Tumor Growth Rate Informs Treatment Efficacy in Metastatic Pancreatic Adenocarcinoma: Application of a Growth and Regression Model to Pivotal Trial and Real-World Data.


Journal

The oncologist
ISSN: 1549-490X
Titre abrégé: Oncologist
Pays: England
ID NLM: 9607837

Informations de publication

Date de publication:
08 02 2023
Historique:
received: 13 04 2022
accepted: 26 09 2022
pubmed: 12 11 2022
medline: 11 2 2023
entrez: 11 11 2022
Statut: ppublish

Résumé

Methods for screening agents earlier in development and strategies for conducting smaller randomized controlled trials (RCTs) are needed. We retrospectively applied a tumor growth model to estimate the rates of growth of pancreatic cancer using radiographic tumor measurements or serum CA 19-9 values from 3033 patients with stages III-IV PDAC who were enrolled in 8 clinical trials or were included in 2 large real-world data sets. g correlated inversely with OS and was consistently lower in the experimental arms than in the control arms of RCTs. At the individual patient level, g was significantly faster for lesions metastatic to the liver relative to those localized to the pancreas. Regardless of regimen, g increased toward the end of therapy, often by over 3-fold. Growth rates of PDAC can be determined using radiographic tumor measurement and CA 19-9 values. g is inversely associated with OS and can differentiate therapies within the same trial and across trials. g can also be used to characterize changes in the behavior of an individual's PDAC, such as differences in the growth rate of lesions based on metastatic site, and the emergence of chemoresistance. We provide examples of how g can be used to benchmark phase II and III clinical data to a virtual reference arm to inform go/no go decisions and consider novel trial designs to optimize and accelerate drug development.

Sections du résumé

BACKGROUND
Methods for screening agents earlier in development and strategies for conducting smaller randomized controlled trials (RCTs) are needed.
METHODS
We retrospectively applied a tumor growth model to estimate the rates of growth of pancreatic cancer using radiographic tumor measurements or serum CA 19-9 values from 3033 patients with stages III-IV PDAC who were enrolled in 8 clinical trials or were included in 2 large real-world data sets.
RESULTS
g correlated inversely with OS and was consistently lower in the experimental arms than in the control arms of RCTs. At the individual patient level, g was significantly faster for lesions metastatic to the liver relative to those localized to the pancreas. Regardless of regimen, g increased toward the end of therapy, often by over 3-fold.
CONCLUSIONS
Growth rates of PDAC can be determined using radiographic tumor measurement and CA 19-9 values. g is inversely associated with OS and can differentiate therapies within the same trial and across trials. g can also be used to characterize changes in the behavior of an individual's PDAC, such as differences in the growth rate of lesions based on metastatic site, and the emergence of chemoresistance. We provide examples of how g can be used to benchmark phase II and III clinical data to a virtual reference arm to inform go/no go decisions and consider novel trial designs to optimize and accelerate drug development.

Identifiants

pubmed: 36367377
pii: 6823566
doi: 10.1093/oncolo/oyac217
pmc: PMC9907043
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Pagination

139-148

Informations de copyright

Published by Oxford University Press 2022.

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Auteurs

Celine Yeh (C)

Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA.

Mengxi Zhou (M)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Keith Sigel (K)

Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Gayle Jameson (G)

Department of Medical Oncology/Hematology, HonorHealth Research Institute, Scottsdale, AZ, USA.

Ruth White (R)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Rachael Safyan (R)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Yvonne Saenger (Y)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Elizabeth Hecht (E)

Department of Radiology, Weill Cornell Medicine, New York, NY, USA.

John Chabot (J)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Stephen Schreibman (S)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Béata Juzyna (B)

R&D UNICANCER, Fédération Nationale des Centres de Lutte Contre le Cancer, Paris, France.

Marc Ychou (M)

Department of Medical Oncology, Institut Régional du Cancer de Montpellier (ICM), Montpellier, France.

Thierry Conroy (T)

Department of Medical Oncology, Institut de Cancérologie de Lorraine, Vandoeuvre-lès-Nancy Cedex, France.

Tito Fojo (T)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.
Hematology/Oncology, James J. Peters VA Medical Center, Bronx, NY, USA.

Gulam A Manji (GA)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.

Daniel Von Hoff (D)

Virginia G. Piper Cancer Center Clinical Trials, HonorHealth Research Institute, Scottsdale, AZ, USA.
Translational Genomics Research Institute, Clinical Translational Research Division, Phoenix, AZ, USA.

Susan E Bates (SE)

Department of Medicine, Division of Hematology/Oncology, Columbia University Herbert Irving Comprehensive Cancer Center, New York, NY, USA.
Hematology/Oncology, James J. Peters VA Medical Center, Bronx, NY, USA.

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