Anorectal gastrointestinal stromal tumors: a retrospective multicenter analysis of 15 cases emphasizing their high local recurrence rate and the need for standardized therapeutic approach

Agaimy, Abbas; Vassos, Nikolaos; Märkl, Bruno; Meidenbauer, Norbert; Köhler, Jens; Spatz, Johann; Hohenberger, Werner; Haller, Florian; Croner, Roland S.; Schneider‐Stock, Regine; Matzel, Klaus E.

doi:10.1007/s00384-013-1655-3

Cited by 23 publications

(18 citation statements)

References 28 publications

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“…In patients undergoing local excision in the absence of perioperative imatinib treatment, high local recurrence rates have been reported compared to radical resection 18,24,25 . However, our data suggest this does not hold true in the modern era.…”

Section: Discussionmentioning

confidence: 99%

Rectal Gastrointestinal Stromal Tumor (GIST) in the Era of Imatinib: Organ Preservation and Improved Oncologic Outcome

et al. 2017

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INTRODUCTION About 5% of GISTs originate in the rectum and historically radical resection was commonly performed. Little is known about the outcome of rectal GIST in the era of imatinib. METHODS Using a prospectively maintained database, we retrospectively analyzed 47 localized, primary rectal GISTs treated at our center from 1982 to 2016, stratified by when imatinib became available in 2000. Overall, disease-specific, and recurrence-free survival (OS, DSS, and RFS) were analyzed by the Kaplan-Meier method. RESULTS Rectal GISTs represented 7.1% of 663 primary GISTs. There were 17 patients in the pre-imatinib era and 30 in the imatinib era. The 2 groups had similar follow-up, age, gender, Miettinen risk, and distance to the anal verge. In the imatinib era, tumors were smaller at diagnosis (median 4.0 vs. 5.0 cm, p=0.029) and 24 of 30 patients received perioperative imatinib. In high-risk patients, organ-preservation and negative margins were more common in the 13 patients treated with neoadjuvant imatinib compared to the 21 treated directly with surgery. High-risk patients who received perioperative imatinib (n=15) had greater (or nearly significantly greater) 5yr OS, DSS, local RFS, and distant RFS than those (n=19) who did not (91, 100, 100, and 71% compared to 47, 65, 74, and 41%, p=0.049, 0.052, 0.077, 0.051, respectively). In the imatinib era, no patient has had a local recurrence or death due to GIST. CONCLUSIONS The use of imatinib is associated with organ-preservation and improved oncologic outcome in rectal GIST.

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Section: Discussionmentioning

confidence: 99%

Rectal Gastrointestinal Stromal Tumor (GIST) in the Era of Imatinib: Organ Preservation and Improved Oncologic Outcome

et al. 2017

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“…Incomplete resection, particularly in the area of the rectum, is also associated with a higher risk of recurrence. 120 As a group, PDGFRA-mutant GISTs appear to be less aggressive than KIT-mutant GISTs, 49 yet PDGFRA-mutant tumors can still progress and kill patients. These tumors are assessed using the same criteria as other GISTs.…”

Section: Assessing Gist Prognosismentioning

confidence: 99%

Gastrointestinal stromal tumors: what do we know now?

2014

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Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the GI tract, arising from the interstitial cells of Cajal, primarily in the stomach and small intestine. They manifest a wide range of morphologies, from spindle cell to epithelioid, but are immunopositive for KIT (CD117) and/or DOG1 in essentially all cases. Although most tumors are localized at presentation, up to half will recur in the abdomen or spread to the liver. The growth of most GISTs is driven by oncogenic mutations in either of two receptor tyrosine kinases: KIT (75% of cases) or PDGFRA (10%). Treatment with tyrosine kinase inhibitors (TKIs) such as imatinib, sunitinib, and regorafenib is effective in controlling unresectable disease; however, drug resistance caused by secondary KIT or PDGFRA mutations eventually develops in 90% of cases. Adjuvant therapy with imatinib is commonly used to reduce the likelihood of disease recurrence after primary surgery, and for this reason assessing the prognosis of newly resected tumors is one of the most important roles for pathologists. Approximately 15% of GISTs are negative for mutations in KIT and PDGFRA. Recent studies of these so-called wild-type GISTs have uncovered a number of other oncogenic drivers, including mutations in neurofibromatosis type I, RAS genes, BRAF, and subunits of the succinate dehydrogenase complex. Routine genotyping is strongly recommended for optimal management of GISTs, as the type and dose of TKI used for treatment is dependent on the mutation identified.

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“…There are a few studies that have shown that the use of IM adjuvant therapy and subsequent local resection is better than, or at least not inferior to, LAR or APR for anorectal GISTs (9,10,11). The present study reports the cases of two rectal GISTs that were treated by IM adjuvant therapy and subsequent transsacral local resections.…”

Section: Discussionmentioning

confidence: 99%

Transsacral excision with pre-operative imatinib mesylate treatment and approach for gastrointestinal stromal tumors in the rectum: A report of two cases

Sun

et al. 2014

Oncology Letters

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Gastrointestinal stromal tumors (GISTs) are rare in the rectum. Radical surgery, such as an abdominoperineal resection, is necessary for large rectal GISTs, which can result in the loss of function of involved organs. Imatinib mesylate can be used as perioperative therapy and may reduce tumor size, and it is now approved for use in the adjuvant therapy of locally resected anorectal GISTs. The present study describes two cases of large rectal GISTs, for which abdominoperineal resections were initially planned. The two patients received pre-operative imatinib mesylate treatment, and the therapeutic response was assessed by magnetic resonance imaging. Finally, transsacral local resection was successfully performed for these two GISTs. A macroscopically complete resection was achieved, and microscopically, the resection margin was negative. One patient experienced the complication of rectal leakage, which was successfully managed by drainage. No recurrence occurred in the two patients after more than two years. Pre-operative imatinib mesylate therapy with subsequent transsacral local resection for selected rectal GISTs is a feasible treatment modality and can prevent extended surgery.

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Anorectal gastrointestinal stromal tumors: a retrospective multicenter analysis of 15 cases emphasizing their high local recurrence rate and the need for standardized therapeutic approach

Cited by 23 publications

References 28 publications

Rectal Gastrointestinal Stromal Tumor (GIST) in the Era of Imatinib: Organ Preservation and Improved Oncologic Outcome

Rectal Gastrointestinal Stromal Tumor (GIST) in the Era of Imatinib: Organ Preservation and Improved Oncologic Outcome

Gastrointestinal stromal tumors: what do we know now?

Transsacral excision with pre-operative imatinib mesylate treatment and approach for gastrointestinal stromal tumors in the rectum: A report of two cases

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