Evidence map›Paper›PMID 40554426›Full record

ArticleBlood advances2025

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

Samuel John, Kevin J Curran, Erin M Hall, Amy K Keating, Susanne H C Baumeister, Sarah Nikiforow, Timothy Driscoll, Amy Moskop, Kevin O McNerney, Christine L Phillips and 10 more

Abstract read
In one paragraph

Article in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

21 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Advances in targeted and cellular therapies for relapsed/refractory mantle cell lymphoma: immunotherapeutic strategies and challenges.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2026
    Review
  6. Review
  7. Article
  8. Review
  9. Review
  10. Review
  11. Article
  12. Review
  13. Frontiers in immunology · 2026
    Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. With BiTEs at the kiddie table, where do CARs come in for pediatric B-ALL?Hematology. American Society of Hematology. Education Program · 2025
    Review
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

20 authors.

Samuel JohnDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX.ORCID 0000-0002-4167-2466
Kevin J CurranDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.ORCID 0000-0002-3337-9956
Erin M HallDivision of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Children's Mercy Kansas City, Kansas City, MO.
Amy K KeatingDepartment of Pediatrics, University of Colorado School of Medicine and Children's Hospital of Colorado, Aurora, CO.ORCID 0000-0002-4187-8456
Susanne H C BaumeisterDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA.ORCID 0000-0003-3846-5156
Sarah NikiforowDepartment of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.ORCID 0009-0001-2711-5575
Timothy DriscollDepartment of Pediatrics, Pediatric Transplant and Cellular Therapy, Duke University, Durham, NC.
Amy MoskopCenter for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0002-5110-004X
Kevin O McNerneyDivision of Hematology, Oncology, Neuro-oncology, and Stem Cell Transplantation, Lurie Children's Hospital, Chicago, IL.ORCID 0000-0002-4359-3569
Christine L PhillipsDepartment of Pediatrics, Cincinnati Children''s Hospital Medical Center, University of Cincinnati, Cincinnati, OH.
Michael A PulsipherDivision of Hematology and Oncology, Intermountain Primary Children's Hospital, Huntsman Cancer Institute, Spencer Fox Eccles School of Medicine at The University of Utah, Salt Lake City, UT.ORCID 0000-0003-3030-8420
Emily M HsiehDivision of Hematology/Oncology, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA.ORCID 0000-0001-5088-7621
Rayne H RouceDepartment of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
Marcelo C PasquiniCenter for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI.ORCID 0000-0003-1579-2293
Ranjan TiwariNovartis Healthcare Pvt Ltd, Hyderabad, India.
Jennifer WillertNovartis Pharmaceuticals Corporation, East Hanover, NJ.
Roberto RamosNovartis Pharmaceuticals Corporation, East Hanover, NJ.
Joerg KruegerDivision of Haematology/Oncology/Bone Marrow Transplantation and Cellular Therapy, Department of Paediatrics, The Hospital for Sick Children, Toronto, ON, Canada.
Erin GuestDivision of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Children's Mercy Kansas City, Kansas City, MO.ORCID 0000-0003-2482-5608
Stephan A GruppDivision of Oncology, Cell Therapy and Transplant Section, Children's Hospital of Philadelphia, Philadelphia, PA.ORCID 0000-0001-8030-7595

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Consortium for Advancing Management and Prevention of Cancer in People with HIVUM1CA121947 · NCI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Joseph A. Sparano · 2015 to 2026
$253.7M
Data Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2M
Blood and Marrow Transplant Clinical Trials Network DCC- The Medical College of Wisconsin, Inc.U24HL138660 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI Steven M. DeVine, Mehdi Hussain Hamadani · 2017 to 2026
$71.4M
Therapeutic Potential of Adaptive NK Cells in Cancer and TransplantationP01CA111412 · NCI · UNIVERSITY OF MINNESOTA TWIN CITIES · PI FELICES, MARTIN · 2005 to 2025
$38.2M
Cure Sickle CellOT3HL147741 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI MARY EAPEN, Mary Maresca Horowitz · 2018 to 2026
$37.7M
Hematopopietic Stem Cell TransplantationU01AI069197 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Effie W Petersdorf · 2005 to 2026
$18.9M
Clinical Significance of MHC Haplotypes in Hematopoietic Cell TransplantationR01CA100019 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI PETERSDORF, EFFIE W · 2003 to 2025
$9.1M
Immuno-Oncology Translation Network (IOTN): Cellular Immunotherapy Data Resource (CIDR)U24CA233032 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI PASQUINI, MARCELO C. · 2018 to 2019
$9.0M
Randomized study of low versus moderate dose busulfan in transplant for severe combined immunodeficiencyU01AI126612 · NIAID · CHILDREN'S HOSPITAL OF LOS ANGELES · PI KEAN, LESLIE S, PULSIPHER, MICHAEL A · 2017 to 2023
$8.5M
Immuno and Epigenetics of Hematopoietic Cell TransplantationR01CA218285 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Effie W Petersdorf · 2017 to 2026
$5.6M
1/2A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic AnemiaU24HL157560 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI SHAW, BRONWEN · 2022 to 2025
$5.0M
CLC NIH HHS C0000008NCI NIH HHS P01 CA111412NCI NIH HHS P30 CA008748NCI NIH HHS R01 CA100019NCI NIH HHS R01 CA218285NCI NIH HHS R01 CA231141NCI NIH HHS R01 CA231838NCI NIH HHS R01 CA262899NCI NIH HHS U24 CA076518NCI NIH HHS U24 CA233032NCI NIH HHS UM1 CA121947NHLBI NIH HHS OT3 HL147741NHLBI NIH HHS R01 HL155741NHLBI NIH HHS U24 HL138660NHLBI NIH HHS U24 HL157560NHLBI NIH HHS UG1 HL069254NIAID NIH HHS R01 AI128775NIAID NIH HHS R01 AI150999NIAID NIH HHS R01 AI158861NIAID NIH HHS U01 AI069197NIAID NIH HHS U01 AI126612NIEHS NIH HHS 27305C0011NIEHS NIH HHS 27307C0009NIEHS NIH HHS 27307C0011NIEHS NIH HHS 27398C0011
6 · The paper itself

Abstract

abstractSince 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.

Indexed as

Immunotherapy, AdoptivePrecursor B-Cell Lymphoblastic Leukemia-LymphomaReceptors, Antigen, T-CellAdolescentAdultChildChild, PreschoolFemaleHumansInfantMaleMiddle AgedRegistriesTreatment OutcomeYoung AdultReceptors, Antigen, T-Celltisagenlecleucel

Identifiers

PMID40554426
PMCPMC12552949

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.