0
Views
0
CrossRef citations to date
0
Altmetric
Immunotherapy - Cancer

Approved CAR-T therapies have reproducible efficacy and safety in clinical practice

, , , &
Article: 2378543 | Received 24 Apr 2024, Accepted 07 Jul 2024, Published online: 05 Aug 2024

References

  • Sadelain M, Brentjens R, Rivière I. The basic principles of chimeric antigen receptor design. Cancer Discov. 2013;3(4):388–10. doi:10.1158/2159-8290.CD-12-0548.
  • Blonde L, Khunti K, Harris SB, Meizinger C, Skolnik NS. Interpretation and impact of real-world clinical data for the practicing clinician. Adv Ther. 2018;35(11):1763–74. doi:10.1007/s12325-018-0805-y.
  • Pui CH, Yang JJ, Bhakta N, Rodriguez-Galindo C. Global efforts toward the cure of childhood acute lymphoblastic leukaemia [published correction appears in lancet child adolesc health]. Lancet Child Adolesc Health. 2018 Nov;2(6):440–54. doi:10.1016/S2352-4642(18)30066-X.
  • Chang JH, Poppe MM, Hua CH, Marcus KJ, Esiashvili N. Acute lymphoblastic leukemia. Pediatr Blood Cancer. 2021;68(Suppl S2):e28371. doi:10.1002/pbc.28371.
  • National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology (NCCN guidelines®): acute lymphoblastic leukemia. Version 1; 2024 [accessed 2024 Jun 4]. https://www.nccn.org/professionals/physician_gls/pdf/all.pdf.
  • Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, et al. Tisagenlecleucel in children and young adults with B-Cell lymphoblastic leukemia. N Engl J Med. 2018;378(5):439–48. doi:10.1056/NEJMoa1709866.
  • Pasquini MC, Hu ZH, Curran K, Laetsch T, Locke F, Rouce R, Pulsipher MA, Phillips CL, Keating A, Frigault MJ, et al. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma [published correction appears in blood adv]. Blood Adv. 2021 Feb 23 [2022 Mar 22];5(4):1731. doi:10.1182/bloodadvances.2020003092.
  • Barsan V, Li Y, Prabhu S, Baggott C, Nguyen K, Pacenta H, Phillips CL, Rossoff J, Stefanski H, Talano J-A, et al. Tisagenlecleucel utilisation and outcomes across refractory, first relapse and multiply relapsed B-cell acute lymphoblastic leukemia: a retrospective analysis of real-world patterns. EClinicalMed. 2023 [accessed 2023 Oct 26];65:102268. doi:10.1016/j.eclinm.2023.102268.
  • Brissot E, Peczynski C, Labopin M, Lussana F, Hough R, Kuball J, Roesler W, Furst S, Barba P, Daskalakis M, et al. CD19 chimeric antigen receptor (CAR) T-Cell therapy for adults with B-Cell acute lymphoblastic leukemia (B-ALL): a large real-world series from acute leukemia working party of the European society for blood and marrow transplantation (EBMT) blood. Blood. 2022;140(Supplement 1):10389–90. doi:10.1182/blood-2022-158880.
  • Bader P, Rossig C, Hutter M, Ayuk FA, Baldus CD, Bücklein VL, Bonig H, Cario G, Einsele H, Holtick U, et al. CD19 CAR T cells are an effective therapy for posttransplant relapse in patients with B-lineage ALL: real-world data from Germany. Blood Adv. 2023;7(11):2436–48. doi:10.1182/bloodadvances.2022008981.
  • Ghorashian S, Jacoby E, De Moerloose B, Rives S, Bonney D, Shenton G, Bader P, Bodmer N, Quintana AM, Herrero B, et al. Tisagenlecleucel therapy for relapsed or refractory B-cell acute lymphoblastic leukaemia in infants and children younger than 3 years of age at screening: an international, multicentre, retrospective cohort study. Lancet Haematol. 2022;9(10):766–75. doi:10.1016/S2352-3026(22)00225-3.
