Roubaud, G., Kind, M., Coindre, J. M., Maki, R. G., Bui, B., Italiano, A. (March 2012) Clinical activity of sorafenib in patients with advanced gastrointestinal stromal tumor bearing PDGFRA exon 18 mutation: a case series. Ann Oncol, 23 (3). pp. 804-5. ISSN 1569-8041 (Electronic)0923-7534 (Linking)
Abstract
PDGFRA is mutated in 5%–10% of gastrointestinal stromal tumors (GISTs), the PDGFRA D842V mutation accounting for ∼60% of all PDGFRA mutations known in GISTs [1]. The DIMH842–845 and the IMH843–846 deletions represent ∼15% of all PDGFRA mutations. While the latter have been associated with sensitivity to imatinib, the PDGFRA D842V mutation confers primary resistance to imatinib [1–4], sunitinib [5], and nilotinib [6]. In vitro data suggested that dasatinib is a potent inhibitor of PDGFRA D842V [7]. Sorafenib also displayed some intermediate efficacy even if it was significantly lower than for PDGFRA ΔDIM842–844 [7]. Given the rarity of metastatic GIST with PDGFRA mutation, clinical data in this setting are almost nonexistent [8]. We report here a series of five patients with metastatic GIST bearing a mutation of the exon 18 of PDGFRA and treated with sorafenib (400 mg twice daily).
| Item Type: | Paper |
|---|---|
| Uncontrolled Keywords: | Antineoplastic Agents/*therapeutic use Benzenesulfonates/*therapeutic use Exons/genetics Gastrointestinal Neoplasms/*drug therapy/*genetics Gastrointestinal Stromal Tumors/*drug therapy/*genetics Humans *Mutation Niacinamide/analogs & derivatives Phenylurea Compounds Pyridines/*therapeutic use Receptor, Platelet-Derived Growth Factor alpha/*genetics |
| Subjects: | bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > exons diseases & disorders > cancer > cancer types > gastrointestinal stromal tumors |
| CSHL Authors: | |
| Communities: | CSHL labs > Maki lab |
| Depositing User: | Matt Covey |
| Date: | March 2012 |
| Date Deposited: | 20 Oct 2016 18:52 |
| Last Modified: | 20 Oct 2016 18:52 |
| Related URLs: | |
| URI: | https://repository.cshl.edu/id/eprint/33744 |
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