BACKGROUND: The objective of this study is to explore the off-label use of targeted therapies (TTs) for @entity1 with @entity421 registered within the @entity242 Group - Bone Tumor Study Group (GSF-GETO) national registry. METHODS: All @entity1 with an @entity421 , registered between January 1, 2009 and July 15, 2013 were analyzed. RESULTS: Twenty-nine @entity1 with refractory relapsed @entity421 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 @entity1 were in second complete remission, 26 were in progression for metastatic relapse. Twenty-three @entity1 received @entity3446 (in combination with @entity786 for 18); 5, @entity2240 ; 4, @entity2857 ; and one, @entity6219 . Stable disease was observed for 45.5  % of @entity1 (95  % Confidence Interval (CI) [20-52.8]). The median Progression-Free Survival (PFS) was 3  months (95  % CI [2-5.4]) for @entity1 treated by @entity3446 and 1.8  months (95  % CI [1.3-2.8]) for @entity1 receiving multi-targeted @entity4149 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 @entity1 receiving @entity3446 compared to other TTs (Hazard Ratio (HR)   =   2.7, 95  % CI [1.05-7.1]). No toxic @entity204 was reported. Grade 3 and 4 @entity137 were observed in 27 and 6  % of cases respectively. CONCLUSION: Off-label TTs, especially @entity3446 , reported benefit in the treatment of @entity421 with an acceptable @entity137 profile, including in pediatric population.

Off-label use of targeted therapies in XXXX : data from the French registry OUTC'S (Observatoire de l'Utilisation des Th  rapies Cibl  es dans les Sarcomes).

@entity1 :: ('9606', 'Species') :: ['patients'] @entity137 :: ('MESH:D064420', 'Disease') :: ['toxicity', 'toxicities'] @entity6219 :: ('MESH:C516667', 'Chemical') :: ['pazopanib'] @entity242 :: ('MESH:D012509', 'Disease') :: ['French Sarcoma'] @entity2240 :: ('MESH:C473478', 'Chemical') :: ['sunitinib'] @entity786 :: ('MESH:D003520', 'Chemical') :: ['cyclophosphamide'] @entity2857 :: ('MESH:C471405', 'Chemical') :: ['sorafenib'] @entity3446 :: ('MESH:D020123', 'Chemical') :: ['sirolimus'] @entity421 :: ('MESH:D012516', 'Disease') :: ['osteosarcomas', 'refractory osteosarcomas', 'osteosarcoma'] @entity4149 :: ('D014443', 'Chemical') :: ['tyrosine'] @entity204 :: ('MESH:D003643', 'Disease') :: ['death']
@entity421 :: (MESH:D012516,Disease) :: ['osteosarcomas']
