Patient-reported outcomes following ciltacabtagene autoleucel or standard of care in patients with lenalidomide-refractory multiple myeloma (CARTITUDE-4): results from a randomised, open-label, phase 3 trial
- Author(s)
- Mina, R; Mylin, AK; Yokoyama, H; Magen, H; Alsdorf, W; Minnema, MC; Shune, L; Isufi, I; Harrison, SJ; Shah, UA; Schecter, JM; Vogel, M; Lendvai, N; Gries, KS; Katz, EG; Slaughter, A; Lonardi, C; Gilbert, J; Li, Q; Deraedt, W; Filho, OC; Patel, N; Florendo, E; Karlin, L; Weisel, K;
- Details
- Publication Year 2025-01,Volume 12,Issue #1,Page e45-e56
- Journal Title
- Lancet Haematology
- Publication Type
- Research article
- Abstract
- BACKGROUND: In CARTITUDE-4, ciltacabtagene autoleucel (cilta-cel) significantly improved progression-free survival (primary endpoint; previously reported) versus standard of care in patients with relapsed, lenalidomide-refractory multiple myeloma. We report here patient-reported outcomes. METHODS: In the ongoing, phase 3, open-label CARTITUDE-4 study, patients were recruited from 81 sites in the USA, Europe, Asia, and Australia, and were randomly assigned 1:1 to cilta-cel (target, 0·75 × 10(6) CAR-T cells/kg) or standard of care (daratumumab, pomalidomide, and dexamethasone; pomalidomide, bortezomib, and dexamethasone). Eligible patients had relapsed, lenalidomide-refractory multiple myeloma, received one to three previous treatment lines including a proteasome inhibitor and an immunomodulatory drug, and had an ECOG performance status of 0 or 1. Secondary endpoints reported here include time to sustained worsening of symptoms (Multiple Myeloma Symptom and Impact Questionnaire [MySIm-Q]; a key secondary endpoint) and change in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life (QoL) Questionnaire Core C30 (intention-to-treat population) and EuroQol 5-Dimension 5-Level (EQ-5D-5L; intention-to-treat population). This study is registered with ClinicalTrials.gov number NCT04181827 and is ongoing. FINDINGS: Patients were enrolled from July 10, 2020, to Nov 17, 2021, and 419 of 516 screened patients were randomly assigned (cilta-cel, n=208; standard of care, n=211; median follow-up, 15·9 months [IQR 12·4 to 17·8]); median age was 61 years. 191 (92%) of 208 patients in the cilta-cel group and 190 (91%) of 209 evaluable patients in the standard- of-care group completed baseline assessments. MySIm-Q compliance post-baseline was 70 to 81% (cilta-cel) and 79 to 89% (standard of care). MySIm-Q median time to sustained symptom worsening with cilta-cel versus standard of care was 23·7 versus 18·9 months (HR 0·42; 95% CI 0·26 to 0·68). 12-month mean changes for EORTC global health status (GHS) were +10·1 (95% CI 7·0 to 13·1) and -1·5 (95% CI -5·3 to 2·3) points and were +8·0 (95% CI 5·2 to 10·7) and +1·4 (95% CI -1·9 to 4·7) points for EQ-5D-5L visual analogue scale (VAS). Rates of clinically meaningful improvements in GHS and VAS were higher with cilta-cel than with standard of care. INTERPRETATION: Health-related QoL improvements and delayed symptom worsening support cilta-cel's clinical efficacy in lenalidomide-refractory disease. FUNDING: Janssen Research & Development, Legend Biotech USA.
- Publisher
- Elsevier
- Keywords
- Humans; *Multiple Myeloma/drug therapy; *Lenalidomide/therapeutic use; Male; Female; Middle Aged; *Patient Reported Outcome Measures; Aged; *Standard of Care; Thalidomide/analogs & derivatives/therapeutic use; Immunotherapy, Adoptive/methods; Quality of Life; Dexamethasone/therapeutic use; Adult; Antineoplastic Combined Chemotherapy Protocols/therapeutic use; Bortezomib/therapeutic use; Antibodies, Monoclonal
- Department(s)
- Haematology
- Publisher's Version
- https://doi.org/10.1016/s2352-3026(24)00320-x
- Terms of Use/Rights Notice
- Refer to copyright notice on published article.
Creation Date: 2025-01-21 06:45:02
Last Modified: 2025-01-21 06:46:48