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Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma

Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen–directed chimeric antigen receptor T cell therapy, showed deep, durable responses in patients with triple-class exposed, relapsed and refractory multiple myeloma (RRMM) in the phase 2 KarMMa (Efficacy and Safety Study of bb2121 in Subject...

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Autores principales: Delforge, Michel, Shah, Nina, Miguel, Jesús San F., Braverman, Julia, Dhanda, Devender S., Shi, Ling, Guo, Shien, Yu, Peiwen, Liao, Weiqin, Campbell, Timothy B., Munshi, Nikhil C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Hematology 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8864645/
https://www.ncbi.nlm.nih.gov/pubmed/34933328
http://dx.doi.org/10.1182/bloodadvances.2021005913
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author Delforge, Michel
Shah, Nina
Miguel, Jesús San F.
Braverman, Julia
Dhanda, Devender S.
Shi, Ling
Guo, Shien
Yu, Peiwen
Liao, Weiqin
Campbell, Timothy B.
Munshi, Nikhil C.
author_facet Delforge, Michel
Shah, Nina
Miguel, Jesús San F.
Braverman, Julia
Dhanda, Devender S.
Shi, Ling
Guo, Shien
Yu, Peiwen
Liao, Weiqin
Campbell, Timothy B.
Munshi, Nikhil C.
author_sort Delforge, Michel
collection PubMed
description Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen–directed chimeric antigen receptor T cell therapy, showed deep, durable responses in patients with triple-class exposed, relapsed and refractory multiple myeloma (RRMM) in the phase 2 KarMMa (Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma) trial. We assessed health-related quality of life (HRQoL) among KarMMa patients. The European Organization for Research and Treatment of Cancer Quality of Life C30 Questionnaire and its supplementary 20-item multiple myeloma module, as well as the EuroQol 5-dimension 5-level instrument, were administered at screening, baseline (≤72 hours before or same day as lymphodepletion), day of ide-cel treatment, and after ide-cel treatment. Mean changes from baseline that exceeded the predetermined threshold of minimally important difference were deemed clinically meaningful. The proportions of patients experiencing clinically meaningful changes in HRQoL were assessed using within-patient change thresholds. Time to stable improvement (≥2 consecutive visits with clinically meaningful HRQoL improvements) was analyzed by using the Kaplan-Meier method. A total of 126 (98%) of 128 patients treated with ide-cel were included in the HRQoL analysis. Pretreatment baseline RRMM burden was high and meaningfully worse than that in the age- and sex-weighted general population. Statistically significant and clinically meaningful improvements from baseline were observed by month 1 for pain (−8.9) and disease symptoms (−10.2), and by month 2 for fatigue (−7.2), physical functioning (6.1), cognitive functioning (6.7), and global health status/QoL (8.0). Clinically meaningful improvements in fatigue, pain, and physical functioning were most prominent at months 9, 12, and 18, respectively, and were sustained through 15 to 18 months after ide-cel treatment. For triple-class exposed patients with RRMM with a poor prognosis and few treatment options, a single ide-cel infusion provides early, sustained, statistically significant, and clinically meaningful improvements in HRQoL. This study was registered at Clinicaltrials.gov as #NCT03361748.
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spelling pubmed-88646452022-02-23 Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma Delforge, Michel Shah, Nina Miguel, Jesús San F. Braverman, Julia Dhanda, Devender S. Shi, Ling Guo, Shien Yu, Peiwen Liao, Weiqin Campbell, Timothy B. Munshi, Nikhil C. Blood Adv Health Services and Outcomes Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen–directed chimeric antigen receptor T cell therapy, showed deep, durable responses in patients with triple-class exposed, relapsed and refractory multiple myeloma (RRMM) in the phase 2 KarMMa (Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma) trial. We assessed health-related quality of life (HRQoL) among KarMMa patients. The European Organization for Research and Treatment of Cancer Quality of Life C30 Questionnaire and its supplementary 20-item multiple myeloma module, as well as the EuroQol 5-dimension 5-level instrument, were administered at screening, baseline (≤72 hours before or same day as lymphodepletion), day of ide-cel treatment, and after ide-cel treatment. Mean changes from baseline that exceeded the predetermined threshold of minimally important difference were deemed clinically meaningful. The proportions of patients experiencing clinically meaningful changes in HRQoL were assessed using within-patient change thresholds. Time to stable improvement (≥2 consecutive visits with clinically meaningful HRQoL improvements) was analyzed by using the Kaplan-Meier method. A total of 126 (98%) of 128 patients treated with ide-cel were included in the HRQoL analysis. Pretreatment baseline RRMM burden was high and meaningfully worse than that in the age- and sex-weighted general population. Statistically significant and clinically meaningful improvements from baseline were observed by month 1 for pain (−8.9) and disease symptoms (−10.2), and by month 2 for fatigue (−7.2), physical functioning (6.1), cognitive functioning (6.7), and global health status/QoL (8.0). Clinically meaningful improvements in fatigue, pain, and physical functioning were most prominent at months 9, 12, and 18, respectively, and were sustained through 15 to 18 months after ide-cel treatment. For triple-class exposed patients with RRMM with a poor prognosis and few treatment options, a single ide-cel infusion provides early, sustained, statistically significant, and clinically meaningful improvements in HRQoL. This study was registered at Clinicaltrials.gov as #NCT03361748. American Society of Hematology 2022-02-17 /pmc/articles/PMC8864645/ /pubmed/34933328 http://dx.doi.org/10.1182/bloodadvances.2021005913 Text en © 2022 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.
spellingShingle Health Services and Outcomes
Delforge, Michel
Shah, Nina
Miguel, Jesús San F.
Braverman, Julia
Dhanda, Devender S.
Shi, Ling
Guo, Shien
Yu, Peiwen
Liao, Weiqin
Campbell, Timothy B.
Munshi, Nikhil C.
Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title_full Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title_fullStr Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title_full_unstemmed Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title_short Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
title_sort health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
topic Health Services and Outcomes
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8864645/
https://www.ncbi.nlm.nih.gov/pubmed/34933328
http://dx.doi.org/10.1182/bloodadvances.2021005913
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