Table 2 –

Treatment efficacy in BCG-unresponsive trials (top) and all patients following adequate BCG (bottom).

Authors	Year	Phase	Agent	n	Age	M (%)/F (%)	CIS vs papillary	Tumor stage			Tumor grade			Biopsy required	Recurrence definition	3-mo CRR/RFR/DFR (%)	6-mo CRR/RFR/DFR (%)	12-mo CRR/RFR/DFR (%)	18-mo CRR/RFR/DFR (%)	24-mo CRR/RFR/DFR (%)
								Ta (%)	T1 (%)	CIS (%)	Low (%)	Int (%)	High (%)
Li et al [48]	2017	II	MCNA	94	67.9	69/31	Both	42	4	72	0	0	100	Yes	HG recurrences		48.9	34.8		28.3
Li et al [48] (CIS cohort)				68						100					HG recurrences		44.8	26.5		16.6
Navai et al [13]	2016	Ib	Instiladrin	7	76.2	100/0	Both	57	14	71	0	0	100	No	Positive biopsy or cytology	28.6	14.3	0.0		0.0
Shore et al [14]	2017	II	Instiladrin	40	70.5	83/17	Both	20	28	75	0	0	100	Yes	HG recurrences	57.5	42.5	35.0
Gacci et al [7]	2006	II/III	Gemcitabine	9	75	78/22	Papillary	44	56	0	11	33	56	No	Positive biopsy or cytology	100.0	66.7	44.4	11.1	0.0
Gacci et al [7]	2006	II/III	BCG	10	75.5	80/20	Papillary	70	30	0	10	60	30	No	Positive biopsy or cytology	80.0	60.0	10.0	10.0	10.0
Gunelli et al [22]	2007	II	Gemcitabine	40	66	95/5	Papillary	10	90	0	0	53	43	No	Positive biopsy or cytology		95.0	77.5	70.0	70.0
Perdona et al [23]	2010	II	Gemcitabine	20	68.3	65/35	Both	20	80	35	25	0	75	No	Positive biopsy	75.0	65.0	50.0	40.0	35.0
Skinner et al [24]	2013	II	Gemcitabine	47	70	70/30	Both	36	4	60	11	0	89	Yes	Positive biopsy or cytology	46.8		27.7		21.0
Dinney et al [12]	2013	I	Instiladrin	17	72	94/6	Both	59	6	65	12	0	88	Yes	Positive biopsy or cytology	41.2	41.2	29.4	29.4	23.5

BCG = bacillus Calmette-Guérin; CIS = carcinoma in situ; CRR = complete response rate; DFR = disease-free rate; HG = high grade; Int = intermediate; MCNA = M. phlei cell wall–nucleic acid complex; M/F = male/female; RFR = recurrence-free rate.