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

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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Publication Details

Journal
Blood Advances
Published
2025-06-24
DOI
https://doi.org/10.1182/bloodadvances.2025015881
Citations
9
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
9.41

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article

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

Sarah Nikiforow, Michael A. Pulsipher, Erin Hall, Jennifer Willert et al.
9 citations
Blood Advances
CAR-T cell therapy research
9.41
article

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

Sarah Nikiforow, Michael A. Pulsipher, Erin Hall, Jennifer Willert, Rayne H. Rouce, Kevin O. McNerney, Emily M. Hsieh, Kevin J. Curran, Ranjan Tiwari, Amy K. Keating, Erin Guest, Samuel John, Susanne H.C. Baumeister, Joerg Krueger, Stephan A. Grupp, Marcelo C. Pasquini, Amy Moskop, Christine L. Phillips, Roberto Ramos, Timothy Driscoll
article en
9 citations

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.

Blood AdvancesVol. 9(20)
Cincinnati Children's Hospital Medical Center (US), Boston Children's Hospital (US), Primary Children's Hospital (US), Children's Mercy Hospital (US), Memorial Sloan Kettering Cancer Center (US), Children's Hospital of Philadelphia (US), Children's Hospital of Los Angeles (US), Harvard University (US), Duke University (US), Medical College of Wisconsin (US), Huntsman Cancer Institute (US), Hospital for Sick Children (CA), Novartis (India) (IN), Children's Hospital Colorado (US), Southwestern Medical Center (US), Versiti Blood Center of Wisconsin (US), Dana-Farber Cancer Institute (US), Novartis (United States) (US), Lurie Children's Hospital (US), Duke Medical Center (US), Texas Children's Hospital (US), Boston Children's Museum (US), Southwestern Medical Center (US), University of Missouri–Kansas City (US), University of Pennsylvania (US), The University of Texas Southwestern Medical Center (US), University of Colorado Denver (US)
U.S. Department of Defense, Gateway for Cancer Research, Amgen, Bristol-Myers Squibb, Pfizer, Astellas Pharma US, AstraZeneca, GlaxoSmithKline, Sanofi, Gilead Sciences, HistoGenetics, University of Pittsburgh, Novartis Pharmaceuticals Corporation, CSL Behring, StemCyte, Actinium Pharmaceuticals, Vertex Pharmaceuticals, Jazz Pharmaceuticals, Mallinckrodt Pharmaceuticals, Atara Biotherapeutics, CareDx, bluebird bio, Adaptive Biotechnologies, BeiGene, Incyte, Syndax Pharmaceuticals, Omeros Corporation, Kiadis Pharma, Astellas Pharma, Servier, Swedish Orphan Biovitrum, MorphoSys, National Institutes of Health, Health Resources and Services Administration, U.S. Navy, Takeda Oncology, Kite Pharma, Pharmacyclics, Legend Biotech, National Heart, Lung, and Blood Institute, Office of Naval Research, National Cancer Institute, National Institute of Allergy and Infectious Diseases, Moonshot Research and Development Program, Cancer Moonshot
Openalex Percentile: Top 2%
CAR-T cell therapy research
9.41
DOIOpen Access PDFOpenAlex
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