Gastrointestinal Stromal Tumor

P3, N=800, Not yet recruiting, GlaxoSmithKline

Despite adjuvant doxorubicin chemotherapy, follow-up imaging revealed tumor recurrence two months postoperatively. This case underscores the highly aggressive biological behavior and dismal prognosis associated with primary gastric UPS. The rapid recurrence following multimodal therapy highlights the critical importance of considering UPS in the differential diagnosis of gastric soft tissue masses and emphasizes the urgent need for more effective, comprehensive therapeutic strategies.

P2, N=818, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: May 2026 --> May 2027 | Trial primary completion date: May 2026 --> May 2027

P=N/A, N=60, Recruiting, Affiliated Hospital to Academy of Military Medical Sciences

Postoperatively, the patient recovered well and was treated with adjuvant imatinib...Comprehensive workup, wide excision with negative margins, and targeted therapy are essential. Increased awareness and reporting are needed to guide management.

P2, N=40, Enrolling by invitation, Peking University People's Hospital | Trial completion date: Mar 2028 --> Mar 2031

Small intestinal GISTs are relatively uncommon, and epithelioid-type GISTs that are KIT-negative and DOG1-negative are extremely rare. This case highlights the importance of considering atypical GISTs in the differential diagnosis of small bowel tumors with unusual immunohistochemical features.

P1, N=278, Recruiting, IDRX, Inc., a wholly owned subsidiary of GSK, LLC | Trial completion date: Nov 2027 --> Jun 2028 | Trial primary completion date: Nov 2027 --> Mar 2027

Owing to their rarity and small size, appendiceal GISTs are often discovered incidentally and may present diagnostic challenges. Herein, we report a rare case of appendiceal GIST detected preoperatively and successfully treated by laparoscopic appendectomy, with long-term recurrence-free survival.

The patient recovered uneventfully and was started on treatment with imatinib mesylate...GISTs should be considered in cases of unexplained SBV. This case emphasizes the diagnostic challenges, the need for prompt surgical palliation, and the role of systemic tyrosine kinase inhibitors in advanced presentation of GIST.

Activating mutations in KIT or PDGFRA characterize most GISTs and confer sensitivity to imatinib, whereas succinate dehydrogenase (SDH)–deficient GISTs lack these mutations and are typically imatinib resistant...Pathology demonstrated spindle cell GIST with significant treatment effect and retained SDHB expression. This case suggests that oncogenic KIT signaling may remain the dominant driver of GIST behavior despite the presence of a germline SDHC mutation and highlights the importance of integrated molecular interpretation in GIST management.

Although tyrosine kinase inhibitors (TKIs), particularly imatinib, have substantially improved outcomes, most patients with advanced disease eventually develop resistance, resulting in disease progression...Molecular stratification and personalized approaches dominate ongoing research, but evidence generation remains limited by small sample sizes and slow recruitment. Future trials integrating innovative therapeutic platforms and patient-centered outcomes are essential to improve long-term disease control and quality of life.