Abstract
Background
Rectal gastrointestinal stromal tumors are a rare subset of gastrointestinal stromal tumors with malignant potential, making their management clinically challenging. This study sought to assess the feasibility, safety, and efficacy of endoscopic resection as a primary treatment for these tumors.
Methods
This study conducted a retrospective analysis and follow-up of clinical data from 18 patients who were diagnosed with rectal gastrointestinal stromal tumors (GISTs) and who underwent endoscopic resection at Zhongshan Hospital, affiliated with Fudan University, from February 2018 to May 2025. This study aimed to outline the clinicopathological characteristics, endoscopic diagnostic approaches, and therapeutic insights specific to rectal GIST patients.
Results
A series of 18 rectal GIST patients underwent endoscopic resection with 100% success in achieving en bloc and R0 resection. The cohort had a 1:1 gender ratio and a mean age of 56. Most (77.8%) were asymptomatic. All the tumors were solitary, peri-anal lesions (4–35 mm). The procedures were efficient (median: 37.5 min) and safe, with no intraoperative complications. Three minor postoperative issues arose but were self-limiting. The median hospitalization duration was 3 days, and notably, no recurrence was found after a median 29-month follow-up.
Conclusion
Although based on a small cohort, endoscopic resection proved feasible and safe for small rectal GISTs (≤ 3 cm), achieving high complete resection and no recurrence. This provides preliminary support for its use, pending confirmation in larger comparative trials.
Keywords: Rectal gastrointestinal stromal tumors, Clinical manifestation, Endoscopic features, Endoscopic resection
Introduction
Gastrointestinal stromal tumors (GISTs) are the most common gastrointestinal mesenchymal tissue tumors that mainly originate from Cajal cells or their precursors, and can occur in any part of the digestive tract [1]. Clinical symptoms are often nonspecific and depend on the location, size, and growth pattern. GISTs are mainly located in the muscular propria, and submucosal masses can be observed via endoscopy. Endoscopic ultrasound can identify the source of masses. Smaller GISTs(< 2 cm) are often referred to as miniGISTs (1–2 cm) or microGISTs (< 1 cm) and are usually found by chance during surgery or endoscopy [2]. Although these small GISTs have mutations that resemble those of larger GISTs, they are typically not removed other than at the rectum’s primary site [3]. Rectal GIST diagnosis rates have steadily increased in recent years due to the widespread use of endoscopy, which has drawn attention because of the propensity of tumors for malignancy. Although GISTs are the most common mesenchymal tumors found in the GI tract, they predominantly occur in the stomach, with colorectal gastrointestinal stromal tumors being infrequent [4]. In addition, there is a lack of prospective data on long-term oncological outcomes after endoscopic resection of rectal GIST.
his pilot study retrospectively analyzed data from 18 patients with rectal GISTs who underwent endoscopic resection at our institution over a 7-year period. We therefore conducted this single-center, retrospective pilot study to collect and preliminarily analyze the clinicopathological characteristics and outcomes of patients with rectal GISTs undergoing endoscopic resection, aiming to provide initial insights into the feasibility, safety, and efficacy of these techniques, acknowledging the inherent limitations of a small sample size for drawing definitive conclusions. For the purpose of this study, we defined “small” rectal GISTs as those with a maximum diameter of ≤ 3 cm, a size threshold commonly used in clinical practice to consider local resection techniques.
Methods
Patient selection
This single-center, retrospective pilot study was conducted by reviewing pathological databases to identify patients who underwent endoscopic resection for small (defined as ≤ 3 cm in maximum diameter) rectal subepithelial lesions between February 2018 and May 2025 and were subsequently confirmed to have GISTs on histopathological examination. A total of 18 patients were included. A preoperative histopathological diagnosis (e.g., via fine-needle aspiration) was not routinely pursued. The decision for primary endoscopic resection was based on a combination of suggestive endoscopic and EUS features (well-demarcated, hypoechoic lesions arising from the muscularis propria) and clinical judgment, aiming for a diagnostic and therapeutic resection in a single procedure. Comprehensive medical records, including demographic data, clinical presentations, endoscopic findings, procedural details, and pathological characteristics, were collected. The study was approved by the Ethics Committee of Zhongshan Hospital, Fudan University. Written informed consent was obtained from all individual participants prior to their enrollment in the study and the endoscopic procedure.
