Original Research Article
Small-sized gastrointestinal stromal tumors: Is there an association between expectant management and overall survival?

https://doi.org/10.1016/j.amjsurg.2021.12.015Get rights and content

Highlights

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    Tumor location is a predictor of expectant management (EM) for small (≤3CM) GIST.

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    No significant difference in OS between EM and resection for small GIST.

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    Female sex and private insurance are predictors of improved survival for small GIST.

Abstract

Background

Small-sized gastrointestinal stromal tumors (GISTs) have limited malignant potential. Few studies evaluate the safety and efficacy of expectant management (EM) for patients presenting with small GIST.

Methods

We queried the National Cancer Database to identify patients ≤65 years presenting with GISTs smaller than 3 cm in size between 2004 and 2015. Patients undergoing EM were 1:3 propensity score matched for relevant covariates to patients undergoing resection. Kaplan-Meier (KM) analysis of matched cohorts was used to evaluate the association between EM and overall survival (OS).

Results

1330 patients met inclusion criteria; 966 (72.6%) had gastric GISTs. 1196 (89.9%) underwent resection; 134 (10.1%) EM. 117 patients undergoing EM were propensity-matched to 356 patients undergoing resection. There was no difference in 5-year OS between patients undergoing EM and those undergoing resection (95.7% vs 92.6%, p = 0.4882).

Conclusions

Survival for small GISTs is similar with expectant management or resection.

Introduction

With the increase in the use of endoscopy and axial imaging to evaluate vague abdominal symptomatology, there has been an increase in the discovery of small gastrointestinal stromal tumors (GISTs).1 While these tumors are typically low grade with very limited potential for aggressive behavior, many are referred for surgical resection. The most recent guidelines from the National Comprehensive Cancer Network (NCCN) recommend resection of very small gastric GISTs in patients that are symptomatic or have high-risk features (irregular borders, echogenic foci or sonographic heterogeneity) on endoscopic ultrasound (EUS).2 Non-gastric GIST are thought to be characterized by a higher potential for disease progression, and these tumors are uniformly resected regardless of symptoms or radiographic features. For patients without high-risk features on EUS, the NCCN recommends interval surveillance by either serial EUS or axial CT or MR imaging. The evidence supporting these NCCN guidelines is sparse with the best available clinical studies including relatively few patients managed expectantly and relatively few patients with small but moderate to high grade tumors. Although complete resection of very small GISTs is thought to be curative, the actual overall survival benefit of resection over expectant management (EM) remains to be determined.3

We queried the National Cancer Data Base (NCDB) in an effort to identify factors associated with use of EM for GIST ≤3CM and to compare survival outcomes between patients with small-sized tumors who underwent curative resection to those undergoing EM. Our hypothesis was that patients with non-gastric GISTs would be less likely to be managed with surveillance only and that there would be no significant difference in survival between patients who underwent surgery and patients who did not.

Section snippets

Data source, sample selection, and variables examined

The National Cancer Data Base (NCDB) is a facility-based dataset that is a joint project of the American Cancer Society and the Commission on Cancer (CoC) of the American College of Surgeons (ACS).4 The NCDB captures an estimated 70% of newly diagnosed cancers being treated in the United States annually at nearly 1500 accredited hospitals. The ACS and CoC have not reviewed and are not responsible for the statistical analysis and conclusions discussed in this manuscript. This project was

Univariate analysis of patient outcomes by treatment approach

1330 patients met inclusion criteria. 966 (72.6%) patients had tumors located in the stomach; 304 (22.9%) patients had small bowel tumors; 43 (3.2%) patients had colonic tumors; 17 (1.3%) patients had rectal tumors. 1196 (89.9%) underwent surgical resection; 134 (10.1%) EM. Of those undergoing resection, 884 had a recorded histologic grade with 861 (97.4%) having low-grade tumors. 1270 (95.5%) patients received histologic confirmation of their tumors either by surgical pathology, preoperative

Discussion

In this study, we report the largest comparison to date of EM and resection for GISTs ≤3CM. While sex and insurance status were both found to be associated with overall survival, there was no statistical difference in overall survival between those patients undergoing resection and those undergoing observation for GIST ≤3CM. This trend was consistent ever after matching for covariates that would be expected to impact overall survival.

There is one finding from our results that ran counter to our

Conclusion

We report the largest and most contemporary comparison between EM and resection for GISTs ≤3CM. Despite the inherent limitations, we find that patients who received EM had no statistically significant reduced overall survival compared to patients who underwent resection. The decision to operate on tumors within this size category should be made after consideration for symptomatic status and imaging features.

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Published by Elsevier Inc.