Management and survival of patients with cancer of unknown primary discussed by a French national multidisciplinary tumour board: a retrospective analysis
Year: 2025
Type: article
Study design: Observational Study
Abstract: Background: Recent clinical trials have shown that molecularly-guided treatments can improve survival in patients with cancers of unknown primary (CUP). However, the feasibility and clinical benefit of these treatments for CUP in a real-life setting remain uncertain. In France, a national multidisciplinary tumour board dedicated to patients with CUP (CUP MTB) was created in 2020, with the aims of coordinating pathological and molecular diagnostic analyses and providing a centralised expertise for therapeutic orientation. This study aimed at evaluating the diagnostic and therapeutic impact of the CUP MTB on patients with CUP in a national real-life setting. Methods: Patient and tumour characteristics, treatments and outcomes are collected prospectively. This study reports the diagnostic and therapeutic impact of all patients discussed in CUP_MTB between July 2020 and December 2023. The diagnostic impact was defined as the identification of a putative tissue of origin, and the initiation of a MTB-oriented treatment. Overall survival was estimated using the Kaplan-Meier method, and hazard ratios were calculated using Cox proportional hazard models. Findings: A total of 246 CUP patients were referred to CUP_MTB (124 females and 122 men); 187 (76%) underwent pathological and molecular characterizations as recommended by the MTB. Tumour profiling enabled the identification of a putative tissue of origin (TOO) in 130/187 (70%) patients. The most frequent TOO were gastrointestinal (n = 29; 22%), lung (n = 22; 17%), breast (n = 21; 16%), and kidney (n = 19; 15%). 149 (61%) patients received a treatment based on MTB recommendation. 111/149 (74.5%) patients received MTB-oriented treatment, including systemic treatment oriented towards the putative TOO (n = 95, 63.8%), or treatment directed towards a targetable molecular alteration (n = 16, 10.7%). 38 (25.5%) patients for whom no MTB-oriented treatment could be recommended were treated with empiric treatment according to international guidelines. The median overall survival of patients treated with MTB-oriented treatment was 18.6 (IQR = 12.0) months, compared to 11.0 (IQR = 10.5) months in patients with empiric treatment (HR = 0.61, 95% CI 0.38-0.98, p = 0.04). Interpretation: Integration of clinical, pathological and molecular data within an expert MTB is feasible in a real-life setting, enables access to molecularly guided treatments and improves survival for a large proportion of CUP patients. Our findings highlight the benefits of dedicated MTB and reference centres to improve the management of CUP. Funding: Institut Curie and the 2025 French Genomic Medicine Initiative.
Authors: Célia Dupain, Nicolas Jacquin, Aurélien Latouche, Zoé Nevière, Pierre Gestraud, Abderaouf Hamza, Kenza Nedara, Vincent Cockenpot, Janick Selves, Yves Allory, Laëtitia Chanas, Maud Milder, Isabelle Soubeyran, Hélène Blons, Anna Patrikidou, Axel de Bernardi, Julien Masliah-Planchon, Odette Mariani, Etienne Rouleau, Fabienne Escande, Sandrine Boyault, Pierre Saintigny, Florence de Fraipont, Pierre Blanc, Jennifer Wong, Camille Tlemsani, Isabelle Guillou, Julie Flavius, Noemie Fuentealba, Maud Kamal, Ivan Bièche, Nicolas Servant, Christophe Le Tourneau, Sarah Watson
Institutions: Institut Curie, Institut Jean Godinot, Conservatoire National des Arts et Métiers, Inserm, Centre François Baclesse, École Nationale Supérieure des Mines de Paris, Centre National de la Recherche Scientifique, Université Paris Cité, Institut Cochin, Université Toulouse III - Paul Sabatier, Centre Hospitalier Universitaire de Toulouse, Institut universitaire du cancer de Toulouse Oncopole, Institut Bergonié, Assistance Publique – Hôpitaux de Paris, Hôpital Européen Georges-Pompidou, Université Paris-Saclay, Institut Gustave Roussy, Centre Léon Bérard, Centre Hospitalier Universitaire de Lille, Lyon 1 Université, Centre de Recherche en Cancérologie de Lyon, Centre Hospitalier Universitaire de Grenoble, Hôpital Cochin, Université Paris Sciences et Lettres, Chimie et Biologie de la Cellule
Language: English
Subfield: Oncology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $4,930
Funder: Institut Curie
OpenAlex ID: W4416025065