Vandetanib for the treatment of advanced medullary thyroid cancer outside a trial: results from a French cohort.
Fiche publication
Date publication
janvier 2015
Auteurs
Membres identifiés du Cancéropôle Est :
Dr ZANETTA Sylvie
Tous les auteurs :
Chougnet CN, Borget I, Leboulleux S, de la Fouchardiere C, Bonichon F, Criniere L, Niccoli P, Bardet S, Schneegans O, Zanetta S, Schvartz C, Drui D, Chauffert B, Rohmer V, Schlumberger M
Lien Pubmed
Résumé
A randomized phase III trial demonstrated that vandetanib treatment is effective in patients with metastatic medullary thyroid cancer (MTC), leading to Regulatory Approvals, but its use may be associated with toxicities that require specific monitoring and management. The objective of the present study performed in France was to describe the toxicity profile and efficacy of vandetanib treatment when given outside any trial. Methods: 68 patients were treated with vandetanib in the frame of a temporary use authorization (ATU) in France from 08/2010 to 02/2012, when the drug was available on request for patients with locally advanced or metastatic MTC. Patients were registered by the French health authorities and characteristics, treatment parameters, toxicity profile and efficacy were retrospectively reviewed. Eight patients were excluded from the analysis because vandetanib treatment was not administered (n=3) or had been given in a trial before ATU (n=3), or was given for a non-MTC cancer (n=2). Results: Data from the 60 MTC patients were analyzed. Mean age was 58 [range 11-83] years, 39 patients were male, and 6 had hereditary MTC. 56 (93%) had metastatic disease in the mediastinum (82%), bones (65%), liver (53%) or lung (53%), and 4 had only locally advanced disease. At the time of study evaluation, with a median follow-up of 20 months and a median duration of treatment of 9.7 months (range: 0.3-36 months), 15 patients are continuing vandetanib treatment (range: 18-36 months). Median progression-free survival was 16.1 months. 25 patients discontinued treatment for disease progression (range: 0.3-29 months). Best tumor response was a complete response in one patient, a partial response in 12 (20%), stable disease in 33 (55%) and progression in 7 patients (12%). All patients had at least one AE during treatment: main AEs were skin toxicity, diarrhea and asthenia. 16 patients (27%) discontinued treatment for toxicity, and one patient died from vandetanib induced cardiac toxicity. Vandetanib is an effective option for patients with advanced MTC. AEs should be monitored carefully and should be minimized by educating both patients and care providers and by applying symptomatic treatment and dose reduction.
Référence
Thyroid. 2015 Jan 27.