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Peer-reviewedCC BY-NC-ND 4.0Journal of Clinical Oncology

Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL

  • Stephan A. Grupp1
  • Shannon L. Maude1
  • Susana Rives2,3,4
  • Hidefumi Hiramatsu5
  • Andre Baruchel6
  • Peter Bader7
  • Henrique Bittencourt8
  • Jochen Buechner9
  • Theodore W. Laetsch1
  • Barbara De Moerloose10
  • Muna Qayed11
  • Heather E. Stefanski12
17 more authors
  • Kara L. Davis13
  • Timothy A. Driscoll14
  • Eneida Nemecek15
  • Christina Peters16
  • Gregory Yanik17
  • Adriana Balduzzi18,19
  • Nicolas Boissel20
  • Seong Lin Khaw21
  • Joerg Krueger22
  • John E. Levine23
  • Gary Douglas Myers24
  • Ranjan Tiwari25
  • Darragh O'Donovan26
  • Rakesh Awasthi27
  • Roberto Ramos27
  • Jennifer Willert28
  • Michael A. Pulsipher29
29 affiliations
  1. 1Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
  2. 2Pediatric Cancer Center Barcelona (PCCB), Hospital Sant Joan de Déu de Barcelona, Institut de Recerca Sant Joan de Déu, Leukemia, Barcelona, Spain
  3. 3Pediatric Hematology Disorders, Developmental Tumors Biology Group, Barcelona, Spain
  4. 4Instituto de Salud Carlos III, Centro de Investigación Biomédica en Red De Enfermedades Raras (CIBERER), Madrid, Spain
  5. 5Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan
  6. 6University Hospital Robert Debré (APHP) and Université Paris Cité, Paris, France
  7. 7Division of Stem Cell Transplantation and Immunology, Department of Pediatrics, Goethe University Frankfurt, Frankfurt, Germany
  8. 8Department of Pediatrics, Hematology-Oncology Division, CHU Sainte-Justine, Montreal, QC, Canada
  9. 9Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway
  10. 10Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium
  11. 11Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University, Atlanta, GA
  12. 12Center for International Blood and Marrow Transplant Research, NMDP, Minneapolis, MN
  13. 13Department 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
  14. 14Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC
  15. 15Center for Hematological Malignancies, Knight Cancer Institute, Oregon Health & Science University, Portland, OR
  16. 16St Anna Children's Hospital, St Anna Children's Cancer Research Institute, Vienna, Austria
  17. 17Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI
  18. 18Hematopoietic Stem Cell Transplantation Unit, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
  19. 19School of Medicine and Surgery, Milano-Bicocca University, Monza, Italy
  20. 20Service d'hématologie Adolescents et Jeunes Adultes, Paris Cité University, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France
  21. 21Children's Cancer Centre, The Royal Children's Hospital Melbourne and Murdoch Children's Research Institute, Parkville, VIC, Australia
  22. 22Division of Haematology/Oncology, Blood and Marrow Transplant/Cellular Therapy, The Hospital for Sick Children, Toronto, ON, Canada
  23. 23BMT Clinical Research, The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY
  24. 24Division of Hematology/Oncology/Bone Marrow Transplant, Children's Mercy Kansas City, Kansas City, MO
  25. 25Novartis Healthcare Private Limited, Hyderabad, India
  26. 26Novartis Pharmaceuticals Corporation, Dublin, Ireland
  27. 27Novartis Pharmaceuticals Corporation, East Hanover, NJ
  28. 28Novartis Pharmaceuticals Corporation, San Franscisco, CA
  29. 29Division 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
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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.

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Cite

BibTeXRIS
BibTeX
@article{grupp2026longterm,
  title = {{Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL}},
  author = {Stephan A. Grupp and Shannon L. Maude and Susana Rives and Hidefumi Hiramatsu and Andre Baruchel and Peter Bader and Henrique Bittencourt and Jochen Buechner and Theodore W. Laetsch and Barbara De Moerloose and Muna Qayed and Heather E. Stefanski and Kara L. Davis and Timothy A. Driscoll and Eneida Nemecek and Christina Peters and Gregory Yanik and Adriana Balduzzi and Nicolas Boissel and Seong Lin Khaw and Joerg Krueger and John E. Levine and Gary Douglas Myers and Ranjan Tiwari and Darragh O'Donovan and Rakesh Awasthi and Roberto Ramos and Jennifer Willert and Michael A. Pulsipher},
  journal = {Journal of Clinical Oncology},
  year = {2026},
  month = jul,
  doi = {10.1200/jco-25-01471},
  url = {https://doi.org/10.1200/jco-25-01471},
  note = {CC BY-NC-ND 4.0},
}
RIS
TY  - JOUR
TI  - Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL
AU  - Stephan A. Grupp
AU  - Shannon L. Maude
AU  - Susana Rives
AU  - Hidefumi Hiramatsu
AU  - Andre Baruchel
AU  - Peter Bader
AU  - Henrique Bittencourt
AU  - Jochen Buechner
AU  - Theodore W. Laetsch
AU  - Barbara De Moerloose
AU  - Muna Qayed
AU  - Heather E. Stefanski
AU  - Kara L. Davis
AU  - Timothy A. Driscoll
AU  - Eneida Nemecek
AU  - Christina Peters
AU  - Gregory Yanik
AU  - Adriana Balduzzi
AU  - Nicolas Boissel
AU  - Seong Lin Khaw
AU  - Joerg Krueger
AU  - John E. Levine
AU  - Gary Douglas Myers
AU  - Ranjan Tiwari
AU  - Darragh O'Donovan
AU  - Rakesh Awasthi
AU  - Roberto Ramos
AU  - Jennifer Willert
AU  - Michael A. Pulsipher
PY  - 2026
DA  - 2026/07/29
JO  - Journal of Clinical Oncology
DO  - 10.1200/jco-25-01471
UR  - https://doi.org/10.1200/jco-25-01471
AB  - 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.
SN  - 0732-183X
SN  - 1527-7755
ER  -