Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy. This study aims to investigate the safety and viability of an organ-preserving approach involving preoperative imatinib mesylate treatment in conjunction with local resection for rectal GIST, specifically targeting patients with c-KIT gene mutations.
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy. Previous studies have established that preoperative administration of imatinib mesylate effectively diminishes the size of rectal gastrointestinal stromal tumors (GIST) and enhances the likelihood of sphincter preservation. After initiating preoperative imatinib mesylate treatment, the sphincter preservation rate has notably escalated from 4.2% to 33.0%-94.9%. In theory, lymph node resection is not required for Gastrointestinal Stromal Tumors (GIST); the local excision of rectal GIST enables sphincter preservation and yields satisfactory anal function and quality of life (QoL). Various surgical techniques are utilized for local excision, including traditional transanal (TA) and transanal minimally invasive surgery (TAMIS) approaches. This study aims to explore the safety and feasibility of an organ-preservation strategy of preoperative imatinib mesylate combined with local resection in rectal gastrointestinal stromal tumor (GIST), specifically for patients with c-KIT gene mutations.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
23
1. For patients with c-KIT exon 11 mutation, imatinib mesylate, 400mg, qd. 2. For patients with c-KIT exon 9 mutation, imatinib mesylate, 600mg or 800mg, qd.
According to the characteristics of the location of the tumor, the surgeon decides the surgical approach based on the existing literature and the availability of surgical equipment, including: 1. Local transanal resection (TA) 2. Local resection transsacralapproach 3. Local resection via perineal approach 4. Local resection transvaginal approach
Weizhong Jiang
Fuzhou, Fujian, China
Organ preservation
Rectum intact, owing to no total mesorectal excision (TME), no locoregional regrowth unless amenable to limited, curative (R0) salvage surgery by local excision (LE) and no permanent stoma (including a never reversed protective stoma, or a stoma owing to toxicities and/or poor functional outcomes)
Time frame: 18 months
3-year disease-free survival
The proportion of participants who remain disease-free at 3 years after surgery
Time frame: 36 months
Local recurrence rate
The local recurrence rate is defined as the incidence detection of a tumor involving the bowel wall only that occurs after LE or TME
Time frame: 36 months
Overall survival
The proportion of participants who remain survival at 3 years after surgery
Time frame: 36 months
R0 resection rate
The R0 resection rate is defined as the rate of R0 resection
Time frame: 18 months
Quality of life based on EORTC-QLQs-C30 and EORTC-QLQs-CR29
Quality of life accessed by EORTC-QLQs-C30 and EORTC-QLQs-CR29 questionnaire
Time frame: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
Anorectal function
Anorectal function based on LARS score
Time frame: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
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