References
  1. 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;378(5):439-448. doi:10.1056/NEJMoa1709866
  2. Gardner RA, Finney O, Annesley C, Brakke H, Summers C, Leger K, et al. Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults. Blood . 2017;129(25):3322-3331. doi:10.1182/blood-2017-02-769208
  3. Pasquini MC, Hu ZH, Curran K, Laetsch T, Locke F, Rouce R, et al. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma [published correction appears in Blood Adv. 2021 Feb 23;5(4):1136] [published correction appears in Blood Adv. 2022 Mar 22;6(6):1731]. Blood Adv . 2020;4(21):5414-5424. doi:10.1182/bloodadvances.2020003092
  4. Rives S, Maude SL, Hiramatsu, H, Baruchel A, Bader P, Bittencourt H, et al. S112: Tisagenlecleucel in Pediatric and Young Adult Patients (Pts) with Relapsed/Refractory (R/R) B-cell Acute Lymphoblastic Leukemia (B-ALL): Final Analyses from the ELIANA Study.HemaSphere . 2022;6:13-14.
  5. Grupp SA, Maude SL, Rives S, Baruchel A, Boyer M, Bittencourt H, et al. Updated Analysis of the Efficacy and Safety of Tisagenlecleucel in Pediatric and Young Adult Patients with Relapsed/Refractory (r/r) Acute Lymphoblastic Leukemia. Blood  2018; 132 (Supplement 1): 895. doi:10.1182/blood-2018-99-112599
  6. Maude SL, Teachey DT, Rheingold SR, Shaw PA, Aplenc R, Barrett DM, et al. Sustained remissions with CD19-specific chimeric antigen receptor (CAR)-modified T cells in children with relapsed/refractory ALL.J Clin Oncol . 2016;34(Supplement 15): 3011.
  7. Maude SL, Frey N, Shaw PA, Aplenc R, Barrett DM, Bunin NJ, et al. Chimeric antigen receptor T cells for sustained remissions in leukemia [published correction appears in N Engl J Med. 2016 Mar 10;374(10):998]. N Engl J Med . 2014;371(16):1507-1517. doi:10.1056/NEJMoa1407222
  8. Park JH, Rivière I, Gonen M, Wang X, Sénéchal B, Curran KJ, et al. Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia. N Engl J Med . 2018;378(5):449-459. doi:10.1056/NEJMoa1709919
  9. Myers RM, Li Y, Barz Leahy A, Barrett DM, Teachey DT, Callahan C, et al. Humanized CD19-Targeted Chimeric Antigen Receptor (CAR) T Cells in CAR-Naive and CAR-Exposed Children and Young Adults with Relapsed or Refractory Acute Lymphoblastic Leukemia. J Clin Oncol . 2021;39(27):3044-3055. doi:10.1200/JCO.20.03458
  10. Shah NN, Lee DW, Yates B, Yuan C, Shalabi H, Martin S, et al. Long-Term Follow-Up of CD19-CAR T-Cell Therapy in Children and Young Adults With B-ALL. J Clin Oncol . 2021;39(15):1650-1659. doi:10.1200/JCO.20.02262
  11. Zhang X, Lu XA, Yang J, Zhang G, Li J, Song L, et al. Efficacy and safety of anti-CD19 CAR T-cell therapy in 110 patients with B-cell acute lymphoblastic leukemia with high-risk features. Blood Adv . 2020;4(10):2325-2338. doi:10.1182/bloodadvances.2020001466
  12. Schultz LM, Baggott C, Prabhu S, Pacenta HL, Phillips CL, Rossoff J, et al. Disease Burden Affects Outcomes in Pediatric and Young Adult B-Cell Lymphoblastic Leukemia After Commercial Tisagenlecleucel: A Pediatric Real-World Chimeric Antigen Receptor Consortium Report. J Clin Oncol . 2022;40(9):945-955. doi:10.1200/JCO.20.03585
  13. Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, et al. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia . 2022;36(6):1508-1515. doi:10.1038/s41375-022-01550-z
