OBJECTIVES: To assess clinical benefit of plitidepsin (Aplidine) in patients with advanced medullary thyroid carcinoma (MTC). MATERIALS AND METHODS: We retrospectively reported the outcome of 10 patients with advanced MTC among 215 patients who have entered the phase I program with plitidepsin. RESULTS: Median number of cycles was 5. Using World Health Organization criteria, 1 among 5 patients with measurable disease displayed a confirmed partial response, whereas 8 patients experienced a stable disease, and 1 patient had a progressive disease, corresponding to a disease control rate of 90%. Two patients treated at the maximum tolerated dose experienced muscular dose-limiting toxicity possibly related to palmitoyl transferase inhibition. One of these 2 patients was able to continue therapy with no dose reduction with the prophylactic addition of l-carnitine, which is used in the treatment of the carnitine palmitoyl transferase deficiency type 2. DISCUSSION: Plitidepsin seems to be able to induce clinical benefit in patients with pretreated MTC, and its toxicity has been manageable at the recommended dose.
OBJECTIVES: To assess clinical benefit of plitidepsin (Aplidine) in patients with advanced medullary thyroid carcinoma (MTC). MATERIALS AND METHODS: We retrospectively reported the outcome of 10 patients with advanced MTC among 215 patients who have entered the phase I program with plitidepsin. RESULTS: Median number of cycles was 5. Using World Health Organization criteria, 1 among 5 patients with measurable disease displayed a confirmed partial response, whereas 8 patients experienced a stable disease, and 1 patient had a progressive disease, corresponding to a disease control rate of 90%. Two patients treated at the maximum tolerated dose experienced muscular dose-limiting toxicity possibly related to palmitoyl transferase inhibition. One of these 2 patients was able to continue therapy with no dose reduction with the prophylactic addition of l-carnitine, which is used in the treatment of the carnitine palmitoyl transferase deficiency type 2. DISCUSSION: Plitidepsin seems to be able to induce clinical benefit in patients with pretreated MTC, and its toxicity has been manageable at the recommended dose.
Authors: Pablo E Morande; Samanta R Zanetti; Mercedes Borge; Paula Nannini; Carolina Jancic; Raimundo F Bezares; Alicia Bitsmans; Miguel González; Andrea L Rodríguez; Carlos M Galmarini; Romina Gamberale; Mirta Giordano Journal: Invest New Drugs Date: 2011-09-02 Impact factor: 3.850
Authors: Leslie D Alexander; Robert P Sellers; Melinda R Davis; Veronica C Ardi; Victoria A Johnson; Robert C Vasko; Shelli R McAlpine Journal: J Med Chem Date: 2009-12-24 Impact factor: 7.446
Authors: Sara Alonso-Álvarez; Emilia Pardal; Diego Sánchez-Nieto; Miguel Navarro; Maria Dolores Caballero; Maria Victoria Mateos; Alejandro Martín Journal: Drug Des Devel Ther Date: 2017-01-19 Impact factor: 4.162