Clinical Trial Endpoints in Metastatic Cancer: Using Individual Participant Data to Inform Future Trials Methodology
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- Richard M Goldberg
- West Virginia University Cancer Institute , Morgantown, WV, USA
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- Richard Adams
- Cardiff University and Velindre UNHS Trust , UK
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- Marc Buyse
- International Drug Development Institute (IDDI) , Louvain-la-Neuve, Belgium
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- Cathy Eng
- Vanderbilt-Ingram Cancer Center , Nashville, TN, USA
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- Axel Grothey
- West Cancer Center and Research Institute , Germantown, TN, USA
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- Thierry André
- Hôpital Saint Antoine , Paris, France
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- Alberto F Sobrero
- Ospedale S. Martin , Genova, Italy
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- Stuart M Lichtman
- Memorial Sloan Kettering Cancer Center , NY, USA
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- Al B Benson
- Robert H. Lurie Comprehensive Cancer Center of Northwestern University , Chicago, IL, USA
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- Cornelis J A Punt
- University Medical Centre Utrecht , the Netherlands
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- Tim Maughan
- Gray Institute of Radiation Oncology and Biology, University of Oxford , UK
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- Tomasz Burzykowski
- International Drug Development Institute (IDDI) , Louvain-la-Neuve, Belgium
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- Dirkje Sommeijer
- University of Amsterdam Academic Medical Centre and Flevohospital , Almere, the Netherlands
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- Everardo D Saad
- International Drug Development Institute (IDDI) , Louvain-la-Neuve, Belgium
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- Qian Shi
- Mayo Clinic , Rochester, MN, USA
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- Elisabeth Coart
- International Drug Development Institute (IDDI) , Louvain-la-Neuve, Belgium
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- Benoist Chibaudel
- Hôpital Franco-Britannique , Paris, France
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- Miriam Koopman
- University Medical Centre Utrecht , the Netherlands
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- Hans-Joachim Schmoll
- Martin Luther University , Halle, Germany
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- Takayuki Yoshino
- National Cancer Center Hospital East , Kashiwa, Japan
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- Julien Taieb
- Georges Pompidou European Hospital , Paris, France
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- Niall C Tebbutt
- Austin Health , Heidelberg, Victoria, Australia
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- John Zalcberg
- Monash University, School of Public Health , Australia
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- Josep Tabernero
- Vall d’Hebron Hospital Campus and Institute of Oncology (VHIO) , Barcelona, Spain
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- Eric Van Cutsem
- University Hospital Gasthuisberg , Leuven, Belgium
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- Alastair Matheson
- Fondation ARCAD , Paris, France
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- Aimery de Gramont
- Hôpital Franco-Britannique , Paris, France
抄録
<jats:title>Abstract</jats:title> <jats:p>Meta-analysis based on individual participant data (IPD) is a powerful methodology for synthesizing evidence by combining information drawn from multiple trials. Hitherto, its principal application has been in questions of clinical management, but an increasingly important use is in clarifying trials methodology, for instance in the selection of endpoints, as discussed in this review. In oncology, the Aide et Recherche en Cancérologie Digestive (ARCAD) Metastatic Colorectal Cancer Database is a leader in the use of IPD-based meta-analysis in methodological research. The ARCAD database contains IPD from more than 38 000 patients enrolled in 46 studies and continues to collect phase III trial data. Here, we review the principal findings of the ARCAD project in respect of endpoint selection and examine their implications for cancer trials. Analysis of the database has confirmed that progression-free survival (PFS) is no longer a valid surrogate endpoint predictive of overall survival in the first-line treatment of colorectal cancer. Nonetheless, PFS remains an endpoint of choice for most first-line trials in metastatic colorectal cancer and other solid tumors. Only substantial PFS effects are likely to translate into clinically meaningful benefits, and accordingly, we advocate an oncology research model designed to identify highly effective treatments in carefully defined patient groups. We also review the use of the ARCAD database in assessing clinical response including novel response metrics and prognostic markers. These studies demonstrate the value of IPD as a tool for methodological studies and provide a reference point for the expansion of this approach within clinical cancer research.</jats:p>
収録刊行物
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- JNCI: Journal of the National Cancer Institute
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JNCI: Journal of the National Cancer Institute 114 (6), 819-828, 2021-12-04
Oxford University Press (OUP)
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キーワード
詳細情報 詳細情報について
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- CRID
- 1360298763130962688
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- ISSN
- 14602105
- 00278874
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- データソース種別
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- Crossref