2,921
Views
1
CrossRef citations to date
0
Altmetric
Original Articles

Indirect treatment comparison of brexucabtagene autoleucel (ZUMA-2) versus standard of care (SCHOLAR-2) in relapsed/refractory mantle cell lymphoma

, , , , , , , , ORCID Icon, , , , , , , , , , ORCID Icon, , , , , & show all
Pages 14-25 | Received 21 Jul 2023, Accepted 02 Oct 2023, Published online: 16 Oct 2023

References

  • Owen C, Berinstein NL, Christofides A, et al. Review of Bruton tyrosine kinase inhibitors for the treatment of relapsed or refractory mantle cell lymphoma. Curr Oncol. 2019;26(2):e233–e240. doi:10.3747/co.26.4345
  • Wang M, Schuster SJ, Phillips T, et al. Observational study of lenalidomide in patients with mantle cell lymphoma who relapsed/progressed after or were refractory/intolerant to ibrutinib (MCL-004). J Hematol Oncol. 2017;10(1):171. doi:10.1186/s13045-017-0537-5
  • McKay P, Leach M, Jackson B, et al. Guideline for the management of mantle cell lymphoma. Br J Haematol. 2018;182(1):46–62. doi:10.1111/bjh.15283
  • Dreyling M, Jurczak W, Jerkeman M, et al. Ibrutinib versus temsirolimus in patients with relapsed or refractory mantle-cell lymphoma: an international, randomised, open-label, phase 3 study. Lancet. 2016;387(10020):770–778. doi:10.1016/S0140-6736(15)00667-4
  • Wang M, Munoz J, Goy A, et al. KTE-X19 CAR T-cell therapy in relapsed or refractory mantle-cell lymphoma. N Engl J Med. 2020;382(14):1331–1342. doi:10.1056/NEJMoa1914347
  • Eyre TA, Walter HS, Iyengar S, et al. Efficacy of venetoclax monotherapy in patients with relapsed, refractory mantle cell lymphoma after Bruton tyrosine kinase inhibitor therapy. Haematologica. 2019;104(2):e68–e71. doi:10.3324/haematol.2018.198812
  • McCulloch R, Visco C, Eyre TA, et al. Efficacy of R-BAC in relapsed, refractory mantle cell lymphoma post BTK inhibitor therapy. Br J Haematol. 2020;189(4):684–688. doi:10.1111/bjh.16416
  • Jain P, Kanagal-Shamanna R, Zhang S, et al. Long-term outcomes and mutation profiling of patients with ­mantle cell lymphoma (MCL) who discontinued ibrutinib. Br J Haematol. 2018;183(4):578–587. doi:10.1111/bjh.15567
  • Martin P, Maddocks K, Leonard JP, et al. Postibrutinib outcomes in patients with mantle cell lymphoma. Blood. 2016;127(12):1559–1563. doi:10.1182/blood-2015-10-673145
  • Hess G, Dreyling M, Oberic L, et al. Real‐world experience among patients with relapsed/refractory mantle cell lymphoma after Bruton tyrosine kinase inhibitor failure in Europe: the SCHOLAR‐2 retrospective chart review study. Br J Haematol. 2023;202(4):749–759. doi:10.1111/bjh.18519
  • Patel D, Grimson F, Mihaylova E, et al. Use of external comparators for health technology assessment submissions based on Single-Arm trials. Value Health. 2021;24(8):1118–1125. doi:10.1016/j.jval.2021.01.015
  • Wang M, Munoz J, Goy A, 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. 2022;2022:JCO2102370.
  • Hernán MA, Sauer BC, Hernández-Díaz S, et al. Specifying a target trial prevents immortal time bias and other self-inflicted injuries in observational analyses. J Clin Epidemiol. 2016;79:70–75. doi:10.1016/j.jclinepi.2016.04.014
  • Faria R, Hernandez Alava M, Manca A, et al. NICE DSU Technical Support Document 17: the use of observational data to inform estimates of treatment effectiveness for technology appraisal: methods for comparative individual patient data; 2015.
  • Phillippo DM, Ades AE, Dias S, et al. NICE DSU technical support document 18: methods for population-adjusted indirect comparisons in submission to NICE. 2016.
  • Austin PC. An introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivariate Behav Res. 2011;46(3):399–424. doi:10.1080/00273171.2011.568786
  • Desai RJ, Franklin JM. Alternative approaches for confounding adjustment in observational studies using weighting based on the propensity score: a primer for practitioners. BMJ. 2019;367:l5657. doi:10.1136/bmj.l5657
  • Austin PC, Stuart EA. Moving towards best practice when using inverse probability of treatment weighting (IPTW) using the propensity score to estimate causal treatment effects in observational studies. Stat Med. 2015;34(28):3661–3679. doi:10.1002/sim.6607
