Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma.

Autor:	Pasquini MC; Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI., Hu ZH; Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI., Curran K; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY., Laetsch T; Cancer Center, Children's Hospital of Philadelphia, Philadelphia, PA., Locke F; Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center, Tampa, FL., Rouce R; Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, TX., Pulsipher MA; Children's Hospital Los Angeles/Pediatrics Department, Keck School of Medicine, University of Southern California, Los Angeles, CA., Phillips CL; Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH., Keating A; Pediatric Hematology, Oncology and Bone Marrow Transplantation, University of Colorado School of Medicine, Aurora, CO., Frigault MJ; Cellular Therapy Service, Massachusetts General Hospital, Boston, MA., Salzberg D; Pediatric Hematologic Oncology, Phoenix Children's Hospital, Phoenix, AZ., Jaglowski S; Bone and Marrow Transplant Program, Ohio State University, Columbus, OH., Sasine JP; Department of Medicine, University of California Los Angeles, Los Angeles, CA., Rosenthal J; Department of Pediatrics, City of Hope, Duarte, CA., Ghosh M; Division of Hematology and Oncology, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI., Landsburg D; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Margossian S; Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Martin PL; Pediatrics Department, Duke University Medical Center, Durham, NC., Kamdar MK; Division of Hematology, University of Colorado School of Medicine, Aurora, CO., Hematti P; Section of Hematology/Oncology, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI., Nikiforow S; Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute, Boston, MA., Turtle C; Fred Hutchinson Cancer Research Center, Seattle, WA., Perales MA; Adult Bone Marrow Transplant Program, Memorial Sloan Kettering Cancer Center, New York, NY., Steinert P; Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI., Horowitz MM; Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI., Moskop A; Department of Medicine, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI., Pacaud L; Novartis Pharmaceuticals, New York, NY., Yi L; Novartis Pharmaceuticals, New York, NY., Chawla R; Novartis Institutes for BioMedical Research, Basel, Switzerland., Bleickardt E; Novartis, Hamden, CT; and., Grupp S; Cancer Center, Children's Hospital of Philadelphia, Philadelphia, PA.; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
Jazyk:	angličtina
Zdroj:	Blood advances [Blood Adv] 2020 Nov 10; Vol. 4 (21), pp. 5414-5424.
DOI:	10.1182/bloodadvances.2020003092
Abstrakt:	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. (© 2020 by The American Society of Hematology.)
Databáze:	MEDLINE
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