  • Shah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, Leguay T, Bishop MR, Topp MS, Tzachanis D, et al. KTE-X19 for relapsed or refractory adult B-cell acute lymphoblastic leukaemia: phase 2 results of the single-arm, open-label, multicentre ZUMA-3 study. The Lancet. 2021;398(10299):491–502. doi:10.1016/S0140-6736(21)01222-8.
  • Bezerra E, Itani T, Wudhikarn K, Perales M-A, Yang Y, Gergis U, Aribi A, Hill LC, Jain N, Leonard JT, et al. Real-world outcomes of brexucabtagene autoleucel (brexu-cel) for relapsed or refractory (R/R) adult B-cell acute lymphoblastic leukemia (B-cell ALL): evidence from the CIBMTR registry. Blood. 2023;142(Supplement 1):1029–1029. doi:10.1182/blood-2023-177693.
  • Roloff G, Faramand R, Aldoss I, Kopmar NE, Schwartz M, Dekker SE, Lin C, Tracy S, Mercadal Vilchez S, Dykes K, et al. Outcomes following brexucabtagene autoleucel administered as an FDA-approved therapy for adults with relapsed/refractory B-all. J Clin Oncol. 2023;41(16_suppl):7001–7001. doi:10.1200/jco.2023.41.16_suppl.7001.
  • Kurz KS, Ott M, Kalmbach S, Steinlein S, Kalla C, Horn H, Ott G, Staiger AM. Large B-Cell lymphomas in the 5th edition of the WHO-Classification of haematolymphoid neoplasms—updated classification and new concepts. Cancers (Basel). 2023 [accessed 2023 Apr 13];15(8):2285. doi:10.3390/cancers15082285.
  • Li S, Young KH, Medeiros LJ. Diffuse large B-cell lymphoma. Pathol. 2018;50(1):74–87. doi:10.1016/j.pathol.2017.09.006.
  • National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology (NCCN guidelines®): B-Cell lymphomas. Version 2; 2024 [accessed 2024 June 4]. https://www.nccn.org/professionals/physician_gls/pdf/b-cell.pdf.
  • Neelapu SS, Locke FL, Bartlett NL, Lekakis LJ, Miklos DB, Jacobson CA, Braunschweig I, Oluwole OO, Siddiqi T, Lin Y, et al. Axicabtagene ciloleucel CAR T-Cell therapy in refractory large B-Cell lymphoma. N Engl J Med. 2017;377(26):2531–44. doi:10.1056/NEJMoa1707447.
  • Schuster SJ, Tam CS, Borchmann P, Worel N, McGuirk JP, Holte H, Waller EK, Jaglowski S, Bishop MR, Damon LE, et al. Long-term clinical outcomes of tisagenlecleucel in patients with relapsed or refractory aggressive B-cell lymphomas (JULIET): a multicentre, open-label, single-arm, phase 2 study. Lancet Oncol. 2021;22(10):1403–15. doi:10.1016/S1470-2045(21)00375-2.
  • Abramson JS, Palomba ML, Gordon LI, Lunning MA, Wang M, Arnason J, Mehta A, Purev E, Maloney DG, Andreadis C, et al. Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study. The Lancet. 2020;396(10254):839–52. doi:10.1016/S0140-6736(20)31366-0.
  • Locke FL, Miklos DB, Jacobson CA, Perales M-A, Kersten M-J, Oluwole OO, Ghobadi A, Rapoport AP, McGuirk J, Pagel JM, et al. Axicabtagene ciloleucel as second-line therapy for large B-cell lymphoma. N Engl J Med. 2022;386(7):640–54. doi:10.1056/NEJMoa2116133.