Intervention
Under intravenous anesthesia, all patients received endoscopic therapy, and throughout the procedure, vital signs were tracked. All of the treatments were carried out by experienced endoscopists. Endoscopic submucosal dissection (ESD), endoscopic full-thickness resection (EFTR), and submucosal tunneling endoscopic resection (STER) were employed based on the following explicit criteria (summarized in Fig. 1):
ESD was primarily selected for intraluminal GISTs located within 2 cm of the anal verge.
EFTR was used for lesions situated 2–5 cm from the anal verge with ≥ 50% extraluminal growth.
STER was applied for GISTs ≤ 3 cm from the anal verge with < 50% extraluminal growth, or for lesions located ≥ 5 cm above the dentate line.
Fig. 1.
Selection of surgical methods for endoscopic resection of rectal stromal tumors
ESD involves creating a submucosal cushion by injecting saline solution, followed by a circumferential mucosal incision and stepwise dissection of the lesion from the underlying tissue, with final hemostasis and prevention of perforation [5]. EFTR involves submucosal injection to lift the lesion, followed by an incision through the mucosa and submucosa to the muscularis propria, resection of the full-thickness lesion, and closure of the defect with clips or sutures [6]. STER provides favorable exposure for GIST resection, as the knife approaches the lesion at a more tangential axis compared to the “exposed ESD technique,” which features a perpendicular axis that carries a higher risk of R1 resection. It involves creating a submucosal tunnel above the lesion, advancing the endoscope into the tunnel for direct visualization and resection of the lesion, and closing the tunnel entry with metallic clips to maintain gastrointestinal integrity [6]. The treatment strategies depend on the size, location, and growth pattern of the lesion, and local expertise. The selection process of different surgical methods is shown in the figure below [7]. (Fig. 1)
Intraoperative bleeding, postoperative bleeding, intraoperative perforation, postoperative infection, and other procedure-related adverse events were recorded.
Histologic evaluation
The tumor size was reported by a pathologist who measured the largest diameter of the tumor specimen. Postoperative samples were sectioned one after the other for HE staining after being embedded in paraffin and preserved with formaldehyde. Immunohistochemistry was used to detect the protein expression of S-100, SOX10, CD34, CD117, SMA, desmin, DOG-1, and Ki-67. Every rectal GIST sample was routinely subjected to a histologic investigation in our hospital’s pathology department. The modified National Institutes of Health (NIH) criteria were used to evaluate the malignancy risk of GISTs by calculating the maximum tumor size and the number of mitoses per 50 high-power fields (HPFs) [8]. The key findings boiled down to en bloc resection, R0 resection, tumor recurrence, and residual disease. En bloc resection simply meant that the tumor was removed in one case. R0, R1, and R2 resections, built upon the en bloc concept, were defined as having negative margins all around (both lateral and vertical), microscopic residual disease, and macroscopic residual disease, respectively. Furthermore, the terms ER0 and ER1 were used to denote the absence or presence, respectively, of residual tumor as observed endoscopically [9, 10].
Follow-up
All of the patients underwent clinical follow-up. After surgery, wound healing was examined endoscopically at 3 and 6 months to look for any leftover tissue or recurrence. Colonoscopy and abdominopelvic CT were then performed once a year or after that. In-depth telephone interviews were conducted with patients from distant provinces and those who were unwilling to return for follow-up. These interviews included questions regarding symptoms and tests or treatments obtained at other hospitals. The follow-up was due on May 30, 2025.
Statistical analysis
All of the statistical analyses were performed via the Excel software package. The enumeration data conforming to a normal distribution are described as the mean ± SD, and the measurement data are described as the frequency (composition ratio).
