The Absolute Effect / Evidence
RATIONALE-301
Clinical question: In first-line unresectable hcc, what is the efficacy and safety of the evaluated intervention?
Registry: NCT03412773 · One underlying study; aliases and follow-up publications consolidated.
Phase III, open-label randomized trial; 674 patients. Evidence cut-off: 13 November 2026.
Design, population and eligibility
Phase III, open-label randomized trial; 674 patients.
Previously untreated unresectable HCC; patients generally had preserved liver function and ECOG 0–1.
Tislelizumab monotherapy versus sorafenib.
Tislelizumab 200 mg IV every 3 weeks versus sorafenib 400 mg orally twice daily.
sorafenib.
Primary endpoint: OS primary; ORR, PFS, DoR and safety secondary.
Key efficacy outcomes
Median OS 15.9 vs 14.1 months; HR 0.85 (95.003% CI 0.71–1.02). Met prespecified non-inferiority; superiority was not demonstrated. ORR 14.3% vs 5.4%; median PFS 2.1 vs 3.4 months (HR 1.11).
Comparator: 14.1. The bars are a simple display of the first reported pair only; consult the endpoint and time horizon above.
Safety and treatment delivery
Grade ≥3 treatment-related AEs 22.2% vs 53.4%; treatment-related discontinuation 6.2% vs 10.2%.
Clinical interpretation and limitations
Interpret the reported hazard ratios in the context of the prespecified endpoint, statistical hierarchy, follow-up maturity, crossover/subsequent therapy and enrolled population. Median survival differences are not risk differences and must not be converted into NNT.
Absolute effect
No NNT/NNH is displayed unless an absolute event-risk difference at a clearly stated fixed timepoint can be calculated from reported trial data. Median survival differences and hazard ratios alone do not support an NNT. The visual above uses reported outcome data only.
Primary publication and follow-up
Registry / follow-up / authoritative source
Source-level verification status: primary publication identified and key quoted outcomes transcribed; where full-text toxicity or procedural details were not available, the page explicitly flags the limitation rather than inferring values.
Current-practice context · 13 November 2026
This page separates historical trial results from contemporary practice. Verify the applicable jurisdictional guideline, regulatory approval, biomarker assay, liver function, comorbidity and treatment availability before applying the evidence. NCCN Guidelines Insights are not a substitute for the full NCCN guideline.
Guideline/regulatory reconciliation and browser-rendered QA remain scheduled for the subsequent quality-control pass.