Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma
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- Marcelo C. Pasquini
- Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI;
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- Zhen-Huan Hu
- Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI;
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- Kevin Curran
- Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY;
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- Theodore Laetsch
- Cancer Center, Children’s Hospital of Philadelphia, Philadelphia, PA;
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- Frederick Locke
- Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center, Tampa, FL;
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- Rayne Rouce
- Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, TX;
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- Michael A. Pulsipher
- Children’s Hospital Los Angeles/Pediatrics Department, Keck School of Medicine, University of Southern California, Los Angeles, CA;
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- Christine L. Phillips
- Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;
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- Amy Keating
- Pediatric Hematology, Oncology and Bone Marrow Transplantation, University of Colorado School of Medicine, Aurora, CO;
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- Matthew J. Frigault
- Cellular Therapy Service, Massachusetts General Hospital, Boston, MA;
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- Dana Salzberg
- Pediatric Hematologic Oncology, Phoenix Children’s Hospital, Phoenix, AZ;
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- Samantha Jaglowski
- Bone and Marrow Transplant Program, Ohio State University, Columbus, OH;
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- Joshua P. Sasine
- Department of Medicine, University of California Los Angeles, Los Angeles, CA;
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- Joseph Rosenthal
- Department of Pediatrics, City of Hope, Duarte, CA;
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- Monalisa Ghosh
- Division of Hematology and Oncology, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI;
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- Daniel Landsburg
- Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA;
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- Steven Margossian
- Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA;
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- Paul L. Martin
- Pediatrics Department, Duke University Medical Center, Durham, NC;
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- Manali K. Kamdar
- Division of Hematology, University of Colorado School of Medicine, Aurora, CO;
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- Peiman Hematti
- Section of Hematology/Oncology, University of Wisconsin–Madison School of Medicine and Public Health, Madison, WI;
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- Sarah Nikiforow
- Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute, Boston, MA;
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- Cameron Turtle
- Fred Hutchinson Cancer Research Center, Seattle, WA;
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- Miguel-Angel Perales
- Adult Bone Marrow Transplant Program, Memorial Sloan Kettering Cancer Center, New York, NY
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- Patricia Steinert
- Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI;
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- Mary M. Horowitz
- Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI;
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- Amy Moskop
- Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI;
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- Lida Pacaud
- Novartis Pharmaceuticals, New York, NY;
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- Lan Yi
- Novartis Pharmaceuticals, New York, NY;
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- Raghav Chawla
- Novartis Institutes for BioMedical Research, Basel, Switzerland;
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- Eric Bleickardt
- Novartis, Hamden, CT; and
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- Stephan Grupp
- Cancer Center, Children’s Hospital of Philadelphia, Philadelphia, PA;
抄録
<jats:title>Abstract</jats:title> <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 ≥3 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 <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>
収録刊行物
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- Blood Advances
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Blood Advances 4 (21), 5414-5424, 2020-11-04
American Society of Hematology