Long-term Outcomes of Oral Vinorelbine in Advanced, Progressive Desmoid Fibromatosis and Influence of CTNNB1 Mutational Status
Autor: | Françoise Rimareix, Olivier Mir, Leila Haddag-Miliani, Etienne Rouleau, Charles Honoré, Olaf Mercier, Carine Ngo, Anaïs Brunet, Andrea Cavalcanti, Charles Court, Sarah Dumont, Sylvain Briand, Axel Le Cesne, Cécile Le Péchoux, Julien Domont, Antonin Levy, Elie Fadel, Julien Adam, Ali N. Chamseddine, Matthieu Faron, Arnaud Bayle |
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Rok vydání: | 2020 |
Předmět: |
0301 basic medicine
Cancer Research medicine.medical_specialty business.industry Fibromatosis Cancer Neutropenia Vinorelbine medicine.disease Gastroenterology Confidence interval 03 medical and health sciences Regimen 030104 developmental biology 0302 clinical medicine Oncology 030220 oncology & carcinogenesis Concomitant Internal medicine Medicine business Progressive disease medicine.drug |
Zdroj: | Clinical Cancer Research. 26:6277-6283 |
ISSN: | 1557-3265 1078-0432 |
Popis: | Purpose: Desmoid-type fibromatosis (DF) are locally aggressive neoplasms, with a need for effective systemic treatment in case of progression to avoid the short- and long-term complications of local treatments. Experimental Design: We retrospectively analyzed the outcomes of adult patients with DF treated with oral vinorelbine (90 mg once weekly) at Gustave Roussy Cancer Institute (Villejuif, Paris, France). Only patients with documented progressive disease according to RECIST v1.1 for more than 3 months (±2 weeks) before treatment initiation were included. Results: From 2009 to 2019, 90 out of 438 patients with DF were eligible for this analysis. Vinorelbine was given alone in 56 patients (62%), or concomitantly with endocrine therapy in 34 patients, for a median duration of 6.7 months. A partial response was observed in 29% and stable disease in another 57%. With a median follow-up of 52.4 months, the median time to treatment failure (TTF) was not reached. Progression-free rates at 6 and 12 months were 88.7% and 77.5%, respectively. Concomitant endocrine therapy was associated with longer TTF in women [HR, 2.16; 95% confidence interval (CI), 1.06–4.37; P = 0.03). Among 64 patients with documented CTNNB1 mutational status, p.S45F or p.S45P mutations were associated with longer TTF compared with p.T41A or wild-type tumors (HR, 2.78; 95% CI, 1.23–6.27; P = 0.04). Toxicity profile was favorable, without grade 3–4 toxicity, except for one grade 3 neutropenia. Conclusions: Oral vinorelbine is an effective, affordable, and well-tolerated regimen in patients with advanced, progressive DF. Prolonged activity was observed in patients with tumors harboring CTNNB1 p.S45F or p.S45P mutations. |
Databáze: | OpenAIRE |
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