  • Kamdar M, Solomon SR, Arnason J, Johnston PB, Glass B, Bachanova V, Ibrahimi S, Mielke S, Mutsaers P, Hernandez-Ilizaliturri F, et al. Lisocabtagene maraleucel versus standard of care with salvage chemotherapy followed by autologous stem cell transplantation as second-line treatment in patients with relapsed or refractory large B-cell lymphoma (TRANSFORM): results from an interim analysis of an open-label, randomised, phase 3 trial [published correction appears in lancet]. Lancet. 2022 Jul 16;400(10347):2294–308. doi:10.1016/S0140-6736(22)00662-6.
  • Jacobson CA, Munoz J, Sun F, Kanters S, Limbrick-Oldfield EH, Spooner C, Mignone K, Ayuk F, Sanderson R, Whitmore J, et al. Real-world outcomes with chimeric antigen receptor T cell therapies in large B cell lymphoma: a systematic review and meta-analysis. Transpl Cell Ther. 2024;30(1):77.1–5. doi:10.1016/j.jtct.2023.10.017.
  • Jacobson CA, Locke FL, Ma L, Asubonteng J, Hu Z-H, Siddiqi T, Ahmed S, Ghobadi A, Miklos DB, Lin Y, et al. Real-world evidence of axicabtagene ciloleucel for the treatment of large B cell lymphoma in the United States. Transpl Cell Ther. 2022;28(9):581.1–8. doi:10.1016/j.jtct.2022.05.026.
  • Riedell PA, Hwang WT, Nastoupil LJ, Pennisi M, McGuirk JP, Maziarz RT, Bachanova V, Oluwole OO, Brower J, Flores OA, et al. Patterns of use, outcomes, and resource utilization among recipients of commercial axicabtagene ciloleucel and tisagenlecleucel for relapsed/refractory aggressive B cell lymphomas. Transpl Cell Ther. 2022;28(10):669–76. doi:10.1016/j.jtct.2022.07.011.
  • Bachy E, Le Gouill S, Di Blasi R, Sesques P, Manson G, Cartron G, Beauvais D, Roulin L, Gros FX, Rubio MT, et al. A real-world comparison of tisagenlecleucel and axicabtagene ciloleucel CAR T cells in relapsed or refractory diffuse large B cell lymphoma. Nat Med. 2022;28(10):2145–54. doi:10.1038/s41591-022-01969-y.
  • Bethge WA, Martus P, Schmitt M, Holtick U, Subklewe M, von Tresckow B, Ayuk F, Wagner-Drouet EM, Wulf GG, Marks R, et al. GLA/DRST real-world outcome analysis of CAR T-cell therapies for large B-cell lymphoma in Germany. Blood. 2022;140(4):349–58. doi:10.1182/blood.2021015209.
  • Kwon M, Iacoboni G, Reguera JL, Corral LL, Morales RH, Ortiz-Maldonado V, Guerreiro M, Caballero AC, Domínguez MLG, Pina JMS, et al. Axicabtagene ciloleucel compared to tisagenlecleucel for the treatment of aggressive B-cell lymphoma. Haematologica. 2023 [accessed 2023 Jan 1];108(1):110–21. doi:10.3324/haematol.2022.280805.
  • Kuhnl A, Roddie C, Kirkwood AA, Tholouli E, Menne T, Patel A, Besley C, Chaganti S, Sanderson R, O’Reilly M, et al. A national service for delivering CD19 CAR-T in large B-cell lymphoma – the UK real-world experience. Br J Haematol. 2022;198(3):492–502. doi:10.1111/bjh.18209.
  • Hess LM, Chen Y, Abada PB, Konig H, Walgren RA, Rice L. Outcomes among patients with mantle cell lymphoma post-covalent BTK inhibitor therapy in the United States: a real-world electronic medical records study. Adv Hematol. 2022 [accessed 2022 Dec 28];2022:1–8. doi:10.1155/2022/8262787.
  • Wang M, Munoz J, Goy A, Locke FL, Jacobson CA, Hill BT, Timmerman JM, Holmes H, Jaglowski S, Flinn IW, et al. KTE-X19 CAR T-cell therapy in relapsed or refractory mantle-cell lymphoma. N Engl J Med. 2020;382(14):1331–42. doi:10.1056/NEJMoa1914347.
