The off-label use of targeted therapies in sarcomas: the OUTC’S program

BMC Cancer

Lauriane Eberst¹²  Claire Cropet¹¹  Axel Le Cesne⁸  Patricia Pautier⁸  Nicolas Penel⁶  Antoine Adenis⁶  Christine Chevreau¹⁴  Jacques-Olivier Bay⁷  Olivier Collard¹⁰  Didier Cupissol⁵  Florence Duffaud⁹  Jean-Claude Gentet⁹  Sophie Piperno-Neumann³  Perrine Marec-Berard¹¹  Emmanuelle Bompas⁴  Antoine Thyss²  Loic Chaigneau¹  Philippe Cassier¹¹  François Bertucci¹³  Jean-Yves Blay¹²  Isabelle Ray-Coquard¹² 

[1] CHU Jean Minjoz, Besançon, France

[2] Centre Antoine Lacassagne, Nice, France

[3] Institut Curie, Paris, France

[4] Centre René Gauducheau, Nantes, France

[5] Institut régional du Cancer, Montpellier, France

[6] Centre Oscar Lambret, Lille, France

[7] CHU Estaing, Clermont-Ferrand, France

[8] Institut Gustave Roussy, Villejuif, France

[9] CHU La Timone, Marseille, France

[10] Institut de Cancérologie Lucien Neuwirth, Saint-Priest-en-Jarez, France

[11] Centre Léon Bérard, 28 rue Laennec 69 373, LYON CEDEX 08, France

[12] Université Claude Bernard Lyon 1, Lyon, France

[13] Institut Paoli-Calmettes, Marseille, France

[14] Institut Claudius Regaud, Toulouse, France

关键词: Tumor board;    Register;    Targeted therapy;    Sarcoma;    Off-label;   

Others  :  1118014
DOI  :  10.1186/1471-2407-14-870

 received in 2014-04-29, accepted in 2014-10-30,  发布年份 2014

【 摘 要 】

Background

Few targeted therapies (TTs) are registered for sarcoma treatment despite numerous phase II studies and yet there are potential treatment options for patients after standard treatment escape. The French Sarcoma Group - Bone Tumor Study Group (GSF-GETO) created a national registry to evaluate the outcome of patients treated with off-label TTs.

Methods

Every consecutive sarcoma-patient receiving an off-label TT outside a clinical trial was included. The objective was to describe this patient efficacy and safety data in routine practice.

Results

From October 2008 to October 2011, 249 patients in 24 centers received 278 treatment lines with TTs. Twenty-five histological subtypes were included: most frequent were leiomyosarcoma (n = 48, receiving sorafenib in 63%, and sunitinib in 27%), GIST (n = 39, receiving sorafenib in 79%), and angiosarcoma (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 toxic death occurred, grade 3 and 4 toxicities were reported in 74 (27%) and 14 patients (5%) respectively.

Conclusion

Off-label TTs can be used for sarcoma patients in routine practice with an acceptable toxicity profile and efficacy similar to that reported in non-randomized clinical trials.

【 授权许可】

   
2014 Eberst et al.; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

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