Clinical Trial Summary
Gastro intestinal stromal tumors (GIST) are rare mesenchymal tumors of the gastrointestinal
tract characterized by somatic mutations in the gene encoding the KIT or the PDGFR alpha
protein1. Treatment of localized forms relies on adequate surgery without tumor spillage and
systemic treatment with imatinib according to risk of relapse defined by localization, tumor
size and mitotic count, as well as mutational status. Advanced and relapsing forms are
currently treated with oral tyrosine-kinase inhibitors (TKI) of KIT and PDGFR such as
Imatinib, Sunitinib and Regorafenib. Over two decades significant changes in drug discovery
have impacted treatment strategies, notably via patient's access to various clinical trials.
The use of focal treatments such as surgery or interventional radiology with mini invasive
procedure of oligometastasis is also being proposed in some cases.
There is no precise data on patterns of sequential treatments used, especially proportions of
patients with metastatic GIST eventually benefiting from access to a clinical trial or a
focal treatment strategy in the course of their disease, and their results in terms of
survival on a real life national level.
Using the French sarcoma Group national database we aim at describing treatments strategies
proposed patients with metastatic GIST in the real life setting.
Objectives include : (i) Description of clinico-biological profiles, patterns of care and
modalities of treatment of patients with metastatic GIST in a real-life national setting and
(ii) evaluation of impact of each treatment line on patients outcome in terms of time to next
treatment (TNT) and survival