Results
Patient characteristics
Nine men and nine women, with ages ranging from 33 to 69 years (mean: 56.22 ± 10.43 years), composed the group. The majority of rectal GISTs were detected incidentally [14 (77.8%)], followed by hematochezia 2 (11.1%)] and abdominal distension [1 (5.6%)]. Table 1 provides a summary of patient background information and procedures. The mean tumor size was 12.6 ± 7.7 mm. According to the modified NIH criteria, 2 (11.1%), 3 (16.7%), 4 (22.2%), and 9 (50.0%) patients were identified as high, intermediate, low, and very low risk, respectively. Only the two high-risk patients received adjuvant imatinib therapy (400 mg/day for 3 years), both of whom had KIT exon 11 mutations. The remaining patients, including those with low and very low risk, did not receive adjuvant therapy, consistent with current guideline recommendations.
Table 1.
Characteristics of 18 rectal gists patients
| Clinical characteristics | n | % |
|---|---|---|
| Gender | ||
| Male | 9 | 50.0 |
| Female | 9 | 50.0 |
| Age(year) | ||
| 30–49 | 4 | 22.2 |
| 50–69 | 14 | 77.8 |
| Symptoms | ||
| abdominal distension | 1 | 5.6 |
| change in bowel habits | 1 | 5.6 |
| hematochezia | 2 | 11.1 |
| none | 14 | 77.8 |
| Location | ||
| ≤ 3 cm from anal margin | 6 | 33.3 |
| 4–6 cm from anal margin | 4 | 22.2 |
| ≥7 cm from anal margin | 8 | 44.4 |
| Maximum diameter (mm) | ||
| ≤20 | 16 | 88.9 |
| 20–50 | 2 | 11.1 |
Endoscopic and EUS performance
All 18 lesions presented as isolated occurrences. The lesions ranged in maximum diameter from 4 to 35 mm, with a mean of (12.6 ± 7.7) mm. Endoscopic examination revealed that the masses were smooth, and raised.
Preoperative endoscopic ultrasound (EUS) was performed on ten patients. In all patients, the ultrasound results indicated hypoechoic lesions originating from the muscularis propria layer.
Treatment procedures and postoperative complications
Lesions were removed by ESD in nine cases, EFTR in seven cases, and STER in two cases. Complete lesion removal was achieved, and the lesions were confirmed endoscopically. The postoperative incision following EFTR is large and involves total resection. Therefore, a nylon cord combined with a metal clip purse-string suture was utilized for incision closure in the four patients who underwent EFTR. Metal clip closure was employed in all patients who underwent STER, whereas electrocautery cauterization or metal clip closure was used in ESD patients, according to the size of the incision [11]. During the perioperative phase, none of the patients experienced any problems, including bleeding, or infection. Figure 2 shows endoscopic images of a patient with a GIST during surgery and at follow-up. In total, all patients underwent an en bloc resection. The mean operative time was 37.5 ± 10.68 min; and the mean hospital stay duration was 3.60 ± 1.36 days.
Fig. 2.
Process and postoperative follow-up chart of a GIST patient undergoing EFTR surgery. A Endoscopic view of submucosal tumors, (B) EUS view of submucosal tumors, (C) Endoscopic view of incision mucosa, (D) The submucosal tumor dissected endoscopically, (E) Endoscopic view of the wound after the tumor was removed F,The tumor G. The mucosal entry closed using endoscopic clips, (H) Pictures from endoscopy during patient follow-upSTER was applied for GISTs ≤ 3 cm from the anal verge with < 50% extraluminal growth, or for lesions located ≥ 5 cm above the dentate line.
Overall, 3 (16.7%) patients had complication: two patients (both underwent EFTR) developed post-resection syndrome manifesting as abdominal pain, which resolved with conservative management (intravenous fluids, analgesics) within 48 h; one patient (following STER) developed a local inflammatory reaction with fever (peak 38.2 °C) and elevated CRP (45 mg/L), which resolved after a 5-day course of oral antibiotics (levofloxacin 500 mg daily) following discharge. The tumor sizes in these three cases were 18 mm, 35 mm, and 12 mm, respectively. All patients with complications fully recovered following conservative treatment. An overview of patient treatment procedures and postoperative complications is summarized in Table 2.
Table 2.
