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1030 Brexucabtagene Autoleucel in Adults with Relapsed/Refractory B-Cell ALL: Outcomes and Novel Insights from the Real-World Outcomes Collaborative of CAR T in Adult ALL (ROCCA)

Program: Oral and Poster Abstracts
Type: Oral
Session: 705. Cellular Immunotherapies: Late Phase and Commercially Available Therapies: Cellular Therapy for Multiple Myeloma, B-cell Acute Lymphoblastic Leukemia and B Cell Lymphomas: Clinical Trial and Real World Evidence
Hematology Disease Topics & Pathways:
Biological therapies, Lymphoid Leukemias, ALL, Chimeric Antigen Receptor (CAR)-T Cell Therapies, Diseases, Therapies, Lymphoid Malignancies
Monday, December 11, 2023: 5:15 PM

Gregory W Roloff, MD1, Ibrahim Aldoss, MD2, Noam E. Kopmar, MD3, Chenyu Lin, MD4, Simone E. Dekker, MD, PhD5, Vishal K Gupta, MD6, Nikeshan Jeyakumar, MD7*, Timothy E O'Connor, MD8, Amy Zhang, PhD9*, Katharine Miller, PhD, MPH9*, Kaitlyn C Dykes, MD10*, Mohamed Ahmed11, Hector Zambrano12*, Danielle Bradshaw, MD13*, Santiago Mercadal, MD14*, Marc Schwartz, MD15*, Sean Tracy, MD, PhD16, Bhagirathbhai Dholaria, MBBS17, Michal Kubiak, MD18, Akash Mukherjee, MD19*, Navneet Majhail, MD, MS, FASTCT20, Minoo Battiwalla, MD21, Luke Mountjoy, DO22, Shahbaz A. Malik, MD23, John Mathews, MD24*, Paul Shaughnessy, MD25, Aaron C. Logan, MD, PhD26, Abdullah Ladha, MD27*, George Yaghmour, MD27, Anjali S. Advani, MD28, Maryann Stefan28*, Caitlin Guzowski, MBA29*, Rasmus T. Hoeg, MD30*, Talal Hilal, MD31, Jozal Moore, MD32, Kristen M. O'Dwyer, MD32, Stephanie B. Tsai, MD, MS8, Joshua Sasine, MD11, Melhem M. Solh, MD29, Catherine J. Lee, MD33, Vamsi K. Kota, MD18, Divya Koura, MD10, Muthu Veeraputhiran, MD19*, Betsy Blunk12*, Jessica T. Leonard, MD34, Veronika Bachanova, MD, PhD35, Wendy Stock, MD1, Ahmed Galal, MD4, Vinod Pullarkat, MD2, Ryan D Cassaday, MD3, Bijal D. Shah, MD36, Rawan Faramand, MD36 and Lori S. Muffly, MD9

1University of Chicago, Chicago, IL
2City of Hope, Duarte, CA
3University of Washington, Seattle, WA
4Duke University School of Medicine, Durham, NC
5Department of Hematology & Medical Oncology, Oregon Health & Science University, Portland, OR
6University of California Los Angeles, Los Angeles, CA
7Division of Blood and Marrow Transplantation & Cellular Therapy, Stanford University School of Medicine, Palo Alto, CA
8Loyola University, Maywood, IL
9Stanford University School of Medicine, Stanford, CA
10University of California San Diego, La Jolla, CA
11Cedars-Sinai Medical Center, Los Angeles, CA
12Sarah Cannon Research Institute, Nashville, TN
13Georgia Cancer Center, Augusta University Medical Center, Augusta, GA
14University of Utah, Salt Lake City, UT
15Division of Hematology, University of Colorado, Aurora, CO
16Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis
17Division of Hematology/Oncology, Vanderbilt University Medical Center, Nashville, TN
18Georgia Cancer Center, Augusta University, Augusta, GA
19University of Arkansas for Medical Sciences, Little Rock, AR
20Sarah Cannon Transplant and Cellular Therapy Network, Nashville, TN
21Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Medical Center, Nashville, TN
22Colorado Blood Cancer Institute and Sarah Cannon Cancer Institute at Presbyterian/St Luke’s Medical Center, Denver, CO
23St. David's South Austin Medical Center, Austin, TX
24Sarah Cannon and Texas Oncology Transplant and Cellular Therapy Program at Medical City Dallas, Dallas, TX
25Sarah Cannon Transplant and Cellular Therapy Program at Methodist Hospital, San Antonio, TX
26Helen Diller Family Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA
27Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA
28Cleveland Clinic Foundation, Cleveland, OH
29Northside Hospital Cancer Institute, Atlanta, GA
30Department of Internal Medicine, Division of Cellular Therapy, Bone Marrow Transplantation and Malignant Hematology, University of California Davis, Sacramento, CA
31Mayo Clinic, Phoenix, AZ
32University of Rochester, Rochester, NY
33Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA
34Oregon Health & Science University, Portland, OR
35Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN
36Department of Blood and Marrow Transplantation and Cellular Immunotherapy, Moffitt Cancer Center, Tampa, FL


In October 2021, brexucabtagene autoleucel (brexu-cel) received U.S. FDA approval as the first CAR T-cell therapy for adults with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) based on the 55-patient ZUMA-3 Phase II study. We subsequently established ROCCA and now report on the largest cohort of patients treated with commercial brexu-cel for r/r B-ALL to date.


