Title |
Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC’S (Observatoire de l’Utilisation des Thérapies Ciblées dans les Sarcomes)
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Published in |
BMC Cancer, November 2015
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DOI | 10.1186/s12885-015-1894-5 |
Pubmed ID | |
Authors |
Mathilde Penel-Page, Isabelle Ray-Coquard, Julie Larcade, Magali Girodet, Laure Bouclier, Muriel Rogasik, Nadège Corradini, Natacha Entz-Werle, Laurence Brugieres, Julien Domont, Cyril Lervat, Sophie Piperno-Neumann, Helène Pacquement, Jacques-Olivier Bay, Jean-Claude Gentet, Antoine Thyss, Loic Chaigneau, Bérangère Narciso, Helène Cornille, Jean-Yves Blay, Perrine Marec-Bérard |
Abstract |
The objective of this study is to explore the off-label use of targeted therapies (TTs) for patients with osteosarcoma registered within the French Sarcoma Group - Bone Tumor Study Group (GSF-GETO) national registry. All patients with an osteosarcoma, registered between January 1, 2009 and July 15, 2013 were analyzed. Twenty-nine patients with refractory relapsed osteosarcomas received 33 treatment lines of TTs. The median age at the beginning of treatment was 19 years (range 9-72). The median number of previous lines of chemotherapy was 3 (range 1-8). Before inclusion, 3 patients were in second complete remission, 26 were in progression for metastatic relapse. Twenty-three patients received sirolimus (in combination with cyclophosphamide for 18); 5, sunitinib; 4, sorafenib; and one, pazopanib. Stable disease was observed for 45.5 % of patients (95 % Confidence Interval (CI) [20-52.8]). The median Progression-Free Survival (PFS) was 3 months (95 % CI [2-5.4]) for patients treated by sirolimus and 1.8 months (95 % CI [1.3-2.8]) for patients receiving multi-targeted tyrosine kinase inhibitors; 6-month PFS 15 %. The median Overall Survival (OS) was 6.8 months (95 % CI [4.7-12.1]), and one-year OS was 24 %. In a multivariate analysis, PFS was superior for patients receiving sirolimus compared to other TTs (Hazard Ratio (HR) = 2.7, 95 % CI [1.05-7.1]). No toxic death was reported. Grade 3 and 4 toxicities were observed in 27 and 6 % of cases respectively. Off-label TTs, especially sirolimus, reported benefit in the treatment of refractory osteosarcomas with an acceptable toxicity profile, including in pediatric population. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 46 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Other | 9 | 20% |
Researcher | 9 | 20% |
Student > Master | 4 | 9% |
Student > Ph. D. Student | 3 | 7% |
Student > Postgraduate | 3 | 7% |
Other | 8 | 17% |
Unknown | 10 | 22% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 22 | 48% |
Pharmacology, Toxicology and Pharmaceutical Science | 4 | 9% |
Agricultural and Biological Sciences | 3 | 7% |
Biochemistry, Genetics and Molecular Biology | 2 | 4% |
Business, Management and Accounting | 1 | 2% |
Other | 3 | 7% |
Unknown | 11 | 24% |