Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study

Abstract: We report final analysis results from the PILOT study of lisocabtagene maraleucel (liso-cel) for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). Sixty-one adults with R/R LBCL who had received 1 previous line of therapy and met ≥1 hematopoietic stem cell transplantati...

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Main Authors: Alison Sehgal, Daanish Hoda, Peter A. Riedell, Nilanjan Ghosh, Mehdi Hamadani, Gerhard C. Hildebrandt, John E. Godwin, Patrick M. Reagan, Nina D. Wagner-Johnston, James Essell, Rajneesh Nath, Scott R. Solomon, Rebecca Champion, Edward Licitra, Suzanne Fanning, Neel K. Gupta, Victor A. Chow, Brenda Yuan, Zhi Yang, Ken Ogasawara, Jerill Thorpe, Leo I. Gordon
Format: Article
Language:English
Published: Elsevier 2025-08-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S247395292500254X
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author Alison Sehgal
Daanish Hoda
Peter A. Riedell
Nilanjan Ghosh
Mehdi Hamadani
Gerhard C. Hildebrandt
John E. Godwin
Patrick M. Reagan
Nina D. Wagner-Johnston
James Essell
Rajneesh Nath
Scott R. Solomon
Rebecca Champion
Edward Licitra
Suzanne Fanning
Neel K. Gupta
Victor A. Chow
Brenda Yuan
Zhi Yang
Ken Ogasawara
Jerill Thorpe
Leo I. Gordon
author_facet Alison Sehgal
Daanish Hoda
Peter A. Riedell
Nilanjan Ghosh
Mehdi Hamadani
Gerhard C. Hildebrandt
John E. Godwin
Patrick M. Reagan
Nina D. Wagner-Johnston
James Essell
Rajneesh Nath
Scott R. Solomon
Rebecca Champion
Edward Licitra
Suzanne Fanning
Neel K. Gupta
Victor A. Chow
Brenda Yuan
Zhi Yang
Ken Ogasawara
Jerill Thorpe
Leo I. Gordon
author_sort Alison Sehgal
collection DOAJ
description Abstract: We report final analysis results from the PILOT study of lisocabtagene maraleucel (liso-cel) for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). Sixty-one adults with R/R LBCL who had received 1 previous line of therapy and met ≥1 hematopoietic stem cell transplantation (HSCT) not intended criterion. Overall response rate (primary end point) was 80%; 54% achieved complete response. After median on-study follow-up of 18.2 months, median duration of response was 23.3 months (95% confidence interval [CI], 6.2 to not reached [NR]). Median progression-free survival (PFS) was 9.0 months (95% CI, 4.2 to NR), median overall survival (OS) was NR (95% CI, 16.3 to NR), and 18-month PFS and OS rates were 43% (95% CI, 30-55) and 59% (95% CI, 45-70), respectively. In the treatment-emergent (TE) period (≤90 days after liso-cel administration), 79% had grade ≥3 adverse events (AEs), 38% had cytokine release syndrome (2% grade 3; no grade 4/5), 31% had neurological events (5% grade 3; no grade 4/5), and 7% had grade ≥3 infections. Of 57 patients in the post-TE period (≥91 days after liso-cel administration), 18% experienced grade ≥3 AEs; 1 patient had grade ≥3 infections. Thirty patients in the leukapheresis set (n = 74) died, mostly of disease progression (n = 24). In this population with high incidence of high-grade B-cell lymphoma, primary-refractory disease, advanced age, and comorbidities, liso-cel demonstrated durable efficacy and a favorable safety profile, consistent with previous reports. These results support liso-cel as second-line therapy for this underserved population of patients with R/R LBCL not intended for HSCT. This trial was registered at www.clinicaltrials.gov as #NCT03483103.
