Management and survival of patients with cancer of unknown primary discussed by a French national multidisciplinary tumour board: a retrospective analysis
Menée en France à partir de données clinico-pathologiques portant sur 246 patients atteints d'un cancer d'origine primitive inconnue, cette étude examine l'utilisation des données moléculaires et clinico-pathologiques pour orienter la prise en charge thérapeutique ainsi que le recours à un traitement basé sur les recommandations du comité national multidisciplinaires des tumeurs (CNMT) puis compare la survie globale des patients en fonction de la manière dont le traitement a été sélectionné (choix en fonction des recommandations du CNMT ou non)
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.
The Lancet Regional Health - Europe , article en libre accès, 2025