KHBO1401-MITSUBA
Regimen
- Experimental
- Gemcitabine 1000 mg/m2 d1, d8 + cisplatin 25 mg/m2 d1, d8 q3w + S-1 60-80 mg/m2/day d1-14 (GCS triplet)
- Control
- Gemcitabine 1000 mg/m2 d1, d8 + cisplatin 25 mg/m2 d1, d8 q3w (GemCis doublet)
Population
Advanced BTC (unresectable/metastatic), 1L treatment-naive; ICC ~35%, ECC ~35%, GBC ~30%; Japan multicenter (Kansai Hepatobiliary Oncology Group, KHBO); all Japanese patients
Key finding
KHBO1401-MITSUBA tested adding S-1 to GemCis as a Japan-specific triplet (GCS) in advanced BTC. Like BILCAP in the adjuvant setting, it narrowly missed its ITT primary endpoint but showed a borderline significant result in per-protocol analysis. The 0.9-month median OS advantage is small but the per-protocol HR of 0.73 suggests potential benefit in patients tolerating full-dose therapy. Japan-specific tolerability of S-1 (East Asian DPD pharmacogenomics) may limit extrapolation. Note: candidate list referenced 'KHBO1202' but that trial registration name corresponds to a different protocol; the principal published phase 3 GCS vs GemCis result is KHBO1401-MITSUBA (Ioka T, J Hepatobiliary Pancreat Sci 2023).
Source: PMID 35900311