Health-related quality of life in patients given ciltacabtagene autoleucel for relapsed or refractory multiple myeloma (CARTITUDE-1): A phase 1b–2, open-label study Journal Article


Authors: Martin, T.; Lin, Y.; Agha, M.; Cohen, A. D.; Htut, M.; Stewart, A. K.; Hari, P.; Berdeja, J. G.; Usmani, S. Z.; Yeh, T. M.; Olyslager, Y.; Goldberg, J. D.; Schecter, J. M.; Madduri, D.; Jackson, C. C.; Deraedt, W.; Gries, K. S.; Fastenau, J. M.; Trudeau, J. J.; Akram, M.; Pacaud, L.; Jakubowiak, A.; Jagannath, S.
Article Title: Health-related quality of life in patients given ciltacabtagene autoleucel for relapsed or refractory multiple myeloma (CARTITUDE-1): A phase 1b–2, open-label study
Abstract: Background: CARTITUDE-1 is a phase 1b–2 study evaluating ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor T cell therapy with two B-cell maturation antigen–targeting single-domain antibodies, in patients with relapsed or refractory multiple myeloma. Primary efficacy outcomes have previously been reported. Here, we report health-related quality of life (HRQOL) secondary outcomes evaluated using patient-reported outcomes. Methods: This single-arm, open-label, phwase 1b–2 study was done at 16 centres in the USA. Patients were aged 18 years or older with diagnosis of multiple myeloma and Eastern Cooperative Oncology Group performance status of 1 or less with three or more previous lines of therapy, or were double refractory to a proteasome inhibitor and immunomodulatory drug, and had received a proteasome inhibitor, immunomodulatory drug, and anti-CD38 antibody. A single cilta-cel infusion (target dose 0·75 × 106 CAR+ T cells per kg) was administered 5–7 days after lymphodepletion. Patient-reported outcomes were assessed using the European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire core 30-item, pre-specified items from the EORTC myeloma module, and EuroQol five-dimensional descriptive system questionnaire. Clinically meaningful changes in patient-reported outcomes were defined by anchor-based minimally important differences. This trial is registered with ClinicalTrials.gov, NCT03548207. This trial is completed but feeding into a long-term follow-up study. Findings: Between July 16, 2018, and Oct 7, 2019, 78 patients were enrolled and underwent apheresis in phase 2 of the study. 68 patients were treated (43 [63%] male, 49 [72%] White), and their patient-reported outcomes assessed (median follow-up 16·9 months, IQR 15·7–17·5). After infusion, a transient decline was observed, followed by improvements in global health status (mean change from baseline to day 464 +8·0 points, SD 20·9), physical (+4·6 points, 21·1), and emotional functional scales (+1·9 points, 23·7) over time, and declines for symptom-based scores (−14·1 pain, SD 31·5 and –15·4 fatigue; SD 29·5), indicating improved patient HRQOL following treatment with cilta-cel. Interpretation: These durable HRQOL improvements are consistent with clinical findings, in which a single cilta-cel infusion led to substantial and durable responses in heavily pre-treated patients with relapsed or refractory multiple myeloma. These results support the use of cilta-cel in patients with relapsed or refractory multiple myeloma. Funding: Janssen Research & Development and Legend Biotech USA. © 2022 Elsevier Ltd
Keywords: adult; protein expression; aged; unclassified drug; major clinical study; fludarabine; clinical trial; cancer growth; drug efficacy; flow cytometry; outcome assessment; follow up; follow-up studies; antineoplastic agent; cell viability; quality of life; proteasome inhibitor; multiple myeloma; phase 2 clinical trial; antineoplastic combined chemotherapy protocols; clinical assessment; cyclophosphamide; multicenter study; immunogenicity; immunomodulating agent; phase 1 clinical trial; visual analog scale; apheresis; patient-reported outcome; proteasome inhibitors; humans; human; male; female; article; b cell maturation antigen; ecog performance status; b-cell maturation antigen; european organization for research and treatment of cancer quality of life questionnaire core 30; european quality of life 5 dimensions questionnaire; ciltacabtagene autoleucel; cd38 antibody
Journal Title: The Lancet Haematology
Volume: 9
Issue: 12
ISSN: 2352-3026
Publisher: Elsevier Science, Inc.  
Date Published: 2022-12-01
Start Page: e897
End Page: e905
Language: English
DOI: 10.1016/s2352-3026(22)00284-8
PUBMED: 36215989
PROVIDER: scopus
DOI/URL:
Notes: Article -- Export Date: 3 January 2023 -- Source: Scopus
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  1. Saad Zafar Usmani
    327 Usmani