Sorafenib in patients with locally advanced and metastatic chordomas: a phase II trial of the French Sarcoma Group (GSF/GETO)
Mené en France sur 27 patients atteints d'un chordome de stade localement avancé ou métastatique, cet essai multicentrique de phase II évalue l'efficacité, du point de vue du taux de survie sans progression à 9 mois, et la toxicité du sorafénib
Purpose : There is no consensual treatment for locally advanced or metastatic chordomas. Methods : We conducted a multicenter, open-label, uncontrolled phase II trial of sorafenib (800 mg/d). The primary endpoint was the 9-month progression-free rate according to RECIST 1.1. All patients had documented progressive disease at the time of study entry. Results : Twenty-seven patients were enrolled between May 2011 and January 2014. The median age was 64 (range, 30-86). There were 17 men and 10 women. Twelve patients had been previously treated with chemotherapy and molecularly targeted agents. The maximum toxicity grade per patient was Grade 3 in 21 cases (77.8%) and Grade 4 in 4 cases (14.8%). Sorafenib provided an intent-to-treat best objective response of 1/27 (3.7%; 95% CI: 0.1-19.0%), a 9-month progression-free rate of 73.0% (95% CI: 46.1-88.0) and a 12-month overall survival rate of 86.5% (95%-CI: 55.8-96.5). Survival curves were similar in pre-treated and not pre-treated patients. Discussion : Additional clinical trials further exploring sorafenib as a treatment of locally advanced or metastatic chordomas are warranted.