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Multicenter Study
. 2025 Jan 2;145(1):85-97.
doi: 10.1182/blood.2024025945.

Safety and efficacy of standard-of-care ciltacabtagene autoleucel for relapsed/refractory multiple myeloma

Surbhi Sidana  1 , Krina K Patel  2 , Lauren C Peres  3 , Radhika Bansal  4 , Mehmet H Kocoglu  5 , Leyla Shune  6 , Shebli Atrash  7 , Kinaya Smith  8 , Shonali Midha  9 , Christopher Ferreri  7 , Binod Dhakal  8 , Danai Dima  10 , Patrick Costello  9 , Charlotte Wagner  11 , Ran Reshef  12 , Hitomi Hosoya  1 , Lekha Mikkilineni  1 , Djordje Atanackovic  5 , Saurabh Chhabra  13 , Ricardo Parrondo  14 , Omar Nadeem  9 , Hashim Mann  15 , Nilesh Kalariya  2 , Vanna Hovanky  1 , Gabriel De Avila  3 , Ciara L Freeman  3 , Frederick L Locke  3 , Melissa Alsina  3 , Sandy Wong  16 , Megan Herr  17 , Myo Htut  18 , Joseph McGuirk  6 , Douglas W Sborov  11 , Jack Khouri  10 , Thomas Martin  16 , Murali Janakiram  18 , Yi Lin  4 , Doris K Hansen  3
Affiliations
Multicenter Study

Safety and efficacy of standard-of-care ciltacabtagene autoleucel for relapsed/refractory multiple myeloma

Surbhi Sidana et al. Blood. .

Abstract

Ciltacabtagene autoleucel (cilta-cel) was approved in 2022 for patients with relapsed/refractory multiple myeloma (RRMM). We report outcomes with cilta-cel in the standard-of-care setting. Patients with RRMM who underwent leukapheresis for cilta-cel manufacturing between 1 March 2022 and 31 December 2022 at 16 US academic medical centers were included. Overall, 255 patients underwent leukapheresis and 236 (92.5%) received cilta-cel, of which 54% would not have met CARTITUDE-1 eligibility criteria. In treated patients (N = 236), cytokine release syndrome was seen in 75% (grade ≥3, 5%), immune effector cell-associated neurotoxicity syndrome in 14% (grade ≥3, 4%), and delayed neurotoxicity in 10%. Overall and complete response rates were as follows: all patients who received cilta-cel (N = 236), 89% and 70%; patients receiving conforming cilta-cel (n = 191), 94% and 74%; and conforming cilta-cel with fludarabine/cyclophosphamide lymphodepletion (n = 152), 95% and 76%, respectively. Nonrelapse mortality was 10%, most commonly from infection. After a median follow-up of 13 months from cilta-cel, the median progression-free survival (PFS) was not reached, with 12-month estimate being 68% (95% confidence interval, 62-74). High ferritin levels, high-risk cytogenetics, and extramedullary disease were independently associated with inferior PFS, with a signal for prior B-cell maturation antigen-targeted therapy (P = .08). Second primary malignancies excluding nonmelanoma skin cancers were seen in 5.5% and myeloid malignancies/acute leukemia in 1.7%. We observed a favorable efficacy profile of standard-of-care cilta-cel in RRMM, despite more than half the patients not meeting the CARTITUDE-1 eligibility criteria.

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Conflict of interest statement

Conflict-of-interest disclosure: S.S. reports consulting or advisory fees from Janssen, Bristol Myers Squibb (BMS), Legend, Magenta Therapeutics, Sanofi, Pfizer, Takeda, Kite, AbbVie, and Regeneron and research funding from Janssen, Magenta Therapeutics, Allogene Therapeutics, Novartis, and BMS. K.K.P. reports consulting or advisory role fees from BMS, Janssen, Pfizer, Arcellx, and Karyopharm Therapeutics; research funding from BMS, Poseida Therapeutics, Takeda, Janssen, Cellectis, Nektar, AbbVie/Genentech, Precision Biosciences, and Allogene Therapeutics; and travel, accommodations, and expenses from BMS. L.C.P. reports research funding from BMS. D.W.S. reports consulting or advisory role fees from Sanofi, GlaxoSmithKline (GSK), BMS, Legend Biotech, Janssen, Pfizer, Bioline, AstraZeneca, Arcellx, and AbbVie. S.A. reports honoraria from Janssen and research funding from GSK, Amgen, Karyopharm Therapeutics, Janssen, and BMS. C.F. reports consulting or advisory role fees from Sanofi and stock/other ownership in Affimed Therapeutics. J.K. reports honoraria from OncLive. C.L.F. reports honoraria/consulting fees from BMS, Seattle Genetics, Celgene, AbbVie, Sanofi, Incyte, Amgen, and ONK Therapeutics/Janssen and research funding from BMS, Janssen, and Roche/Genentech. F.L.L. reports consulting or advisory role fees from Allogene, Amgen, Bluebird Bio, BMS/Celgene, Calibr, Cellular Biomedicine Group, Cowen, EcoR1, Emerging Therapy Solutions, Gerson Lehman Group, GammaDelta Therapeutics, Iovance, Janssen, Kite (a Gilead Company), Legend Biotech, Novartis, Umoja, and Wugen; research funding from Allogene, Kite, and Novartis; and patents, royalties, and other intellectual property in the field of cellular immunotherapy. M.A. reports consulting or advisory role fees from BMS and Janssen and speakers’ bureau fees and honoraria from Janssen. J.M. reports honoraria from Kite Pharma, Juno Therapeutics, AlloVir, Magenta Therapeutics, and EcoR1 Capital; speakers’ bureau fees from Kite/Gilead; research funding from Novartis, Fresenius Biotech, Astellas Pharma, Bellicum Pharmaceuticals, Novartis, Gamida Cell, Pluristem Therapeutics, Kite Pharma, and AlloVir; honoraria from Kite, AlloVir, Juno Therapeutics, and Magenta Therapeutics; and travel, accommodations, and expenses from Kita Pharma. S.W. is a current employee of BMS. D.K.H. reports honoraria from OncLive and Multiple Myeloma Hub; research funding from BMS and Adaptive Biotech; and consulting or advisory role fees from BMS, Janssen, and Pfizer. The remaining authors declare no competing financial interests.

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