  • Wang M, Munoz J, Goy A, Locke FL, Jacobson CA, Hill BT, Timmerman JM, Holmes H, Jaglowski S, Flinn IW, et al. Three-year follow-up of KTE-X19 in patients with relapsed/refractory mantle cell lymphoma, including high-risk subgroups, in the ZUMA-2 study. J Clin Oncol. 2023;41(3):555–67. doi:10.1200/JCO.21.02370.
  • Wang Y, Jain P, Locke FL, Maurer MJ, Frank MJ, Munoz JL, Dahiya S, Beitinjaneh AM, Jacobs MT, Mcguirk JP, et al. Brexucabtagene autoleucel for relapsed or refractory mantle cell lymphoma in standard-of-care practice: results from the US lymphoma CAR T consortium. J Clin Oncol. 2023;41(14):2594–606. doi:10.1200/JCO.22.01797.
  • Dreyling M, Fowler NH, Dickinson M, Martinez-Lopez J, Kolstad A, Butler J, Ghosh M, Popplewell L, Chavez JC, Bachy E, et al. Durable response after tisagenlecleucel in adults with relapsed/refractory follicular lymphoma: Elara trial update. Blood J. 2024 Jan 9];143(17):1713–25. doi:10.1182/blood.2023021567.
  • Iacoboni G, Rejeski K, Villacampa G, van Doesum JA, Chiappella A, Bonifazi F, Lopez-Corral L, van Aalderen M, Kwon M, Martínez-Cibrian N, et al. Real-world evidence of brexucabtagene autoleucel for the treatment of relapsed or refractory mantle cell lymphoma. Blood Adv. 2022;6(12):3606–10. doi:10.1182/bloodadvances.2021006922.
  • Herbaux C, Bret C, Di Blasi R, Bachy E, Beauvais D, Gat E, Gastinne T, Broussais F, Cartron G, Cuffel A, et al. KTE-X19 in relapsed or refractory mantle-cell lymphoma, a “real-life” study from the descar-T registry and Lysa group. Blood. 2021;138(Supplement 1):743–743. doi:10.1182/blood-2021-148626.
  • O’Reilly MA, Sanderson R, Wilson W, Burns D, Besley C, Creasey T, Uttenthal B, Elliot J, Roddie C, Kuhnl A, et al. Brexucabtagene autoleucel for relapsed/refractory mantle cell lymphoma: real-world outcomes in the United Kingdom. Blood. 2022;140(Supplement 1):7519–21. doi:10.1182/blood-2022-165031.
  • Mohty R, Kharfan-Dabaja MA. CAR T-cell therapy for follicular lymphoma and mantle cell lymphoma. Ther Adv Hematol. 2022;13:13. doi:10.1177/20406207221142133.
  • Fowler NH, Dickinson M, Dreyling M, Martinez-Lopez J, Kolstad A, Butler J, Ghosh M, Popplewell L, Chavez JC, Bachy E, et al. Tisagenlecleucel in adult relapsed or refractory follicular lymphoma: the phase 2 ELARA trial. Nat Med. 2022;28(2):325–32. doi:10.1038/s41591-021-01622-0.
  • Salles G, Schuster SJ, Dreyling M, Fischer L, Kuruvilla J, Patten PEM, von Tresckow B, Smith SM, Jiménez-Ubieto A, Davis KL, et al. Efficacy comparison of tisagenlecleucel vs usual care in patients with relapsed or refractory follicular lymphoma. Blood Adv. 2022;6(22):5835–43. doi:10.1182/bloodadvances.2022008150.
  • Hao Y, Hsu WC, Parzynski CS, Degtyarev E, Hampson LV, Masood A, Wu WH. Effectiveness of tisagenlecleucel versus real-world standard of care in relapsed/refractory follicular lymphoma. J Comp Eff Res. 2023;12(7):e220173. doi:10.57264/cer-2022-0173.
