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

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.

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Publication Details

Journal
Blood
Published
2025-04-08
DOI
https://doi.org/10.1182/blood.2024026216
Citations
11
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
11.50

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article

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

Heather Jolie Landau, Hamza Hashmi, Aimaz Afrough, Siddhartha Ganguly et al.
11 citations
Blood
CAR-T cell therapy research
11.50
article

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

Heather Jolie Landau, Hamza Hashmi, Aimaz Afrough, Siddhartha Ganguly, Saad Z. Usmani, Matthew Bye, Abu‐Sayeef Mirza, Muzaffar H. Qazilbash, Surbhi Sidana, Othman Salim Akhtar, Pallavi Patwardhan, Larry D. Anderson, Binod Dhakal, Krina K. Patel, Ciara L. Freeman, Taiga Nishihori, Ruta Brazauskas, Amani Kitali, Temitope Oloyede, Christopher J. Ferreri, Marcelo C. Pasquini, Nausheen Ahmed, Lohith Gowda, Devender Dhanda, Doris K. Hansen, Melanie J. Harrison
article en
11 citations

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.

BloodVol. 146(2)
Houston Methodist (US), Memorial Sloan Kettering Cancer Center (US), The University of Texas MD Anderson Cancer Center (US), Palo Alto University (US), Medical College of Wisconsin (US), Yale Cancer Center (US), Moffitt Cancer Center (US), Yale University (US), Bristol-Myers Squibb (United States) (US), The University of Kansas Cancer Center (US), Levine Cancer Institute (US), Methodist Hospital (US), University of Kansas Medical Center (US), The University of Texas Southwestern Medical Center (US), Stanford University (US)
Doris Duke Charitable Foundation, Gateway for Cancer Research, Amgen, Bristol-Myers Squibb, Pfizer, Astellas Pharma US, AstraZeneca, GlaxoSmithKline, Sanofi, Gilead Sciences, Alexion Pharmaceuticals, HistoGenetics, Novartis Pharmaceuticals Corporation, CSL Behring, Vertex Pharmaceuticals, Jazz Pharmaceuticals, Mallinckrodt Pharmaceuticals, Atara Biotherapeutics, CareDx, bluebird bio, Adaptive Biotechnologies, BeiGene, Incyte, Omeros Corporation, Seagen, Kiadis Pharma, Astellas Pharma, Swedish Orphan Biovitrum, MorphoSys, Medac, Health Resources and Services Administration, Takeda Oncology, Pharmacyclics, Legend Biotech, National Heart, Lung, and Blood Institute, Office of Naval Research, National Cancer Institute, National Institute of Allergy and Infectious Diseases, Stanford Cancer Institute
Good health and well-being
Openalex Percentile: Top 1%
CAR-T cell therapy research
11.50
DOIOpen Access PDFOpenAlex
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