Titre :

Efficacy and safety of regorafenib in patients with metastatic or locally advanced chondrosarcoma: Results of a non-comparative, randomised, double-blind, placebo controlled, multicentre phase II study

Auteur(s) :

Duffaud, F. [Auteur]
Italiano, A. [Auteur]
Bompas, E. [Auteur]
Rios, M. [Auteur]
Penel, Nicolas [Auteur]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Mir, O. [Auteur]
Piperno-Neumann, S. [Auteur]
Chevreau, C. [Auteur]
Delcambre, C. [Auteur]
Bertucci, F. [Auteur]
Boudou-Rouquette, P. [Auteur]
Cancel, M. [Auteur]
Perrin, C. [Auteur]
Saada-Bouzid, E. [Auteur]
Monard, L. [Auteur]
Schiffler, C. [Auteur]
Chaigneau, L. [Auteur]
Hervieu, A. [Auteur]
Collard, O. [Auteur]
Bouvier, C. [Auteur]
Vidal, V. [Auteur]
Chabaud, S. [Auteur]
Blay, J. Y. [Auteur]

Titre de la revue :

European Journal of Cancer

Nom court de la revue :

Eur J Cancer

Numéro :

150

Pagination :

p. 108-118

Date de publication :

2021-06

ISSN :

1879-0852

Mot(s)-clé(s) en anglais :

Metastatic chondrosarcoma
Progression after standard chemotherapy
Regorafenib

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Background This multi-cohort trial explored the efficacy and safety of regorafenib for patients with advanced sarcomas of bone origin; this report details the cohort of patients with metastatic or locally advanced ...
Lire la suite >Background This multi-cohort trial explored the efficacy and safety of regorafenib for patients with advanced sarcomas of bone origin; this report details the cohort of patients with metastatic or locally advanced chondrosarcoma (CS), progressing after prior chemotherapy. Patients and methods Patients with CS, progressing despite prior standard therapy, were randomised (2:1) to receive regorafenib or placebo. Patients on placebo could crossover to receive regorafenib after centrally confirmed progressive disease. The primary endpoint was progression-free rate (PFR) at 12 weeks. With one-sided α of 0.05, and 80% power, at least 16/24 progression-free patients at 12 weeks were needed for success (P0 = 50%, P1 = 75%). Results From September 2014 to February 2019, 46 patients were included in the CS cohort, and 40 patients were evaluable for efficacy: 16 on placebo and 24 on regorafenib. Thirteen patients (54.2%; 95% CI [35.8%-[) were non-progressive at 12 weeks on regorafenib versus 5 (31.3%; 95% CI [13.2%-[);) on placebo. Median PFS was 19.9 weeks on regorafenib, and 8.0 on placebo. Fourteen placebo patients crossed over to regorafenib after progression. The most common grade ≥3 treatment-related adverse events on regorafenib included hypertension (12%), asthenia (8%), thrombocytopenia (8%) and diarrhoea (8%). One episode of fatal liver dysfunction occurred on regorafenib. Conclusion Although the primary endpoint was not met statistically in this small randomised cohort, there is modest evidence to suggest that regorafenib might slow disease progression in patients with metastatic CS after the failure of prior chemotherapy.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
CHU Lille

Collections :

• METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694

Date de dépôt :

2023-11-15T06:50:09Z
2024-04-19T12:01:21Z