Surgical and pathology-related data of 18 patients
| Surgical and pathologically related | n | % |
|---|---|---|
| Therapeutic methods | ||
| ESD | 9 | 50.0 |
| EFTR | 7 | 38.9 |
| STER | 2 | 11.1 |
| Postoperative complications | ||
| abdominal pain | 2 | 11.1 |
| fever | 1 | 5.6 |
| Mitotic index, n (%) | ||
| ≤5/50 HPF | 15 | 83.3 |
| 6–10/50 HPF | 2 | 11.1 |
| >10/50 HPF | 1 | 5.6 |
| NIH risk category | ||
| High | 2 | 11.1 |
| Intermediate | 3 | 16.7 |
| Low | 4 | 22.2 |
| Very Low | 9 | 50.0 |
| Pathological cutting edge | ||
| R1 | 0 | |
| R0 | 18 | 100.0 |
| ER0 | 18 | 100.0 |
Pathological and immunohistochemical features
Gross examination revealed unencapsulated masses with grayish-yellow or grayish-white cut surfaces. The tumor cells were spindle-shaped and abundant, and some cases presented with mitotic signs. Immunohistochemistry was performed on all lesions. The results revealed that 16 lesions were positive for CD117, 17 lesions were positive for DOG-1, and were partially positive for CD34, SMA, Des. Two patients had positive Ki-67 rates of 10% and 15%, whereas the remaining lesions had positive Ki-67 rates of less than 5%. In examining the specimens microscopically, 100% of the margins were negative. Table 3 summarizes the pathological characteristics stratified by resection technique.
Table 3.
Pathological characteristics by resection technique
| Characteristic | ESD (n = 9) | EFTR (n = 7) | STER (n = 2) |
|---|---|---|---|
| Tumor size, mm | |||
| Median (range) | 10 (4–20) | 18 (10–35) | 12 (8–16) |
| Growth pattern, n (%) | |||
| Intraluminal | 9 (100%) | 0 (0%) | 1 (50%) |
| Extraluminal ≥ 50% | 0 (0%) | 7 (100%) | 0 (0%) |
| Extraluminal < 50% | 0 (0%) | 0 (0%) | 1 (50%) |
| Mitotic count, n (%) | |||
| ≤ 5/50 HPF | 9 (100%) | 5 (71.4%) | 1 (50%) |
| 6–10/50 HPF | 0 (0%) | 1 (14.3%) | 1 (50%) |
| > 10/50 HPF | 0 (0%) | 1 (14.3%) | 0 (0%) |
| NIH risk category, n (%) | |||
| Very low | 6 (66.7%) | 2 (28.6%) | 1 (50%) |
| Low | 3 (33.3%) | 1 (14.3%) | 0 (0%) |
| Intermediate | 0 (0%) | 2 (28.6%) | 1 (50%) |
| High | 0 (0%) | 2 (28.6%) | 0 (0%) |
| Margin distance, mm | |||
| Median (range) | 1.2 (0.5–2.0) | 1.0 (0.3–1.8) | 1.5 (1.0–2.0) |
Follow-up results
All patients were scheduled for a standardized clinical follow-up protocol. Endoscopic examination to assess wound healing and check for local residue or recurrence was performed at 3 and 6 months postoperatively. Subsequently, surveillance via colonoscopy and abdominopelvic CT was conducted annually. All 18 patients completed these mandatory endoscopic and/or imaging evaluations for at least one year, ensuring the collection of primary oncological outcome data. For patients from distant provinces or those reluctant to return to our center, in-depth telephone interviews were conducted as a supplement to the scheduled examinations. These interviews specifically inquired about relevant symptoms and gathered reports of any tests or treatments received at other local hospitals to capture any potential adverse events or recurrence signals. The follow-up period concluded on May 30, 2025.
Limitations and generalizability
This study has several limitations that must be considered when interpreting its results. Firstly, as a retrospective pilot study from a single, high-volume tertiary center, the small cohort size (n = 18) significantly limits its statistical power. While the 100% en bloc and R0 resection rates and the absence of recurrence are highly encouraging, these outstanding outcomes may not be fully generalizable and could be influenced by selection bias or the expertise of the operating endoscopists. The findings, particularly regarding margin status and recurrence, should be considered preliminary. Definitive conclusions regarding efficacy and safety require validation in larger, prospective multicenter studies, such as the recent population-based analysis by Ding et al., which included 239 patients to robustly compare endoscopic and surgical outcomes [12].
