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

Danique L M van Broekhoven, Dirk J. Grünhagenl, Thijs van Dalen, Frits van Coevorden, Han J. Bonenkamp, Lukas B. Been, Marc H A Bemelmans, Sander D S Dijkstra, Chiara Colombo, Alessandro Gronchi, Cornelis Verhoef

Research output: Contribution to journalArticle

Abstract

Background: The 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 methods: GRAFITI 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. Discussion: This study will provide insight in tumor behavior, the effect on quality of life and clinicopathological factors predictive of tumor progression. Trial registration: The GRAFITI trial is registered in the Netherlands National Trial Register (NTR), number 4714.

Original languageEnglish
Article number686
JournalBMC Cancer
Volume16
Issue number1
DOIs
Publication statusPublished - Aug 26 2016

Keywords

  • Aggressive fibromatosis
  • Desmoid
  • Desmoid-type fibromatosis
  • Growth
  • Progression
  • Wait-and-see
  • Watchful waiting

ASJC Scopus subject areas

  • Genetics
  • Oncology
  • Cancer Research

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    van Broekhoven, D. L. M., Grünhagenl, D. J., van Dalen, T., van Coevorden, F., Bonenkamp, H. J., Been, L. B., Bemelmans, M. H. A., Dijkstra, S. D. S., Colombo, C., Gronchi, A., & Verhoef, C. (2016). Tailored Beta-catenin mutational approach in extra-abdominal sporadic desmoid tumor patients without therapeutic intervention. BMC Cancer, 16(1), [686]. https://doi.org/10.1186/s12885-016-2704-4