Current insights and future perspectives of treatment strategies for biliary tract cancer

Table 3 Phase III trials in advanced biliary tract cancer

Trial	Sample size	Primary endpoint	Regimen and median OS	Enrollment period	Results	Ref.
ABC-02	410	OS	GC: 11.7 months; GEM: 8.1 months	2005-2008	Significant OS benefit with GC (HR = 0.64, P < 0.001)	[30]
JCOG1113	354	OS	GS: 15.1 months; GC: 13.4 months	2013-2017	GS noninferior to GC (HR = 0.945, P = 0.046)	[40]
KHBO1401	246	OS	GCS: 13.5 months; GC: 12.6 months	2013-2017	OS benefit with GCS (HR = 0.79, P = 0.046)	[41]
TOPAZ-1	685	OS	D + GC: 12.8 months; GC: 11.5 months	2019-2021	OS benefit with D + GC (HR = 0.80, P = 0.021)	[44]
KEYNOTE-966	1069	OS	P + GC: 12.7 months; GC: 10.9 months	2019-2022	OS benefit with P + GC (HR = 0.83, P = 0.0034)	[46]

OS: Overall survival; GC: Gemcitabine + cisplatin; GEM: Gemcitabine; GS: Gemcitabine + S1; GCS: Gemcitabine + cisplatin + S1; D: Durvalumab; P: Pembrolizumab; HR: Hazard ratio.