jochen Buechner [ELIANA trial investigators] (2018). Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. New England Journal of Medicine. https://doi.org/10.1056/NEJMoa1709866

BibTeX
@article{maude2018tisagenlecleucel,
  author    = {Shannon L. Maude and Theodore W. Laetsch and Jochen Buechner and {ELIANA trial investigators}},
  title     = {Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia},
  journal   = {New England Journal of Medicine},
  year      = {2018},
  doi       = {10.1056/NEJMoa1709866},
  url       = {https://doi.org/10.1056/NEJMoa1709866}
}

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