ASCOT
Adjuvant S-1 compared with observation in resected biliary tract cancer (JCOG1202, ASCOT): a multicentre, open-label, randomised, controlled, phase 3 trial
Phase 3N = 440
- Population
- Patients aged 20 to 80 years treated at 38 Japanese hospitals after resection of extrahepatic or intrahepatic cholangiocarcinoma, gallbladder carcinoma or ampullary carcinoma (no residual tumour or microscopic residual tumour); 440 enrolled.
- Design
- Open-label, multicentre randomised phase 3 trial (JCOG1202) at 38 Japanese hospitals. Patients aged 20 to 80 years with histologically confirmed extrahepatic cholangiocarcinoma, gallbladder carcinoma, ampullary carcinoma or intrahepatic cholangiocarcinoma in the resected specimen, with no residual tumour or only microscopic residual tumour, were randomly assigned 1:1 (minimisation by institution, primary tumour site and lymph node metastasis) to observation or to S-1 (40, 50 or 60 mg according to body surface area, orally twice daily for 4 weeks followed by 2 weeks of rest, four cycles). Primary endpoint overall survival, assessed for all randomised patients on an intention-to-treat basis. 440 patients enrolled between 9 September 2013 and 22 June 2018; data cutoff 23 June 2021. Registered UMIN000011688 (University hospital Medical Information Network, Japan); the abstract gives no ClinicalTrials.gov number.
- Primary endpoint
- OS
- Arms
- Observation (control, n = 222): Observation after resection
- S-1 (experimental, n = 218): S-1 40, 50 or 60 mg by body surface area orally twice daily for 4 weeks then 2 weeks of rest, four cycles
- People
- Nakachi K, Hepatobiliary and Pancreatic Oncology Group of the Japan Clinical Oncology Group (JCOG-HBPOG)
OSHR 0.69 (95% CI 0.51–0.94)Δ 36-month OS+9.5 pointsRFSHR 0.80 (95% CI 0.61–1.04)Δ 36-month RFS+11.5 points
Show the numbers
| Endpoint | Analysis | S-1 | Observation | Effect |
|---|---|---|---|---|
| OS | primarymedian follow-up 45.4 monthsNakachi 2023 | 77.1% at 3 y (70.9–82.1%) | 67.6% at 3 y (61.0–73.3%) | HR 0.69 (95% CI 0.51–0.94)P=0.0080 (one-sided)Primary analysis: 3-year overall survival. Adjusted hazard ratio as labelled in the abstract; the p-value is one-sided. Median duration of follow-up 45.4 months. Main grade 3-4 adverse events in the S-1 group: decreased neutrophil count 29 (14%) and biliary tract infection 15 (7%). |
| RFSRandomised patients (analysis population for relapse-free survival not stated in the abstract) | primarymedian follow-up 45.4 monthsNakachi 2023 | 62.4% at 3 y (55.6–68.4%) | 50.9% at 3 y (44.1–57.2%) | HR 0.80 (95% CI 0.61–1.04)P=0.088 (two-sided)3-year relapse-free survival; the difference was not statistically significant. |
| Endpoint | Analysis | S-1 | Observation | Effect |
|---|---|---|---|---|
| OS | primarymedian follow-up 45.4 monthsNakachi 2023 | 77.1% at 3 y (70.9–82.1%) | 67.6% at 3 y (61.0–73.3%) | HR 0.69 (95% CI 0.51–0.94)P=0.0080 (one-sided)Primary analysis: 3-year overall survival. Adjusted hazard ratio as labelled in the abstract; the p-value is one-sided. Median duration of follow-up 45.4 months. Main grade 3-4 adverse events in the S-1 group: decreased neutrophil count 29 (14%) and biliary tract infection 15 (7%). |
| RFSRandomised patients (analysis population for relapse-free survival not stated in the abstract) | primarymedian follow-up 45.4 monthsNakachi 2023 | 62.4% at 3 y (55.6–68.4%) | 50.9% at 3 y (44.1–57.2%) | HR 0.80 (95% CI 0.61–1.04)P=0.088 (two-sided)3-year relapse-free survival; the difference was not statistically significant. |
Data table
| Endpoint | HR | 95% CI |
|---|---|---|
| OS | 0.69 | 0.51–0.94 |
| RFS | 0.8 | 0.61–1.04 |