{
  "abstract": "We report the 5-year analysis of tisagenlecleucel in 79 pediatric and young adult patients with relapsed or refractory (r/r) B-cell ALL (B-ALL) from the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), with a median follow-up of 79.4 months. Tisagenlecleucel was administered as a single infusion with dosing normalized by weight in patients ≤50 kg. Key long-term end points included relapse-free survival (RFS), overall survival (OS), and safety. Censoring included loss to follow-up, withdrawal of consent, new anticancer therapy (± stem cell transplantation [SCT]), and death. The estimated 5-year RFS among responders (n = 70) with and without inclusion of SCT in censoring for new anticancer therapies was 47.3% and 51.0%, respectively. The median time to B-cell recovery was not reached with censoring for all further anticancer therapies, including SCT. The median OS was not reached. The estimated OS at 5 years was 55.0% and 62.4% with and without inclusion of SCT in censoring for new anticancer therapies, respectively. In total, 17 responders received a postinfusion SCT, 14 while still in complete remission. No new or unexpected adverse events were reported. These findings continue to support the potential of tisagenlecleucel as definitive therapy for many heavily pretreated pediatric and young adult patients with r/r B-ALL.",
  "abstract_shown": true,
  "affiliations": [
    "Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA",
    "Pediatric Cancer Center Barcelona (PCCB), Hospital Sant Joan de Déu de Barcelona, Institut de Recerca Sant Joan de Déu, Leukemia, Barcelona, Spain",
    "Pediatric Hematology Disorders, Developmental Tumors Biology Group, Barcelona, Spain",
    "Instituto de Salud Carlos III, Centro de Investigación Biomédica en Red De Enfermedades Raras (CIBERER), Madrid, Spain",
    "Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan",
    "University Hospital Robert Debré (APHP) and Université Paris Cité, Paris, France",
    "Division of Stem Cell Transplantation and Immunology, Department of Pediatrics, Goethe University Frankfurt, Frankfurt, Germany",
    "Department of Pediatrics, Hematology-Oncology Division, CHU Sainte-Justine, Montreal, QC, Canada",
    "Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway",
    "Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium",
    "Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University, Atlanta, GA",
    "Center for International Blood and Marrow Transplant Research, NMDP, Minneapolis, MN",
    "Department of Pediatrics, Hematology, Oncology, Stem Cell Transplant and Regenerative Medicine Stanford University, Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA",
    "Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC",
    "Center for Hematological Malignancies, Knight Cancer Institute, Oregon Health & Science University, Portland, OR",
    "St Anna Children's Hospital, St Anna Children's Cancer Research Institute, Vienna, Austria",
    "Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI",
    "Hematopoietic Stem Cell Transplantation Unit, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy",
    "School of Medicine and Surgery, Milano-Bicocca University, Monza, Italy",
    "Service d'hématologie Adolescents et Jeunes Adultes, Paris Cité University, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France",
    "Children's Cancer Centre, The Royal Children's Hospital Melbourne and Murdoch Children's Research Institute, Parkville, VIC, Australia",
    "Division of Haematology/Oncology, Blood and Marrow Transplant/Cellular Therapy, The Hospital for Sick Children, Toronto, ON, Canada",
    "BMT Clinical Research, The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY",
    "Division of Hematology/Oncology/Bone Marrow Transplant, Children's Mercy Kansas City, Kansas City, MO",
    "Novartis Healthcare Private Limited, Hyderabad, India",
    "Novartis Pharmaceuticals Corporation, Dublin, Ireland",
    "Novartis Pharmaceuticals Corporation, East Hanover, NJ",
    "Novartis Pharmaceuticals Corporation, San Franscisco, CA",
    "Division of Pediatric 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"
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 29,
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        0
      ],
      "name": "Stephan A. Grupp",
      "orcid": "0000-0001-8030-7595"
    },
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        0
      ],
      "name": "Shannon L. Maude",
      "orcid": "0000-0003-2210-8736"
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        2,
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      "name": "Susana Rives",
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      "name": "Hidefumi Hiramatsu",
      "orcid": "0000-0003-3136-5670"
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      "name": "Andre Baruchel",
      "orcid": "0000-0003-0120-6089"
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      "name": "Peter Bader"
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      "name": "Henrique Bittencourt",
      "orcid": "0000-0002-5507-8310"
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      "name": "Jochen Buechner",
      "orcid": "0000-0001-5848-4501"
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      ],
      "name": "Theodore W. Laetsch",
      "orcid": "0000-0002-8497-3138"
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      "name": "Barbara De Moerloose",
      "orcid": "0000-0002-2449-539X"
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      "name": "Muna Qayed",
      "orcid": "0000-0001-7689-343X"
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        11
      ],
      "name": "Heather E. Stefanski"
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        12
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      "name": "Kara L. Davis",
      "orcid": "0000-0002-7182-2592"
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        13
      ],
      "name": "Timothy A. Driscoll",
      "orcid": "0000-0002-3053-8683"
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    {
      "affs": [
        14
      ],
      "name": "Eneida Nemecek"
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    {
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        15
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      "name": "Christina Peters",
      "orcid": "0000-0003-0369-8515"
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      "name": "Gregory Yanik"
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        18
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      "name": "Adriana Balduzzi",
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      "name": "Nicolas Boissel",
      "orcid": "0000-0003-2091-7927"
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    {
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        20
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      "name": "Seong Lin Khaw",
      "orcid": "0000-0002-9084-8974"
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      "name": "Joerg Krueger",
      "orcid": "0000-0003-1225-1977"
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      "name": "John E. Levine",
      "orcid": "0000-0002-5611-7828"
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      "name": "Gary Douglas Myers"
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      "name": "Ranjan Tiwari"
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      "name": "Darragh O'Donovan"
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      "name": "Rakesh Awasthi"
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      "name": "Roberto Ramos"
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      "name": "Jennifer Willert"
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    {
      "affs": [
        28
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      "name": "Michael A. Pulsipher",
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  "bibtex": "https://research.draconitic.com/paper/fphf0j0hg4hde.bib",
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  "doi": "10.1200/jco-25-01471",
  "fulltext_url": "https://pmc.ncbi.nlm.nih.gov/articles/PMC13718899/",
  "id": "fphf0j0hg4hde",
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    "0732-183X",
    "1527-7755"
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  "landing_url": "https://doi.org/10.1200/jco-25-01471",
  "language": "en",
  "licence": "CC-BY-NC-ND-4.0",
  "licence_label": "CC BY-NC-ND 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
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  "native_subjects": [
    [
      "heading",
      "CLINICAL TRIAL UPDATES"
    ],
    [
      "heading",
      "Pediatric Oncology"
    ],
    [
      "openalex",
      "2730|CAR-T cell therapy research"
    ]
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  "openalex": "W7171707931",
  "pdf_url": "https://ascopubs.org/doi/pdfdirect/10.1200/JCO-25-01471",
  "pmcid": "PMC13718899",
  "pmid": "42525896",
  "published": "2026-07-29",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/fphf0j0hg4hde.ris",
  "sources": [
    {
      "first_seen": "2026-11-02T12:22:50Z",
      "native_id": "PMC13718899",
      "source": "pmc",
      "url": "https://doi.org/10.1200/jco-25-01471"
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  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2730|CAR-T cell therapy research",
      "subfield": "oncology"
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  ],
  "title": "Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL",
  "title_text": "Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL",
  "type": "peer-reviewed",
  "updated_at": "2026-11-02T12:22:50Z",
  "url": "https://research.draconitic.com/paper/fphf0j0hg4hde",
  "venue": "Journal of Clinical Oncology",
  "versions": []
}