Regorafenib in patients with advanced Ewing ...
Document type :
Article dans une revue scientifique: Article original
PMID :
Permalink :
Title :
Regorafenib in patients with advanced Ewing sarcoma: results of a non-comparative, randomised, double-blind, placebo-controlled, multicentre Phase II study.
Author(s) :
Duffaud, Florence [Auteur]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Blay, Jean-Yves [Auteur]
Centre Léon Bérard [Lyon]
Le Cesne, Axel [Auteur]
Institut Gustave Roussy [IGR]
Chevreau, Christine [Auteur]
Institut Universitaire du Cancer de Toulouse - Oncopole [IUCT Oncopole - UMR 1037]
Boudou-Rouquette, Pascaline [Auteur]
Hôpital Cochin [AP-HP]
Kalbacher, Elsa [Auteur]
Centre Hospitalier Régional Universitaire de Besançon [CHRU Besançon]
Penel, Nicolas [Auteur]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Perrin, Christophe [Auteur]
CRLCC Eugène Marquis [CRLCC]
Laurence, Valérie [Auteur]
Institut Curie [Paris]
Bompas, Emmanuelle [Auteur]
CRLCC René Gauducheau
Saada Bouzid, Esma [Auteur]
Centre de Lutte contre le Cancer Antoine Lacassagne [Nice] [UNICANCER/CAL]
Delcambre, Corinne [Auteur]
Centre Régional de Lutte contre le Cancer François Baclesse [Caen] [UNICANCER/CRLC]
Bertucci, Francois [Auteur]
Institut Paoli-Calmettes [IPC]
Cancel, Mathilde [Auteur]
Centre Hospitalier Régional Universitaire de Tours [CHRU Tours]
Schiffler, Camille [Auteur]
Centre Léon Bérard [Lyon]
Monard, Laure [Auteur]
UNICANCER
Bouvier, Corinne [Auteur]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Vidal, Vincent [Auteur]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Gaspar, Nathalie [Auteur]
Institut Gustave Roussy [IGR]
Chabaud, Sylvie [Auteur]
Centre Léon Bérard [Lyon]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Blay, Jean-Yves [Auteur]
Centre Léon Bérard [Lyon]
Le Cesne, Axel [Auteur]
Institut Gustave Roussy [IGR]
Chevreau, Christine [Auteur]
Institut Universitaire du Cancer de Toulouse - Oncopole [IUCT Oncopole - UMR 1037]
Boudou-Rouquette, Pascaline [Auteur]
Hôpital Cochin [AP-HP]
Kalbacher, Elsa [Auteur]
Centre Hospitalier Régional Universitaire de Besançon [CHRU Besançon]
Penel, Nicolas [Auteur]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Perrin, Christophe [Auteur]
CRLCC Eugène Marquis [CRLCC]
Laurence, Valérie [Auteur]
Institut Curie [Paris]
Bompas, Emmanuelle [Auteur]
CRLCC René Gauducheau
Saada Bouzid, Esma [Auteur]
Centre de Lutte contre le Cancer Antoine Lacassagne [Nice] [UNICANCER/CAL]
Delcambre, Corinne [Auteur]
Centre Régional de Lutte contre le Cancer François Baclesse [Caen] [UNICANCER/CRLC]
Bertucci, Francois [Auteur]
Institut Paoli-Calmettes [IPC]
Cancel, Mathilde [Auteur]
Centre Hospitalier Régional Universitaire de Tours [CHRU Tours]
Schiffler, Camille [Auteur]
Centre Léon Bérard [Lyon]
Monard, Laure [Auteur]
UNICANCER
Bouvier, Corinne [Auteur]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Vidal, Vincent [Auteur]
Hôpital de la Timone [CHU - APHM] [TIMONE]
Gaspar, Nathalie [Auteur]
Institut Gustave Roussy [IGR]
Chabaud, Sylvie [Auteur]
Centre Léon Bérard [Lyon]
Journal title :
British Journal of Cancer
Abbreviated title :
Br J Cancer
Publication date :
2023-11-04
ISSN :
1532-1827
English abstract : [en]
Background
The REGOBONE multi-cohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the Ewing sarcoma (ES) cohort.
Methods
Patients with relapsed ...
Show more >Background The REGOBONE multi-cohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the Ewing sarcoma (ES) cohort. Methods Patients with relapsed ES 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 progression. The primary endpoint was the progression-free rate at 8 weeks. With one-sided α of 0.05, and 80% power, at least 14/24 progression-free patients at 8 weeks were needed for success. Results From September 2014 to November 2019, 41 patients were accrued. 36 patients were evaluable for efficacy: 23 on regorafenib and 13 on placebo. Thirteen patients (56%; one-sided 95% CI [37.5%–[)) were progression-free at 8 weeks on regorafenib vs. 1 (7.7%; 95% CI [0.4%–[) on placebo. Median PFS was 11.4 weeks on regorafenib, and 3.9 weeks on placebo. Ten placebo patients crossed over to receive regorafenib after progression. The most common grade ≥3 regorafenib-related adverse events were pain (22%), asthenia (17%), thrombocytopenia (13%) and diarrhoea (13%). Conclusion Although the primary endpoint was not met statistically in this randomised cohort, there is evidence to suggest that regorafenib might modestly delay tumour progression in relapsed ES after failure of prior chemotherapy.Show less >
Show more >Background The REGOBONE multi-cohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the Ewing sarcoma (ES) cohort. Methods Patients with relapsed ES 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 progression. The primary endpoint was the progression-free rate at 8 weeks. With one-sided α of 0.05, and 80% power, at least 14/24 progression-free patients at 8 weeks were needed for success. Results From September 2014 to November 2019, 41 patients were accrued. 36 patients were evaluable for efficacy: 23 on regorafenib and 13 on placebo. Thirteen patients (56%; one-sided 95% CI [37.5%–[)) were progression-free at 8 weeks on regorafenib vs. 1 (7.7%; 95% CI [0.4%–[) on placebo. Median PFS was 11.4 weeks on regorafenib, and 3.9 weeks on placebo. Ten placebo patients crossed over to receive regorafenib after progression. The most common grade ≥3 regorafenib-related adverse events were pain (22%), asthenia (17%), thrombocytopenia (13%) and diarrhoea (13%). Conclusion Although the primary endpoint was not met statistically in this randomised cohort, there is evidence to suggest that regorafenib might modestly delay tumour progression in relapsed ES after 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 :
2024-01-20T22:04:20Z
2024-04-09T09:33:56Z
2024-04-09T09:33:56Z