Published online Sep 28, 2017. doi: 10.4329/wjr.v9.i9.365
Peer-review started: February 9, 2017
First decision: June 12, 2017
Revised: July 24, 2017
Accepted: August 2, 2017
Article in press: August 2, 2017
Published online: September 28, 2017
Processing time: 258 Days and 22.1 Hours
Familial gastrointestinal stromal tumor (GIST) is a rare autosomal dominant disorder associated with mutations in the KIT gene in the majority of cases. Although, exon 11 appears to be the hot spot region for approximately 95% of germline mutations, pathogenic variations have also been identified in exon 8, 13 and 17. Exon 13 germline mutations are extremely rare amongst familial GISTs and seven families with a germline mutation have been reported to date. Moreover, the role of imatinib mesylate in this rare familiar settings is not completely known so far. We describe here clinical, imaging, pathological and genetic findings of a family with four affected members; grandmother, his son and two grand-sons having a germline gain-of-function mutation of KIT in exon 13 and discuss the imatinib mesylate treatment surveillance outcomes towards disease management.
Core tip: Familial gastrointestinal stromal tumor (GIST) with exon 13 germline mutations are extremely rare. Moreover, there are only a few reports describing the response to imatinib in familial GISTs. The data on the role of imatinib in familial GISTs is still limited. Understanding the role of imatinib is important for the appropriate management of mutation positive familial GISTs. It is also crucial to be able to determine the role of specific germline KIT mutations in. We hereby report our findings in consideration of up-to-date information.