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

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

収録刊行物

  • Blood Advances

    Blood Advances 4 (21), 5414-5424, 2020-11-04

    American Society of Hematology

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