Secondly, the lack of a comparative control group (e.g., patients undergoing traditional surgical resection like transanal excision) prevents any definitive claims regarding the superiority or non-inferiority of the endoscopic approach. Our study describes outcomes from an endoscopic cohort but cannot assess comparative metrics such as long-term oncological survival, functional outcomes, or quality of life relative to surgery. As highlighted in a systematic review by Khan et al. [13]comparative studies are crucial to elucidate the differences in outcomes like R0 rates and hospital stay between local excision and radical resection. Future research should prioritize such comparative designs to precisely define the role of endoscopic resection within the treatment arsenal for rectal GISTs.
Discussion
Rectal GISTs occur across a wide age range but are most common in males. Study results show that the average age of onset in patients with rectal GIST is 49.7 years old [14], which is slightly lower than the mean age of 56.2 years in our cohort. Most patients with rectal GISTs have no clinical symptoms and are found incidentally during endoscopy. The clinical symptoms of rectal stromal tumors (GISTs) are usually nonspecific and are related to size, location, and whether the tumor has invaded surrounding organs. Common clinical symptoms include changes in bowel habits, hematochezia, and abdominal discomfort [15]. As the tumor size increases, symptoms such as compression, intestinal obstruction, and digestive tract bleeding may also occur [16].
Rectal stromal tumors typically develop in the middle to lower regions of the rectum [17], which aligns with the findings of this study. Endoscopic intervention is generally recommended for tumors measuring less than 5 cm in diameter; however, the average size of the lesions observed here was 12.6 ± 7.7 mm, with the largest measuring 35 × 29 × 22 mm. Endoscopic ultrasound (EUS) evaluations of rectal gastrointestinal stromal tumors (GISTs) predominantly reveal hypoechoic masses that exhibit a uniform internal echogenic pattern, primarily arising from the muscularis propria layer. In our findings, all identified lesions were hypoechoic and originated from the muscularis propria.
Given the malignant potential of rectal GISTs, current clinical guidelines recommend early intervention upon diagnosis [18]. All suspected rectal GISTs should be biopsied and preferably excised after EUS assessment, regardless of tumor size [2]. There is a lack of definitive recommendations regarding the optimal surgical approach for these lesions. However, several studies have shown that endoscopic treatment of rectal submucosal tumors can significantly improve the rate of local tumor control and the preservation of anal function [19].
Preoperative evaluation remains challenging, and preoperative examinations to assess tumor risk are critical. The diagnosis of rectal GISTs requires a combination of methods, including endoscopy, imaging examination (e.g. endoscopic ultrasound, CT, and MRI), and pathological examination (e.g. immunohistochemistry and genetic testing). These methods can help to accurately distinguish rectal GIST from other rectal tumors (e.g. neuroendocrine tumors and leiomyoma) [20]. In one study, Okai et al. [21] compared the endoscopic ultrasound characteristics of gastrointestinal stromal tumors with those of other gastrointestinal submucosal tumors, and reported that a marginal halo and relatively higher echogenicity on EUS might suggest a GIST. Marginal lobulation and a short doubling time may be signs of a malignant GIST. The increasing popularity of advanced endoscopic techniques has led to the increasing acceptance of diagnostic resection [22]. This approach enables clinicians to secure a comprehensive specimen in a single intervention, thereby not only confirming the diagnosis but also eliminating the lesion itself [23]. This dual benefit significantly alleviates both the financial strain and emotional toll on patients. Consequently, when dealing with submucosal tumors in the rectal area—particularly if gastrointestinal stromal tumors remain a possibility in the preoperative assessment—endoscopic resection may be considered a preferred method in carefully selected cases (e.g., small tumors ≤ 3 cm, favorable growth pattern, and availability of advanced endoscopic expertise), as it enables both diagnosis and treatment in a single procedure. This approach aligns with the NCCN Guidelines, which recommend R0 resection as the primary goal for resectable GISTs, while emphasizing the importance of preserving function in anatomically challenging sites such as the rectum [24]. Endoscopic resection should be undertaken in expert centers with contingency plans for salvage surgery if needed.
