Standard-of-care idecabtagene vicleucel for relapsed/refractory multiple myeloma

S Surbhi Sidana (Stanford University School of Medicine, Palo Alto, CA) N Nausheen Ahmed (5University of Kansas Health System, Division of Hematological Malignancy and Cellular Therapeutics, Kansas City, United States) O Othman Salim Akhtar (3Department of Medicine, Medical College of Wisconsin, Milwaukee, WI) R Ruta Brazauskas (Medical College of Wisconsin, Milwaukee, WI) T Temitope Oloyede (1Medical College of Wisconsin, Milwaukee, United States) M Matthew Bye (4Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI) D Doris Hansen (1H. Lee Moffitt Cancer Center and Research Institute, Tampa, United States) C Christopher Ferreri (7Atrium Health Levine Cancer Institute, Charlotte, United States) C Ciara L. Freeman (7Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL) A Aimaz Afrough (Myeloma, Waldenstrom’s, and Amyloidosis Program, Hematologic Malignancies and Cellular Therapy Program, Simmons Comprehensive Cancer Center (A.A.), University of Texas Southwestern Medical Center, Dallas, TX.) L Larry D. Anderson (1Myeloma, Waldenstrom’s, and Amyloidosis Program, Section of Hematologic Malignancies and Cellular Therapy, Simmons Comprehensive Cancer Center, The University of Texas Southwestern Medical Center, Dallas, TX) B Binod Dhakal (2Division of Hematology/Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI) D Devender Dhanda (8Bristol Myers Squibb, Princeton, United States) L Lohith Gowda H Hamza Hashmi (Memorial Sloan Kettering Cancer Center, New York) M Melanie J. Harrison (9Bristol Myers Squibb, Summit, NJ) A Amani Kitali (9Bristol Myers Squibb, Summit, NJ) H Heather Landau (Adult Bone Marrow Transplantation Service Memorial Sloan Kettering Cancer Center New York New York USA) A Abu-Sayeef Mirza (6Moffitt Cancer Center, Tampa, FL) P Pallavi Patwardhan (9Bristol Myers Squibb, Summit, NJ) M Muzaffar Qazilbash (1MD Anderson Cancer Center, Department of Stem Cell Transplantation and Cellular Therapy, Houston, United States) S Saad Usmani (8Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY) K Krina Patel (4The University of Texas MD Anderson Cancer Center, Houston, United States) T Taiga Nishihori (Moffitt Cancer Center, Tampa, Florida, United States) S Siddhartha Ganguly (6Houston Methodist Hospital, Houston, United States) M Marcelo C. Pasquini (18Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI)

Abstract

Abstract Idecabtagene vicleucel (ide-cel) was the first US Food and Drug Administration–approved chimeric antigen receptor T-cell (CAR-T) therapy for multiple myeloma (MM). However, because clinical trials are highly selective with stringent eligibility criteria, the objective of this study was to evaluate the safety and effectiveness of standard-of-care (SOC) ide-cel in the real world. Using the Center for International Blood and Marrow Transplant Research registry, we evaluated 821 patients who received SOC ide-cel. Median follow-up was 11.6 months. Median age was 66 years, and the cohort included 31% patients aged ≥70 years, with 15% Black and 7% Hispanic, and 77% of patients with ≥1 significant comorbidity. The median number of prior lines of therapy was 7, 15% patients previously received B-cell maturation antigen–directed therapy, 17% had extramedullary disease, and 27% had high-risk cytogenetics. Overall response rate was 73%, and complete response rate was 25%. Median progression-free survival was 8.8 months. Treatment-related mortality was reported in 6% of patients. Cytokine release syndrome was diagnosed in 80% of patients (grade ≥3, 3%). Immune effector cell–associated neurotoxicity syndrome was observed in 28% (grade ≥3, 5%), with no cases of Parkinsonism reported. Clinically significant infections were seen in 45% of patients. Second primary malignancies were reported in 4%, including 1% myeloid malignancies. This is, to our knowledge, the largest real-world study of ide-cel CAR-T therapy in patients with relapsed/refractory (R/R) MM. We observed a favorable safety and efficacy profile that mirrors trial experience, even in the setting of significant comorbidities in 77% of patients, many of which would have made them ineligible for the registrational KarMMa clinical trial. This trial was registered at www.clinicaltrials.gov as #NCT03361748.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 2
Published July 10, 2025
Pages 167-177
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (26)

S

Surbhi Sidana

Stanford University School of Medicine, Palo Alto, CA

N

Nausheen Ahmed

5University of Kansas Health System, Division of Hematological Malignancy and Cellular Therapeutics, Kansas City, United States

O

Othman Salim Akhtar

3Department of Medicine, Medical College of Wisconsin, Milwaukee, WI

R

Ruta Brazauskas

Medical College of Wisconsin, Milwaukee, WI

T

Temitope Oloyede

1Medical College of Wisconsin, Milwaukee, United States

M

Matthew Bye

4Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI

D

Doris Hansen

1H. Lee Moffitt Cancer Center and Research Institute, Tampa, United States

C

Christopher Ferreri

7Atrium Health Levine Cancer Institute, Charlotte, United States

C

Ciara L. Freeman

7Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

A

Aimaz Afrough

Myeloma, Waldenstrom’s, and Amyloidosis Program, Hematologic Malignancies and Cellular Therapy Program, Simmons Comprehensive Cancer Center (A.A.), University of Texas Southwestern Medical Center, Dallas, TX.

L

Larry D. Anderson

1Myeloma, Waldenstrom’s, and Amyloidosis Program, Section of Hematologic Malignancies and Cellular Therapy, Simmons Comprehensive Cancer Center, The University of Texas Southwestern Medical Center, Dallas, TX

B

Binod Dhakal

2Division of Hematology/Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI

D

Devender Dhanda

8Bristol Myers Squibb, Princeton, United States

L

Lohith Gowda

H

Hamza Hashmi

Memorial Sloan Kettering Cancer Center, New York

M

Melanie J. Harrison

9Bristol Myers Squibb, Summit, NJ

A

Amani Kitali

9Bristol Myers Squibb, Summit, NJ

H

Heather Landau

Adult Bone Marrow Transplantation Service Memorial Sloan Kettering Cancer Center New York New York USA

A

Abu-Sayeef Mirza

6Moffitt Cancer Center, Tampa, FL

P

Pallavi Patwardhan

9Bristol Myers Squibb, Summit, NJ

M

Muzaffar Qazilbash

1MD Anderson Cancer Center, Department of Stem Cell Transplantation and Cellular Therapy, Houston, United States

S

Saad Usmani

8Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

K

Krina Patel

4The University of Texas MD Anderson Cancer Center, Houston, United States

T

Taiga Nishihori

Moffitt Cancer Center, Tampa, Florida, United States

S

Siddhartha Ganguly

6Houston Methodist Hospital, Houston, United States

M

Marcelo C. Pasquini

18Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI