BACKGROUND: Few targeted therapies (TTs) are registered for @entity242 treatment despite numerous phase II studies and yet there are potential treatment options for @entity1 after standard treatment escape. The @entity242 Group - Bone Tumor Study Group (GSF-GETO) created a national registry to evaluate the outcome of @entity1 treated with off-label TTs. METHODS: Every consecutive @entity242 - @entity1 receiving an off-label TT outside a clinical trial was included. The objective was to describe this @entity1 efficacy and safety data in routine practice. RESULTS: From October 2008 to October 2011, 249 @entity1 in 24 centers received 278 treatment lines with TTs. Twenty-five histological subtypes were included: most frequent were @entity1822 (n = 48, receiving sorafenib in 63%, and sunitinib in 27%), @entity677 (n = 39, receiving sorafenib in 79%), and @entity5426 (n =18, receiving sorafenib in 78%). The overall response rate to TTs was 15% (95% CI [10,6-20,2]), the disease control rate at 2  months was 59%. The median progression-free survival was 4,1  months (IC 95% [3,2-4,8]). Three complete responses were observed. No @entity137 occurred, grade 3 and 4 @entity137 were reported in 74 (27%) and 14 @entity1 (5%) respectively. CONCLUSION: Off-label TTs can be used for @entity242 @entity1 in routine practice with an acceptable @entity137 profile and efficacy similar to that reported in non-randomized clinical trials.

The off-label use of targeted therapies in XXXX : the OUTC'S program.

@entity1 :: ('9606', 'Species') :: ['patients', 'patient'] @entity137 :: ('MESH:D064420', 'Disease') :: ['toxicity', 'toxic death', 'toxicities'] @entity677 :: ('MESH:C564650', 'Disease') :: ['GIST'] @entity242 :: ('MESH:D012509', 'Disease') :: ['sarcoma', 'French Sarcoma'] @entity5426 :: ('MESH:D006394', 'Disease') :: ['angiosarcoma'] @entity1822 :: ('MESH:D007890', 'Disease') :: ['leiomyosarcoma']
@entity242 :: (MESH:D012509,Disease) :: ['sarcomas']