The current endoscopic resection methods for rectal GISTs are mainly ESD and EFTR [25]. Within this cohort, EFTR was the predominant technique employed. The successful complete resection with no residual tumor in all cases can be attributed to the high efficacy of both ESD and EFTR in eradicating subepithelial lesions. A minority of cases (n = 2) were managed with STER, a technique that facilitates the resection of muscularis propria lesions via a submucosal tunnel, thereby ensuring a safe buffer zone between the lesion itself and the mucosal entry point. The method lessens the chance of infection, gas-related problems, and perforation while also making endoscopic wound closure easier [26]. However, there is currently insufficient evidence to demonstrate the effectiveness and safety of the STER technique for rectal submucosal lesions. In our series, three patients experienced self-limiting complications: two cases of post-resection syndrome after EFTR and one case of localized inflammatory reaction after STER. These events may be attributed to the extent of tissue resection or transient microbial exposure. To minimize such risks, we recommend meticulous wound closure—particularly in EFTR—using techniques such as purse-string suturing with clips, and consider prophylactic antibiotics in cases with larger resection areas or prolonged procedure times. Additionally, gentle manipulation and avoidance of excessive electrocautery may reduce thermal injury and subsequent inflammation. The average operating time of the 18 patients was 37.5 min, and the average hospital stay was 3 days. These findings indicate that endoscopic resection of rectal GISTs is a safe and effective treatment method.
The gold standard for diagnosing GISTs is pathological examination. The predominant spindle cell type (70%), epithelial-like cell type (20%), and the mixed spindle-epithelial cell type (10%) are morphological characteristics of GIST. Even within the same subtype, the appearance of GIST varies greatly in terms of tumor cell density, tumor cell dysplasia, mitotic figure count, and growth pattern [25]. Alpha-smooth muscle actin, CD34, and S-100 proteins are immunohistochemically positive in 60–80%, 20–40%, and 5% of cases, respectively, whereas KIT is immunohistochemically positive in approximately 95% of GISTs [27]. In terms of the potential risk of malignant transformation, GISTs are usually divided into four levels, extremely low-risk, low-risk, moderate and high-risk, on the basis of factors such as tumor size and mitotic figure count [28]. In the present study, 2 patients were at high risk, one of whom had a mitotic count of 150/50 HPF. All patients received adjuvant treatment after surgery and had a good prognosis.
A critical aspect of managing rectal GISTs is risk stratification and subsequent therapy. In our cohort, two patients (11.1%) were classified as high-risk according to the modified NIH criteria. For such high-risk GISTs, current clinical guidelines, including those from ESMO-EURACAN and the GEIS, strongly recommend adjuvant therapy with imatinib to reduce the risk of recurrence [29]. In our study, both high-risk patients received adjuvant imatinib treatment following endoscopic resection, which likely contributed to their favorable outcomes and the overall zero recurrence rate observed during follow-up. Importantly, no patients with low or very low risk received adjuvant therapy, as it is not indicated per guidelines. This underscores that endoscopic resection for high-risk lesions should not be viewed as a standalone curative procedure but rather as a component of a multidisciplinary management strategy. The decision for adjuvant therapy must be individualized based on rigorous risk assessment. Some experts advocate neoadjuvant imatinib therapy for larger or marginally resectable rectal GISTs to downsize the tumor and facilitate organ-preserving approaches [30]. This strategy may enhance the feasibility of endoscopic resection, reduce the risk of tumor rupture, and improve functional outcomes. Although not employed in our cohort (all tumors were ≤ 3.5 cm), neoadjuvant therapy represents a valuable option for more complex cases and should be considered in a multidisciplinary setting. Our limited experience suggests that endoscopic resection can successfully achieve local tumor removal, even in high-risk cases, facilitating subsequent systemic treatment; however, long-term follow-up and larger studies are needed to confirm the adequacy of endoscopic local control for high-risk rectal GISTs within a multimodal approach.
A pivotal finding of our study is the discrepancy between endoscopic (ER0: 100%) and histopathological (R0: 100%) assessments of resection margins. Importantly, none of the patients experienced recurrence during the follow-up period (median 29 months). This finding is consistent with a large study of endoscopically resected gastric mesenchymal tumors, where margin status (R0 vs. R1) did not significantly influence recurrence rates [9]. Several factors may explain this observation. First, the thermal artifacts from electrosurgical dissection during endoscopy can devitalize a thin layer of tissue at the resection margin, potentially eradicating microscopic residual cells. Second, the natural history of small, low- to very-low-risk GISTs (which constituted 88.9% of our cohort) is often indolent, and the risk of recurrence from microscopically positive margins might be negligible over the short to medium term. Nevertheless, careful long-term follow-up remains essential, especially for high-risk tumors.