  14. Laetsch TW, Maude SL, Grupp SA, Boyer MW, Harris AC, Qayed M, et al. CTL019 Therapy Appears Safe and Effective in Pediatric Patients with Down Syndrome with Relapsed/ Refractory (r/r) Acute Lymphoblastic Leukemia. Blood  2017; 130 (Supplement 1): 1280. doi:10.1182/blood.V130.Suppl_1.1280.1280
  15. Pulsipher MA, Han X, Maude SL, Laetsch TW, Qayed M, Rives S, et al. Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia. Blood Cancer Discov . 2022;3(1):66-81. doi:10.1158/2643-3230.BCD-21-0095
  16. Lee DW, Santomasso BD, Locke FL, Ghobadi A, Turtle CJ, Brudno JN, et al. ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells. Biol Blood Marrow Transplant . 2019;25(4):625-638. doi:10.1016/j.bbmt.2018.12.758
  17. Orlando EJ, Han X, Tribouley C, Wood PA, Leary RJ, Riester M, et al. Genetic mechanisms of target antigen loss in CAR19 therapy of acute lymphoblastic leukemia. Nat Med . 2018;24(10):1504-1506. doi:10.1038/s41591-018-0146-z
  18. Sotillo E, Barrett DM, Black KL, Bagashev A, Oldridge D, Wu G, et al. Convergence of Acquired Mutations and Alternative Splicing of CD19 Enables Resistance to CART-19 Immunotherapy. Cancer Discov . 2015;5(12):1282-1295. doi:10.1158/2159-8290.CD-15-1020
  19. Gauthier J, Bezerra ED, Hirayama AV, Fiorenza S, Sheih A, Chou CK, et al. Factors associated with outcomes after a second CD19-targeted CAR T-cell infusion for refractory B-cell malignancies. Blood . 2021;137(3):323-335. doi:10.1182/blood.2020006770
  20. Myers RM, Devine K, Li Y, Lawrence S, Leahy AB, Liu H, et al. Outcomes after Reinfusion of CD19-Specific Chimeric Antigen Receptor (CAR)-Modified T Cells in Children and Young Adults with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia. Blood  2021; 138 (Supplement 1): 474. doi:10.1182/blood-2021-147299
  21. Boyer MW, Chaudhury S, Davis KL, Triscoll TA, Grupp SA, Hermiston M, et al. HESTER: A Phase II Study Evaluating Efficacy and Safety of Tisagenlecleucel Reinfusion in Pediatric and Young Adult Patients with Acute Lymphoblastic Leukemia Experiencing Loss of B-Cell Aplasia. Blood  2020; 136 (Supplement 1): 23–24. doi:10.1182/blood-2020-136340
  22. Holland EM, Molina JC, Dede K, Moyer D, Zhou T, Yuan CM, et al. Efficacy of second CAR-T (CART2) infusion limited by poor CART expansion and antigen modulation. J Immunother Cancer . 2022;10(5):e004483. doi:10.1136/jitc-2021-004483
  23. Fabrizio VA, Boelens JJ, Mauguen A, Baggott C, Prabhu S, Egeler E, et al. Optimal fludarabine lymphodepletion is associated with improved outcomes after CAR T-cell therapy. Blood Adv . 2022;6(7):1961-1968. doi:10.1182/bloodadvances.2021006418
  24. Li AM, Hucks GE, Dinofia AM, Seif AE, Teachey DT, Baniewicz D, et al. Checkpoint Inhibitors Augment CD19-Directed Chimeric Antigen Receptor (CAR) T Cell Therapy in Relapsed B-Cell Acute Lymphoblastic Leukemia. Blood  2018; 132 (Supplement 1): 556. doi:10.1182/blood-2018-99-112572
  25. Stefanski H, Eaton A, Baggott C, Rossoff J, Verneris MR, Keating AK, et al. Higher doses of tisagenlecleucel associate with improved outcomes: a report from the pediatric real-world CAR consortium [published online ahead of print, 2022 Aug 8]. Blood Adv . 2022;bloodadvances.2022007246. doi:10.1182/bloodadvances.2022007246
  26. Brentjens RJ, Santos E, Nikhamin Y, Yeh R, Matsushita M, La Perle K, et al. Genetically targeted T cells eradicate systemic acute lymphoblastic leukemia xenografts. Clin Cancer Res . 2007;13(18 Pt 1):5426-5435. doi:10.1158/1078-0432.CCR-07-0674