{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T17:14:25Z","timestamp":1793121265253,"version":"4.5.0"},"update-to":[{"DOI":"10.1182\/bloodadvances.2021005069","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2020,11,4]],"date-time":"2020-11-04T00:00:00Z","timestamp":1604448000000}},{"DOI":"10.1182\/bloodadvances.2021004265","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2020,11,4]],"date-time":"2020-11-04T00:00:00Z","timestamp":1604448000000}}],"reference-count":18,"publisher":"American Society of Hematology","issue":"21","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,11,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for treatment of pediatric and young adult patients with relapsed\/refractory acute lymphoblastic leukemia (ALL) and adults with non-Hodgkin lymphoma (NHL). The initial experience with tisagenlecleucel in a real-world setting from a cellular therapy registry is presented here. As of January 2020, 511 patients were enrolled from 73 centers, and 410 patients had follow-up data reported (ALL, n = 255; NHL, n = 155), with a median follow-up of 13.4 and 11.9 months for ALL and NHL, respectively. Among patients with ALL, the initial complete remission (CR) rate was 85.5%. Twelve-month duration of response (DOR), event-free survival, and overall survival (OS) rates were 60.9%, 52.4%, and 77.2%, respectively. Among adults with NHL, the best overall response rate was 61.8%, including an initial CR rate of 39.5%. Six-month DOR, progression-free survival, and OS rates were 55.3%, 38.7%, and 70.7%, respectively. Grade \u22653 cytokine release syndrome and neurotoxicity were reported in 11.6% and 7.5% of all patients, respectively. Similar outcomes were observed in patients with in-specification and out-of-specification products as a result of viability &amp;lt;80% (range, 61% to 79%). This first report of tisagenlecleucel in the real-world setting demonstrates outcomes with similar efficacy and improved safety compared with those seen in the pivotal trials.<\/jats:p>","DOI":"10.1182\/bloodadvances.2020003092","type":"journal-article","created":{"date-parts":[[2020,11,4]],"date-time":"2020-11-04T11:54:34Z","timestamp":1604490874000},"page":"5414-5424","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":434,"title":["Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma"],"prefix":"10.1182","volume":"4","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1579-2293","authenticated-orcid":false,"given":"Marcelo C.","family":"Pasquini","sequence":"first","affiliation":[{"name":"Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, 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Oncology and Bone Marrow Transplantation, University of Colorado School of Medicine, Aurora, CO;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew J.","family":"Frigault","sequence":"additional","affiliation":[{"name":"Cellular Therapy Service, Massachusetts General Hospital, Boston, MA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dana","family":"Salzberg","sequence":"additional","affiliation":[{"name":"Pediatric Hematologic Oncology, Phoenix Children\u2019s Hospital, Phoenix, AZ;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4335-2554","authenticated-orcid":false,"given":"Samantha","family":"Jaglowski","sequence":"additional","affiliation":[{"name":"Bone and Marrow Transplant Program, Ohio State University, Columbus, OH;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9866-462X","authenticated-orcid":false,"given":"Joshua P.","family":"Sasine","sequence":"additional","affiliation":[{"name":"Department of Medicine, University of California Los Angeles, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, City of Hope, Duarte, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monalisa","family":"Ghosh","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Landsburg","sequence":"additional","affiliation":[{"name":"Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven","family":"Margossian","sequence":"additional","affiliation":[{"name":"Dana-Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Boston, MA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul L.","family":"Martin","sequence":"additional","affiliation":[{"name":"Pediatrics Department, Duke University Medical Center, Durham, NC;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manali K.","family":"Kamdar","sequence":"additional","affiliation":[{"name":"Division of Hematology, University of Colorado School of Medicine, Aurora, CO;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peiman","family":"Hematti","sequence":"additional","affiliation":[{"name":"Section of Hematology\/Oncology, University of Wisconsin\u2013Madison School of Medicine and Public Health, Madison, 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