Efficacy and safety of regorafenib in ...
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Article dans une revue scientifique: Article original
PMID :
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Title :
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
Author(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]
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]
Journal title :
European Journal of Cancer
Abbreviated title :
Eur J Cancer
Volume number :
150
Pages :
p. 108-118
Publication date :
2021-06
ISSN :
1879-0852
English keyword(s) :
Metastatic chondrosarcoma
Progression after standard chemotherapy
Regorafenib
Progression after standard chemotherapy
Regorafenib
HAL domain(s) :
Sciences du Vivant [q-bio]
English abstract : [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 ...
Show more >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.Show less >
Show more >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.Show less >
Language :
Anglais
Audience :
Internationale
Popular science :
Non
Administrative institution(s) :
Université de Lille
CHU Lille
CHU Lille
Submission date :
2023-11-15T06:50:09Z
2024-04-19T12:01:21Z
2024-04-19T12:01:21Z
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