title: Local excision after preoperative chemoradiotherapy for rectal cancer: results of a multicenter phase II clinical trial.
abstract: 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.
  
  
  METHODS
  Sequential 2-stage phase II study for early efficacy.
  
  
  METHODS
  Multicenter study.
  
  
  METHODS
  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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: 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.
authors.forename: Salvatore
authors.initials: S
authors.lastname: Pucciarelli
authors.forename: Antonino
authors.initials: A
authors.lastname: De Paoli
authors.forename: Mario
authors.initials: M
authors.lastname: Guerrieri
authors.forename: Giuseppe
authors.initials: G
authors.lastname: La Torre
authors.forename: Isacco
authors.initials: I
authors.lastname: Maretto
authors.forename: Francesco
authors.initials: F
authors.lastname: De Marchi
authors.forename: Giovanna
authors.initials: G
authors.lastname: Mantello
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: Gambacorta
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Canzonieri
authors.forename: Donato
authors.initials: D
authors.lastname: Nitti
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Valentini
authors.forename: Claudio
authors.initials: C
authors.lastname: Coco
mesh_terms: D000230:Adenocarcinoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D059248:Chemoradiotherapy / Q000379:methods; D013505:Digestive System Surgical Procedures / Q000379:methods; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D020360:Neoadjuvant Therapy / Q000379:methods; D009364:Neoplasm Recurrence, Local / Q000401:mortality; D012004:Rectal Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D016896:Treatment Outcome