1. Regosarc: regorafenib versus placebo in doxorubicin-refractory soft-tissue sarcoma-a quality-adjusted time without symptoms of progression or toxicity analysis
- Author
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Berry, V. (Vincent), Basson, L. (Laurent), Bogart, E. (Emilie), Mir, O. (Olivier), Blay, J-Y. (Jean-Yves), Italiano, A. (Antoine), Bertucci, F. (François), Chevreau, C. (Christine), Clisant-Delaine, S. (Stephanie), Liegl-Atzwanger, B. (Bernadette), Tresch-Bruneel, E. (Emmanuelle), Wallet, J. (Jennifer), Taieb, S. (Sophie), Decoupigny, E. (Emilie), Le Cesne, A. (Axel), Brodowicz, T. (Thomas), Penel, N. (Nicolas), Site de Recherche Intégrée en Cancérologie (SIRIC-ONCOLille), Université de Lille, Sciences et Technologies-Université de Lille, Sciences Humaines et Sociales-Centre Régional de Lutte contre le Cancer Oscar Lambret [Lille] (UNICANCER/Lille), Université de Lille-UNICANCER-Université de Lille-UNICANCER-Cancéropole Nord-Ouest-Université de Lille, Droit et Santé-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Centre Régional de Lutte contre le Cancer Oscar Lambret [Lille] (UNICANCER/Lille), Université de Lille-UNICANCER, Institut Gustave Roussy (IGR), Centre Léon Bérard [Lyon], Institut Bergonié [Bordeaux], UNICANCER, Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Institut Claudius Regaud, Karl-Franzens-Universität Graz, University of Vienna [Vienna], Evaluation des technologies de santé et des pratiques médicales - ULR 2694 (METRICS), Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Université Lille Nord de France (COMUE)-UNICANCER-Université Lille Nord de France (COMUE)-UNICANCER-Cancéropole Nord-Ouest-Université de Lille, Droit et Santé-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Université Lille Nord de France (COMUE)-UNICANCER, Karl-Franzens-Universität [Graz, Autriche], Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille)-Université de Lille, CHU Lille, Université de Lille, Site de Recherche Intégrée en Cancérologie [SIRIC-ONCOLille], METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694, Institut Gustave Roussy [IGR], and Evaluation des technologies de santé et des pratiques médicales - ULR 2694 [METRICS]
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[SDV]Life Sciences [q-bio] ,Mesh:Diarrhea/chemically induced ,Mesh:Alopecia/chemically induced ,Mesh:Phenylurea Compounds/therapeutic use ,Mesh:Aged ,Mesh:Synovial/drug therapy ,Mesh:Treatment Outcome ,Mesh:Anorexia/chemically induced ,Mesh:Quality of Life ,Mesh:Proportional Hazards Models ,Mesh:Severity of Illness Index ,Mesh:Female ,quality-adjusted survival ,Mesh:Fecal Incontinence/chemically induced ,Mesh:Male ,Mesh:Sarcoma ,Mesh:Middle Aged ,quality-adjusted time without symptoms of progression or toxicity (Q-TWiST) ,Mesh:Asthenia/chemically induced ,Mesh:Liposarcoma/drug therapy ,metastatic soft-tissue sarcoma ,placebo ,regorafenib ,Mesh:Mucositis/chemically induced ,Mesh:Hospitalization ,Mesh:Sarcoma/drug therapy ,Mesh:Hand-Foot Syndrome/etiology ,Mesh:Pyridines/therapeutic use ,Mesh:Antineoplastic Agents/therapeutic use ,Mesh:Leiomyosarcoma/drug therapy ,Mesh:Humans ,Mesh:Hypertension/chemically induced ,Mesh:Double-Blind Method - Abstract
International audience; BACKGROUND: In a placebo-controlled, randomized phase 2 trial (ClinicalTrials.gov identifier NCT01900743), regorafenib improved progression-free survival (PFS) for patients with doxorubicin-pretreated advanced nonadipocytic sarcoma. A quality-adjusted time without symptoms of progression or toxicity (Q-TWiST) post hoc exploratory analysis was applied to provide an integrated measure of its clinical benefit.METHODS: In the base-case analysis, each patient's overall survival (OS) was partitioned into 3 mutually exclusive health states: the time with a grade 3 or 4 adverse event (TOX), the time without symptoms of disease or grade 3 or 4 toxicity from treatment, and the time after tumor progression or relapse. The time spent in each state was weighted with a health-state utility associated with that state and was summed to calculate the Q-TWiST. The stability of the base-case analysis was explored with several sensitivity analyses.RESULTS: In nonadipocytic sarcoma, the PFS was (4.0 months [2.6-5.5 months] with regorafenib vs 1.0 month [0.9-1.8 months] with a placebo; hazard ratio, 0.36 [0.25-0.53]; P < .0001); the OS was 13.4 months (8.6-17.3 months) with regorafenib and 9.0 months (6.8-12.5 months) with a placebo (hazard ratio, 0.67 [0.44-1.02]). With the classic definition of TOX (including all grade 3 and 4 clinical adverse events), the Q-TWiSTs were 8.0 months (7.0-9.0 months) with regorafenib and 5.7 months (4.9-6.4 months) with a placebo (P
- Published
- 2017