Table 3.
Implication of gastrointestinal stromal tumors mutations and response to targeted therapy
| Imatinib[23] | Sunitinib[25] | Regorafenib[28] | |
| KIT mutation | |||
| Exon 11 | OR 63% | CB 34% | Increased sensitivity |
| Exon 9 | OR 37%. Intermediate sensitivity. Higher dose 800 mg more effective in metastatic disease than 400 mg daily | CB 34% | Unknown |
| Exon 13 | OR 40%. Sensitivity as primary mutation. Resistance as secondary mutation | CB 100% | Unknown |
| Exon 14 | Resistance as secondary mutation | Unknown | Unknown |
| Exon 17 | OR 25%. Primary mutation sensitive in vitro. Resistance as secondary mutation | CB 0% | Unknown |
| PDGFRA mutation | |||
| Exon 18 | OR 50% | CB 0% | Unknown |
| Exon 12 | Increased sensitivity | CB 0% | Unknown |
| Exon 14 | Increased sensitivity in vitro | Unknown | Unknown |
| Exon 18 D842V | Decreased sensitivity | Decreased sensitivity | Unknown |
| BRAF mutation | Resistance | Resistance | Unknown |
| SDH mutation | Decreased sensitivity | Unknown | Increased sensitivity |
| No KIT, PDGFRA or BRAF mutation | OR 28% | CB 56% | Some activity |
Objective response (OR) is defined as a complete or partial response by Response Evaluation Criteria for Solid Tumors (RECIST) criteria, excludes non-evaluable patients. Clinical benefit (CB) is defined as response or stable disease for 6 mo or more according to RECIST. PDGFRA: Platelet-derived growth factor receptor alpha; SDH: Succinate dehydrogenase.