BACKGROUND: Transanal local excision has been suggested as an attractive approach for patients with rectal cancer who show a major clinical response after preoperative chemoradiotherapy.
OBJECTIVE: To evaluate the impact of transanal local excision on the local recurrence of rectal cancer in patients who had a major clinical response after preoperative chemoradiotherapy.
DESIGN: Sequential 2-stage phase II study for early efficacy.
SETTING: Multicenter study.
PATIENTS: Patients with clinical T3 or low-lying T2 rectal adenocarcinoma that showed a major clinical response after a preoperative chemoradiotherapy. Eligible patients underwent a full-thickness transanal local excision. According to their histopathology, the patients staged as ypT0-1 were observed, while the remaining patients were recommended to undergo a subsequent total mesorectal excision.
MAIN OUTCOME MEASURES: A local recurrence rate of ≤5% was set as a successful rate for stopping the trial early after the first stage.
RESULTS: The study group included 63 patients. Before chemoradiotherapy, patients were staged as clinical T3 (n = 42) and T2 (n = 21). After the local excision, 43 patients fulfilled the criteria to be observed with no further treatment. Nine of the remaining 20 patients for whom a subsequent total mesorectal excision was recommended refused surgery. Two of these patients who refused surgery had intraluminal local recurrence; both had a ypT2 tumor and underwent salvage surgery. The estimated cumulative 3-year overall survival, disease-free survival and local disease-free survival were 91.5% (95% CI: 75.9–97.2), 91.0% (95% CI: 77.0–96.6) and 96.9% (95% CI: 80.3–99.5), respectively.
LIMITATIONS: The time of follow-up is still short and the sample size is limited.
CONCLUSIONS: Our data suggest that local excision is a good option for patients with a major clinical response after chemoradiotherapy. A longer period of follow-up is required to confirm these findings.
1Department of Surgery, Oncology and Gastroenterology, University of Padua, Padua, Italy
2Department of Radiation Oncology, National Cancer Institute, Aviano, Italy
3General Surgery, University Politecnica delle Marche, Ancona, Italy
4Department of Public Health and Infectious Diseases, Sapienza University of Rome, Rome, Italy
5Department of Surgical Oncology, National Cancer Institute, Aviano, Italy
6Department of Radiotherapy, State Hospital, Ancona, Italy
7Department of Radiotherapy, Catholic University of Rome, Rome, Italy
8Department of Pathology, National Cancer Institute, Aviano, Italy
9Department of Surgical Sciences, Catholic University of Rome, Rome, Italy
Funding/Support: This work was partially supported by the AIRC (Associazione Italiana Ricerca sul Cancro) Regional Grant (2008).
Financial disclosure: the authors have no competing interests to declare.
Podium presentation at the meeting of The American Society of Colon and Rectal Surgeons, Phoenix, AZ, April 27 to May 1, 2013.
Correspondence: Salvatore Pucciarelli, Department of Surgery, Oncology and Gastroenterology, University of Padova, Via Giustiniani, 2, 35128 Padova, Italy. E-mail: email@example.com