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    CANCER:

    Gastrointestinal Stromal Tumor

    3d
    Molecular pathology of gastrointestinal neoplasms (PubMed, Magy Onkol)
    The responsiveness of gastrointestinal stromal tumors to imatinib requires validation via molecular testing. Patients diagnosed with pancreatic cancer may see enhanced survival rates by targeted therapy addressing microsatellite instability and BRCA mutations. In bile duct malignancies, especially intrahepatic cholangiocarcinoma of the small duct variant, the analysis of IDH1 mutations and FGFR2 fusions presents new treatment prospects.
    Review • Journal • BRCA Biomarker
    |
    HER-2 (Human epidermal growth factor receptor 2) • KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • NRAS (Neuroblastoma RAS viral oncogene homolog) • MSI (Microsatellite instability) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • CLDN18 (Claudin 18) • BRCA (Breast cancer early onset)
    |
    KRAS mutation • BRAF mutation • HER-2 amplification • NRAS mutation • IDH1 mutation • FGFR2 mutation • FGFR2 fusion • BRCA mutation • IDH1 mutation + FGFR2 fusion
    |
    imatinib
    6d
    Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015) (clinicaltrials.gov)
    P2, N=322, Recruiting, Merck Sharp & Dohme LLC | Trial completion date: Feb 2027 --> Jun 2029 | Trial primary completion date: Feb 2027 --> Jun 2029
    Trial completion date • Trial primary completion date • Stroma • Metastases
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • VHL (von Hippel-Lindau tumor suppressor) • EPAS1 (Endothelial PAS domain protein 1)
    |
    PDGFRA mutation
    |
    Welireg (belzutifan)
    6d
    A Drug-drug Interaction Study of Avapritinib and Midazolam (clinicaltrials.gov)
    P1, N=10, Completed, Blueprint Medicines Corporation | Active, not recruiting --> Completed | Trial completion date: Sep 2025 --> May 2024
    Trial completion • Trial completion date • Stroma • Metastases
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    |
    Ayvakit (avapritinib) • midazolam hydrochloride
    6d
    Phase II Study of Sunitinib in Tumors With c-KIT Mutations: Results From the NCI MATCH ECOG-ACRIN Trial (EAY131) Subprotocol V. (PubMed, JCO Precis Oncol)
    Sunitinib for c-KIT mutations did not meet the primary end point, but in this small sample size, a potential signal cannot be ruled out. Rate of eligible c-KIT mutations was low, affecting accrual to this arm.
    P2 data • Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    |
    KIT mutation
    |
    sunitinib
    13d
    Prognostic Significance of C-MYC and EGFR Overexpression in Gastrointestinal Stromal Tumors: An Immunohistochemical Study. (PubMed, Appl Immunohistochem Mol Morphol)
    Our findings suggest that both C-MYC and EGFR may be overexpressed and/or amplified in GISTs, indicating their potential prognostic role. This could also pave the way for therapeutic strategies targeting these proteins.
    Journal • Stroma
    |
    EGFR (Epidermal growth factor receptor) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    EGFR expression • KIT mutation • MYC expression • PDGFRA mutation
    14d
    Pathologic diagnosis and molecular features of gastrointestinal stromal tumors: a mini-review. (PubMed, Front Oncol)
    The standard treatment includes surgery and imatinib for metastatic cases; however, resistance to tyrosine kinase inhibitors remains a significant hurdle, especially in pediatric and wildtype GISTs. This highlights the need for advanced therapeutic strategies and emphasizes the importance of molecular profiling in guiding treatment decisions and improving outcomes for GIST patients.
    Review • Journal • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • ANO1 (Anoctamin 1)
    |
    KIT mutation • KIT expression
    |
    imatinib
    15d
    MEK162 in Combination With Imatinib Mesylate in Patients With Untreated Advanced Gastrointestinal Stromal Tumor (GIST) (clinicaltrials.gov)
    P1/2, N=75, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Nov 2024 --> Nov 2025 | Trial primary completion date: Nov 2024 --> Nov 2025
    Trial completion date • Trial primary completion date • Combination therapy • Stroma • Metastases
    |
    imatinib • Mektovi (binimetinib)
    16d
    Primary Small Intestinal Leiomyosarcoma Presenting With Small Bowel Perforation: A Case Report. (PubMed, Cureus)
    Due to the rarity of LMSs and their potential for misclassification, accurate diagnosis and multidisciplinary management are crucial. Given the high risk of recurrence and metastasis, particularly due to perforation, long-term follow-up is also recommended.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    |
    KIT expression
    19d
    In Vitro Assessment of the Role of Endoplasmic Reticulum Stress in Sunitinib-Induced Liver and Kidney Toxicity. (PubMed, Toxicol Lett)
    The research findings indicate that sunitinib may induce cytotoxic effects in hepatocytes through mechanisms involving oxidative stress, endoplasmic reticulum stress, and mitochondrial damage. However, in the kidney, the toxicity mechanism is different from that of liver, and the endoplasmic reticulum stress does not seem to be involved in this mechanism.
    Preclinical • Journal
    |
    NFE2L2 (Nuclear Factor, Erythroid 2 Like 2) • CASP3 (Caspase 3) • ATF4 (Activating Transcription Factor 4) • ATF6 (Activating Transcription Factor 6)
    |
    sunitinib
    20d
    Immunohistochemical Expression of ROR2 in Gastrointestinal Stromal Tumor. (PubMed, J Gastrointest Cancer)
    GIST cases displaying ROR2 positivity exhibit a reduced likelihood of disease progression and demonstrate favorable prognostic characteristics.
    Journal • Stroma
    |
    ROR2 (Receptor Tyrosine Kinase Like Orphan Receptor 2)
    23d
    Study for Patients Previously Treated in Avapritinib Clinical Trials (clinicaltrials.gov)
    P4, N=2, Completed, Blueprint Medicines Corporation | Active, not recruiting --> Completed
    Trial completion
    |
    Ayvakit (avapritinib)
    24d
    Mean platelet volume/platelet count ratio can predict the recurrence-free survival rate of patients after complete resection of gastrointestinal stromal tumors. (PubMed, Front Oncol)
    Patients with higher PVPR have better RFS. The nomogram including PVPR could be used to assist clinical treatment decision-making.
    Journal • Stroma
    |
    CD34 (CD34 molecule)
    24d
    Identification and validation of serum MUC17 as a non-invasive early warning biomarker for screening of gastric intraepithelial neoplasia. (PubMed, Transl Oncol)
    These findings suggest that plasma MUC17 levels hold substantial promise as a screening biomarker for individuals with GIN and EGC, effectively identifying high-risk groups that necessitate further gastroscopy.
    Journal
    |
    MUC17 (Mucin 17)
    28d
    Exophytic Gastric Mass Posing a Diagnostic Dilemma: A Case of Mesenteric Fibromatosis. (PubMed, Cureus)
    Beta-catenin is a key marker for MF; although immunohistochemistry markers were negative for beta-catenin in this case, it is important to note that beta-catenin positivity is observed in only 70-80% of cases. Differentiating these two distinct tumors can be challenging due to potential overlaps in clinical presentation, macroscopic appearance, and even histological features.
    Journal
    |
    CTNNB1 (Catenin (cadherin-associated protein), beta 1)
    30d
    YAP acts as an independent prognostic marker and regulates growth and metastasis of gastrointestinal stromal tumors via FBXW7-YAP pathway. (PubMed, J Gastroenterol)
    YAP expression serves as a predictive marker of recurrence for GIST patients with curative resection, highlighting its potential as a novel therapeutic target that warrants further investigation.
    Journal • Stroma
    |
    FBXW7 (F-Box And WD Repeat Domain Containing 7)
    |
    imatinib
    1m
    Appendiceal neurofibroma after resection of multiple gastrointestinal stromal tumors of the small intestine in a patient with neurofibromatosis type 1: a case report. (PubMed, Surg Case Rep)
    Patients with NF1 can develop a variety of gastrointestinal lesions. Appendiceal neurofibroma can be difficult to diagnose preoperatively and differentiate from malignancy. Hence, surgical resection should be considered.
    Journal • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • NF1 (Neurofibromin 1)
    |
    KIT positive
    1m
    Updated overall survival and safety with ripretinib vs sunitinib in patients with advanced gastrointestinal stromal tumor previously treated with imatinib and harboring KIT exon 11 + 17/18 mutations: ctDNA analysis from INTRIGUE (AIOM 2024)
    In this updated exploratory analysis from INTRIGUE, OS was longer for ripretinib vs sunitinib for pts with KIT exon 11 + 17/18 mutations identified by baseline ctDNA. The safety profile was consistent and more favorable for ripretinib vs sunitinib in these pts. Previously presented at the 2024 ESMO Sarcoma and Rare Cancers Congress.
    Clinical • Circulating tumor DNA • Stroma • Metastases
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    |
    KIT mutation • KIT exon 11 mutation • KIT exon 17 mutation
    |
    Guardant360® CDx
    |
    imatinib • sunitinib • Qinlock (ripretinib)
    1m
    Different efficacy of tyrosine kinase inhibitors by KIT and PGFRA mutations identified in circulating tumor DNA for the treatment of refractory gastrointestinal stromal tumors. (PubMed, BJC Rep)
    ctDNA-based analysis facilitates assessment of disease status and genomic profiles, thus potentially assisting in identifying optimal therapeutic strategies for advanced GIST patients.
    Journal • Circulating tumor DNA • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation • PDGFRA mutation
    1m
    Safety and Efficacy of Avapritinib in Chinese Patients With Gastrointestinal Stromal Tumor (GIST) in the Real World (clinicaltrials.gov)
    P=N/A, N=61, Completed, CStone Pharmaceuticals | Active, not recruiting --> Completed | Trial completion date: Oct 2025 --> Sep 2024 | Trial primary completion date: May 2025 --> Sep 2024
    Trial completion • Trial completion date • Trial primary completion date • Real-world evidence • Real-world • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation
    |
    imatinib • sunitinib • Ayvakit (avapritinib)
    1m
    Application of dual-energy computed tomography in gastrointestinal stromal tumors (ChiCTR2400090297)
    P=N/A, N=70, Not yet recruiting, Renji Hospital affiliated to Shanghai Jiaotong University School of Medicine; Renji Hospital affiliated to Shanghai Jiaotong University School of Medi
    New trial • Stroma
    1m
    Analysis of case characteristics of patients with gastrointestinal stromal tumors using clinical data (ChiCTR2400090130)
    P=N/A, N=107, Not yet recruiting, Department of General Surgery, The First Affiliated Hospital of Nanjing Medical University; The First Affiliated Hospital of Nanjing Medical Universit
    New trial • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    1m
    Genomic and transcriptomic landscape of human gastrointestinal stromal tumors. (PubMed, Nat Commun)
    Finally, four prominent subtypes are proposed with different genomic features, expression profiles, immune characteristics, clinical characteristics and subtype-specific treatment strategies. This large-scale analysis depicts the landscape and provides further insights into GIST pathogenesis and precise treatment.
    Journal • Tumor mutational burden • Stroma
    |
    TMB (Tumor Mutational Burden)
    |
    TMB-L
    2ms
    The challenge of diagnosing neuroendocrine neoplasms: experience from a national reference center. (PubMed, Virchows Arch)
    Selecting appropriate immunohistochemical stains based on H&E morphology is the key to avoiding diagnostic pitfalls. Medical history and molecular genomic information greatly assist in correctly diagnosing NENs and their mimics.
    Journal
    |
    SYP (Synaptophysin)
    2ms
    Gene of the month: C-KIT. (PubMed, Indian J Cancer)
    C-KIT has been studied as the first biomarker for targeted therapy. Herein we review its structure, function and role in various non-neoplastic and neoplastic diseases.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    2ms
    Gastrointestinal Stromal Tumors: Variants and Some Pitfalls That They Create. (PubMed, Adv Anat Pathol)
    It is also important to consider morphologic and immunophenotypic changes associated with treatment, including the potential absence of kit expression, particularly in GISTs that have metastasized. Therefore, obtaining clinical information regarding prior therapy with a tyrosine kinase inhibitor (TKI) is crucial.
    Review • Journal • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • ANO1 (Anoctamin 1)
    |
    PDGFRA mutation • KIT expression
    2ms
    Primary gastric epithelioid angiosarcoma: A case report of diagnostic biopsy pitfall. (PubMed, Medicine (Baltimore))
    Primary gastric epithelioid angiosarcoma is a rare gastric tumor. Given the epithelioid cell features displayed by tumor cells and the high expression of epithelial markers in tumor cells (83%), preoperative diagnosis is difficult and should be differentiated from adenocarcinoma or gastrointestinal stromal tumor.
    Journal • Biopsy
    |
    CD34 (CD34 molecule) • CD31 (Platelet and endothelial cell adhesion molecule 1) • PECAM1 (Platelet And Endothelial Cell Adhesion Molecule 1)
    2ms
    Gastrointestinal stromal tumors regulate macrophage M2 polarization through the MIF/CXCR4 axis to immune escape. (PubMed, Front Immunol)
    The MIF/CXCR4 axis is the most common ligand-receptor interaction between macrophages and tumor cells. GIST cells can regulate macrophage M2 polarization through the MIF/CXCR4 axis.
    Journal • Stroma
    |
    IL10 (Interleukin 10) • CD68 (CD68 Molecule) • MRC1 (Mannose Receptor C-Type 1)
    2ms
    Gastrointestinal stromal tumours (GIST) in children: An update of this orphan disease. (PubMed, Bull Cancer)
    International collaborations have been deployed in order to increase the clinical and biological knowledge of this orphan pathology in pediatrics.
    Journal • Stroma
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    2ms
    Comprehensive Genomic Assessment of Advanced-Stage GI Stromal Tumors Using the Japanese National Center for Cancer Genomics and Advanced Therapeutics Database. (PubMed, JCO Precis Oncol)
    This study highlights the genomic landscape of advanced GISTs and the important role of CGP in identifying rational molecular-targeted therapeutic options.
    Retrospective data • Journal • Tumor mutational burden • PD(L)-1 Biomarker • Stroma • Metastases
    |
    HER-2 (Human epidermal growth factor receptor 2) • TP53 (Tumor protein P53) • TMB (Tumor Mutational Burden) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • STK11 (Serine/threonine kinase 11) • HRD (Homologous Recombination Deficiency) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • RB1 (RB Transcriptional Corepressor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • ETV6 (ETS Variant Transcription Factor 6) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • NTRK (Neurotrophic receptor tyrosine kinase) • SDHA (Succinate Dehydrogenase Complex Flavoprotein Subunit A)
    |
    TMB-H • NTRK3 fusion • HRD • ETV6-NTRK3 fusion • PDGFRA mutation • PDGFR wild-type
    |
    Keytruda (pembrolizumab)
    2ms
    Increased plasma imatinib exposure and toxicity in chronically treated GIST patients with SARS-CoV-2 infection: a case series. (PubMed, Front Immunol)
    In 3 cases, this increase reflected the occurrence or worsening of imatinib side effects. This case-series highlights the clinical impact of SARS-CoV-2 infection on the management of patients with GIST treated with imatinib.
    Journal
    |
    ABCB1 (ATP Binding Cassette Subfamily B Member 1) • ABCG2 (ATP Binding Cassette Subfamily G Member 2) • CYP3A4 (Cytochrome P450, family 3, subfamily A, polypeptide 4) • CYP3A5 (Cytochrome P450 Family 3 Subfamily A Member 5)
    |
    imatinib
    2ms
    A Master Protocol to Evaluate DCC-3009 in Gastrointestinal Stromal Tumor (GIST) (clinicaltrials.gov)
    P1/2, N=120, Recruiting, Deciphera Pharmaceuticals, LLC | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    Current Drug Resistance Mechanisms and Treatment Options in Gastrointestinal Stromal Tumors: Summary and Update. (PubMed, Curr Treat Options Oncol)
    Despite the significant improvement in treatment outcomes with adjuvant imatinib therapy for patients, drug resistance remains a major challenge for GIST therapy...The OXPHOS pathway is a promising therapeutic target in combination with TKI against sensitive KIT/PDGFRA mutants. Comprehensive understanding of the above resistance mechanisms, experimental drugs/strategies and metabolic changes is critical to implement the proper therapy strategy and improve the clinical therapy outcomes for GIST.
    Review • Journal • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    PDGFRA mutation
    |
    imatinib
    2ms
    Gastrointestinal stromal tumor in Carney's triad with laparoscopic total gastrectomy: a case report. (PubMed, Surg Case Rep)
    This case underscores the necessity of total gastrectomy with lymph node dissection due to the high incidence of metastasis in GISTs associated with Carney's triad. Further research is required to determine the optimal extent of lymph node dissection in such cases.
    Journal • Stroma
    |
    SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • ANO1 (Anoctamin 1)
    |
    KIT positive
    |
    imatinib
    2ms
    A Study of Ziftomenib, an Oral Menin Inhibitor, in Combination with Imatinib in Patients with Advanced Gastrointestinal Stromal Tumors (GIST) (clinicaltrials.gov)
    P1, N=157, Not yet recruiting, Kura Oncology, Inc.
    New P1 trial • Combination therapy • Stroma • Metastases
    |
    imatinib • ziftomenib (KO-539)
    2ms
    A Study of Olverembatinib in SDH-deficient GIST. (clinicaltrials.gov)
    P3, N=40, Not yet recruiting, Ascentage Pharma Group Inc.
    New P3 trial • Stroma
    |
    Nailike (olverembatinib)
    2ms
    VISIONARY: Targeted Agent Evaluation in Digestive Cancers in China Based on Molecular Characteristics (clinicaltrials.gov)
    P=N/A, N=600, Active, not recruiting, Peking University | Recruiting --> Active, not recruiting | Trial completion date: Feb 2024 --> Mar 2025 | Trial primary completion date: Feb 2024 --> Nov 2024
    Enrollment closed • Trial completion date • Trial primary completion date • Real-world evidence • Tumor mutational burden • Real-world
    |
    FoundationOne® CDx
    2ms
    Clinical utility of liquid-based comprehensive genomic profiling (CGP) in gastrointestinal stromal tumors (GIST) (DGHO 2024)
    55% (42/77) of liquid samples with a KIT -driver mutation had a co-occurring imatinib-resistant alteration, and a minority of cases harbored non- KIT mechanisms of resistance such as FGFR2 fusion, BRAF or EGFR alterations... Known driver and TKI-resistant mutations are identified in liquid biopsies of patients with GIST, with high concordance to tissue in the presence of elevated TF. Liquid biopsy may be valuable in the molecular classification of GIST during the medical management of advanced disease.
    Clinical • Stroma
    |
    EGFR (Epidermal growth factor receptor) • BRAF (B-raf proto-oncogene) • FGFR2 (Fibroblast growth factor receptor 2) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1)
    |
    KIT mutation • FGFR2 mutation • FGFR2 fusion • NF1 mutation • KIT exon 13 mutation • KIT exon 17 mutation
    |
    FoundationOne® CDx • FoundationOne® Liquid CDx
    |
    imatinib
    2ms
    Updated overall survival and safety data on ripretinib vs. sunitinib in patients with advanced gastrointestinal stromal tumor harboring KIT exon 11 + 17/18 mutations after prior treatment with imatinib: ctDNA analysis by INTRIGUE (DGHO 2024)
    In this updated exploratory analysis from INTRIGUE, OS was longer for ripretinib vs sunitinib for pts with KIT exon 11 + 17/18 mutations identified by baseline ctDNA. The safety profile was favorable for pts with KIT exon 11 + 17/18 mutations in the ripretinib arm.
    Clinical • Circulating tumor DNA • Stroma • Metastases
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase)
    |
    KIT mutation • KIT exon 11 mutation • KIT exon 17 mutation
    |
    Guardant360® CDx
    |
    imatinib • sunitinib • Qinlock (ripretinib)
    2ms
    Clinical characteristics of gastrointestinal stromal tumors with hypoglycemia. (PubMed, Oncol Lett)
    Furthermore, a chronological analysis was performed using multiple resected archived samples from the same case, and revealed that diffuse IGF-II expression may have occurred in the early phase of tumor development. The present study catalogued the characteristics of GIST with hypoglycemia with a focus on NICTH-GIST.
    Journal • Stroma
    |
    IGF2 (Insulin-like growth factor 2)
    2ms
    A Master Protocol to Evaluate DCC-3009 in Gastrointestinal Stromal Tumor (GIST) (clinicaltrials.gov)
    P1/2, N=120, Not yet recruiting, Deciphera Pharmaceuticals, LLC
    New P1/2 trial • Stroma
    2ms
    Correlation Between Imatinib Trough Concentration and Efficacy in Advanced GIST Patients with Different Genotypes (clinicaltrials.gov)
    P=N/A, N=168, Completed, First Affiliated Hospital, Sun Yat-Sen University
    New trial • Stroma • Metastases
    |
    imatinib
    3ms
    Oncogenetic Panel and Integrated Clinical Data Registry Study for Wild Type Gastrointestinal Stromal Tumor Patients (clinicaltrials.gov)
    P=N/A; Trial primary completion date: Dec 2025 --> Dec 2024
    Trial primary completion date • Stroma
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    PDGFRA mutation • KIT wild-type • PDGFR wild-type
    |
    OncoPanel™ Assay