While several reports have described endoscopic resection for rectal GISTs, our study contributes to the literature by providing a detailed, technique-stratified analysis of pathological outcomes and explicit criteria for procedure selection based on tumor location and growth pattern. Furthermore, we report a homogeneous cohort of small (≤ 3.5 cm) rectal GISTs with 100% R0 resection and no recurrence at a median follow-up of 29 months, including two high-risk cases successfully managed with adjuvant imatinib. Our findings underscore the feasibility of endoscopic resection in carefully selected patients within a multidisciplinary framework, and provide practical insights into complication management and technique selection that may guide other centers in developing similar programs.
Conclusion
Within the limitations of this pilot study, our experience suggests that endoscopic resection is a feasible and promising primary treatment for carefully selected, small (≤ 3 cm) rectal GISTs, allowing for complete tumor removal and pathological diagnosis with a favorable safety profile. The excellent short-term oncological outcomes in our small cohort, despite potential challenges in margin assessment, merit further investigation. Importantly, management must be individualized, with high-risk patients requiring a multidisciplinary approach that includes consideration of adjuvant imatinib therapy. These preliminary findings support the prospective evaluation of endoscopic resection in larger, comparative studies to better define its precise role in the management paradigm of rectal GISTs.
Acknowledgements
We extend our sincere gratitude to the nursing staff and assistants at the Endoscopy Center of Zhongshan Hospital for their invaluable support and collaboration during the endoscopic procedures. We also thank all the patients who participated in this study.
Institutional review board statement
The Committee of Medical Ethics (Zhongshan Hospital Fudan University, No. B2020-265R) approved this study.
Abbreviations
- GISTs
Gastrointestinal stromal tumors
- ESD
Endoscopic submucosal dissection
- EFTR
Endoscopic full-thickness resection
- STER
Submucosal tunneling endoscopic resection
- EUS
Endoscopic ultrasound
- NIH
National Institutes of Health
- HPF
High-power fields
- ER0
Endoscopic complete resection
- R0
Histopathological complete resection
Authors’ contributions
Shi Q, Zhong YS, and Zhou PH designed the research study; Kedinuer Abulaiti, Bing Li, Yingjie Liu, Bahejuan Jiaerken, Yongbing Li, and Zhipeng Qi performed the research; Kedinuer Abulaiti and Bing Li wrote the main manuscript text. All authors read and approved the final manuscript.
Funding
This study was supported by the Shanghai Municipal Commission of Science and Technology (No. 22JC1403003, No. 22XD1402200, No. 22S31903800), the Innovation Fund of Zhongshan Hospital Affiliated to Fudan University (No. 2023-2ZSCX21), and the Jiangsu Provincial Association of Chinese Medicine Research Project (No. XYLD2024058).
Data availability
The datasets used and/or analyzed during the current study are available from the corresponding author upon reasonable request at shi.qiang@zs-hospital.sh.cn.
Declarations
Ethics approval and consent to participate
This study was conducted in accordance with the ethical principles of the Declaration of Helsinki. The study protocol was reviewed and approved by the Ethics Committee of Zhongshan Hospital, Fudan University (Approval No. B2020-265R). Written informed consent was obtained from all individual participants prior to their enrollment in the study and the endoscopic procedure.
Consent for publication
All participants provided written informed consent for the publication of their anonymized data and images.
Competing interests
The authors declare no competing interests.
Footnotes
Publisher’s Note
Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Kedinuer Abulaiti, Bing Li and Yingjie Liu contributed equally to this work and should be considered co-first authors.
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Associated Data
This section collects any data citations, data availability statements, or supplementary materials included in this article.
Data Availability Statement
The datasets used and/or analyzed during the current study are available from the corresponding author upon reasonable request at shi.qiang@zs-hospital.sh.cn.


