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Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma.

Publication ,  Journal Article
Pasquini, MC; Hu, Z-H; Curran, K; Laetsch, T; Locke, F; Rouce, R; Pulsipher, MA; Phillips, CL; Keating, A; Frigault, MJ; Salzberg, D; Ghosh, M ...
Published in: Blood Adv
November 10, 2020

Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL) and adults with non-Hodgkin lymphoma (NHL). The initial experience with tisagenlecleucel in a real-world setting from a cellular therapy registry is presented here. As of January 2020, 511 patients were enrolled from 73 centers, and 410 patients had follow-up data reported (ALL, n = 255; NHL, n = 155), with a median follow-up of 13.4 and 11.9 months for ALL and NHL, respectively. Among patients with ALL, the initial complete remission (CR) rate was 85.5%. Twelve-month duration of response (DOR), event-free survival, and overall survival (OS) rates were 60.9%, 52.4%, and 77.2%, respectively. Among adults with NHL, the best overall response rate was 61.8%, including an initial CR rate of 39.5%. Six-month DOR, progression-free survival, and OS rates were 55.3%, 38.7%, and 70.7%, respectively. Grade ≥3 cytokine release syndrome and neurotoxicity were reported in 11.6% and 7.5% of all patients, respectively. Similar outcomes were observed in patients with in-specification and out-of-specification products as a result of viability <80% (range, 61% to 79%). This first report of tisagenlecleucel in the real-world setting demonstrates outcomes with similar efficacy and improved safety compared with those seen in the pivotal trials.

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Published In

Blood Adv

DOI

EISSN

2473-9537

Publication Date

November 10, 2020

Volume

4

Issue

21

Start / End Page

5414 / 5424

Location

United States

Related Subject Headings

  • Receptors, Antigen, T-Cell
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Lymphoma, Non-Hodgkin
  • Immunotherapy, Adoptive
  • Humans
  • Antigens, CD19
  • Adult
  • 3201 Cardiovascular medicine and haematology
 

Citation

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Pasquini, M. C., Hu, Z.-H., Curran, K., Laetsch, T., Locke, F., Rouce, R., … Grupp, S. (2020). Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Adv, 4(21), 5414–5424. https://doi.org/10.1182/bloodadvances.2020003092
Pasquini, Marcelo C., Zhen-Huan Hu, Kevin Curran, Theodore Laetsch, Frederick Locke, Rayne Rouce, Michael A. Pulsipher, et al. “Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma.Blood Adv 4, no. 21 (November 10, 2020): 5414–24. https://doi.org/10.1182/bloodadvances.2020003092.
Pasquini MC, Hu Z-H, Curran K, Laetsch T, Locke F, Rouce R, et al. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Adv. 2020 Nov 10;4(21):5414–24.
Pasquini, Marcelo C., et al. “Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma.Blood Adv, vol. 4, no. 21, Nov. 2020, pp. 5414–24. Pubmed, doi:10.1182/bloodadvances.2020003092.
Pasquini MC, Hu Z-H, Curran K, Laetsch T, Locke F, Rouce R, Pulsipher MA, Phillips CL, Keating A, Frigault MJ, Salzberg D, Jaglowski S, Sasine JP, Rosenthal J, Ghosh M, Landsburg D, Margossian S, Martin PL, Kamdar MK, Hematti P, Nikiforow S, Turtle C, Perales M-A, Steinert P, Horowitz MM, Moskop A, Pacaud L, Yi L, Chawla R, Bleickardt E, Grupp S. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Adv. 2020 Nov 10;4(21):5414–5424.

Published In

Blood Adv

DOI

EISSN

2473-9537

Publication Date

November 10, 2020

Volume

4

Issue

21

Start / End Page

5414 / 5424

Location

United States

Related Subject Headings

  • Receptors, Antigen, T-Cell
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Lymphoma, Non-Hodgkin
  • Immunotherapy, Adoptive
  • Humans
  • Antigens, CD19
  • Adult
  • 3201 Cardiovascular medicine and haematology