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.