Case report reveals an uncommon germline KIT exon 11 deletion in familial multifocal GIST, demonstrating favorable response to imatinib therapy.
Familial Gastrointestinal Stromal tumors (GIST) are rare neoplasms but GIST tumors are some of the most common soft tissue sarcoma subtypes. The incidence is between 10 to 15 cases per million worldwide and approximately 500 cases in the USA [1]. GIST is often diagnosed based on clinical presentation, the tumor's anatomic location and immunohistochemistry (IHC) pattern [1]. Patients with familial GIST develop GIST tumors that are numerous, smaller in size, and occur in the background of intestinal cells of Cajal hyperplasia [2]. The first family with features of inherited GIST was reported in the 1990s which was the first time Exon 11 KIT variant was reported [3]. We present two cases of family members affected with multifocal GIST tumors who underwent germline genetic analysis and were found to have germline pathogenic variants in Exon 11 of the KIT gene [c.1735_1737del (p.Asp579del)]. Both patients were treated with Imatinib with good response.
No takes yet. Share an insight, caveat, or question.
Ganesan et al. (2026) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: