Development and External Validation of a Prognostic Model for DFS After Curative Resection of Duodenal GIST

NCT07837271 | Active Not Recruiting

• 1 other identifier: 20260912022335098
• Study Type: observational
• Target: 288
• Locations: 1 country
• Sites: 2

Brief Summary

Gastrointestinal stromal tumour (GIST) is the most common mesenchymal neoplasm of the digestive tract, originating from the interstitial cells of Cajal. GIST can arise anywhere along the gastrointestinal tract, but most commonly occurs in the stomach (50%-60%) and small intestine (20%-30%), with the colorectal region accounting for approximately 10%. Duodenal GIST is a relatively rare subtype, comprising approximately 5% of all GISTs. Due to its low incidence, clinical understanding of duodenal GIST has long been limited, and its clinical presentation is non-specific, varying considerably with tumour size, location, and growth pattern. The most common symptoms are related to gastrointestinal bleeding, including melaena, haematemesis, haematochezia, or symptomatic anaemia; abdominal pain is also a frequent presentation. With regard to anatomical distribution, the second portion of the duodenum (descending part) is the most commonly involved site. Surgery is the first-line treatment for localised GIST, and R0 resection (negative margins) is the key to a favourable prognosis. However, because the duodenum is in close proximity to the pancreas, biliary tract, and major vascular structures, surgical decision-making for GIST at this site is far more complex than at other locations. Surgeons must carefully balance oncological radicality against preservation of pancreaticoduodenal function. Despite advances in surgical technique, the risk of recurrence after surgery for duodenal GIST remains substantial. Studies have shown that compared with GIST at other sites, duodenal GIST has a significantly poorer prognosis, and even among patients with the same NIH risk classification, clinical outcomes differ. This suggests that duodenal GIST may exhibit distinct biological behaviour and should not be equated with GIST at other sites. Accurate risk stratification is therefore critical for determining individualised follow-up intervals, the intensity of adjuvant therapy, and informed patient decision-making. The most widely used risk stratification tool for GIST is the modified National Institutes of Health (NIH) classification, which is based on tumour size, mitotic count, and primary site. However, this classification was developed primarily from data on gastric GIST, and its performance in non-gastric GIST remains highly controversial. Previous studies have reported a C-statistic of 0.73 for the modified NIH criteria in gastric GIST, but only 0.62 in non-gastric GIST; the TNM staging system has demonstrated superior prognostic discrimination compared with the modified NIH criteria in intestinal GIST. Given the substantial differences in surgical complexity, recurrence risk, and treatment decision-making between duodenal GIST and GIST at other sites, the application of generic risk stratification tools to this anatomical subgroup is clearly inadequate. In addition to conventional prognostic factors such as tumour size, mitotic count, and primary site, the prognosis of duodenal GIST may also be influenced by molecular biomarkers and dynamic clinical factors such as perioperative complications. At the genomic level, KIT exon 9 mutations occur at a slightly higher frequency in duodenal GIST than in GIST overall, and have been confirmed as an independent risk factor for overall survival in patients with duodenal GIST. Compared with KIT exon 11 mutations, exon 9 mutations are associated with poorer response to imatinib targeted therapy and a higher risk of metastasis. This molecular difference may partly explain the poorer clinical prognosis observed in duodenal GIST. Given the limitations of existing risk stratification tools for duodenal GIST, together with the unique clinicopathological features, surgical complexity, and molecular biological background of tumours at this site, the development of a prognostic assessment tool specifically for duodenal GIST is of considerable clinical importance. In recent years, nomogram models have been widely applied to individualised prognostic prediction in various tumours and have demonstrated good predictive performance in the field of GIST. However, an externally validated prediction model specifically for disease-free survival after radical resection of duodenal GIST is currently lacking. This study, based on multicentre data, aims to develop and externally validate a generalisable nomogram model for predicting disease-free survival (DFS) after radical resection of duodenal GIST, thereby providing a quantitative reference for individualised follow-up and treatment decision-making in clinical practice.

Conditions

Duodenal; Gastrointestinal Stromal Tumors

Outcome Measures

Primary Outcomes (1)

• Disease-free survival (DFS)

  Disease-free survival (DFS) was defined as the time from radical resection to tumour recurrence, metastasis, or death from any cause, whichever occurred first. Patients who were alive without recurrence at the last follow-up were censored. DFS was estimated using the Kaplan-Meier method, and the predictive performance of the nomogram was evaluated by discrimination (C-index and time-dependent AUC) and calibration in both the training and external validation cohorts.

  From the date of radical resection to the date of recurrence, metastasis, or death from any cause, whichever occurred first, assessed up to 10 years after surgery.

Secondary Outcomes (4)

• Independent prognostic factors for disease-free survival

  Up to 10 years from the date of surgery (baseline).

• Discrimination of the nomogram model by concordance index (C-index)

  From the date of surgery through the last follow-up, assessed up to 10 years.

• Calibration of the nomogram model

  Assessed at 5 years after surgery.

• Discrimination of the nomogram model by time-dependent AUC at 5 years

  At 5 years after surgery.

Study Arms (2)

Training Cohort

Patients with primary duodenal gastrointestinal stromal tumour (GIST) who underwent radical resection at Zhongshan Hospital, Fudan University between January 2010 and January 2015. This cohort was used to identify independent prognostic factors and to develop the nomogram prediction model for disease-free survival (DFS).

External Validation Cohort

Patients with primary duodenal gastrointestinal stromal tumour (GIST) who underwent radical resection at the First Affiliated Hospital of Fujian Medical University between January 2015 and January 2026. This independent cohort was used for external validation of the nomogram, including assessment of discrimination (C-index and time-dependent AUC) and calibration.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study included patients with primary duodenal gastrointestinal stromal tumour (GIST) who underwent radical surgical resection at two tertiary referral centres in China. The training cohort comprised 204 patients treated at Zhongshan Hospital, Fudan University between January 2010 and January 2015. The external validation cohort comprised 84 patients treated at the First Affiliated Hospital of Fujian Medical University between January 2015 and January 2026. Eligible patients had histopathologically confirmed primary duodenal GIST, underwent radical resection, and had complete clinicopathological and follow-up data. Patients with concurrent or previous other malignancies, metastatic disease or multiple lesions at initial diagnosis, or those who received preoperative systemic therapy were excluded. This study population represents a real-world cohort of duodenal GIST patients treated with curative-intent surgery in routine clinical practice.

You may qualify if:

• Histopathologically confirmed primary duodenal gastrointestinal stromal tumour (GIST).
• Underwent radical surgical resection.
• Complete clinicopathological and follow-up data available.

You may not qualify if:

• Concurrent or previous other malignancies.
• Metastatic disease or multiple lesions at initial diagnosis.
• Received preoperative systemic therapy.

Sponsors & Collaborators

• First Affiliated Hospital of Fujian Medical University: lead

Study Sites (2)

The First Affiliated Hospital of Fujian Medical University

Fuzhou, Fujian, 350005, China

Location

Zhongshan Hospital, Fudan University

Shanghai, Shanghai Municipality, 200032, China

Location

MeSH Terms

Conditions

Gastrointestinal Stromal Tumors

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: September 12, 2026
• First Posted: September 23, 2026
• Study Start: January 1, 2026
• Primary Completion: June 1, 2026
• Study Completion: September 20, 2026
• Last Updated: September 23, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ANALYTIC CODE
• Time Frame: The data will become available starting 3 months after publication and will remain available for 3 years after publication.

Locations

CN (2)