Blood Advances (Jun 2025)
Real-world outcomes for young adult patients receiving CD19 CAR T-cell therapy
- Hannah Lust,
- Liora M. Schultz,
- Soyang Kwon,
- Gregory W. Roloff,
- Ibrahim Aldoss,
- Christina Baggott,
- Samuel John,
- Jenna E. Rossoff,
- Kevin O. McNerney,
- Vanessa A. Fabrizio,
- Julie-An Talano,
- Amy Moskop,
- Kevin J. Curran,
- Christine L. Phillips,
- Nicole Karras,
- Susanne H.C. Baumeister,
- Stacy L. Cooper,
- Michelle Hermiston,
- Prakash Satwani,
- Muna Qayed,
- Sunil S. Raikar,
- Margaret MacMillan,
- Erin Hall,
- Khanh Nguyen,
- Ryan D. Cassaday,
- Noam E. Kopmar,
- Vamsi K. Kota,
- John Mathews,
- Paul Shaughnessy,
- Marc S. Schwartz,
- Abdullah Ladha,
- George Yaghmour,
- Muthu V. Kumaran,
- Veronika Bachanova,
- Sean Tracy,
- Tamer Othman,
- Marlise R. Luskin,
- Evan C. Chen,
- Anjani S. Advani,
- Nikeshan Jeyakumar,
- Katharine Miller,
- Amy Zhang,
- Katherine C. Sutherland,
- Bijal D. Shah,
- Lori Muffly,
- Rawan Faramand
Affiliations
- Hannah Lust
- Division of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL; Correspondence: Hannah Lust, Division of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital, 225 E Chicago Ave, Box 30, Chicago, IL 60611;
- Liora M. Schultz
- Department of Pediatrics, Bass Center for Childhood Cancer and Blood Disorders, Stanford University School of Medicine, Stanford, CA
- Soyang Kwon
- Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
- Gregory W. Roloff
- Division of Hematology/Oncology, University of Chicago, Chicago, IL
- Ibrahim Aldoss
- Division of Leukemia, Hematology and Blood Stem Cell and Marrow Transplant, City of Hope, Duarte, CA
- Christina Baggott
- Department of Pediatrics, Bass Center for Childhood Cancer and Blood Disorders, Stanford University School of Medicine, Stanford, CA
- Samuel John
- Division of Pediatric Hematology/Oncology, Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX
- Jenna E. Rossoff
- Division of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
- Kevin O. McNerney
- Division of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
- Vanessa A. Fabrizio
- Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplant, University of Colorado Anschutz Medical Campus, Colorado Children's Hospital, Aurora, CO
- Julie-An Talano
- Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
- Amy Moskop
- Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
- Kevin J. Curran
- Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY
- Christine L. Phillips
- Division of Oncology, Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital, Cincinnati, OH
- Nicole Karras
- Department of Pediatrics, City of Hope, Duarte, CA
- Susanne H.C. Baumeister
- Division of Hematology/Oncology, Department of Pediatric Oncology, Harvard Medical School, Boston, MA
- Stacy L. Cooper
- Department of Oncology, Division of Pediatric Oncology, Johns Hopkins, Baltimore, MD
- Michelle Hermiston
- Division of Pediatric Allergy, Immunology, and Blood & Marrow Transplantation, University of California San Francisco Benioff Children’s Hospital, San Francisco, CA
- Prakash Satwani
- Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Department of Pediatrics, Columbia University Medical Center, New York, NY
- Muna Qayed
- Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
- Sunil S. Raikar
- Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
- Margaret MacMillan
- Division of Pediatric Blood and Marrow Transplantation and Cellular Therapy, University of Minnesota, Minneapolis, MN
- Erin Hall
- Division of Pediatric Hematology, Oncology, and Blood & Marrow Transplantation, Children’s Mercy, Kansas City, KS
- Khanh Nguyen
- Department of Pediatrics, Bass Center for Childhood Cancer and Blood Disorders, Stanford University School of Medicine, Stanford, CA
- Ryan D. Cassaday
- Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA
- Noam E. Kopmar
- Division of Medical Oncology, Department of Medicine, University of Washington, Seattle, WA
- Vamsi K. Kota
- Department of Medicine, Division of Hematology and Oncology, Augusta University Medical Center, Augusta, GA
- John Mathews
- Department of Transplant and Cellular Therapy, Sarah Cannon and Texas Oncology Transplant and Cellular Therapy Program, Medical City Dallas, Dallas, TX
- Paul Shaughnessy
- Department of Transplant and Cellular Therapy, Sarah Cannon Transplant and Cellular Therapy Program, Methodist Hospital, HCA Healthcare, San Antonio, TX
- Marc S. Schwartz
- Department of Hematology and Oncology, University of Colorado, Aurora, CO
- Abdullah Ladha
- Jane Anne Nohl Division of Hematology and Center for the Study of Blood Disease, University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA
- George Yaghmour
- Jane Anne Nohl Division of Hematology and Center for the Study of Blood Disease, University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA
- Muthu V. Kumaran
- Division of Hematology and Oncology, University of Arkansas for Medical Sciences, Little Rock, AR
- Veronika Bachanova
- Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis, MN
- Sean Tracy
- Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis, MN
- Tamer Othman
- Division of Hematology and Oncology, University of California San Francisco, San Francisco, CA
- Marlise R. Luskin
- Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
- Evan C. Chen
- Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
- Anjani S. Advani
- Leukemia Program, Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
- Nikeshan Jeyakumar
- Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA
- Katharine Miller
- Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA
- Amy Zhang
- Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA
- Katherine C. Sutherland
- Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA
- Bijal D. Shah
- Division of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, FL
- Lori Muffly
- Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University, Stanford, CA
- Rawan Faramand
- Division of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, FL
- DOI
- https://doi.org/10.1182/bloodadvances.2024014846
- Journal volume & issue
-
Vol. 9,
no. 11
pp. 2763 – 2772
Abstract
Abstract: Tisagenlecleucel (tisa-cel) and brexucabtagene autoleucel (brexu-cel) are approved CD19 chimeric antigen receptor T-cell therapy (CAR T) products for young adults (YA) with relapsed/refractory B-cell acute lymphoblastic leukemia. A distinct analysis of YAs receiving commercial CD19 CAR T has not been reported. Using retrospective data from the Pediatric Real-World CAR T Consortium and the Real-World Outcomes of CAR T in Adult ALL collaboration, we describe the efficacy and safety of tisa-cel and brexu-cel in 70 YAs (18-26 years; tisa-cel, n = 50; brexu-cel, n = 20). Cytokine release syndrome (CRS) and immune effector cell–associated neurotoxicity syndrome (ICANS) were observed more frequently for brexu-cel vs tisa-cel (CRS, 85% vs 56%; ICANS, 40% vs 18%). Complete response rates were similar between products at 80% for brexu-cel and 88% for tisa-cel. Relapse-free survival (RFS) at 12 months was 46% for brexu-cel and 36% for tisa-cel. Durability of remission over 12 months was 61% for brexu-cel vs 41% for tisa-cel; 12-month overall survival (OS) for brexu-cel was 84% vs 68% for tisa-cel. In multivariate analysis, low disease burden was associated with improved OS, whereas inotuzumab before CAR T was associated with inferior outcomes. This study demonstrates comparable real-world efficacy among YAs receiving CD19 CAR T irrespective of CAR T construct; however, rates of toxicity seem higher with brexu-cel.
