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. 2025 Oct 28;9(20):5249-5262.
doi: 10.1182/bloodadvances.2025015881.

Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry

Samuel John  1 , Kevin J Curran  2 , Erin M Hall  3   4 , Amy K Keating  5 , Susanne H C Baumeister  6   7   8 , Sarah Nikiforow  9 , Timothy Driscoll  10 , Amy Moskop  11   12 , Kevin O McNerney  13 , Christine L Phillips  14 , Michael A Pulsipher  15 , Emily M Hsieh  16 , Rayne H Rouce  17 , Marcelo C Pasquini  11 , Ranjan Tiwari  18 , Jennifer Willert  19 , Roberto Ramos  19 , Joerg Krueger  20 , Erin Guest  3 , Stephan A Grupp  21   22
Affiliations

Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry

Samuel John et al. Blood Adv. .

Abstract

Since the first approval of tisagenlecleucel in 2017, pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) may receive this CD19-directed chimeric antigen receptor T-cell therapy. We report real-world data from the Center for International Blood and Marrow Transplant Research (>2.5 years of follow-up). As of 4 May 2022, 768 patients with B-ALL had received tisagenlecleucel. Patients aged ≥18 and <18 years of age (including those <3 years) were treated during first relapse (26.6% and 26.7% [<3 years, 44.8%], respectively) or primary refractory disease (12.4% and 12.1% [<3 years, 15.5%], respectively) with 17.6% and 11.6% (<3 years, 13.8%), respectively, having high disease burden (≥50% bone marrow [BM] blasts) and 20.2% and 20.2% (<3 years, 13.8%), respectively, having low disease burden (>0 to <5% BM blasts). Among patients with ≥12 months postinfusion follow-up (n = 578; median follow-up, 32.1 months), the best overall response of complete remission/complete remission with incomplete blood count recovery was 86.0%. The 12-month relapse-free survival (RFS) and overall survival (OS) were 61.8% and 79.4%, respectively, whereas the 24-month RFS and OS were 50.3% and 63.8%, respectively. Age (<18 years) and disease burden (<50% BM blasts) were associated with better outcomes. Previous inotuzumab therapy and KMT2A rearrangement were associated with worse outcomes. Older patients (≥18 years) experienced a higher rate of any-grade cytokine release syndrome (CRS) associated with higher disease burden before infusion. Any-grade CRS and neurotoxicity were lower in patients aged <3 years. Extended follow-up continues to demonstrate high rates of RFS and favorable safety in this population.

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Conflict of interest statement

Conflict of interest disclosure: K.J.C. reports serving as a consultant for Novartis; participating as board member and shareholder for Turn Bio and PromiCell; and research funding from Novartis, Cellectis, and Atara Bio. E.M.H.’s spouse reports participating in a speaker's bureau for Boehringer Ingelheim; honoraria from Vida Diagnostics; and participating on a scientific advisory committee for Polarean. S.H.C.B.’s spouse reports former employment for Alexion and Takeda Pharmaceuticals and holds Takeda and AstraZeneca stock and stock options. S.N. reports participating in ad hoc advisory boards for A2 Bio, Legend Biotech, Kite/Gilead, Iovance, SmartImmune, and GlaxoSmithKline. C.L.P. reports serving as a member on the board of directors or advisory committees for Novartis. M.A.P. reports participating in scientific advisory boards for Medexus, Equillium, Mesoblast, and Novartis; participating in sponsored invited educational talks for Novartis and Miltenyi Biotec; and study support from Miltenyi Biotec and Adaptive Biotechnologies. R.H.R. reports honoraria from Novartis; research funding from Tessa Therapeutics; and serving as a consultant for Pfizer. M.C.P. reports research funding from Bristol Myers Squibb, Novartis, Kite Pharma, and GlaxoSmithKline; and serving as a consultant for Bristol Myers Squibb. R.T., J.W., and R.R. report current employment with Novartis. J.K. reports serving as a consultant for Kite/Gilead and Novartis. E.G. reports participating in scientific advisory boards for Syndax and Jazz Pharmaceuticals. S.A.G. reports research and/or clinical trial support from Novartis, Servier, Vertex, and Kite Pharma; and participating in study steering committees or serving as a consultant or on scientific advisory boards for Novartis, Allogene, Cellectis, CRISPR/Vertex, Jazz Pharmaceuticals, TCR2, Allogene, BioLineRx, Mckesson, and Cabaletta. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
RFS and OS Kaplan-Meier curves across the age categories. (A) RFS∗. (B) OS. ∗Censored for allo-SCT. P values represent comparisons between patients aged <18 and ≥18 years. NE, not estimable.
Figure 2.
Figure 2.
Forest plots for OS and RFS by multivariate analysis without censoring for allo-SCT. Covariates with multivariate P value of < .05 are shown in red (dotted line). HCT, hematopoietic stem cell transplant; Ph+, Philadelphia chromosome positive.
See this image and copyright information in PMC

References

    1. Schwab C, Harrison CJ. Advances in B-cell precursor acute lymphoblastic leukemia genomics. HemaSphere. 2018;2(4) - PMC - PubMed
    1. Siegel RL, Miller KD, Jemal A. Cancer statistics, 2020. CA Cancer J Clin. 2020;70(1):7–30. - PubMed
    1. Inaba H, Greaves M, Mullighan CG. Acute lymphoblastic leukaemia. Lancet. 2013;381(9881):1943–1955. - PMC - PubMed
    1. Locatelli F, Schrappe M, Bernardo ME, Rutella S. How I treat relapsed childhood acute lymphoblastic leukemia. Blood. 2012;120(14):2807–2816. - PubMed
    1. Oskarsson T, Söderhäll S, Arvidson J, et al. Relapsed childhood acute lymphoblastic leukemia in the Nordic countries: prognostic factors, treatment and outcome. Haematologica. 2016;101(1):68–76. - PMC - PubMed

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