Tailored Beta-catenin mutational approach in extra-abdominal sporadic desmoid tumor patients without therapeutic intervention

BMC Cancer

Study Protocol

Dirk J. Grünhagenl¹  Danique L.M. van Broekhoven¹  Cornelis Verhoef¹  Chiara Colombo²  Alessandro Gronchi²  Sander D.S. Dijkstra³  Marc H.A. Bemelmans⁴  Frits van Coevorden⁵  Han J. Bonenkamp⁶  Lukas B. Been⁷  Thijs van Dalen⁸ 

[1] Erasmus MC Cancer Institute, Rotterdam, The Netherlands;Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy;Leiden University Medical Center, Leiden, The Netherlands;Maastricht University Medical Center, Maastricht, The Netherlands;Netherlands Cancer Institute, Amsterdam, The Netherlands;Radboud University Medical Center, Nijmegen, The Netherlands;University Medical Center Groningen, Groningen, The Netherlands;University Medical Center Utrecht, Utrecht, The Netherlands;

关键词: Aggressive fibromatosis;    Desmoid;    Desmoid-type fibromatosis;    Watchful waiting;    Wait-and-see;    Growth;    Progression;   

DOI  :  10.1186/s12885-016-2704-4

 received in 2015-11-19, accepted in 2016-08-10,  发布年份 2016

来源: Springer

【 摘 要 】

BackgroundThe efficacy of the classical treatment modalities surgery and radiotherapy in the treatment of aggressive fibromatosis is presently disputed and there is a shift towards a more conservative approach. The aim of the present study is to objectify tumor growth in patients with extra-abdominal or abdominal wall aggressive fibromatosis, while adhering to a “watchful waiting” policy. Other objectives are to investigate quality of life and to identify factors associated with tumor growth, in particular the relation with the presence of a CTNNB1-gene mutation in the tumor.Design and methodsGRAFITI is a nationwide, multicenter, prospective registration trial. All patients with extra-abdominal or abdominal wall aggressive fibromatosis are eligible for inclusion in the study. Main exclusion criteria are: history of familiar adenomatous polyposis, severe pain, functional impairment, life/limb threating situations in case of progressive disease. Patients included in the study will be treated with a watchful waiting policy during a period of 5 years. Imaging studies with ultrasound and magnetic resonance imaging scan will be performed during follow-up to monitor possible growth: the first years every 3 months, the second year twice and the yearly. In addition patients will be asked to complete a quality of life questionnaire on specific follow-up moments. The primary endpoint is the rate of progression per year, defined by the Response Evaluation Criteria In Solid Tumors (RECIST). Secondary endpoints are quality of life and the rate of influence on tumor progression for several factors, such as CTNNB1-mutations, age and localization.DiscussionThis study will provide insight in tumor behavior, the effect on quality of life and clinicopathological factors predictive of tumor progression.Trial registrationThe GRAFITI trial is registered in the Netherlands National Trial Register (NTR), number 4714.

【 授权许可】

CC BY   
© The Author(s). 2016