Advanced chordoma treated by first-line molecular targeted therapies: Outcomes and prognostic factors. A retrospective study of the French Sarcoma Group (GSF/GETO) and the Association des Neuro-Oncologues d'Expression Française (ANOCEF)

Loïc Lebellec, Bruno Chauffert, Jean Yves Blay, Axel Le Cesne, Christine Chevreau, Emmanuelle Bompas, François Bertucci, Didier Cupissol, Michel Fabbro, Esma Saada-Bouzid, Florence Duffaud, Loïc Feuvret, Alice Bonneville-Levard, Jacques Olivier Bay, Elodie Vauleon, Armelle Vinceneux, Georges Noel, Nicolas Penel, Olivier Mir

    Résultats de recherche: Contribution à un journalArticleRevue par des pairs

    46 Citations (Scopus)

    Résumé

    Background To assess the role of first-line Molecular Targeted Therapies (MTTs) in Advanced chordoma (AC) patients. Methods Retrospective study of 80 patients treated between January 2004 and December 2015 at 15 major French Sarcoma or Neurooncology Centres. Results The sex ratio M/F was 46/34. The median age was 59 (6–86) years. The primary sites were the sacrum (50, 62.5%), mobile spine (12, 15.0%), and skull base (18, 22.5%). Metastases were present in 28 patients (36.0%). The first line of MTTs consisted of imatinib (62, 77.5%), sorafenib (11, 13.7%), erlotinib (5, 6.3%), sunitinib (1, 1.2%) and temsirolimus (1, 1.2%). The reported responses were: partial response (5, 6.3%), stable disease (58, 72.5%), or progressive disease (10, 12.5%). Symptomatic improvement was seen in 28/66 assessable patients (42.4%) and was associated with an objective response occurrence (p = 0.005), imatinib (p = 0.020) or erlotinib use (p = 0.028). The median progression-free survival (PFS) was 9.4°months (95% CI, [6.8–16.1]). Two independent factors of poor prognosis for PFS were identified: a skull-based primary location (HR = 2.5, p = 0.019), and the interval between diagnosis and MTT of <52months (HR = 2.8, p < 0.001). The median overall survival (OS) was 4.4°years (95% CI, [3.8–5.6]). Four independent factors of poor prognosis for OS were identified: the presence of liver metastases (HR = 13.2, p < 0.001), pain requiring opioids (HR = 2.9, p = 0.012), skull-based primary location (HR = 19.7, p < 0.001), and prior radiotherapy (photon alone) (HR = 2.5, p = 0.024). The PFS and OS did not significantly differ between the MTT. Conclusions The prognostic factors identified require validation in an independent database but are potently useful to guide treatment decisions and design further clinical trials.

    langue originaleAnglais
    Pages (de - à)119-128
    Nombre de pages10
    journalEuropean Journal of Cancer
    Volume79
    Les DOIs
    étatPublié - 1 juil. 2017

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