Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.

Real world effectiveness, toxicity and costs analyses from chimeric antigen receptor (CAR)-T cell therapy are of utmost relevance to determine whether and how to offer patients highly personalized immunotherapy. In this study, we aimed at describing CAR T-cells effectiveness, safety and costs in a P...

Full description

Bibliographic Details
Main Authors: Sérgio Chacim, Teresa Monjardino, José Luís Cunha, Pedro Medeiros, Patrícia Redondo, Maria José Bento, José Mário Mariz
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2022-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0278950
_version_ 1811177206408282112
author Sérgio Chacim
Teresa Monjardino
José Luís Cunha
Pedro Medeiros
Patrícia Redondo
Maria José Bento
José Mário Mariz
author_facet Sérgio Chacim
Teresa Monjardino
José Luís Cunha
Pedro Medeiros
Patrícia Redondo
Maria José Bento
José Mário Mariz
author_sort Sérgio Chacim
collection DOAJ
description Real world effectiveness, toxicity and costs analyses from chimeric antigen receptor (CAR)-T cell therapy are of utmost relevance to determine whether and how to offer patients highly personalized immunotherapy. In this study, we aimed at describing CAR T-cells effectiveness, safety and costs in a Portuguese Comprehensive Cancer Center. We performed a retrospective descriptive study of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma and transformed follicular lymphoma referred to CAR T-cell therapy, between May 2019 and February 2021. Rates of treatment response, toxicity and survival (Kaplan-Meier method) were analyzed by intention-to-treat. Direct medical costs stratified by inpatient-care, outpatient-care, and diagnostic-therapeutic procedures (DTP) were derived based on resources used and their respective unit costs. In twenty patients (median age 49.5y; 55%male; 70%DLBCL; 50% with primary refractory disease), best overall and complete response rates were 65.0% and 45.0%, respectively. Median overall (OS) and progression-free survivals were 9.2 and 7.3 months; 12-month OS rate was 42.6% (95%CI:23.2-78.3). Grade≥3 cytokine release syndrome and neurotoxicity occurred in 5.6% and 11.1% of patients, respectively. CAR T-cell therapy expenditure, including adverse events costs, was 7 176 196€, or 286 238€ when excluding drug cost. Median cost for treated patient was 355 165€ with CAR T-cell drug cost accounting for 97.0% of the overall expense. Excluding CAR T-cell acquisition cost, inpatient-care and DTP accounted for 57% and 38% of total cost/patient, respectively. Our findings highlight the heavy economic burden of CAR T-cell therapy driven by drug acquisition costs.
first_indexed 2024-04-10T22:57:52Z
format Article
id doaj.art-429cbcfea92e404fae65b7fa173348ba
institution Directory Open Access Journal
issn 1932-6203
language English
last_indexed 2024-04-10T22:57:52Z
publishDate 2022-01-01
publisher Public Library of Science (PLoS)
record_format Article
series PLoS ONE
spelling doaj.art-429cbcfea92e404fae65b7fa173348ba2023-01-14T05:31:38ZengPublic Library of Science (PLoS)PLoS ONE1932-62032022-01-011712e027895010.1371/journal.pone.0278950Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.Sérgio ChacimTeresa MonjardinoJosé Luís CunhaPedro MedeirosPatrícia RedondoMaria José BentoJosé Mário MarizReal world effectiveness, toxicity and costs analyses from chimeric antigen receptor (CAR)-T cell therapy are of utmost relevance to determine whether and how to offer patients highly personalized immunotherapy. In this study, we aimed at describing CAR T-cells effectiveness, safety and costs in a Portuguese Comprehensive Cancer Center. We performed a retrospective descriptive study of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma and transformed follicular lymphoma referred to CAR T-cell therapy, between May 2019 and February 2021. Rates of treatment response, toxicity and survival (Kaplan-Meier method) were analyzed by intention-to-treat. Direct medical costs stratified by inpatient-care, outpatient-care, and diagnostic-therapeutic procedures (DTP) were derived based on resources used and their respective unit costs. In twenty patients (median age 49.5y; 55%male; 70%DLBCL; 50% with primary refractory disease), best overall and complete response rates were 65.0% and 45.0%, respectively. Median overall (OS) and progression-free survivals were 9.2 and 7.3 months; 12-month OS rate was 42.6% (95%CI:23.2-78.3). Grade≥3 cytokine release syndrome and neurotoxicity occurred in 5.6% and 11.1% of patients, respectively. CAR T-cell therapy expenditure, including adverse events costs, was 7 176 196€, or 286 238€ when excluding drug cost. Median cost for treated patient was 355 165€ with CAR T-cell drug cost accounting for 97.0% of the overall expense. Excluding CAR T-cell acquisition cost, inpatient-care and DTP accounted for 57% and 38% of total cost/patient, respectively. Our findings highlight the heavy economic burden of CAR T-cell therapy driven by drug acquisition costs.https://doi.org/10.1371/journal.pone.0278950
spellingShingle Sérgio Chacim
Teresa Monjardino
José Luís Cunha
Pedro Medeiros
Patrícia Redondo
Maria José Bento
José Mário Mariz
Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
PLoS ONE
title Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
title_full Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
title_fullStr Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
title_full_unstemmed Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
title_short Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center.
title_sort costs effectiveness and safety associated with chimeric antigen receptor car t cell therapy results from a comprehensive cancer center
url https://doi.org/10.1371/journal.pone.0278950
work_keys_str_mv AT sergiochacim costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT teresamonjardino costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT joseluiscunha costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT pedromedeiros costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT patriciaredondo costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT mariajosebento costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter
AT josemariomariz costseffectivenessandsafetyassociatedwithchimericantigenreceptorcartcelltherapyresultsfromacomprehensivecancercenter