  • Jacobson CA, Chavez JC, Sehgal AR, William BM, Munoz J, Salles G, Munshi PN, Casulo C, Maloney DG, de Vos S, et al. Axicabtagene ciloleucel in relapsed or refractory indolent non-Hodgkin lymphoma (ZUMA-5): a single-arm, multicentre, phase 2 trial. Lancet Oncol. 2022;23(1):91–103. doi:10.1016/S1470-2045(21)00591-X.
  • Neelapu SS, Chavez JC, Sehgal AR, Epperla N, Ulrickson M, Bachy E, Munshi PN, Casulo C, Maloney DG, de Vos S, et al. Three-year follow-up analysis of axicabtagene ciloleucel in relapsed/refractory indolent non-Hodgkin lymphoma (ZUMA-5). Blood. 2024;143(6):496–506. doi:10.1182/blood.2023021243.
  • Ghione P, Palomba ML, Patel AR, Bobillo S, Deighton K, Jacobson CA, Nahas M, Hatswell AJ, Jung AS, Kanters S, et al. Comparative effectiveness of zuma-5 (axi-CEL) vs scholar-5 external control in relapsed/refractory follicular lymphoma. Blood. 2022;140(8):851–60. doi:10.1182/blood.2021014375.
  • Jacobson CA, Hemmer MT, Hu Z-H, Frank MJ, Popplewell L, Ahmed N, Lin Y, Best T, Beygi S, Miao HH, et al. Real-world early outcomes of axicabtagene ciloleucel for relapsed or refractory (R/R) follicular lymphoma (FL). J Clin Oncol. 2023;41(16_suppl):7509–7509. doi:10.1200/jco.2023.41.16_suppl.7509.
  • Ysebaert L, Houot R, Casasnovas O, Sylvain C, Jardin F, Loschi M, Brisou G, Thieblemont C, Bay J-O, Castilla-Llorente C, et al. Real-world experience of car T-cells in patients with relapsed/refractory follicular lymphoma: a descart registry analysis from the lysa. Blood. 2023;142(Supplement 1):296–296. doi:10.1182/blood-2023-184653.
  • Padala SA, Barsouk A, Barsouk A, Rawla P, Vakiti A, Kolhe R, Kota V, Ajebo GH. Epidemiology, staging, and management of multiple myeloma. Med Sci (Basel). 2021 [accessed 2021 Jan 20];9(1):3. doi:10.3390/medsci9010003.
  • Munshi NC, Anderson LD, Shah N, Madduri D, Berdeja J, Lonial S, Raje N, Lin Y, Siegel D, Oriol A, et al. Idecabtagene vicleucel in relapsed and refractory multiple myeloma. N Engl J Med. 2021;384(8):705–16. doi:10.1056/NEJMoa2024850.
  • Anderson LD, Munshi NC, Shah N, Jagannath S, Berdeja JG, Lonial S, Raje NS, Siegel DSD, Lin Y, Oriol A, et al. Idecabtagene vicleucel (IDE-Cel, BB2121), a BCMA-directed car T-cell therapy, in relapsed and refractory multiple myeloma: updated KARMMA results. J Clin Oncol. 2021;39(15_suppl):8016–8016. doi:10.1200/jco.2021.39.15_suppl.8016.
  • Berdeja JG, Madduri D, Usmani SZ, Jakubowiak A, Agha M, Cohen AD, Stewart AK, Hari P, Htut M, Lesokhin A, et al. Ciltacabtagene autoleucel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy in patients with relapsed or refractory multiple myeloma (CARTITUDE-1): a phase 1b/2 open-label study [published correction appears in lancet]. Lancet. 2021 Oct 2;398(10307):314–24. doi:10.1016/S0140-6736(21)00933-8.