Adults (18+) with r/r B-ALL infused with commercial brexu-cel across 25 U.S. institutions were included. ASTCT consensus criteria were used to score CRS and ICANS. Methodologies for assessing MRD (minimal threshold of 10-4) included flow cytometry, NGS, or qPCR depending on institution practice. Duration of remission (DOR) was calculated from time of complete response (CR); progression-free survival (PFS) and overall survival (OS) were calculated from day of brexu-cel infusion and were not censored for hematopoietic cell transplant (HCT) or maintenance. All living patients were censored at the time of last follow-up prior to data lock, which occurred on June 30, 2023.


Among 152 infused, the median age was 46 (range, 18-81), 57% were male, and 34% were Hispanic. Most (67%) had Ph- ALL, were heavily pre-treated (median 4 prior lines), and entered apheresis with high disease burden (57%). At time of apheresis, 23% of patients only had MRD+ disease and 15% were in complete molecular remission. While 82% developed CRS, the majority was grade 1-2, with 9% of the overall cohort experiencing grade 3-4 CRS. In contrast, 55% developed ICANS, with 32% of the entire cohort experiencing grade 3-4 ICANS. Eight patients (5%) died of toxicity/infection prior to D+28 response assessment.

Among 133 patients with response assessment, 120 (90%) achieved morphologic CR, of whom 82% were MRD-, 15% were MRD+, and 3% MRD unknown. The median follow-up for survivors was 8.4 months; 45 patients have relapsed and 42 patients died. Median DOR was not reached. Median PFS and OS were 8.6 months and 15.6 months, respectively. Estimated PFS and OS of the entire cohort at 6-months were 61% (95% CI, 52-68) and 81% (95% CI, 73-87) and at 12-months were 47% (95% CI, 37-56) and 63% (95% CI, 53-72), respectively. We found no association between pre-CAR disease burden and post-CAR PFS/OS. However, patients with MRD- response to CAR had superior PFS relative to patients with MRD+ CR (median 14 months vs. 5 months, P=0.002). Forty-four patients received post-CAR consolidation/maintenance therapy while in CR: 25 allogeneic HCT, 15 TKI, 2 POMP, and 3 other/unknown. To examine the effect of consolidation/maintenance following brexu-cel, we performed a landmark analysis of PFS limited to patients alive and in CR at 2-months post-CAR infusion (N=113) and suggests superior PFS in patients receiving either HCT or other forms maintenance, relative to those receiving no further therapy following brexu-cel (P=0.055). We then investigated post-CAR MRD-response in combination with receipt of post-CAR consolidation/maintenance and found that even among patients achieving MRD-negative response, post-CAR consolidation/maintenance led to superior PFS (Figure 1).


Among 152 adults treated with commercial brexu-cel for r/r B-ALL across 25 U.S. institutions, we found very high response rates (CR/CRi: 90%; 82% MRD-) consistent with the Zuma-3 data. While rates of severe CRS are low, grade 3-4 ICANS was observed in 32% of patients and warrants further investigation We demonstrate the prognostic impact of achieving an MRD-negative CR and an emerging role for consolidation/maintenance therapies to enhance the durability of response following brexu-cel in adults r/r B-ALL.

