Skip to main content
construction release_alert
Scholars@Duke will be undergoing maintenance April 11-15. Some features may be unavailable during this time.
cancel

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

Publication ,  Journal Article
Maude, SL; Laetsch, TW; Buechner, J; Rives, S; Boyer, M; Bittencourt, H; Bader, P; Verneris, MR; Stefanski, HE; Myers, GD; Qayed, M; Schlis, K ...
Published in: N Engl J Med
February 1, 2018

BACKGROUND: In a single-center phase 1-2a study, the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel produced high rates of complete remission and was associated with serious but mainly reversible toxic effects in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). METHODS: We conducted a phase 2, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. The primary end point was the overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months. RESULTS: For this planned analysis, 75 patients received an infusion of tisagenlecleucel and could be evaluated for efficacy. The overall remission rate within 3 months was 81%, with all patients who had a response to treatment found to be negative for minimal residual disease, as assessed by means of flow cytometry. The rates of event-free survival and overall survival were 73% (95% confidence interval [CI], 60 to 82) and 90% (95% CI, 81 to 95), respectively, at 6 months and 50% (95% CI, 35 to 64) and 76% (95% CI, 63 to 86) at 12 months. The median duration of remission was not reached. Persistence of tisagenlecleucel in the blood was observed for as long as 20 months. Grade 3 or 4 adverse events that were suspected to be related to tisagenlecleucel occurred in 73% of patients. The cytokine release syndrome occurred in 77% of patients, 48% of whom received tocilizumab. Neurologic events occurred in 40% of patients and were managed with supportive care, and no cerebral edema was reported. CONCLUSIONS: 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 .).

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

February 1, 2018

Volume

378

Issue

5

Start / End Page

439 / 448

Location

United States

Related Subject Headings

  • Young Adult
  • Survival Analysis
  • Remission Induction
  • Receptors, Antigen, T-Cell
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Male
  • Infusions, Intravenous
  • Humans
  • General & Internal Medicine
  • Female
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Maude, S. L., Laetsch, T. W., Buechner, J., Rives, S., Boyer, M., Bittencourt, H., … Grupp, S. A. (2018). Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med, 378(5), 439–448. https://doi.org/10.1056/NEJMoa1709866
Maude, Shannon L., Theodore W. Laetsch, Jochen Buechner, Susana Rives, Michael Boyer, Henrique Bittencourt, Peter Bader, et al. “Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.N Engl J Med 378, no. 5 (February 1, 2018): 439–48. https://doi.org/10.1056/NEJMoa1709866.
Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, et al. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018 Feb 1;378(5):439–48.
Maude, Shannon L., et al. “Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.N Engl J Med, vol. 378, no. 5, Feb. 2018, pp. 439–48. Pubmed, doi:10.1056/NEJMoa1709866.
Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018 Feb 1;378(5):439–448.

Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

February 1, 2018

Volume

378

Issue

5

Start / End Page

439 / 448

Location

United States

Related Subject Headings

  • Young Adult
  • Survival Analysis
  • Remission Induction
  • Receptors, Antigen, T-Cell
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Male
  • Infusions, Intravenous
  • Humans
  • General & Internal Medicine
  • Female