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Identification of Adjustment Variables in Indirect Comparisons: A Rapid Review of CAR-T Therapies for Diffuse Large B-Cell Lymphoma.

作者信息

Riou Sybille, Rungaldier Stefanie, Mahlich Jörg

机构信息

Miltenyi Biomedicine, Friedrich-Ebert-Straße 68, 51429 Bergisch Gladbach, Germany.

Düsseldorf Institute for Competition Economics (DICE), Heinrich-Heine-University Düsseldorf, Universitätsstraße 1, 40225 Düsseldorf, Germany.

出版信息

Cancers (Basel). 2025 Apr 15;17(8):1335. doi: 10.3390/cancers17081335.


DOI:10.3390/cancers17081335
PMID:40282511
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12025827/
Abstract

: Chimeric antigen receptor T-cell (CAR-T) therapies have been approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of diffuse large B-cell lymphoma (DLBCL), primarily based on single-arm trials or indirect comparisons with stem cell transplantation. However, no direct head-to-head comparisons of CAR-T therapies have been conducted, largely due to their high cost. To assess their true value, indirect treatment comparisons (ITCs) are essential. These comparisons, however, are prone to confounding biases, which necessitate careful adjustments through the identification and measurement of relevant variables. : This study aims to identify the variables used for adjustment in ITCs of CAR-T therapies for DLBCL and examine the methodologies employed to select them. A rapid literature review was conducted in PubMed in September 2023, focusing on ITCs involving CAR-T therapies for DLBCL. The search was based on keywords categorized into three groups: techniques (ITCs and related terms), drugs (CAR-T therapies), and indication (DLBCL). : The rapid literature review identified 21 articles, of which 11 were selected for analysis. Exclusions were made for articles that did not identify confounders, were letters to editors, or addressed conditions other than DLBCL. Among the 11 selected publications, 10 did not clearly specify the methodology used to identify adjustment variables. A total of 25 potential confounders were identified across the studies, with substantial variability in the set of variables used, reflecting a lack of standardization in confounder selection. Commonly identified confounders included the number of prior treatment lines and Eastern Cooperative Oncology Group Performance Status (ECOG PS), although their inclusion as adjustment variables in ITCs was inconsistent, often due to missing data. : While the identified confounders are clinically relevant, the methodologies for selecting them remain unclear, resulting in significant variability across studies. Additionally, key variables commonly considered in health technology assessments (HTAs), such as age, sex, and disease severity, were inconsistently incorporated into ITCs. To improve the reliability and consistency of ITC outcomes, there is a pressing need for standardized methodologies for identifying and adjusting for confounders.

摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e69/12025827/c91f617db8a2/cancers-17-01335-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e69/12025827/30d7b94e3c93/cancers-17-01335-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e69/12025827/c91f617db8a2/cancers-17-01335-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e69/12025827/30d7b94e3c93/cancers-17-01335-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e69/12025827/c91f617db8a2/cancers-17-01335-g002.jpg

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Identification of Adjustment Variables in Indirect Comparisons: A Rapid Review of CAR-T Therapies for Diffuse Large B-Cell Lymphoma.

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引用本文的文献

[1]
Systematic literature review to identify prognostic factors of efficacy and safety outcomes of chimeric antigen receptor T-Cell therapies in diffuse large B-Cell lymphoma.

J Cancer Res Clin Oncol. 2025-7-7

本文引用的文献

[1]
Prospective Validation of CAR-HEMATOTOX and a Simplified Version Predict Survival in Patients with Large B-Cell Lymphoma Treated with Anti-CD19 CAR T-Cells: Data from CART-SIE Study.

Transplant Cell Ther. 2025-4

[2]
Two-year follow-up of lisocabtagene maraleucel in relapsed or refractory large B-cell lymphoma in TRANSCEND NHL 001.

Blood. 2024-2-1

[3]
Management Considerations for Patients With Primary Refractory and Early Relapsed Diffuse Large B-Cell Lymphoma.

Am Soc Clin Oncol Educ Book. 2023-1

[4]
Component Costs of CAR-T Therapy in Addition to Treatment Acquisition Costs in Patients with Multiple Myeloma.

Oncol Ther. 2023-6

[5]
Survival outcomes of patients newly diagnosed with diffuse large B-cell lymphoma: real-world evidence from a German claims database.

J Cancer Res Clin Oncol. 2023-8

[6]
Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health.

BMJ Evid Based Med. 2024-1-19

[7]
Effective bridging therapy can improve CD19 CAR-T outcomes while maintaining safety in patients with large B-cell lymphoma.

Blood Adv. 2023-6-27

[8]
Healthcare Resource Utilization and Associated Costs of German Patients with Diffuse Large B-Cell Lymphoma: A Retrospective Health Claims Data Analysis.

Oncol Ther. 2023-3

[9]
A real-world comparison of tisagenlecleucel and axicabtagene ciloleucel CAR T cells in relapsed or refractory diffuse large B cell lymphoma.

Nat Med. 2022-10

[10]
Outcomes of patients with aggressive B-cell lymphoma after failure of anti-CD19 CAR T-cell therapy: a DESCAR-T analysis.

Blood. 2022-12-15

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