Disclosures: Aldoss: Pfizer: Consultancy; Jazz: Consultancy; Sobi: Consultancy; KiTE: Consultancy; Amgen: Consultancy, Honoraria; Takeda: Consultancy. Lin: Biomarin: Current equity holder in publicly-traded company; Rigel Pharmaceuticals: Consultancy. Schwartz: Jazz Pharmaceuticals: Consultancy; Novartis: Consultancy. Dholaria: gamida cel: Consultancy; BMS: Research Funding; Takeda: Research Funding; Pfizer: Research Funding; Wugen: Research Funding; Gilead: Research Funding; ADC therapeutics: Consultancy, Honoraria; Arivan: Consultancy; BEAM therapeutics: Consultancy; Atara: Research Funding; Molecular Templates: Research Funding; AstraZeneca: Research Funding; NCI: Research Funding; Allovir: Research Funding; Poseida: Research Funding; Pluri Biotech: Consultancy; Janssen: Consultancy, Honoraria, Research Funding; Angiocrine: Research Funding; Poseida: Research Funding; Adicet: Research Funding; MEI: Research Funding; Orca Bio: Research Funding; Boxer Capital: Consultancy; Ellipsis pharma: Consultancy; Lumanity: Consultancy. Majhail: Anthem Inc: Membership on an entity's Board of Directors or advisory committees. Battiwalla: Novartis: Research Funding; Fate Therapeutics: Research Funding. Shaughnessy: Sanofi: Speakers Bureau; BMS: Speakers Bureau. Logan: Amgen, Autolus Therapeutics, Kadmon, Kite, Pharmacyclics, Talaris: Research Funding; AbbVie, Amgen, Actinium, BMS, Pfizer, Sanofi, Takeda: Consultancy. Advani: Jazz: Honoraria, Membership on an entity's Board of Directors or advisory committees; Kura: Honoraria; Servier: Research Funding; Beam: Honoraria; Immunogen: Research Funding; OBI: Research Funding; Kite: Honoraria, Other: consulting, Research Funding; Pfizer: Honoraria, Research Funding; Glycomimetics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Seattle Genetics: Research Funding; Macrogenics: Research Funding; Incyte: Research Funding; Taiho: Honoraria, Membership on an entity's Board of Directors or advisory committees; Nkarta: Honoraria; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Honoraria, Other: advisory board, Research Funding. Hoeg: Orca Bio: Research Funding. Tsai: Jazz Pharmaceutical: Speakers Bureau; Bristol Myers Squibb: Speakers Bureau. Solh: Bristol-Myers Squibb: Speakers Bureau. Lee: Kadmon: Honoraria; Fresenius Kabi: Consultancy; BMS: Honoraria; Kite Pharma: Honoraria, Speakers Bureau; Sanofi: Consultancy, Honoraria; Incyte Corp: Consultancy, Research Funding. Kota: Pfizer: Honoraria; Kite: Honoraria; Novartis: Honoraria; Incyte: Research Funding. Koura: BMS: Consultancy, Research Funding. Leonard: Takeda: Consultancy; Pfizer: Consultancy; Adaptive Biotechnologies: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: Travel, accommodations, expenses; Kite/Gilead: Consultancy. Bachanova: BMS: Research Funding; Citius: Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees; ADC: Membership on an entity's Board of Directors or advisory committees; Allogene: Membership on an entity's Board of Directors or advisory committees; Miltenyi: Other: DSMB; Incyte: Research Funding; Gamida Cell: Research Funding. Stock: Kura: Research Funding; Servier: Other: Data Safety Monitoring Board/Advisory Board; Newave: Honoraria; Glaxo Smith Kline: Consultancy; Amgen: Honoraria; Kite: Consultancy; Jazz Pharmaceuticals: Consultancy, Honoraria. Pullarkat: Amgen: Consultancy, Speakers Bureau; Servier: Consultancy, Speakers Bureau; Jazz Pharmaceuticals: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Pfizer: Consultancy, Speakers Bureau; Genentech: Consultancy, Speakers Bureau; AbbVie: Consultancy, Speakers Bureau. Cassaday: Servier: Research Funding; Incyte: Research Funding; Vanda Pharmaceuticals: Research Funding; Pfizer: Consultancy, Honoraria, Research Funding; Merck: Research Funding; Kite/Gilead: Consultancy, Honoraria, Research Funding; Amgen: Consultancy, Honoraria, Research Funding; Seagen: Other: Spouse was employed by and owned stock in Seagen within the last 24 months.; Autolus: Membership on an entity's Board of Directors or advisory committees; Jazz: Consultancy, Honoraria; PeproMene Bio: Membership on an entity's Board of Directors or advisory committees. Shah: Moffitt Cancer Center: Current Employment; Pharmacyclics/Janssen, Spectrum/Acrotech, BeiGene, Gilead Sciences: Honoraria; DSMC, Pepromene Bio: Membership on an entity's Board of Directors or advisory committees; Incyte, Jazz Pharmaceuticals, Kite/Gilead, SERVIER: Research Funding; Celgene, Novartis, Pfizer, Janssen, Seattle Genetics, AstraZeneca, Stemline Therapeutics, Kite/Gilead: Other: Travel, Accommodations, Expenses; Takeda, AstraZeneca, Adaptive Biotechnologies, BMS/Celgene, Novartis, Pfizer, Amgen, Precision Biosciences, Kite/Gilead, Jazz Pharmaceuticals, Century Therapeutics, Deciphera, Autolus Therapeutics, Lilly, Pepromene: Consultancy. Faramand: Kite: Research Funding; Gilead: Research Funding. Muffly: pfizer: Consultancy; autolus: Consultancy; amgen: Consultancy; kite: Consultancy, Honoraria, Research Funding; adaptive: Membership on an entity's Board of Directors or advisory committees, Research Funding; bms: Research Funding; jasper: Research Funding; astellas: Consultancy, Research Funding; orca bio: Research Funding.

*signifies non-member of ASH