  • Bar N, Diels J, van Sanden S, Mendes J, Hernando T, Cost P, Schecter JM, Lendvai N, Patel N, Ishida T, et al. Comparative efficacy of ciltacabtagene autoleucel versus Idecabtagene vicleucel in the treatment of patients with relapsed or refractory multiple myeloma previously treated with 2-4 prior lines of therapy using a matching-adjusted indirect comparison. Blood. 2023;142(Supplement 1):2141. doi:10.1182/blood-2023-182141.
  • Sidana S, Ahmed N, Akhtar OS, Heim M, Brazauskas R, Hansen DK, Ferreri C, Freeman CLL, Afrough A, Anderson LD, et al. Real world outcomes with Idecabtagene vicleucel (IDE-CEL) CAR-T cell therapy for relapsed/refractory multiple myeloma. Blood. 2023;142(Supplement 1):1027–1027. doi:10.1182/blood-2023-181762.
  • Hansen DK, Sidana S, Peres LC, Colin Leitzinger C, Shune L, Shrewsbury A, Gonzalez R, Sborov DW, Wagner C, Dima D, et al. Idecabtagene vicleucel for relapsed/refractory multiple myeloma: real-world experience from the myeloma CAR-T consortium. J Clin Oncol. 2023;41(11):2087–97. doi:10.1200/JCO.22.01365.
  • Dima D, Rashid A, Davis JA, Shune L, Abdallah A-O, Li H, DeJarnette S, Khouri J, Williams L, Hashmi H, et al. Efficacy and safety of idecabtagene vicleucel in patients with relapsed–refractory multiple myeloma not meeting the KarMMa-1 trial eligibility criteria: a real-world multicentre study. Br J Haematol. 2024;204(4):1293–9. doi:10.1111/bjh.19302.
  • Rodriguez-Otero P, Ailawadhi S, Arnulf B, Patel K, Cavo M, Nooka AK, Manier S, Callander N, Costa LJ, Vij R, et al. Ide-cel or standard regimens in relapsed and refractory multiple myeloma. N Engl J Med. 2023;388(11):1002–14. doi:10.1056/NEJMoa2213614.
  • San-Miguel J, Dhakal B, Yong K, Spencer A, Anguille S, Mateos M-V, Fernández de Larrea C, Martínez-López J, Moreau P, Touzeau C, et al. Cilta-cel or standard care in lenalidomide-refractory multiple myeloma. N Engl J Med. 2023;389(4):335–47. doi:10.1056/NEJMoa2303379.
  • National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology (NCCN guidelines®): multiple myeloma. Version 4. 2024 [accessed 2024 June 4]. https://www.nccn.org/professionals/physician_gls/pdf/myeloma.pdf.
  • Zhang X, Zhang H, Lan H, Wu J, Xiao Y. CAR-T cell therapy in multiple myeloma: current limitations and potential strategies. Front Immunol. 2023;14:1101495. doi:10.3389/fimmu.2023.1101495.
  • Mukkamalla SKR, Taneja A, Malipeddi D, Master SR. Chronic lymphocytic leukemia. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Mar 7. https://www.ncbi.nlm.nih.gov/books/NBK470433/.
  • Siddiqi T, Maloney DG, Kenderian SS, Brander DM, Dorritie K, Soumerai J, Riedell PA, Shah NN, Nath R, Fakhri B, et al. Lisocabtagene maraleucel in chronic lymphocytic leukaemia and small lymphocytic lymphoma (TRANSCEND CLL 004): a multicentre, open-label, single-arm, phase 1–2 study. The Lancet. 2023;402(10402):641–54. doi:10.1016/S0140-6736(23)01052-8.
  • U.S. Food and Drug Administration. Commissioner O of the FDA roundup; 2024 Jan 23 [accessed 2024 Apr 10]. https://www.fda.gov/news-events/press-announcements/fda-roundup-january-23-2024.
  • Verdun N, Marks P. Secondary cancers after chimeric antigen receptor t-cell therapy. N Engl J Med. 2024;390(7):584–6. doi:10.1056/NEJMp2400209.