Operative versus nonoperative treatment for stage 0 distal rectal cancer following chemoradiation therapy: long-term results
Phase observationalN = 265
- Population
- Patients with distal rectal adenocarcinoma considered resectable, treated with neoadjuvant chemoradiation
- Design
- Non-randomised comparison. 265 patients with distal rectal adenocarcinoma considered resectable were treated with neoadjuvant chemoradiation (5-fluorouracil, leucovorin and 5040 cGy). Patients with incomplete clinical response were referred for radical resection; those whose specimens were stage p0 were compared with patients with a complete clinical response who were observed without surgery. Groups were defined by response, not by randomisation. According to the full text, the patients were treated between 1991 and 2002 at one institution (Division of Colorectal Surgery, University of São Paulo Medical School); complete clinical response meant no abnormality on clinical, endoscopic and radiological reassessment 8 weeks after chemoradiation, and any significant residual ulcer or positive biopsy meant incomplete response.
- Arms
- Resection group (incomplete clinical response, pT0N0 specimen) (control, n = 22): Chemoradiation followed by radical resection; pT0N0M0 specimen
- Observation group (complete clinical response) (experimental, n = 71): Chemoradiation followed by nonoperative observation after complete clinical response
- People
- Habr-Gama A, senior author Gama-Rodrigues J
Δ 60-month OS+12 pointsΔ 60-month DFS+9 points
Show the numbers
| Endpoint | Analysis | Observation group (complete clinical response) | Resection group (incomplete clinical response, pT0N0 specimen) | Effect |
|---|---|---|---|---|
| Clinical complete responseAll 265 patients treated with chemoradiationProportion of the 265 patients with a complete clinical response after chemoradiation (criteria in the full text: no abnormality on examination, endoscopy and imaging at 8 weeks; none in the abstract) | finalHabr-Gama 2004 | 26.8% | — | 71 of 265 patients; 22 patients (8.3%) had an incomplete clinical response and a pT0N0 resected specimen. |
| OSPatients in the two groups; the groups were not randomisedFive-year overall survival | finalHabr-Gama 2004 | 100% at 5 y | 88% at 5 y | Mean follow-up 57.3 months in the observation group and 48 months in the resection group; two patients in the resection group died of the disease. |
| DFSPatients in the two groups; the groups were not randomisedFive-year disease-free survival | finalHabr-Gama 2004 | 92% at 5 y | 83% at 5 y | There were 3 systemic recurrences in each group and 2 endorectal recurrences in the observation group. |
| Endpoint | Analysis | Observation group (complete clinical response) | Resection group (incomplete clinical response, pT0N0 specimen) | Effect |
|---|---|---|---|---|
| Clinical complete responseAll 265 patients treated with chemoradiationProportion of the 265 patients with a complete clinical response after chemoradiation (criteria in the full text: no abnormality on examination, endoscopy and imaging at 8 weeks; none in the abstract) | finalHabr-Gama 2004 | 26.8% | — | 71 of 265 patients; 22 patients (8.3%) had an incomplete clinical response and a pT0N0 resected specimen. |
| OSPatients in the two groups; the groups were not randomisedFive-year overall survival | finalHabr-Gama 2004 | 100% at 5 y | 88% at 5 y | Mean follow-up 57.3 months in the observation group and 48 months in the resection group; two patients in the resection group died of the disease. |
| DFSPatients in the two groups; the groups were not randomisedFive-year disease-free survival | finalHabr-Gama 2004 | 92% at 5 y | 83% at 5 y | There were 3 systemic recurrences in each group and 2 endorectal recurrences in the observation group. |