  • Austin PC. Balance diagnostics for comparing the distribution of baseline covariates between treatment groups in propensity-score matched samples. Stat Med. 2009;28(25):3083–3107. doi:10.1002/sim.3697
  • Sterne JAC, White IR, Carlin JB, et al. Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls. BMJ. 2009;338(1):b2393–b2393. doi:10.1136/bmj.b2393
  • Kang JDY, Schafer JL. Demystifying double robustness: a comparison of alternative strategies for estimating a population mean from incomplete data. Stat Sci. 2007;22(4):523–539.
  • Schemper M, Smith TL. A note on quantifying follow-up in studies of failure time. Control Clin Trials. 1996;17(4):343–346. doi:10.1016/0197-2456(96)00075-x
  • R Core Team (2021) R: A language and environment for statistical computing. R Foundation for Statistical Computing, Vienna, Austria. URL https://www.R-project.org/.
  • Dreyling M, Shah B, Wu JJ, et al. SA29 efficacy outcomes following treatment with bruton tyrosine kinase inhibitors (BTKI) for relapsed/refractory mantle cell lymphoma (R/R MCL): a literature-based meta-analysis. Value in Health. 2022;25(7):S609–S610. doi:10.1016/j.jval.2022.04.1695
  • Phillips TJ, Dickinson M, Morschhauser F, et al. Glofitamab monotherapy induces high complete response rates in patients with heavily pretreated relapsed or refractory mantle cell lymphoma. Blood. 2022;140(Supplement 1):178–180. doi:10.1182/blood-2022-157777
  • Wang ML, Jurczak W, Zinzani PL, et al. Pirtobrutinib in covalent BTK-Inhibitor pre-treated mantle cell lymphoma. J Clin Oncol. 2023;2023:101200JCO2300562.
  • Kambhampati S, Ahmed N, Hamadani M, et al. Real-world outcomes of brexucabtagene autoleucel (brexu-cel) for relapsed or refractory (R/R) mantle cell lymphoma (MCL): a CIBMTR subgroup analysis by prior treatment. J Clin Oncol. 2023;41(16_suppl):7507–7507. doi:10.1200/JCO.2023.41.16_suppl.7507
  • Downing NS, Aminawung JA, Shah ND, et al. Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005–2012. JAMA. 2014;311(4):368–377. doi:10.1001/jama.2013.282034
  • Tenhunen O, Lasch F, Schiel A, et al. Single‐arm clinical trials as pivotal evidence for cancer drug approval: a retrospective cohort study of centralized European marketing authorizations between 2010 and 2019. Clin Pharmacol Ther. 2020;108(3):653–660. doi:10.1002/cpt.1965
  • Goring S, Taylor A, Müller K, et al. Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review. BMJ Open. 2019;9(2):e024895. doi:10.1136/bmjopen-2018-024895
  • Hatswell AJ, Baio G, Berlin JA, et al. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014. BMJ Open. 2016;6(6):e011666. doi:10.1136/bmjopen-2016-011666
  • Purpura CA, Garry EM, Honig N, et al. The role of real-world evidence in FDA-approved new drug and biologics license applications. Clin Pharmacol Ther. 2022;111(1):135–144. doi:10.1002/cpt.2474
  • Ghione P, Palomba ML, Patel AR, et al. Comparative effectiveness of ZUMA-5 (axi-cel) vs SCHOLAR-5 external control in relapsed/refractory follicular lymphoma. Blood. 2022;140(8):851–860. doi:10.1182/blood.2021014375
  • Neelapu SS, Locke FL, Bartlett NL, et al. Comparison of 2-year outcomes with CAR T cells (ZUMA-1) vs salvage chemotherapy in refractory large B-cell lymphoma. Blood Adv. 2021;5(20):4149–4155. doi:10.1182/bloodadvances.2020003848
  • Salles G, Schuster SJ, Dreyling M, et al. Efficacy comparison of tisagenlecleucel vs usual care in patients with relapsed or refractory follicular lymphoma. Blood Adv. 2022;6(22):5835–5843. doi:10.1182/bloodadvances.2022008150
  • Remiro-Azócar A, Heath A, Baio G. Parametric G-computation for compatible indirect treatment comparisons with limited individual patient data. Res Synth Methods. 2022;13(6):716–744. doi:10.1002/jrsm.1565
  • Daniel R, Zhang J, Farewell D. Making apples from oranges: comparing noncollapsible effect estimators and their standard errors after adjustment for different covariate sets. Biom J. 2021;63(3):528–557. doi:10.1002/bimj.201900297