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spelling doaj-art-57a9d1da74fb4e8f99e18990c3628c692025-08-20T03:08:17ZengElsevierBlood Advances2473-95292025-08-019153694370510.1182/bloodadvances.2024015262Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT studyAlison Sehgal0Daanish Hoda1Peter A. Riedell2Nilanjan Ghosh3Mehdi Hamadani4Gerhard C. Hildebrandt5John E. Godwin6Patrick M. Reagan7Nina D. Wagner-Johnston8James Essell9Rajneesh Nath10Scott R. Solomon11Rebecca Champion12Edward Licitra13Suzanne Fanning14Neel K. Gupta15Victor A. Chow16Brenda Yuan17Zhi Yang18Ken Ogasawara19Jerill Thorpe20Leo I. Gordon21University of Pittsburgh Medical Center, Hillman Cancer Center, Pittsburgh, PALoveland Clinic for Blood Cancer Therapy, Intermountain Healthcare, Salt Lake City, UTDavid and Etta Jonas Center for Cellular Therapy, University of Chicago, Chicago, ILDepartment of Hematologic Oncology and Blood Disorders, Atrium Health Levine Cancer Institute, Charlotte, NCBone and Marrow Transplant and Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, WIDivision of Hematology and Oncology, Department of Medicine, University of Missouri Columbia, Columbia, MOOncology Hematology Clinic, Providence Cancer Center, Earle A. Chiles Research Institute, Portland, ORUniversity of Rochester Medical Center, Rochester, NYJohns Hopkins University, Baltimore, MDDepartment of Medical Oncology, Hematology, Blood and Marrow Transplantation, Oncology Hematology Care, Cincinnati, OHSCT Cellular Therapy & Acute Leukemia, Banner MD Anderson Cancer Center, Gilbert, AZTransplant and Cellular Immunotherapy Program, Northside Hospital Cancer Institute, Atlanta, GANorton Cancer Institute, Louisville, KYAstera Cancer Care, East Brunswick, NJDepartment of Hematology, Prisma Health, Greenville, SCStanford Cancer Center, Palo Alto, CABristol Myers Squibb, Seattle, WABristol Myers Squibb, Princeton, NJBristol Myers Squibb, Seattle, WABristol Myers Squibb, Princeton, NJBristol Myers Squibb, Seattle, WADepartment of Medicine, Division of Hematology/Oncology, Northwestern University Feinberg School of Medicine, and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Correspondence: Leo I. Gordon, Department of Medicine, Division of Hematology/Oncology, Northwestern University Feinberg School of Medicine, and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, 676 North Saint Clair St, Suite 850, Chicago, IL 60611;Abstract: We report final analysis results from the PILOT study of lisocabtagene maraleucel (liso-cel) for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). Sixty-one adults with R/R LBCL who had received 1 previous line of therapy and met ≥1 hematopoietic stem cell transplantation (HSCT) not intended criterion. Overall response rate (primary end point) was 80%; 54% achieved complete response. After median on-study follow-up of 18.2 months, median duration of response was 23.3 months (95% confidence interval [CI], 6.2 to not reached [NR]). Median progression-free survival (PFS) was 9.0 months (95% CI, 4.2 to NR), median overall survival (OS) was NR (95% CI, 16.3 to NR), and 18-month PFS and OS rates were 43% (95% CI, 30-55) and 59% (95% CI, 45-70), respectively. In the treatment-emergent (TE) period (≤90 days after liso-cel administration), 79% had grade ≥3 adverse events (AEs), 38% had cytokine release syndrome (2% grade 3; no grade 4/5), 31% had neurological events (5% grade 3; no grade 4/5), and 7% had grade ≥3 infections. Of 57 patients in the post-TE period (≥91 days after liso-cel administration), 18% experienced grade ≥3 AEs; 1 patient had grade ≥3 infections. Thirty patients in the leukapheresis set (n = 74) died, mostly of disease progression (n = 24). In this population with high incidence of high-grade B-cell lymphoma, primary-refractory disease, advanced age, and comorbidities, liso-cel demonstrated durable efficacy and a favorable safety profile, consistent with previous reports. These results support liso-cel as second-line therapy for this underserved population of patients with R/R LBCL not intended for HSCT. This trial was registered at www.clinicaltrials.gov as #NCT03483103.http://www.sciencedirect.com/science/article/pii/S247395292500254X
spellingShingle Alison Sehgal
Daanish Hoda
Peter A. Riedell
Nilanjan Ghosh
Mehdi Hamadani
Gerhard C. Hildebrandt
John E. Godwin
Patrick M. Reagan
Nina D. Wagner-Johnston
James Essell
Rajneesh Nath
Scott R. Solomon
Rebecca Champion
Edward Licitra
Suzanne Fanning
Neel K. Gupta
Victor A. Chow
Brenda Yuan
Zhi Yang
Ken Ogasawara
Jerill Thorpe
Leo I. Gordon
Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
Blood Advances
title Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
title_full Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
title_fullStr Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
title_full_unstemmed Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
title_short Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study
title_sort lisocabtagene maraleucel for r r lbcl in patients not intended for hsct final results of the phase 2 pilot study
url http://www.sciencedirect.com/science/article/pii/S247395292500254X
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