{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T19:19:26Z","timestamp":1793992766396,"version":"4.8.0"},"reference-count":9,"publisher":"American Society of Clinical Oncology (ASCO)","issue":"9","content-domain":{"domain":["ascopubs.org"],"crossmark-restriction":true},"short-container-title":["JCO"],"published-print":{"date-parts":[[2023,3,20]]},"abstract":"<jats:p>Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.<\/jats:p>\n                  <jats:p>In the primary analysis of the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849 ), tisagenlecleucel provided an overall remission rate of 81% in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R\/R B-ALL), with 59% of responders remaining relapse-free at 12 months. Here, we report an update on efficacy, safety, and patient-reported quality of life in 79 pediatric and young adult patients with R\/R B-ALL following a median follow-up of 38.8 months. The overall remission rate was 82%. The median event-free survival was 24 months, and the median overall survival was not reached. Event-free survival was 44% (95% CI, 31 to 57) and overall survival was 63% (95% CI, 51 to 73) at 3 years overall (most events occur within the first 2 years). The estimated 3-year relapse-free survival with and without censoring for subsequent therapy was 52% (95% CI, 37 to 66) and 48% (95% CI, 34 to 60), respectively. No new or unexpected long-term adverse events were reported. Grade 3\/4 adverse events were reported in 29% of patients &gt; 1 year after infusion; grade 3\/4 infection rate did not increase &gt; 1 year after infusion. Patients reported improvements in quality of life up to 36 months after infusion. These findings demonstrate favorable long-term safety and suggest tisagenlecleucel as a curative treatment option for heavily pretreated pediatric and young adult patients with R\/R B-ALL.<\/jats:p>","DOI":"10.1200\/jco.22.00642","type":"journal-article","created":{"date-parts":[[2022,11,18]],"date-time":"2022-11-18T15:59:26Z","timestamp":1668787166000},"page":"1664-1669","update-policy":"https:\/\/doi.org\/10.1200\/crossmark","source":"Crossref","is-referenced-by-count":389,"title":["Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed\/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial"],"prefix":"10.1200","volume":"41","clinical-trial-number":[{"clinical-trial-number":"nct02435849","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8497-3138","authenticated-orcid":false,"given":"Theodore W.","family":"Laetsch","sequence":"first","affiliation":[{"name":"Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia, PA"},{"name":"Department of Pediatrics, Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2210-8736","authenticated-orcid":false,"given":"Shannon L.","family":"Maude","sequence":"additional","affiliation":[{"name":"Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia, PA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5658-1831","authenticated-orcid":false,"given":"Susana","family":"Rives","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\u2014Oncology and Institut de Recerca, Hospital Sant Joan de D\u00e9u Barcelona, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidefumi","family":"Hiramatsu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5507-8310","authenticated-orcid":false,"given":"Henrique","family":"Bittencourt","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Faculty of Medicine, University of Montreal, Montreal, Canada"},{"name":"The Hematology Oncology Division and Charles-Bruneau Cancer Center, Centre Hospitalier Universitaire Sainte-Justine, Montreal, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4554-0265","authenticated-orcid":false,"given":"Peter","family":"Bader","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Frankfurt, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0120-6089","authenticated-orcid":false,"given":"Andr\u00e9","family":"Baruchel","sequence":"additional","affiliation":[{"name":"University Hospital Robert Debr\u00e9 (APHP) and Universit\u00e9 de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Boyer","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Internal Medicine, University of Utah, Salt Lake City, UT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2449-539X","authenticated-orcid":false,"given":"Barbara","family":"De Moerloose","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7689-343X","authenticated-orcid":false,"given":"Muna","family":"Qayed","sequence":"additional","affiliation":[{"name":"Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5848-4501","authenticated-orcid":false,"given":"Jochen","family":"Buechner","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3030-8420","authenticated-orcid":false,"given":"Michael A.","family":"Pulsipher","sequence":"additional","affiliation":[{"name":"Division of Hematology, Oncology, Blood and Marrow Transplant, Children's Hospital Los Angeles, USC Keck School of Medicine, Los Angeles, CA"},{"name":"At the time of present work, now affiliated with Division of Pediatric Hematology and Oncology, Intermountain Primary Children's Hospital, Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary Douglas","family":"Myers","sequence":"additional","affiliation":[{"name":"Children's Mercy Hospital and Clinics, Kansas City, MO"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather E.","family":"Stefanski","sequence":"additional","affiliation":[{"name":"National Bone Marrow Donor Program, Be the Match, Division of Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul L.","family":"Martin","sequence":"additional","affiliation":[{"name":"Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eneida","family":"Nemecek","sequence":"additional","affiliation":[{"name":"Oregon Health and Science University, Portland, OR"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0369-8515","authenticated-orcid":false,"given":"Christina","family":"Peters","sequence":"additional","affiliation":[{"name":"Stem Cell Transplantation Unit, St Anna Children's Hospital, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory","family":"Yanik","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9084-8974","authenticated-orcid":false,"given":"Seong Lin","family":"Khaw","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre, Royal Children's Hospital and Murdoch Children's Research Institute, Parkville, Victoria, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7182-2592","authenticated-orcid":false,"given":"Kara L.","family":"Davis","sequence":"additional","affiliation":[{"name":"Division of Hematology, Oncology, Stem Cell Transplant and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joerg","family":"Krueger","sequence":"additional","affiliation":[{"name":"Division of Haematology\/Oncology\/Bone Marrow Transplantation, Hospital for Sick Children, Toronto, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5879-0610","authenticated-orcid":false,"given":"Adriana","family":"Balduzzi","sequence":"additional","affiliation":[{"name":"Clinica Pediatrica Universit\u00e0 degli Studi di Milano Bicocca, Monza, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Boissel","sequence":"additional","affiliation":[{"name":"Saint-Louis Hospital (APHP) and Universit\u00e9 de Paris Diderot, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ranjan","family":"Tiwari","sequence":"additional","affiliation":[{"name":"Novartis Healthcare Pvt Ltd, Hyderabad, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Darragh","family":"O'Donovan","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals Corporation, Dublin, Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8030-7595","authenticated-orcid":false,"given":"Stephan A.","family":"Grupp","sequence":"additional","affiliation":[{"name":"Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia, PA"},{"name":"Department 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